Why is high quality health information important for patients?

Medical and healthcare information are sometimes complex for patients, therefore it is important to ensure they receive clear and understandable information

For every two medicines prescribed by doctors, one will be taken in the way the doctor advised and the other will not1. Medicine dosing errors – such as taking medicine at the wrong time, taking too much, or not taking enough – mean a patient’s condition may fail to improve and could get worse1,2. There are many reasons why patients don’t take medicines as advised, which may include forgetfulness, not understanding instructions, or not believing the medicine is helping1,3,4. High quality patient information can help to overcome some of these problems.

Before any medical treatment begins, it is the doctor’s responsibility to ensure that the patient (or a person legally allowed to make decisions on their behalf) understands what it’s for and that they agree to receive it5.This is called informed consent. In practice, this means that the person giving consent must receive clear, understandable and relevant information about what the treatment involves, including expected benefits and risks, any alternatives, and what may happen if the patient chooses not to go ahead with treatment. If the suggested treatment involves taking part in a research study, patients must also understand the research aims, and any extra tests or clinic visits involved in the study.

Informed consent is more than just a legal responsibility for the doctor. Without high quality information upfront, it’s very difficult for any patient to make the right decision when it comes to their health & what treatment plan best suits them. But once involved, a well-informed patient is more likely to stick with their agreed treatment plan than one who doesn’t1,6,7.

What makes patient information effective?

Healthcare professionals often assume that their explanations and instructions are easy to understand8. In reality, they are often misunderstood8. Health literacy is the term used to describe the ability of patients to read and understand the words and numbers they come across in a healthcare setting (e.g. dosing instructions)9. Most adults are comfortable reading health information written in language that could be understood by a typical 12-year-old, but one in every five adults finds this difficult9. Older people may have additional challenges if they have problems with seeing, hearing or reasoning9. People with reduced health literacy can be helped to understand complex ideas if the doctor speaks slowly, using everyday words, or if information is given in short sections rather than all at once. Written information that includes pictures and illustrations can also help. In one study, patients had a better understanding of when to take their daily tablets if they were shown a visual aid (a diagram showing coloured tablets), compared to spoken or written instructions alone5.

Good communication with a healthcare provider makes it more likely that you will get the intended benefit from your treatment, and helps to ensure the care you get is truly ‘patient-centred’

Finding high-quality information online

With the growth of the internet and social media, patients today have access to more information than they could ever hope to read10. With so much available, it can be hard to know which sources to trust. Fortunately, there are some simple ways to check that the information is likely to be reliable,
as outlined in detail in IAPO’s Patient information and health literacy briefing. One is to look at who is responsible for producing the information. Do they have suitable qualifications or experience? Do they have any reason to provide biased information? The information should say when it was last checked or updated, as medical recommendations and treatment options often change over time. It should also be made clear which statements are backed up by published evidence and which are simply personal opinions or personal experience. However, sites which focus on sharing the personal experiences of other patients (e.g. blog sites) can still be reputable information sources, with some valuable insights to offer.

Improving communication with your healthcare team

Medical and healthcare information can be complex, and may take a while to understand fully, even for people with good health literacy. You should never be embarrassed about asking as many questions as you need about why a medicine has been prescribed, how it should be taken, what side effects to look out for and what to do if you are experiencing problems. Some patients find it helpful to use their own words to repeat back what they believe a healthcare professional has said, to confirm they have understood correctly. Good communication with a healthcare provider makes it more likely that you will get the intended benefit from your treatment, and helps to ensure the care you get is truly ‘patient-centred’ – i.e. best suited to you as an individual.

Sources:

[1] Brown MT, Bussell JK. Medication Adherence. Mayo Clin Proc. 2011;86(4):304-314.

[2] Roh YH, Koh Y, Noh JH et al. Effect of health literacy on adherence to osteoporosis treatment among patients with distal radius fracture. Archives of Osteoporosis. 2017;12:42.

[3] Reading SR, Go AS, Fang MC. Health Literacy and Awareness of Atrial Fibrillation J Am Heart Assoc. 2017;6:e005128.

[4] De Vries ST, Keers JC, Visser JC et al. Medication beliefs, treatment complexity, and non-adherence to different drug classes in patients with type 2 diabetes. Journal of Psychosomatic Research 2014;76:134–138.

[5] NHS Choices. Consent to treatment. Available from: http://www.nhs.uk/conditions/Consent-to-treatment/Pages/Introduction.aspx [Accessed 24 April 2017]

[6] Schillinger D, Machtinger EL, Wang F, et al. Language, literacy and communication regarding medication in an anticoagulation clinic: Are pictures better than words? In: Henriksen K, Battles JB, Marks ES, Lewin DI, editors. Advances in Patient Safety: From Research to Implementation (Volume 2: Concepts and Methodology). Rockville (MD): Agency for Healthcare Research and Quality (US); 2005 Feb.

[7] Fan JH, Lyons SA, Goodman MS et al. Relationship Between Health Literacy and Unintentional and Intentional Medication Nonadherence in Medically Underserved Patients With Type 2 Diabetes. Diabetes Educ. 2016;42(2):199-208.

[8] Cornett S. Assessing and Addressing Health Literacy. OJIN: The Online Journal of Issues in Nursing 2009;14(3):2.

[9] Safeer RS, Keenan J. Health Literacy: The Gap Between Physicians and Patients. Am Fam Physician. 2005;72(3):463-468.

[10] HealthIT.gov. eHealth: Find quality resources. Available from: https://www.healthit.gov/patients-families/find-quality-resources [Accessed 24April 2017]

Other Stories

July 3, 2017
How can a patient-centred approach help to achieve high quality healthcare?

How can a patient-centred approach help to achieve high quality healthcare?

It has been shown that a patient-centred approach can imporve the way healthcare is delivered

by Antonio Ciaglia
June 27, 2017
What is patient-centred healthcare?

What is patient-centred healthcare?

Patient-centred healthcare, the healthcare of the future

by Antonio Ciaglia
May 23, 2017
Working towards Universal Health Coverage: the role of research

Working towards Universal Health Coverage: the role of research

Good health allows children to learn and adults to earn, helps people escape from poverty, and provided the basis for long-term economic development

by Antonio Ciaglia
How can a patient-centred approach help to achieve high quality healthcare? - Patient Research Exchange

How can a patient-centred approach help to achieve high quality healthcare?

It has been shown that a patient-centred approach can imporve the way healthcare is delivered

Achieving high quality healthcare is an ambition of governments and health organisations around the world. There are many different opinions on what ‘high quality’ means, how it should be measured, and how it can be achieved 1. As a general rule, ‘high quality’ is taken to mean healthcare that follows the latest recommendations of specialist medical societies and experts who have considered all the latest evidence. However, most people now agree that high quality healthcare must also be based on the needs, preferences and values of each individual patient – in other words, it must be ‘patient centred’2. In addition to receiving the most appropriate treatment, patient-centred care can help to achieve many other things that are considered signs of high quality care, such as feeling listened to, not having to wait too long for treatment and being able to access care easily and conveniently1,2

What is ‘high quality’ patient-centred care and how do we measure it?

Measuring the quality of healthcare is important, as it allows healthcare professionals to understand what they are already doing well and what may still need to be improved3. However, as patient-centred care focuses on individual needs that vary from one person to another, it is not always clear what to measure4. A list of patient-centred quality standards – in terms of goals for healthcare organisations – has been published by the government in the United Kingdom5. These include: ensuring healthcare professionals are trained in communication skills; reducing the number of different doctors and nurses involved during a single period of patient care (e.g.a hospital stay); and ensuring staff introduce themselves and explain their role to the patient before starting to provide care5.

Healthcare organisations are increasingly recognising the value of patient insights and experiences to help them re-design processes and systems in a more patient-centred way

Studies looking at the quality of patient-centred care focus either on the personal experiences of the patient (e.g. whether they feel their personal priorities were taken into account by healthcare teams), or on whether their health improves as a result of the care4,6. Video recordings have shown that patients’ memories of a discussion with their doctor don’t always reflect how the discussion actually went, which makes it difficult to use patient surveys to measure care quality. Measuring health improvement might therefore be the best way of assessing the value of different types of patient-centred care7. For example, a five-year study comparing two hospitals showed that patient-centred care resulted in shorter stays in hospital and a lower overall cost of care. In another study, patient-centred care was associated with better relief from symptoms and fewer medical tests7,9.

How can patients share responsibility for high-quality care?

Patients and their family members often have a lot of valuable insights, both about their own health condition and the way care has been delivered to them in and out of hospital10. These insights mean that patients can play active and important roles in measuring and improving the quality of healthcare at different organisational levels. The University of Montreal in Canada recently set up a new department to involve patients in designing better healthcare services10. The department includes teams of patients and doctors who help design training for medical students, research new ways of delivering patient centred care, and assess the quality of healthcare services in their local area10. Patients who took part in this project have said that it helped them to feel like they were making a difference, as well as improving their own communication and team-working skills10.

Patients and their family members can also draw on specific experiences to suggest simple, practical ideas to improve the way healthcare is delivered. For example, the daughter of a woman with serious memory problems caused by dementia suggested using a logbook to record when her mother had been washed, fed and given her medication each day, to improve communication between family members and different health workers. In another case, a cancer patient noticed that doctors and nurses didn’t always introduce themselves personally, which she found off-putting. She set up a national campaign called “Hello, my name is…”, which encouraged doctors and nurses to interact in a more ‘human’ way and less like anonymous professionals11.

In the past few decades, our understanding of high quality healthcare has changed, becoming a broader definition of care encompassing what takes place at home and in the local community12. Healthcare organisations are increasingly recognising the value of patient insights and experiences to help them re-design processes and systems in a more patient-centred way.

Sources:

1. Committee on Quality of Health Care in America. Institute of Medicine. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington, DC: National Academy Press; 2001.

2. Raleigh VS, Foot C. Getting the Measure of Quality: Opportunities and challenges. The Kings Fund, 2010. Available from: https://www.kingsfund.org.uk/sites/files/kf/Getting-the-measure-of-quality-Veena-Raleigh-Catherine-Foot-The-Kings-Fund-January-2010.pdf [Accessed 27 April 2017]

3. Werner RM, Asch DA. Clinical concerns about clinical performance measurement. Annals of Family Medicine. 2007;5:159–63.

4. Health Foundation, 2014. Helping measure person-centred care. Available from: http://www.health.org.uk/sites/health/files/HelpingMeasurePersonCentredCare.pdf (Accessed 27 April 2017)

5. National Institute for Health and Care Excellence (NICE). Patient experience in adult NHS services. February 2012. Available from: https://www.nice.org.uk/guidance/cg138 [Accessed 27 April 2017]

6. Epstein RM, Street RL. The Values and Value of Patient-Centered Care. Annals of Family Medicine. 2011;9(2):100-3

7. Frampton S, Guastello S, Brady C et al. Picker Institute, October 2008. Patient-centred care Improvement Guide. Available from http://patient-centeredcare.org/ [Accessed 27 April 2017]

8. Zolnierek KBH, DiMatteo MR. Physician communication and patient adherence to treatment: a meta-analysis. Med Care 2009;47(8);826-834.

9. Stewart M, Brown JB, Donner A, et al. The impact of patient-centered care on outcomes. J Fam Pract 2000;49(9):796-804.

10. Pomey MP, Hihat H; Khalifa M, et al. Patient partnership in quality improvement of healthcare services:Patients’ inputs and challenges faced. Patient Experience Journal. 2015; 2(1):6.

11. “Hello, my name is…” Campaign. Available from: www.hellomynameis.org.uk [Accessed 27 April 2017]

12. Royal College of General Practitioners, November 2014. An Inquiry into Patient Centred Care in the 21st Century. Implications for General Practice and Primary Care. Available from: http://www.rcgp.org.uk/policy/rcgp-policy-areas/inquiry-into-patient-centred-care-in-the-21st-century.aspx [Accessed 27 April 2017]

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May 23, 2017
Working towards Universal Health Coverage: the role of research

Working towards Universal Health Coverage: the role of research

Good health allows children to learn and adults to earn, helps people escape from poverty, and provided the basis for long-term economic development

by Antonio Ciaglia
June 27, 2017
What is patient-centred healthcare?

What is patient-centred healthcare?

Patient-centred healthcare, the healthcare of the future

by Antonio Ciaglia
July 10, 2017
Why is high quality health information important for patients?

Why is high quality health information important for patients?

Medical and healthcare information are sometimes complex for patients, therefore it is important to ensure they receive clear and understandable information

by Antonio Ciaglia
What is patient-centred healthcare? - Patient Research Exchange

What is patient-centred healthcare?

Patient-centred healthcare, the healthcare of the future

The concept ‘doctor knows best’ has been accepted for centuries1. This is understandable, as doctors spend many years gaining specialist medical skills and experience. However, while doctors are experts in diseases, no-one understands an individual patient’s own experience of a disease and how they get on with treatments better than the patient themselves. Patients live with their conditions every moment of every day, while a doctor or nurse usually only sees a particular patient every so often. This is why there has been a growing trend towards viewing patients as the most important member of their own healthcare team1.

The internet and social media have made it easier than ever for patients to share experiences with each other and gain a deeper understanding of their own condition. In addition, new health technologies such as wearable devices or heart monitors help patients measure their own health scores and learn what works best for them1. Patients have a unique perspective on their own needs, priorities and reactions to treatment, and should therefore contribute to a process of shared decision-making with healthcare professionals.

What does patient-centred healthcare mean?

According to the International Alliance of Patients’ Organisations (IAPO)2, patient-centred healthcare is based on five core principles2: 1) patients have individual needs and preferences; 2) patients have the right and responsibility to be involved in making decisions that affect their lives; 3) governments and healthcare organisations should involve patients when designing new healthcare policies; 4) patients must have access to safe, quality and appropriate health services, treatments, and preventive care (e.g.
vaccinations); 5) patients should be given the information needed to make decisions about healthcare, treatment and living with their condition.

Small changes can help to achieve care that is more patient-centred3. Some hospitals in England have introduced ‘what matters to me’ boards by patients’ beds, where they can write messages to their healthcare team4. Sharing their preferences (e.g. views about pain relief or the types of food and drinks they prefer) helps hospital staff provide care that takes these personal needs into account. Other health systems are trying to be more patient-centred by introducing a personal health budget, where patients (usually with long-term care needs) are allowed to decide how a certain amount of money may be spent to contribute to their care5.

Patient-centred care relies on well-trained healthcare teams who have the time and resources available to understand patients’ personal needs and educate them on how to start playing a greater role in their own care

What are the benefits of patient-centred healthcare?

Health improvements over the last century mean that more people are living longer with conditions like cancer, heart disease, and diabetes6. Many people also live with more than one condition, which means they may take several types of medicine or see different specialists.;Involving, educating and motivating patients about their own care will enable them to ensure each of their specialist healthcare teams knows what the others have recommended. This can help to avoid communication problems, reduce waste from unused medications, and increase the chance of patients getting the intended benefit from their treatment3. It has been found that people who are more involved in their own care are less likely to use emergency hospital services, more likely to stick to the treatment plans agreed with their doctors, and more likely to take their medication correctly7,8. This may lead not only to better health outcomes but also to more efficient use of healthcare resources.

Are there any challenges with achieving successful patient-centred healthcare?

Patient-centred care relies on well-trained healthcare teams who have the time and resources available to understand patients’ personal needs and educate them on how to start playing a greater role in their own care. It may also mean that healthcare providers need to consider investing in new technology (e.g. electronic health records) to help ensure patient preferences and agreed care plans are known by all health professionals treating them.

Once patients start taking greater control of their own care, the internet is often the first place they go for health information9. However, taking decisions based purely on information accessed online carries risks. Almost half of people responding to one survey admitted to using supplies of a painkiller that had been prescribed to someone else after diagnosing themselves online10. This can be dangerous, not only because you should never take medicine intended for another person, but also because a new diagnosis or change to a treatment plan should always be discussed with a healthcare professional. When reading information online, it is important to consider whether it is reliable, up-to-date and represents the viewpoint of experts rather than just one person’s opinion11. Your doctor should be able to recommend good websites and provide guidance on which types of online information to avoid.

New roles for ‘expert’ patients

Advances in modern technology and an increased focus on patient-centred care have meant that some patients are becoming real experts in their own conditions1. People like this can act as partners with their doctors in making healthcare decisions and may even be invited to help design research studies. In other cases, patients may use the knowledge they have gained through personal experience of a condition to help other patients. People who are able to make this sort of contribution to society are still in the minority of course but it’s possible for almost every patient to play a greater role in ensuring their own personal healthcare is as beneficial as it can be.

Sources:

[1] deBronkart, D. From patient centred to people powered: autonomy on the rise. BMJ 2015;350:h148

[2] International Alliance of Patients’ Organisations, June 2007. What is Patient-Centred Healthcare? A review of definitions and principles. 2nd
Edition. Available from: http://iapo.org.uk/sites/default/files/files/IAPO%20Patient-Centred%20Healthcare%20Review%202nd%20edition.pdf [Accessed 20 April 2017]

[3] The Health Foundation, October 2014. Patient-centred care made simple. Available from: http://www.health.org.uk/publication/person-centred-care-made-simple [Accessed 20 April 2017]

[4] Guy’s and St Thomas Hospital, April 2014. What matters to me today – patients personalise their care. Available from: http://www.guysandstthomas.nhs.uk/news-and-events/2014-news/Named-nurse-board.aspx [Accessed 20 April 2017]

[5] NHS England. NHS Personal Budgets Frequently Asked Questions. Available from: https://www.england.nhs.uk/healthbudgets/understanding/faqs/ [Accessed 20 April 2017]

[6] World Health Organization Western Pacific Region, 2007. People-Centred Health Care: A Policy Framework. ISBN 978 92 9061 317 6 Available from: http://www.wpro.who.int/health_services/people_at_the_centre_of_care/documents/ENG-PCIPolicyFramework.pdf [Accessed 20 April 2017]

[7] National Institute of Health and Care Excellence (NICE), 2009. Medicines adherence: Involving patients in decisions about prescribed medicines and supporting adherence. Available from: http://www.nice.org.uk/guidance/cg76 [Accessed 20 April 2017]

[8] De Silva D. Helping people share decision making, The Health Foundation, June 2012. Available from: www.health.org.uk/publications/evidence-helping-people-help-themselves. [Accessed 20 April 2017]

[9] HealthIT.gov. eHealth: Find quality resources. Available from: https://www.healthit.gov/patients-families/find-quality-resources [Accessed 20 April 2017]

[10] The Guardian, November 2016. DIY diagnosis of illness is dangerous trend, say pharmacists. Available from: https://www.theguardian.com/society/2016/nov/14/diy-diagnosis-of-illness-is-dangerous-trend-say-pharmacists [Accessed 20 April 2017]

[11] US National Library of Medicine. Evaluating health information. Available from: https://medlineplus.gov/evaluatinghealthinformation.html [Accessed 20 April 2017]

Other Stories

May 23, 2017
Working towards Universal Health Coverage: the role of research

Working towards Universal Health Coverage: the role of research

Good health allows children to learn and adults to earn, helps people escape from poverty, and provided the basis for long-term economic development

by Antonio Ciaglia
July 3, 2017
How can a patient-centred approach help to achieve high quality healthcare?

How can a patient-centred approach help to achieve high quality healthcare?

It has been shown that a patient-centred approach can imporve the way healthcare is delivered

by Antonio Ciaglia
July 10, 2017
Why is high quality health information important for patients?

Why is high quality health information important for patients?

Medical and healthcare information are sometimes complex for patients, therefore it is important to ensure they receive clear and understandable information

by Antonio Ciaglia
Working towards Universal Health Coverage: the role of research - Patient Research Exchange

Working towards Universal Health Coverage: the role of research

Good health allows children to learn and adults to earn, helps people escape from poverty, and provided the basis for long-term economic development

Access to good quality, affordable healthcare is often viewed as a basic human need and could greatly improve the health and wellbeing of millions of people worldwide. For example, in developing countries, assistance from doctors and midwives during pregnancy and childbirth could significantly reduce the risk of babies dying soon after birth. Better access to medicines could improve the treatment and reduce the spread of diseases like HIV/AIDS.ii,[iii],[iv] It’s also important to reduce the risk of people falling into poverty after paying for essential healthcare for themselves or their family.iv Health-related poverty isn’t only a problem in developing countries – people in some high-income countries who are uninsured or under-insured may also risk serious financial problems if they need expensive medicine or surgery.[v] Ensuring a basic level of affordable healthcare could improve both life expectancy and quality of life for countless people – and allow them to stay active and productive members of society for longer.

Universal health coverage is a type of government scheme that enables all residents in a country to access medicines, vaccines and other health services that are considered essential without risking financial hardship. This type of medical care is well established in Europe, and includes the UK’s National Health Service (NHS) and the Protection Maladie Universelle (PUMA)
in France, in which most (but not all) medicines and health services are freely available to citizens.

Member states of the United Nations (UN) recently agreed to try to expand universal health coverage to all countries in the world by 2030. The specific type of universal health coverage given in a country (e.g. the particular medicines and services offered as part of the scheme) will depend on the most important causes of disease and death within each population, as well as the money available to each government.i Combining money from funding sources (e.g. taxes or national insurance schemes) could help to spread the financial risks of ill health across a population, making it less likely that an illness will cause serious hardship for an individual.

What could be achieved through universal health coverage?

In 2015, the UN published a list of Sustainable Development Goals, one of which has several targets linked to improving universal health coverage. For example, one target is to reduce deaths of mothers and babies during childbirth by increasing access to good quality antenatal (pregnancy) and midwifery care. Globally, the number of women receiving care from a midwife has increased from 62% in 2000 to 73% in 2013.iv Another UN target for 2030 is to put an end to serious diseases that affect the world’s poorest countries, including HIV/AIDS, tuberculosis and malaria. Achieving these very ambitious targets will involve lots of different activities, such as providing effective, affordable vaccines and medicines, improving early diagnosis of diseases, giving dietary or sexual health advice, and controlling mosquito breeding grounds.xi

The role of research in designing universal health coverage

Wider access to health services and better financial protection can be expected to lead to better overall health, but every country needs to understand the best way(s) to use universal health coverage for its own citizens since the available budget will always be limited.Research can answer important questions that help to design affordable universal healthcare, such as:
Research questions like these may be addressed by international research groups or by the companies that make medicines. Some countries, such as Thailand, have set up new research organisations to answer questions about how best to design universal health coverage for their own populations. The World Bank and the World Health Organisation also support research in many low- and middle-income countries. In many cases, patients and members of the public may be asked to join research studies, or to share their personal medical information, to help answer these questions. Universal health coverage is an important social, economic and health ambition that is supported by most countries around the world. Studies that involve local researchers in looking for answers to specific local health problems can help governments understand how to use their money in a way that best improves basic levels of health for people living in their country.

  • Who will benefit most from a particular type of medicine or medical treatment?
  • What are the benefits and costs involved with offering a medicine or vaccine to everyone in a country who may be at risk from a particular disease?
  • When is the best time to give a certain type of treatment (e.g. should it be given as soon as the disease develops, or only to those who are affected by symptoms)?
  • Where is it best to provide a treatment (e.g. in hospital, at community clinics, or in a patient’s home)?

Research questions like these may be addressed by international research groups or by the companies that make medicines. Some countries, such as Thailand, have set up new research organisations to answer questions about how best to design universal health coverage for their own populations. The World Bank and the World Health Organisation also support research in many low- and middle-income countries. In many cases, patients and members of the public may be asked to join research studies, or to share their personal medical information, to help answer these questions. Universal health coverage is an important social, economic and health ambition that is supported by most countries around the world. Studies that involve local researchers in looking for answers to specific local health problems can help governments understand how to use their money in a way that best improves basic levels of health for people living in their country.

Sources:

  1. World Health Organization Fact Sheet. Website available from: http://www.who.int/mediacentre/factsheets/fs395/en/ (Accessed 3 March 2017).
  2. World Health Organisation and World Bank, 2015. Tracking Universal Health Coverage: First global monitoring report. Available from: http://apps.who.int/iris/bitstream/10665/174536/1/9789241564977_eng.pdf?ua (Accessed 3 March 2017).
  3. Souza JP, Gülmezoglu AM, Vogel J, et al. Moving beyond essential interventions for reduction of maternal mortality (the WHO Multi-country Survey on Maternal and Newborn Health): across-sectional study. Lancet 2013;381(9879):1747–55.
  4. World Health Statistics 2012. Geneva, World Health Organization, 2012.
  5. UNAIDS report on the global AIDS epidemic 2012. Geneva, Joint United Nations Programme on HIV/AIDS, 2012.
  6. United States Census Bureau. Health Insurance Coverage in the United States: 2014. Report Number: P60-253 Available from: https://www.census.gov/library/publications/2015/demo/p60-253.html (Accessed 7 March 2017)
  7. World Bank Universal Health Coverage Overview. Website available from: http://www.worldbank.org/en/topic/universalhealthcoverage/overview#1 (Accessed 3 March 2017).
  8. French Government Public Services Website (in French). Available from: https://www.service-public.fr/particuliers/vosdroits/F34047 (Accessed 7 March 2017)
  9. United Nations Sustainable Development Goals (Health). Website available from: http://www.un.org/sustainabledevelopment/health/ (Accessed 3 March 2017)
  10. United Nations Draft Resolution (A/67/L.36) Global health and foreign policy Global health and foreign policy. Available from: http://www.un.org/ga/search/view_doc.asp?symbol=A/67/L.36&referer=http://www.un.org/en/ga/info/draft/index.shtml&Lang=E (Accessed 3 March 2017)
  11. World Health Report 2013. Available from: - http://apps.who.int/iris/bitstream/10665/85761/2/9789240690837_eng.pdf?ua=1) (Accessed 3 March 2017)
  12. World health statistics 2012. Geneva, World Health Organization, 2012.
  13. Health Intervention and Technology Assessment Programme (Thailand). Website available from: http://www.hitap.net/en/organization/background (Accessed 3 March 2017)

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April 12, 2017
Antibiotic resistance and the challenges of conducting new antibiotic clinical trials

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Deborah's testimonial on her heart failure and how it has affected her life

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Antibiotic resistance and the challenges of conducting new antibiotic clinical trials - Patient Research Exchange

Antibiotic resistance and the challenges of conducting new antibiotic clinical trials

Nowadays, antibiotics are over-used leading to bacteria who become resistant to drugs. In order to avoid an antibiotic resistance, there are ways to contribute against resistance.

Antibiotics are essential medicines for preventing and treating infections caused by bacteria in both humans and animals. Antibiotics not only save lives when dangerous infections develop but they also allow doctors to perform complex surgery and organ transplants where infection is a major risk. In addition, they have played a major role in improving the care of newborn babies and people with cancer whose immune systems are too weak to fight off infections by themselves1.

However, bacteria have developed many ways of adapting to survive the effects of antibiotics. They become ‘resistant’, and infections that were once easy to control become far more difficult ­– or even impossible – to treat2. Antibiotic resistance is growing at an alarming pace around the world, especially in bacteria that cause common infections such as urinary tract infections and pneumonia3. The World Health Organization (WHO) believes that antibiotic resistance is now one of the biggest threats to health worldwide.2
There are several reasons for this worrying situation4. Antibiotics have been over-used and misused for decades. In some countries they are available for people to buy without a prescription and are often wrongly used to treat infections such as winter coughs and colds, which are caused by viruses, not bacteria5. The more people are exposed to antibiotics unnecessarily, the faster the bacteria evolve to become resistant to them. The situation has been made worse by the widespread use of antibiotics in farming, leading to resistant bacteria transferring from animals to humans and antibiotics getting into the environment.4
Tacking the global threat of antibiotic resistance has become a major focus for many governments and other organizations interested in human health. Ways to restrict the use of antibiotics have been proposed, and the urgent need to increase the number of effective antibiotics in the race against resistant ‘super-bugs’ has been highlighted.6 The development of new antibiotics has helped to fight resistant bacteria in the past, but very few new options have become available in recent years and less than 50 potential new antibiotics are currently being tested – a very small number considering that most drugs undergoing testing are not successful.8
There are several reasons why sufficient efforts have not been focused on finding new antibiotics to treat resistant bacteria. Some of these are economic, but many relate to difficulties in conducting clinical trials of potential new antibiotics and getting them approved.

Why has this happened?

What can be done?

What are the main challenges in developing new antibiotics?

Trials of new antibiotics in suitable patients are expensive to run and are thought to account for more than 80% of the cost of antibiotic development.6Drug companies are not permitted to conduct trials comparing new antibiotics with placebo (‘dummy’) medications but instead need to prove that a new antibiotic is more effective than an existing one.6Since the differences between the new and old antibiotic may be fairly small, large numbers of patients need to be enrolled in clinical trials to detect such differences.6

Hundreds of hospitals can be involved in testing a new antibiotic, presenting many practical and legal problems.9 In addition to these difficulties, almost every country in the world outside of the European Union requires new antibiotics to be registered with them individually before doctors in that country can prescribe them.6 This means that, if a new antibiotic is to be made available worldwide, applications for approval must be presented separately to up to 170 different medical authorities – a slow and expensive process. 6

To make it easier and less costly to develop new antibiotics, it has been proposed that research teams club together to form clinical trial networks.6,9 If all studies were conducted in a similar way, it may be possible to share results between studies, meaning fewer people would need to take part. In addition, simpler ways of getting antibiotics approved worldwide would help to encourage the development of new antibiotics.1 Progress has already been made towards this goal in the USA, Japan and Europe.6
Alongside efforts to speed up the availability of effective new antibiotics, it is essential that the public, healthcare professionals, politicians and the drug and farming industries work together to raise awareness of the impact of antibiotic resistance on human health. Greater understanding of this should lead to:6
Each of us at our own level can contribute to efforts against antibiotic resistance. As a patient, actions you can take include the following:
The world has at last woken up to the dangers ahead if we don’t all work together to stop the spread of antibiotic resistance and develop effective new drugs. The WHO and the United Nations have both made tackling antibiotic resistance a high priority. By using antibiotics more wisely – and joining forces to develop new antibiotics – governments, healthcare professionals and the public can turn this global crisis into a much more manageable situation.

What can we do now?

  • Improved hygiene and infection control practices in homes, hospitals and other medical settings
  • Appropriate restrictions on use of antibiotics in medicine and farming
  • Improved global monitoring and surveys of drug resistance and antibiotic use
  • New, rapid tests to identify exactly what is causing an infection to avoid giving an antibiotic which might not work
  • Greater funding of antibiotic research, including clinical trials

What can I do, as a patient?

  • Ensure you discuss with your doctor whether antibiotics are the right treatment for you in a given situation
  • Discuss alternative options to antibiotics with your doctor, if you believe this would be suitable
  • Be mindful of the amount of times you are taking antibiotics as a treatment. If you are wondering whether it is too frequent – ask your doctor
  • Ensure you follow correct hygiene measures to limit the spread of infection. This applies in particular when you are in any medical environment such as the hospital or the doctor’s office, but also at home or at your workplace. Wash your hands frequently and stay home if you are ill to avoid spreading contagious germs
  • For more resources & information, consider checking the World Health Organization (WHO) or your national medical system (for example the NHS in the UK), and talk to your healthcare professionals

Sources:

  1. Gould IM, Bal AM. New antibiotic agents in the pipeline and how they can overcome microbial resistance. Virulence 2013;4(2):185-91.
  2. World Health Organization. Antibiotic resistance fact sheet. October 2016. Available from: http://www.who.int/mediacentre/factsheets/antibiotic-resistance/en/ (Accessed 27 February 2017).
  3. World Health Organization. Antimicrobial Resistance: Global Report on Surveillance 2014. Available from: http://apps.who.int/iris/bitstream/10665/112642/1/9789241564748_eng.pdf (Accessed 27 February 2017).
  4. Ventola CL. The Antibiotic Resistance Crisis. Part 1: Causes and Threats. P & T 2015;40(4):277-83.
  5. Centers for Disease Control and Prevention. Antibiotic Resistance Threats in the United States, 2013. Available from: https://www.cdc.gov/drugresistance/threat-report-2013/ (Accessed 27 February 2017).
  6. O’Neil J on behalf of the Review on Antimicrobial Resistance.Tackling Drug-resistant Infections Globally: Final Report and Recommendations. Available from: https://amr-review.org/sites/default/files/160518_Final%20paper_with%20cover.pdf (Accessed 27 February 2017).
  7. Bartlett JG, Gilbert DN, Spellberg B. Seven ways to preserve the miracle of antibiotics. Clin Infect Dis 2013;56(10):1445-50.
  8. O’Donnell P. Antibiotic-resistance Focus Could Benefit Clinical Trials. Available from: http://www.appliedclinicaltrialsonline.com/antibiotic-resistance-focus-could-benefit-clinical-trials (Accessed 27 February 2017).

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How heart failure changed my life

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How heart failure changed my life - Patient Research Exchange

How heart failure changed my life

Deborah's testimonial on her heart failure and how it has affected her life

The day that changed my life forever

I’m a 45 year old English woman, married to a Dutch man, and we have a 15 year old daughter. In 2010, we moved to the Netherlands where I worked as a teaching assistant and had a pretty normal life: I loved riding my bicycle, going shopping… I never had major health problems, occasionally I caught a cold but that’s it. I had never considered the possibility of heart failure.

On March 23rd, 2013, I remember waking up with a really strange feeling in my throat, almost as if someone inside was pulling at something… It was something I’d never felt before. As the day went on it got worse, so I decided to seek medical advice. My doctor recommended I take a paracetamol and go home to rest, as it was likely I had the flu.

On the Saturday evening, my state got worse, I started to have a burning feeling in my back and pain in my jaws. I called the doctor again but received a similar recommendation as before. Soon after I started vomiting. My husband was really concerned as he knew something was seriously wrong, so he once again called the doctor to make an appointment. The doctor listened to my chest, put the ECD machine on, and within seconds, it was clear that there was a major problem with my heart. He called an ambulance and the heart unit, and within half an hour, I was in the hospital: I was having a heart attack.

Atypical symptoms

Looking back, these were atypical symptoms of a heart attack. It is really difficult to detect the symptoms of heart attack, as they vary from person to person. The common symptoms are usually chest pain, upper body discomfort, shortness of breath, breaking out in a cold sweat, feeling unusually tired for no reason.1

I always thought that a heart attack would manifest with the typical “pain in the chest”, as seen on TV. But actually, I had none of these “typical” symptoms that would have pointed to an issue with my heart. Instead, I started with the strange feeling in my throat, followed by the burning feeling in my back, jaws and vomiting – strange symptoms for what was originally thought to be the flu.

New lifestyle

Following my return home from the hospital, my life changed. It was as if I had aged overnight and went from being 45 to being 90. I still try to do what I can, but it’s not always easy – even the smaller tasks take it out of me: I used to be able to cycle 60 km every day and now I can hardly spend 5 minutes on the bike without getting out of breath or experiencing chest pain.

Going to the supermarket became an exhausting activity for me… even taking a shower is too much from an energy perspective. Making plans with friends is also difficult, I always have to say no as I can’t keep up.

Living with heart failure is to accept the diagnosis. Whilst to start with it was really difficult to understand and accept, once you embrace it and learnt to live with it, you realise you’ve been given a life sentence and not a death sentence

What is heart failure and what does it provoke?

As you have seen, my life changed completely. I did not just have a heart attack, my heart ruptured and was ready to burst, therefore they had to operate. Since then, my heart is fragile and I have heart failure. Most people don’t know what heart failure is and usually confuse it with a heart attack. A heart attack is when the blood flows stops at a part of the heart, damaging the heart muscles. The consequence of this phenomenon can be heart failure, which means that the heart is not pumping enough blood around the body: in other words, the heart cannot keep up with the demands of the body.

In that case, any minimal physical activity is really exhausting. In my case, it took some time getting used to it. Decision making is a way to manage it, having a sense of priorities, and really planning days ahead to know what I need to do and what I can’t do: it’s about accepting and making decisions accordingly.

I really believe that there should be more education, among doctors and the general public, to recognize atypical heart attack symptoms, especially among women. Things might have gone differently if I had been aware of what was happening.

How Pumping Marvellous helped me get through my illness

Since 2014, I am member of Pumping Marvellous – the UK’s patient led Heart Failure charity. It was founded by Nick Hartshorne-Evans, a heart failure patient himself, whose experiences have shaped the Foundation’s goals and principles.

Pumping Marvellous has been a huge source of support for me. I was recently screened for a heart transplant and I am excited to say that I will be put on a transplant list. I will start by going to a cardiac rehab center to get my body up to scratch, and without Pumping Marvellous, I wouldn’t have made it through this experience!

I’ve been highly involved with the charity, regularly around to support other people and offer any advice that I can give and try to make someone else’s life a little bit easier. Through my work, I want to help make people aware of heart failure and offer to others that little bit of support that I unfortunately couldn’t benefit from 3 years ago. In 2015 I was also able to represent Pumping Marvellous at the European Parliament, presenting a heart failure toolkit.

As an active member of the charity, I was recently awarded the Patient Educator Badge Black which is a huge honour and achievement. The award is only handed out 3 times a year – patients put in their votes, depending on how you represent the charity and the work you have put into it.

Important to remember

I believe the most important thing is for everyone to get the same standard of care, and not for it not to be a “lottery” depending on where you are. For example, the care I received in the Netherlands is very different from what I’ve heard about the care offered in England. In my opinion, it should be the same no matter where you are or what country you’re in.

My advice for other people living with heart failure is to accept the diagnosis. Whilst to start with it was really difficult to understand and accept, once you embrace it and learnt to live with it, you realise you’ve been given a life sentence and not a death sentence. If you see it like that, you will better be able to live with it.

Deborah Budding is, a wife and a mother, suffering from heart failure, also black patient educator for the Pumping Marvellous Foundation and if she can save one person going through what she went through, she will be happy.

Sources:

  1. https://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0062989/

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Listening to patients and what matters to them

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The use of Patient Reported Outcomes (PROs) in clinical research

by Patient Research Exchange
October 21, 2016
Information sharing in medical research: Who owns my data?

Information sharing in medical research: Who owns my data?

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From clinical trial to available medicine - why does it take so long - Patient Research Exchange

From clinical trial to available medicine - why does it take so long

How patients are able to access medicines after they’ve been through a clinical trial

More than 20,000 clinical research studies begin each year.1 Previous articles have described how a series of three or more clinical research studies answer questions about the safety of a potential new medicine and how well it works. It often takes as long as 10 years to collect all the information needed from the studies – or even longer if the disease being studied develops very slowly; or if it is difficult to find suitable people to take part.1 Evidence collected in clinical research studies is used to apply for legal approval for the use of new medicines;2 however, legal approval does not necessarily mean the medicine becomes available for doctors to prescribe.

Ensuring medicines are made available at a reasonable cost

Government agencies, healthcare providers and pharmaceutical companies have a shared goal of making safe and effective medicines available to patients who need them as soon as possible. After the legal approval of a new medicine, these organisations work together to understand whether – and in which specific types of patients – the medicine is good value for money. This may include learning more about how the medicine works in the ‘real world’ – i.e. outside the tightly controlled conditions of a clinical research study. Participants taking part in this sort of research may gain personal benefits from accessing approved medicines earlier than they otherwise could.

Most countries have a separate approval step in which the health authorities decide whether they are willing to pay a form of subvention for the new medicine.3,4 The typical length of time between legal approval and the point at which a new medicine becomes available to patients varies from 6 weeks in the United States, to 21 weeks in the United Kingdom and 66 weeks in Italy.5 So what takes so long, and why do some countries take longer than others to make a new medicine available?

Who decides which medicines are made available to patients?

The bodies, known as payors, that pay for prescription medicines vary from country to country. Usually, the cost of medicines is covered by a publicly funded drug plan at national or regional levels, a privately funded drug plan, individual patients, or a combination of these.4Available funding may be limited, for example, by budget allocation or potential impact of the drug plan, so decisions need to be made as to which medicines to fund, often through a process that assesses the added value of a new medicine.

For patients who may benefit from a medicine that is legally approved but not yet available in their country, a few options are available. The possibility for this to occur as well as the different options available will vary from country to country depending on local laws and regulations.

Although the legal approval of a medicine is usually based only on whether it works and is safe, payers are also interested in other information, such as how much additional benefit may be gained from the new medicine compared with the best treatment(s) already available. They want to understand the long-term cost to society, of buying the medicine, compared with potential future cost savings, such as avoiding hospital admissions or reducing the risk of more serious medical conditions. For example, paying the price of the medication may outweigh the cost of having to support a large number of patients through extended periods of hospitalization. It may also be important to learn whether patients outside of a clinical research study have any problems with taking the medicine as instructed. With limited budgets, payors may also consider the value of a medicine for one condition versus another medicine for a different condition, or the overall budget impact of a new medicine. Other factors may include the severity of the condition or unmet need.

The information needed by payors is sometimes, but not always, available from the original clinical research studies.6 Drug manufacturers may also submit economic analyses based on projected costs and benefits. To help collect more information about the costs, risks and benefits of the medicine in a “real-world” healthcare situation for a particular country, a healthcare provider may make the medicine available for a short time (a year or two) while it is studied further – this is sometimes called ‘provisional’ or ‘conditional’ funding.6 As countries have different ways of funding medicines, and different ways of judging value for money, it is usual for medicines to become available to patients at different times.5

Is there a way to access medicines that are legally approved but not yet funded?

For patients who may benefit from a medicine that is legally approved but not yet available in their country, a few options are available. The possibility for this to occur as well as the different options available will vary from country to country depending on local laws and regulations. But below are some indications of how this could work.

  • If no other approved treatment seems suitable, a doctor may be able to apply for special funding of the new medicine as a ‘one-off’ case.7,8
  • Patients who have previously had successful treatment with a medicine as part of a clinical research study may be allowed to continue to take that medicine until it is available for their doctors to prescribe. However, this is not always the case.9
  • Patients may participate in studies known as ‘early access programme’, or ‘post-approval study’, which are different from earlier clinical research because participants will not usually need to undergo any additional medical tests beyond those they would receive as part of their routine medical care. Participants will also know exactly which medicine they are receiving, and that the study medicine has previously been shown to work. These studies are very important because they help researchers to understand how the medicine works for a wide range of people, which may include people with more complex medical needs than those included in the original clinical research studies10
  • Because clinical trials are conducted on a selected sample of patients, even after approval, there may be some “uncertainty” as to which patients in the total population will actually benefit. Manufacturers may enter into “managed access/entry agreements” with payers whereby patients who might benefit are provided access through a registry and only those who respond received continued access through the drug plan. Over time, the access criteria will change to reflect learning from the registry.11

Sources:

  1. Viergever RF, Keyang Li. Trends in global clinical trial registration: an analysis of numbers of registered clinical trials in different parts of the world from 2004 to 2013. BMJ Open 2015;5:e008932
  2. How Drugs are Developed and Approved. United States Food and Drug Administration. Available from: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ (Accessed 1 November 2016)
  3. Saltman D. An Introduction to European Market Access. Seminar Public Health and Primary Care, Imperial College. Available from: https://www1.imperial.ac.uk/resources/7F5B8EAC-E83A-4C71-9192-7BACDFCAFBA7/europeanmafeb27.pdf (Accessed 1 November 2016)
  4. ISPOR Global Healthcare Systems Road Map, February 2015. Available from: http://www.ispor.org/htaroadmaps/ (Accessed 1 November 2016).
  5. Mycka J, Dellamano R, Lobb W et al. Regulatory Approval to Patient Access, and Evaluation of EU5 and US National Timing Differences. ISPOR 6th Asia-Pacific Conference September 6-9 2014
  6. Jarosławski S, Toumi M. Market access agreements for pharmaceuticals in Europe: diversity of approaches and underlying concepts. BMC Health Services Research 2011;11:259
  7. Individual funding requests - A guide for patients and service users. NHS England, 2015. Available from: https://www.england.nhs.uk/commissioning/wp-content/uploads/sites/12/2015/06/individl-fund-reqts-info-pats.pdf (Accessed 1 November 2016)
  8. NHS England Cancer Drugs Fund, 2016. Available from: https://www.england.nhs.uk/cancer/cdf/ (Accessed 1 November 2016)
  9. Cedars-Sinai, 2016. Available from: https://www.cedars-sinai.edu/Patients/Clinical-Trials/Clinical-Trials-Frequently-Asked-Questions.aspx (Accessed 8 December 2016)
  10. Patil S. Early Access programs: Benefits, challenges, and key considerations for successful implementation; Perspect Clin Res; 2016 Jan-Mar; 7(1):4-8
  11. Charles River Associates, Managed entry agreements in the context of medicines adaptive pathways to patients Nov 2016, http://adaptsmart.eu/wp-content/uploads/2016/12/CRA-MEA-in-the-context-of-MAPPs-Final-Report-16-December-2016-STC.pdf

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From molecule to medicine: How are new drugs developed?

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From Sherlock Holmes and Dr. Watson to Abbott and Costello, we know that partnerships between opposites can lead to solved mysteries and hilarious laughs.

by Editorial Team
Making an impact in mental health care and research - Patient Research Exchange

Making an impact in mental health care and research

Mental health problems in our society

About 1 in 4 people live with mental health problems that affect their thoughts, emotions, behaviour, or the way they relate to others1. Depression, schizophrenia, anxiety, autism and dementia are examples, although many people live with problems that have not yet been named or diagnosed. There are effective treatments for many mental health problems, along with techniques to reduce their impact on a person’s day-to-day life. Unfortunately, up to half of all people with mental health problems in developed countries receive no treatment2. This may be because they have decided treatment is not right for them, or because their healthcare system is not able to give them the support they need. As mental health problems are experienced differently by different people, it is important that treatment plans are flexible and designed to support the particular needs of each individual.

Putting the person at the centre of mental health care

Only 50 years ago, mental health care was very different from how it is today. For example, it was usual for people with even mild mental problems to stay in hospital while receiving treatment3. Sometimes people with mental health issues were not treated very considerately or respectfully. Thankfully, we now have a much better understanding of medicines and other therapies (such as counseling), as well as the conditions themselves, and people are generally able to live at home while being treated.

If more specialist care is needed, this may be given in a specially designed care facility. Care facilities are much improved from the mental health hospitals of the past but could be improved even more if patients themselves are consulted on the design. For example, a journalist who had received mental health care herself reported that every door in the facility where she was treated had round metal doorknobs that were difficult to use with sweaty palms4;– a common symptom of anxiety and a side effect of some medicines prescribed by psychiatrists2 . Had patients been consulted on this design feature, it’s likely they could have pointed out its unsuitability. Taking medicines at the right time can also be problematic for people with memory difficulties or disordered thoughts – by discussing these difficulties with patients, evidence shows it is possible to find ways to overcome them, for example using pill boxes or text message reminders5.

Research has helped us to understand the causes and possible treatment of mental health problems. Both in routine health care, and when designing research, we now recognise the importance of getting the input of patients themselves

A ‘patient-centred’ or ‘person-centric’ approach means all aspects of healthcare are designed to meet the needs and preferences of patients6. These terms are used widely by government, doctors and patient organisations, who believe it is important to involve people in decisions about their care, with health seen as part of life as a whole7 . In a large survey of people with mental health problems, the majority said that they wanted to be more involved in decisions about their own care8. Where care is more person-centred, people receiving treatment (and their doctors and nurses) tend to have a better overall experience9.

Getting involved in mental health research

Research is an important part of designing future healthcare; without it, no new medicines and few medical advances could be made. Although research in mental health is just as important as research in conditions like cancer or diabetes, researchers planning a mental health study often struggle to find enough people to take part10. However, when a group of people with depression or schizophrenia was asked if they would be willing in theory to take part in research, almost all said yes11. Could part of the reason for low participation in mental health research be that most people are not aware of opportunities open to them6? Could it also be that people with mental health problems don’t always feel that the studies available are designed with their needs in mind12?

In the UK, a government group called the Mental Health Research Network wants to involve more patients in designing mental health research. This is because it’s been shown that if patients have been involved in designing a study, other patients are far more likely to want to take part8. People involved in designing research also say they feel they have made an important contribution to society7. Helping to design research might include giving an opinion on the treatment(s) being studied, the number of times participants need to visit the clinic, the responsibilities of participants between clinic visits, or the information written for study participants. Another important way to contribute to research is to help researchers to understand what kind of improvement they should look for in the people taking part in the study, because the sort of change seen as ‘meaningful’ often differs from person to person.

Could you or someone you know change the future of mental health care?

In the past 50 years, mental healthcare has changed a lot. A big part of this change is due to research that has helped us understand the causes and possible treatment of mental health problems. Both in routine health care, and when designing research, we now recognise the importance of getting the input of patients themselves. By being given more information and opportunities to make decisions, people often feel more satisfied with the treatment they receive, and may also help to improve care for other people in the future.

Sources:

  1. Fundamental Facts About Mental Health. Mental Health Foundation, 2015. Available from: https://www.mentalhealth.org.uk/publications/fundamental-facts-about-mental-health-2015
  2. Mental Disorders Fact Sheet. World Health Organization, 2016. Available from: http://www.who.int/mediacentre/factsheets/fs396/en/
  3. Chow WS, Priebe S. Understanding psychiatric institutionalization: a conceptual review. BMC Psychiatry 2013;13:169
  4. Mental Health Patients Facilities Design Out Stress. The Guardian, 2014. Available from: https://www.theguardian.com/society/2014/jun/03/mental-health-patients-facilities-design-out-stress
  5. Barkhof E, Meijer CJ, de Sonneville LMJ, et al. Interventions to improve adherence to antipsychotic medication in patients with schizophrenia–A review of the past decade. European Psychiatry 2012;27(1):9-18
  6. Patient-centred healthcare. International Alliance of Patients’ Organizations, 2016. Available from: https://www.iapo.org.uk/patient-centred-healthcare
  7. Starfield B. Is Patient-Centered Care the Same As Person-Focused Care? Perm J. 2011 Spring; 15(2): 63–69.
  8. Survey of mental health inpatient services. Care Quality Commission, 2009. Available from: http://www.nhssurveys.org/Filestore/documents/MH09_RV5.pdf
  9. Crawford,M. et al. Systematic Review of involving patients in the planning and development of healthcare. British Medical Journal. 2002;325:1263-1267.
  10. Mental Health – How much does the UK spend on research? Welcome Trust, 2015. Available from: http://blog.wellcome.ac.uk/2015/04/21/mental-healthhow-much-does-the-uk-spend-on-research/
  11. Schäfer I, Burns T, Fleischhacker WW, et al. Attitudes of patients with schizophrenia and depression to psychiatric research: a study in seven European countries. Soc Psychiatry Psychiatr Epidemiol 2011;46:159–65
  12. Woodall A, Morgan C, Sloan C, et al. Barriers to participation in mental health research: are there specific gender, ethnicity and age related barriers? BMC Psychiatry 2010;10:103.
  13. Ennis L, Wykes T. Impact of patient involvement in mental health research: longitudinal study The British Journal of Psychiatry 2013;203:381-386.

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Social media and the future of medicine - Patient Research Exchange

Social media and the future of medicine

The use of social media in the medical field will continue to increase over the next few years

“Social Media” has become a major trend in recent years. Do we really know what it means? Twitter and Facebook probably come to mind first. In fact, social media, is a tool that allows for the sharing of information in virtual networks. Even text messaging tools such as WhatsApp can be considered a social medium, meaning that people claiming not to be into social media are using it almost daily. It is no longer a toy for the young generations. Social media has also made its way into the Healthcare industry, an interesting example is Dr. Henry Heimlich, cardiovascular surgeon and inventor of the Heimlich Maneuver. Guess where I found out about it? On his twitter feed! He is 96 (!) years old and active on social media. The most fascinating part: I can now directly interact with him on this platform. Social media has made people more accessible than ever, allowing you to talk to peers you may never have met otherwise.

Information overload?

But I share some of the concerns voiced by the critics, this is especially the case for patients. Social media can be very overwhelming, distracting and even addictive. Most non-users argue they simply don’t have the time, but this is precisely the wrong argument. With social media, we have a new technology at hand that we have to use to our advantage, like other previously ground-breaking technologies. Patients can use social media to get information on their symptoms and conditions, as well as interact with physicians and other health care professionals. The information is not always valid, so it is important to primarily rely on trustworthy sources, such as hospitals, health care organizations like WHO, publishers like the BMJ, and expert physicians.

However, patients have to learn how to use social media optimally. New generations will grow up with it, with the risk of using it more for fun, making it a “time waster”. Older generations need a more formal introduction probably. So should we teach the optimal use of social media? Probably yes.

Technology is rapidly evolving and the sharing of medical scientific information is moving from print and on-site presentations to digital online publication (webinars, etc.), social media is the ideal platform for this development, from hosting and sharing information to enabling interaction

Keep your finger at the pulse of clinical science - no matter where you are

I was unfortunately unable to attend this year’s major cardiology conference, the American College of Cardiology Annual Scientific Sessions (ACC) due to clinical commitments. I am sure many other colleagues have similar struggles, considering the increasing number of conferences, commitments, etc. However, I followed the meeting in real time online as much as possible via Twitter and even became one of the top social media influencers of the meeting according to statistics. I was able to discuss new research with peer experts, study authors and even with patients. Often, papers got published simultaneously in peer-reviewed journals, and these can be included in the online discussion and regarded as a novel form of post-publication peer-review.

The ACC 2016 sessions were attended by an incredible number of ~20,000 health professionals. Via social media, patients were also able to follow the news. The digital reach of this meeting was gigantic: 6,733 actively tweeted about the meeting and the tweets were read 140,562,727 (!) times.

Patients are online as well

Of course as a doctor, I have certain thoughts and opinions that might differ from the patients’. Therefore I asked Kaz Aston, a multiple sclerosis (MS) patient and engaged campaigner for this condition, to provide her point of view.

According to Kaz, at a time when any subject can easily be discussed via social media, it makes perfect sense that health related topics are part of the conversation and talked about not only amongst patients, but also between patients and healthcare professionals. Social media has created a community at the tip of your fingers. This can help improve public health globally in settings such as hospitals or in local communities.

Kaz explained that over the last two years, patient conferences have backed the use of social media to support improved engagement. These types of events have become very popular amongst patients and healthcare professionals and are routinely featured at global medical conferences. Today most healthcare patient events accommodate live social media streams that are linked to designated hashtags or conference websites which support collaboration between attendees and people who are following the conference remotely. The latest numbers for one day events have rendered online audiences of over 25 million people while simultaneously delivering worldwide trending hashtags. Kaz explained that the use of social media in healthcare has promoted patient adherence and communication and has received a lot of positive feedback.

To summarize, I want to emphasize that as information technology is rapidly evolving and the sharing of medical scientific information is moving from print and on-site presentations to digital online publication (webinars, etc.), social media is the ideal platform for this development, from hosting and sharing information to enabling interaction. No one should feel obliged to use social media, but I strongly advise colleagues to embrace this technology, it is very useful and once you know how to use it, it is great fun too!

Pascal is an internationally trained cardiologist. He trained in Switzerland, the US and then worked as a consultant in London and at Yale Medical School. His focus is on percutaneous heart procedures, such as coronary angioplasty, aortic valve replacements, etc. He is also the editor-in-chief of the scientific cardiology journal Open Heart (British Medical Journal Group).

Links

www.drpascalmeier.com

Twitter: pascalmeier74

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April 22, 2016
Listening to patients and what matters to them

Listening to patients and what matters to them

The use of Patient Reported Outcomes (PROs) in clinical research

by Patient Research Exchange
July 7, 2016
Health Technology Assessments: Getting the Right Balance in Medicine

Health Technology Assessments: Getting the Right Balance in Medicine

Scientists spend many years collecting evidence about new health technologies to show they work and are suitable to use in people. However, showing that a health technology works is only part of the story!

by Patient Research Exchange
August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
Pascal Meier - Patient Research Exchange

Pascal Meier

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Is it safe to take medicines that are still being tested? - Patient Research Exchange

Is it safe to take medicines that are still being tested?

Medicines and research: the complex procedure behind it

Almost half of all adults regularly take a prescription medicine 1. If your doctor gives you a prescription, it is very likely that the same type of medicine has been taken before by hundreds, or usually thousands, of other people with the same condition as you. Your doctor will be able to tell you how the medicine is expected to work, and what side effects are most often experienced by people who take it.

This information is available because it has been collected in clinical research studies in which volunteers have taken the medicine while being carefully monitored by doctors. Medicines that are taken as part of clinical research studies (which are also known as ‘experimental’ or ‘investigational’ medicines) are less well understood than most of those prescribed, because there are still questions that need to be answered about their safety and how they work.

How is patient safety ensured in research studies?

Government health authorities – the European Medicines Agency in Europe or the Food and Drug Administration in the USA – are responsible for approving medicines to be used in clinical research studies2. Before a research medicine can be taken by people, the health authority looks for evidence that the risk of serious side effects is low and that there is a good chance the new medicine will have benefits. This may include evidence that shows the medicine can be given to animals without serious harmful effects. The first people to take a research medicine are usually healthy volunteers who are given a very small amount of the medicine. The dose is increased very slowly and carefully. As more and more is learnt about the medicine and how it works, larger studies with more people may begin. At each stage of research, the development of the new medicine will be stopped if serious safety problems are found.2

If a research medicine has a high chance of causing harmful side effects, this will almost always be detected the very first time it is given to humans. Of 17,000 early (Phase I) research studies completed in the past 10 years, very few have reported serious problems 3,4. However, in 2006, six young men in this early stage of research experienced life-changing side effects because of taking an investigational medicine5. As a result, the health authorities made strict new rules to ensure such problems were very unlikely to happen again, such as increasing the amount of information that must be known about the medicine before it is given to humans at the dose thought to be needed for treating a disease6,7.

People taking part in clinical research studies are protected by strict laws, and their well-being is the highest priority for study doctors and nurses. By taking part in a research study, you may be able to benefit from early access to medicines that aren’t yet available outside a study.

How is the safety of a medicine tested before it is given to humans?

Studies of a medicine before it is given to humans are called ‘pre-clinical’ or basic research. Much of this research is either done using animal cells in a laboratory, or by giving the potential new medicine to an animal. Animal studies are carefully controlled and only done when needed; scientists are working to reduce the need for animal testing without increasing the risks to people in clinical research studies8.

Another way to research the activity of medicines in humans is called ‘microdosing’2. This involves giving healthy human volunteers very small amounts of the medicine (much less than would be given to treat a disease). This very low dose is unlikely to pose a serious risk to the volunteer, but may be helpful to scientists who can track the medicine as it moves around the body (it is labelled with a marker that can be seen on a scan). Computers are also playing an increasingly important role in the research of new medicines9,10. Using information about the chemical structure of a medicine, computers can predict how it will be taken into the body and how it interacts with living cells. Currently, computer models are limited by our understanding of diseases – as we learn more, predictions will become more accurate. These research methods may improve the safety of early clinical research studies, and reduce the need for some animal testing.

What are the risks and benefits of taking part in research?

Every year hundreds of thousands of people take part in clinical research studies, and serious side effects are rare. Considering whether to take part in research involves weighing up the possible benefits versus the known and unknown risks11. The risk of taking medicines as part of a research study may be higher than taking a prescribed medicine, although a lot is already known about the research medicine before it is given to people.

People taking part in clinical research studies are protected by strict laws, and their well-being is the highest priority for study doctors and nurses. By taking part in a research study, you may be able to benefit from early access to medicines that aren’t yet available outside a study. You will also be making an important contribution to medical knowledge, which could improve the treatments people receive in future.

Sources:

  1. Almost half of all adults take prescription drugs. NHS, 2014 Available from: http://www.nhs.uk/news/2014/12December/Pages/Almost-half-of-all-adults-take-prescription-drugs.aspx (Accessed October 2016)
  2. The FDA’s Drug Review Process: Ensuring Drugs Are Safe and Effective. The United States Food and Drug Administration. Available from: http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htm (Accessed October 2016)
  3. A search for all completed Phase I studies registered on ClinicalTrials.gov since 1 January 2006 returned 17396 entries (Accessed 28 October 2016).
  4. Emanuel EJ, Bedarida G, Macci K, et al. Quantifying the risks of non-oncology phase I research in healthy volunteers: meta-analysis of phase I studies. BMJ 2015;350:h3271
  5. Northwick Park drug trial disaster could it happen again? BBC Health, 2016 Available from: http://www.bbc.co.uk/news/health22556736 (Accessed October 2016)
  6. European Medicines Agency 2008, ICH Topic M 3 (R2) Non-Clinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals.
  7. Milton MN, Horvath CJ. The EMEA Guideline on First-in-Human Clinical Trials and Its Impact on Pharmaceutical Development. Toxicologic Pathology 2009; 37:363-371.
  8. European Union 2010, Protection of animals used for scientific purposes. EU Directive 2010/63/EU
  9. Virtual Drug Tests, Bayer Pharmaceuticals Available from: http://pharma.bayer.com/en/innovation-partnering/technologies-and-trends/research-technologies/virtual-drug-tests/ (Accessed October 2016)
  10. Lounkine E, Keiser MJ, Whitebread S, et al. Large-scale prediction and testing of drug activity on side-effect targets Nature 2012; 486, 361–367.
  11. Advantages & drawbacks of taking part in a clinical trial. Cancer Research UK, 2015. Available from: http://www.cancerresearchuk.org/aboutcancer/findaclinicaltrial/whatyoushouldbetoldaboutaclinicaltrial/advantagesanddrawbacks (Accessed October 2016)

Other Stories

September 27, 2016
Making research everyone’s business

Making research everyone’s business

The expertise of patients and carers is increasingly recognised as an essential component of research and no longer considered just a "nice to have".

by Simon Stones
April 12, 2016
Their future in our hands: clinical research in children

Their future in our hands: clinical research in children

Children are not ‘mini adults’. That's why it is so important to conduct clinical research in children.

by Patient Research Exchange
April 21, 2015
Patients Becoming Partners in Research

Patients Becoming Partners in Research

When it comes to clinical trials, everyone knows the drill: experts create and patients participate. Right?

by Editorial Team
Information sharing in medical research: Who owns my data? - Patient Research Exchange

Information sharing in medical research: Who owns my data?

Data in clinical research studies are helping to improve our lives in many ways

Information is big business in the modern world. Information technology is increasingly used to analyse our habits: where we go, what we buy and who our friends are. Some argue that this is an invasion of privacy, but it also has the potential to improve our lives in many ways. Your medical history represents some of the most sensitive and personal information that exists about you, so healthcare professionals are very careful about protecting it. Your consent is needed before it can be shared with anyone.

People taking part in clinical research studies agree to ‘donate’ their health information to doctors and research organisations to help them better understand the risks and benefits of medicines and other medical treatments being studied. A large amount of personal information is collected within a clinical study, such as blood test results (e.g. blood sugar and cholesterol levels), measurements of weight, heart health and blood pressure, medical images from scans and information about mood and mental health. This information is known as data. Once the study is complete, data collected from each individual are combined to enable scientists to carry out an analysis of all the data and answer specific research questions.

Who owns clinical research study data and how is it used?

For most clinical research studies, data are legally owned by the study sponsor (the organisation(s) that paid for the study to be conducted), which is usually a medicines company or an academic institution like a teaching hospital or school of medicine. Scientific and medical communities agree that the owners of the data have a responsibility to announce the main results from a study (whether these were positive, negative or neutral) as soon as possible after they are known1,2. Study results are usually announced at scientific congresses and in journal articles which present overall summary results (e.g. totals or averages). These do not identify results for individual participants (the very large volume of data collected in a typical clinical research study is one of the reasons why up to half the data tends remain unpublished)3. However, it is increasingly thought that study sponsors should share the full details of individual patients’ results with researchers, on request, as long as every participant’s identity is kept anonymous to protect their privacy3,4. This is because it is believed that ‘raw data’ (data from individual participants which hasn’t yet been processed or analysed) may be valuable to scientists planning future research.

Opening access to data has become normal in most areas of life, sharing all the raw data will help to gain the full benefit from the important contribution made by study participants

In recent years, the idea of data sharing has become more and more prominent and it has become easier to access the full results of research studies and not just the analysis and interpretation that the researchers choose to publish5. One example, the International Stroke Trial (1991-96), was a large study with nearly 20,000 participants. This study collected an enormous amount of raw data, which have since been made anonymous and accessible by other researchers6. Increasing use of crowd-sourced health data (where whole communities of people are asked to provide answers to simple questions about their health) and electronic medical records provide opportunities to study real-life medical situations outside traditional clinical studies7,8. Crowd-sourcing also raises the possibility that research results could ultimately be jointly owned and shared by the people who contribute the data, not just by study sponsors9.

What does data sharing mean for clinical trial participants?

Balancing the scientific importance of using and sharing data with the legal requirement to protect people’s privacy is a priority for research organisations10. When joining a study, participants sign a consent form that explains the various kinds of health information that will be collected during the study, who owns the data, and how it will be stored. You can withdraw your consent to participate at any time, which means that you no longer attend study visits or receive study medication. Usually, data that has been contributed before withdrawing consent will remain part of the study, but no further data will be collected unless you give your permission11. The more data a study can continue to collect, the more valid and useful the results are likely to be11.

Data sharing for the benefit of society

Medical science continually builds on and refines previous knowledge with the help of research findings. In the era of electronic information exchange, when open access to data has become normal in most areas of life, sharing all the raw data collected in clinical studies will help to gain the full benefit from the important contribution made by study participants.

Nick Hartshorne-Evans was diagnosed with Heart Failure in January 2010. His experience as a patient stimulated him into developing the only dedicated patient led Heart Failure charity in the UK – The Pumping Marvellous Foundation. Nick is also President of iHHub, which has attracted significant attention from international partners where it advocates for the global heart failure patient voice and in 6 months has managed to recruit 35 patient groups globally. Nick regularly engages and involves himself in both global, regional, national and local patient advocacy along with delivering patient led collaborative solutions. He is a patient expert with NICE, a mentor within NHS England’s clinical entrepreneur programme, the patient lay voice for the NICE Chronic Guidelines commencing April 2016 and an Honorary Research Fellow at the University of Liverpool’s Management School.

The International Heart Hub (iHHub) is the first global non-profit organisation that brings together and supports the creation of patient groups from every country in the world to raise awareness of heart failure and improve lives. iHHub was founded as an alliance of patient organisations from North America, Latin America, Europe, Middle East and Africa and works from the centre of the global heart failure conversation to amplify the patient voice.

Links:

@pumpinghearts

https://www.facebook.com/heartfailureaware

@GlobalHF

https://www.facebook.com/iHHubGlobalHF

Sources:

  1. Institute of Medicine. Ensuring the Integrity, Accessibility, and Stewardship of Research Data in the Digital Age. Washington, DC: National Academy Press; 2009
  2. Hrynaszkiewicz I, Norton ML, Vickers AJ, Altman DG. Preparing raw clinical data for publication: guidance for journal editors, authors, and peer reviewers. BMJ 2010;340:c181.
  3. Ross JS; Lehman R; Gross CP The Importance of Clinical Trial Data Sharing: Toward More Open Science. Circ Cardiovasc Qual Outcomes 2012;5:238-240.
  4. EFPIA Principles for Responsible Clinical Trial Data Sharing, 2013. Available from: http://transparency.efpia.eu/uploads/Modules/Documents/data-sharing-prin-final.pdf Accessed August 2016.
  5. US Department of Health and Human Services, National Institutes of Health, National Heart Lung and Blood Institute. Biologic Specimen and Data Repository Information Coordinating Center. 2007. Available at: https://biolincc.nhlbi.nih.gov/home/. Accessed August 2016
  6. International Stroke Trial Collaborative Group. The International Stroke Trial (IST): a randomised trial of aspirin, subcutaneous heparin, both, or neither among 19435 patients with a cute ischaemic stroke. International Stroke Trial Collaborative Group. Lancet1997;349:1569 –1581.
  7. Patients Like Me. Website available from: https://www.patientslikeme.com/ Accessed August 2016
  8. Twenty-three and me. Website available from: https://www.23andme.com/en-gb/research/ Accessed August 2016.
  9. Terry SF, Terry PF. Power to the People: Participant Ownership of Clinical Trial Data. Sci Transl Med 2011;3(69):1-4.
  10. National Institutes of Health. Clinical Research and the HIPAA Privacy Rule. Available from: https://privacyruleandresearch.nih.gov/pdf/clin_research.pdf Accessed August 2016
  11. Gabriel AP, Mercado CP. Data retention after a patient withdraws consent in clinical trials. Open Access J Clin Trials. 2011; 3: 15–19.

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August 3, 2015
Clinical Trial Recruiting Tools in the Digital Age

Clinical Trial Recruiting Tools in the Digital Age

Tablets, smartphones, and even self-driving cars likely come to people’s minds when they hear about the information technology revolution affecting the way we work and play.

by Editorial Team
August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
September 22, 2015
Crowdsourcing Clinical Trials

Crowdsourcing Clinical Trials

The notion that too many people involved in a project can ruin it is quite common.

by Editorial Team
Nick Hartshorne-Evans - Patient Research Exchange

Nick Hartshorne-Evans

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Making research everyone’s business - Patient Research Exchange

Making research everyone’s business

The expertise of patients and carers is increasingly recognised as an essential component of research and no longer considered just a "nice to have".

Not that long ago, research was viewed as scary scientists in white coats, injecting solutions into the arms of restrained victims, turning them into Frankenstein. Okay, that’s never really been the case, yet research remains a poorly understood area of healthcare amongst many individuals and groups around the world.

Giving research the importance it deserves

Since the National Institute for Health Research (NIHR) was established in 2006, research really has been placed on the health and social care agenda in the United Kingdom (UK).1 Between 2014 and 2015, more than 260,000 people took part in NIHR research studies, helping to understand new ways of preventing, identifying and treating ill health.2 Although the NIHR has transformed research in the National Health Service (NHS), there’s still a long way to go.

Historically, research was left to the researchers – the experts. Patients were just people who received the treatment or intervention from the professionals, quietly and without question. That’s not the case anymore. The expertise of patients and carers is increasingly recognised as an essential component of research – making it more relevant and cost-effective in the long-term.4

As a patient, it is easy to underestimate the expertise that you have. From treatments, to understanding your disease, and the impact on your quality of life – you have a lot to give.

I first became involved with research in 2012, after attending an open evening for young people with musculoskeletal diseases. There, I met my former paediatric rheumatology consultant, who informed me about a vacancy for a patient representative on the NIHR Clinical Research Network (CRN): Children/Arthritis Research UK Paediatric Rheumatology Clinical Studies Group. It was all a little bit overwhelming as I didn’t really know what it entailed. Although I understood the fundamentals of medicine research and development, I didn’t fully appreciate the complexity and breadth of research. During my first face-to-face meeting on this group, I was sat amongst twenty health professionals and thought ‘How on earth can I contribute the level of expertise that these other people do?’ Four years later, with a vast amount of experience and involvement as a patient representative, my views have completely changed. I’ve grown in confidence to speak about matters important to patients and their families, as well as having a great understanding of research and healthcare in general. However, what hasn’t changed is my personal, lived experience of living with ill health. As a patient, it is easy to underestimate the expertise that you have. From treatments, to understanding your disease, and the impact on your quality of life – you have a lot to give. Although health professionals and researchers are incredibly skilled, unless they’ve lived with certain health problems, it’s impossible for them to fully understand the impact of ill health on your life and that of your family and friends.

Involving the real experts: patients

Since the NIHR was established, there has been an increasing awareness of the positive contribution that patients and the public can make to healthcare research.5 At national and international levels, there are a growing number of individuals and organisations supporting patient and public involvement in healthcare research6-9, encouraging widespread adoption of an integrated model of research between researchers and the public.10-13 Recently, the framework for public involvement in research has been restructured4, to better address the importance of person-centric health research. Historically, the terms consultation, collaboration and user-controlled were used to describe different levels of patient and public involvement in research. However, these have evolved to encompass participation, engagement and involvement in research, representing three different stages of association and commitment, to best meet the needs of individuals contributing to research (Table 1).14

Table 1. Levels of patient and public involvement in health and social care research. Participation, engagement and involvement are three levels of association for patient and public contributors in an integrated model of communication applied to health and social care research.
Table 1. Levels of patient and public involvement in health and social care research. Participation, engagement and involvement are three levels of association for patient and public contributors in an integrated model of communication applied to health and social care research.

Historically, research was viewed as an add-on people only considered when existing treatments had failed. Personally, I envisage research shifting to an integral aspect of modern healthcare delivery, as are consultations and treatment. Whether that be taking part in a clinical trial, completing a questionnaire, becoming a patient research ambassador or being an adviser on a research project. Getting involved in research can help you to learn more about your condition and ways to manage it. It can also boost your confidence – an easy target when you are learning to live with ill health. Research can also help you to have a voice – helping to influence the care you and others may receive in the future.

Patient involvement is more than a “nice to have”

The NIHR stipulates that patients and the public must be involved in all aspects of research, from prioritisation exercises, to designing, conducting, evaluating and disseminating research.5,15 Health and social care research that does not involve patients and the public is now considered flawed. Patient and public involvement should begin at the earliest opportunity. Preferably, any research should be based upon six core values, which integrated teams of researchers and the public must strive to achieve. These are respect, support, transparency, responsiveness, diversity and accountability.16 Ultimately, people who are directly, or indirectly affected by research have a right to say what publicly funded research is undertaken, and the way in which it is conducted. Regardless of profession or experience, individuals designing, engaging and participating in research are contributing to a ‘research active’ nation.16 Researchers must not assume that what they want is what patients want. Researchers, patients and the public have expertise in their own rights, and when such experiences are shared, they can influence research for the better.

Research has helped me to develop a greater appreciation for how we best care for people with ill health. My involvement as a patient representative has evolved to the point where I wanted to pursue a career in research. I am now a PhD student at the University of Leeds, looking at self-management of complex health conditions in children. Despite being at this early stage in my career, I am still a patient. Several people question whether I am a ‘real patient’ now that I am a researcher – something which I certainly take with a pinch of salt. I am still as much a patient as I was before I started my PhD. If anything, I am now more confident in my understanding of my conditions, and I can better articulate the impact and consequences of my health conditions on my daily life.

The NIHR have published their strategic goals, which includes a dashboard of performance indicators to evaluate public participation, engagement and involvement in research.17 Transparency in research practice has been shown to empower people who use health and social care services, providing a route of influencing change and improving issues which concern people the most. The NIHR’s Patient Research Ambassador Initiative and Join Dementia Research Initiative18-19 are two clear examples of how patients and the public are influencing change for the better, whereby patients, members of the public and researchers are at the heart of research, as equal partners. I am proud to be have been involved with the Patient Research Ambassador Initiative, promoting the patient voice, trying to get more people engaged and involved in research. By talking more about research, and helping researchers to better involve patients and families, we can ensure that research is everyone’s business, and high on the agenda in society.

Simon Stones
Simon Stones

Simon is a service-user researcher, having recently graduated from The University of Manchester with a BSc (Hons) in Biomedical Sciences. He is now embarking on a PhD at the University of Leeds, in the field of child health. His research interests include self-management of complex long-term conditions, having learned first-hand what it is like to live with ill health from a young age. Simon was diagnosed with a form of childhood-onset arthritis at the age of three, and has since been diagnosed with fibromyalgia and inflammatory bowel disease. Inspired by his own experiences, Simon is now a health activist, acting as a consumer health advocate and patient research ambassador for people living with long-term conditions, in collaboration with a variety of organisations around the world.

Links:

http://simonstones.wordpress.com

https://uk.linkedin.com/in/simonstones

Sources:

  1. NIHR. (2016). About the NIHR. Available from: http://www.nihr.ac.uk/about/
  2. NIHR. (2016). NIHR at 10. Available from: http://www.nihr.ac.uk/about/nihr-at-10.htm
  3. Office for National Statistics. (2016). Population estimates. Available from: https://www.ons.gov.uk/peoplepopulationandcommunity/populationandmigration/populationestimates
  4. INVOLVE. (2012). Briefing notes for researchers: public involvement in NHS, public health and social care research. NIHR. Available from: http://www.invo.org.uk/wp-content/uploads/2014/11/9938_INVOLVE_Briefing_Notes_WEB.pdf
  5. NIHR. (2015). Going the extra mile: improving the nation’s health and wellbeing through public involvement in research. Available from: http://www.nihr.ac.uk/documents/about-NIHR/NIHR-Publications/Extra%20Mile2.pdf
  6. Arthritis Research UK. (2010). The role of stakeholder representation in research funding: experiences from Arthritis Research UK’s stakeholder panel, ‘USER’. Available from: http://www.twocanassociates.co.uk/perch/resources/files/stakeholdersinresearch(1).pdf
  7. NHS England. (2013). The NHS belong to the people: a call to action. Available from: https://www.england.nhs.uk/wp-content/uploads/2013/07/nhs_belongs.pdf
  8. NHS England. (2013). Transforming participation in health and care: ‘the NHS belongs to us all’. Available from: https://www.england.nhs.uk/wp-content/uploads/2013/09/trans-part-hc-guid1.pdf
  9. Preston, J., Stones, S. R., Barker, L. & George, E. Exciting things happen when involving young people to promote better research for better healthcare, in DPharm Europe. 2016. London, United Kingdom. Available from: http://generationr.org.uk/wp-content/uploads/2016/02/GenRDPharmwithoutvideos.pptx
  10. Lewenstein, B. V. (1995). From fax to facts: communication in the cold fusion saga. Social Studies of Science 25: 403-436.
  11. Schiele, B., Claessens, M. & Shi, S. (2012). Science communication in the world: practices, theories and trends. The Netherlands: Springer.
  12. Bultitude, K. (2011). The why and how of science communication, in Rosulek, P. (ed). Science Communication. European Commission: Pilsen.
  13. Tlili, A. & Dawson, E. (2010). Mediating science and society in the EU and UK: from information-transmission to deliberative democracy? Minerva 48(4): 429-461.
  14. NIHR. (2014). Patient and public involvement in health and social care research: a handbook for researchers. Available from: https://www.rds-yh.nihr.ac.uk/wp-content/uploads/2015/01/RDS_PPI-Handbook_2014-v8-FINAL-11.pdf
  15. Stones, S. R., Majeed-Ariss, R. & Swallow, V. M. Patients and academics as co-researchers: dream or reality, in Leeds Children’s Hospital Conference. 2015. Leeds, United Kingdom.
  16. INVOLVE. (2014). Policy on payment of fees and expenses for members of the public actively involved with INVOLVE. NIHR. Available from: http://www.invo.org.uk/wp-content/uploads/2014/11/INVOLVE-internal-payment-policy-FINAL-August2014.pdf
  17. NIHR. (2014). Promoting a ‘research active’ nation. Available from: http://www.nihr.ac.uk/documents/get-involved/Promoting%20A%20Research%20Active%20Nation_NIHR%20Strategic%20Plan_May%202014.pdf
  18. NIHR. (2016). Patient Research Ambassador Initiative. Available from: https://sites.google.com/a/nihr.ac.uk/patient-research-ambassador-initiative/
  19. NIHR. (2016). Join Dementia Research. Available from: https://www.joindementiaresearch.nihr.ac.uk

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September 7, 2016
Living with COPD - My Story

Living with COPD - My Story

Whether you are a patient, carer, clinician or interested bystander, my hope is that what I share in my story about living with COPD resonates with you in some way.

by Ian Venamore
August 12, 2016
Nothing About Me Without Me

Nothing About Me Without Me

Whereas in the past, patients were only regarded as study "subjects", nowadays they are playing an increasingly important role in healthcare and clinical research.

by Gary Puckrein
May 13, 2015
Towards Patient-Engaged Research

Towards Patient-Engaged Research

Research is finding answers to questions. Patients have lots of questions.

by Durhane Wong-Rieger
Simon Stones - Patient Research Exchange

Simon Stones

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Living with COPD - My Story - Patient Research Exchange

Living with COPD - My Story

Whether you are a patient, carer, clinician or interested bystander, my hope is that what I share in my story about living with COPD resonates with you in some way.

That was then: My way to diagnosis and beyond

I was born in the 40s and like many of my generation, I was a regular smoker until I quit in when I was in my 50s. By quitting I believed that I had dodged a bullet because for eight years I enjoyed good health. In 2003 I began to experience subtle symptoms, such as shortness of breath, cough and chest congestion and infections, which belied the seriousness of the condition that I now live with. It wasn’t until my mid-fifties that I was told I had chronic obstructive pulmonary disease (COPD) and had lost more than 25 % of my lung capacity. At this point, no mention was made of rehabilitation or structured exercise in addition to medication, but I still felt well. I took the required inhalants and medication as prescribed so I thought I had stopped the disease in its tracks. I also still held a demanding and responsible job, and retirement was a distant vision. So much for blissful ignorance!

In 2011 I was forced to confront reality. My breathing became considerably worse and began to impact my everyday life. Things took longer to do and I worked hard to hide the worst of my symptoms from my greatest supporter and most severe critic, my significant other. Fortunately for me, my wife is far smarter than I am and insisted that I consult another respiratory specialist and plan an exit from work, enabling us to share what I had almost lost sight of, a now uncertain future for us to enjoy. With another breathing test result showing a 50 per cent loss of predicted capacity, I faced reality and my own mortality squarely in the eye for the very first time.

My advice: Knowledge is power, so educate yourself on your condition. And be prepared to question your doctors. It’s not just your right, it is your responsibility.

It was good fortune that the specialist had on his desk a copy of LungNet News, the official newsletter of Lung Foundation Australia. I took the copy and contacted them directly and was referred to the Royal Brisbane Hospital Thoracic Department where I soon after began pulmonary rehabilitation. For those not familiar with these programs, they typically comprise an initial assessment, a review of individual physical capability based on age or disability, followed by an eight week, twice weekly class including tailored gym exercise for an hour which is usually complemented by education sessions covering a variety of topics like lung pathology, medication, diet, breathing techniques and management of anxiety.

Following the pulmonary rehabilitation program, I kept up the routine and since then I religiously attend this class twice weekly. I believe this is one of the most critical factors in maintaining good health for people with COPD. Evidence points to exercise being more beneficial or effective than many prescribed medications. The other very important step during pulmonary rehabilitation is the preparation of a patient driven action plan based on a ‘traffic light’ system. The patient and their doctor establish green, amber, red, symptom levels with corresponding escalating levels of action the patient must take. In doing so the patient is empowered to take ownership of their own condition and immediate responsibility for their health. It’s all about listening to your body and getting to know it well.

Giving back

I decided that now retired, I would become a member of Lung Foundation Australia and perhaps help out to give back. I was asked to help update the national data base for patient support groups. This enabled me to talk directly to other sufferers and carers throughout Australia. I learned a lot about lung disease in general, and came to appreciate the great work the people of this organisation do on behalf of the patients, carers and the community as a whole. I was invited to join the national CPAG (COPD Patient Advocacy Group) to support patients and carers to have a voice within the Foundation on goal setting and priorities.

My personal experience at the Lung Foundation opened my eyes to how much patients ourselves can bring a unique perspective to lung disease awareness, research and self-management by virtue of first-hand experience. My continued philosophy was that if I can assist in preventing any future cases of lung disease or through advocacy improve awareness, diagnosis, management and quality of life for my fellow suffers, then I consider my time well spent.

This is now: Taking part in shaping the future of COPD management

Twelve years post diagnosis and after completing pulmonary rehabilitation and commencing my volunteer work with Lung Foundation Australia, I can look back with some satisfaction at where I am and what I have achieved.

My COPD is stable, despite having suffered a couple of exacerbations over the last couple of years. I repeated pulmonary rehabilitation after my first flare up and continue to learn more about lung disease. I have completed a clinical medication trial and will continue to look for further tests or trials in which I can participate. I have also informally counselled others who approached me to help them better understand their condition and recalled my experiences on a similar journey of discovery.

I participated in the COPD Global Foundation Summit in France, which has the goal of driving worldwide change in the recognition, diagnosis and treatment of COPD. It was an honour to represent Australia at the forum. It was very interesting to see how COPD is managed in different cultures and nations.

My hope is that the COPD Global Foundation and its network of patients, carers and clinicians will develop into a well-respected, accessible resource with a voice that will reach the most remote communities to help, and indeed be the catalyst for change, in improving the lives and conditions of people with COPD, no matter where they are in the world.

My advice: Take Responsibility for Your Health

In retrospect, it would be easy but unfair to single out doctors for their often reactive approach to the management of chronic diseases such as COPD. More often than not, they have an abundance of patients, multiple critical cases and time pressures to deal with. As patients we must learn to help ourselves by demanding the time of our doctors, educating ourselves on diseases prior to consultations and preparing questions. We are generally not keen to hear bad news, the rationale being that if we need to hear more, the doctor will tell us all. If he does not then all is well. There can be fatal flaws in that belief. If we do not show initiative and purpose in our dealings with clinicians, or take responsibility for the management of our own health, we deserve the treatment we get.
My advice is simple. Knowledge is power, so educate yourself on your condition. And be prepared to question your doctors: it’s not just your right, it is your responsibility.

"Knowledge is power"
“Knowledge is power”

Ian Venamore was diagnosed with chronic obstructive pulmonary disease (COPD) in his mid-fifties. Today he is an active volunteer member of Lung Foundation Australia and chair of the associated COPD Patient Advocacy Group (CPAG).He also participates in international meetings with the goal to change the future of COPD recognition, diagnosis and treatment. He is a strong advocate for pulmonary rehabilitation and attends his gym classes “religiously.” He is also a firm believer in and proponent of patient education and self management.

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Nothing About Me Without Me - Patient Research Exchange

Nothing About Me Without Me

Whereas in the past, patients were only regarded as study "subjects", nowadays they are playing an increasingly important role in healthcare and clinical research.

What matters to patients?

Nowadays patients are gaining a more active role in healthcare. It is increasingly recognized that even if two people have the same medical condition, they may have different priorities and preferences regarding treatment. For example, many people with diabetes find the need for regular finger pricks to monitor their blood glucose particularly intrusive and inconvenient, but some object more to having to constantly watch their diet or find time for exercise. Similarly, for some people it might be important to avoid a particular side effect, such as weight gain or hair loss, while others are willing to put up with this if it means getting the most effective treatment. All of these things will affect care decisions.

‘Patient-centered medicine’ is the term used to describe medical decisions that a patient makes together with their doctor: the doctor is the expert in the condition whilst the patient knows best what is important to them personally. This is well captured in the phrase ‘nothing about me without me’ meaning medical decisions that directly affect a patient should not be made without consulting them.1,2

Why patient perspectives are relevant to clinical research

Recently, this cultural shift in the way we think about healthcare has started to influence clinical research too. In the past, patients were regarded as ‘subjects’ who had research performed on them. Their opinion was not considered important since research was seen purely as a scientific activity. Now, however, it is seen as desirable to involve patients in designing and planning clinical research studies. If people who have actually lived with a particular condition can share their personal experiences, concerns and hopes with the researchers, the study that is ultimately planned may be more connected to the things that really matter to them. For example, it has been found that 75% of people with diabetes would prefer clinical trials to directly measure the impact of a treatment on the onset of diabetes-related kidney failure or sight problems, rather than just measuring the effect on their blood sugar levels.1 Patients can also look at a proposed study protocol and see if the amount of time, number of clinic visits and planned study procedures are likely to be acceptable to trial participants, as they will have a good idea how well people with their condition are able to cope.3

Patients as research ‘partners’

This has led to the idea of patients as ‘partners’ in research. The Patient-Centered Outcomes Research Institute in the USA and the James Lind Alliance in the UK have highlighted the fact that patients may have useful contributions to make when research priorities are agreed and have started to put plans in place to ensure that patients participate in the decision-making process.1 In addition, the British National Institute of Health Research (NIHR) runs a project called INVOLVE which is designed to encourage patient and public involvement (known as PPI) in clinical research projects.4 PPI can take many forms and varies from study to study. It may include helping to decide how best to spend money available for research, offering advice as members of a study advisory group, helping to write, or comment on, booklets, leaflets and other materials that explain the research and undertaking interviews with research participants.4

In the past, patients were regarded as ‘subjects’ who had research performed on them. However, now it is seen as desirable to involve patients in designing and planning clinical research studies

Even regulatory authorities (who are responsible for approving new medicines) have recognized the importance of PPI and now require patient involvement in the design of new clinical research studies.1 In addition, many leading scientific journals which publish the findings of research now include summaries for patients which explain research results in terms that people with a non-scientific background can understand.1

Considerations and potential issues with PPI

In planning PPI for a particular research study, it is important to consider exactly how best it can help. PPI does entail extra time and expense so this must be used wisely. Sometimes PPI can appear ‘tokenistic’ – which means it seems to be included just so researchers can say they have consulted patients, rather than for genuinely good reasons.2,5,6 Some researchers worry that patients may have personal aims or ideas that could clash with the scientific aims of a study. These concerns should diminish as scientists and patients gain more experience on how to work together effectively. As part of this, it is important that PPI contributors truly represent the population of people with the disease in question; for example involving just one PPI contributor may mean that the study team gets a single opinion, which might not be typical of the patient population as a whole.5,6

Potential benefits of PPI

One key area where PPI is beneficial is in ensuring that the treatment outcomes assessed in studies are not just what a doctor observes or measures, but also include things that patients report themselves – such as how a treatment affects their mood or energy levels. (For more information on this topic, see our article on Patient-Reported Outcomes).

Involving patients in research planning has also been shown to help with study enrollment.6,7 This is not surprising because if potential participants know a study has been designed with the involvement of people like them, they may well be more inclined to enroll.

PPI is becoming more and more embedded in clinical research. Surveys conducted in the UK in the early 2000s showed that fewer than 25% of studies reported any PPI. In contrast, a UK survey run in 2015 found that nearly 80% of studies include PPI in some form.6 This may partly be in response to new national policies introduced by regulatory authorities and funding bodies. However, it is likely also to be due to a growing recognition amongst researchers that PPI really can make their studies more valuable and more relevant to the people who are ultimately meant to benefit from them.

Sources:

  1. Sacristán JA, Aguarón A, Avendaño-Solá C et al. Patient involvement in clinical research: why, when, and how. Pat Pref Adher 2016; 10:631–640.
  2. Fitzgibbon J, Baillie J, Simon N et al. The role of the public in developing interventions: a reflection and critique of a cancer clinical trials unit’s model. Pat Pref Adher 2014; 8:1527–1535.
  3. Sharma NS. Patient centric approach for clinical trials: current trends and new opportunities. Perspect Clin Res 2015; 6(3): 134–138.
  4. National Institute of Health Research INVOLVE http://www.invo.org.uk/frequently-asked-questions/
  5. Gamble C, Dudley L, Allam A et al. An evidence base to optimise methods for involving patient and public contributors in clinical trials: a mixed-methods study. Health Serv Deliv Res 2015; 3(39).
  6. Wilson P, Mathie E, Keenan J et al. ReseArch with Patient and Public invOlvement: a RealisT evaluation – the RAPPORT study. Health Serv Deliv Res 2015; 3(38).
  7. Domecq JP, Prutsky G, Elraiyah T et al. Patient engagement in research: a systematic review. BMC Health Serv Res 2014; 14:89.

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Health Technology Assessments: Getting the Right Balance in Medicine - Patient Research Exchange

Health Technology Assessments: Getting the Right Balance in Medicine

Scientists spend many years collecting evidence about new health technologies to show they work and are suitable to use in people. However, showing that a health technology works is only part of the story!

Every year, scientists and doctors discover new health technologies: new ways of treating or preventing health problems. A health technology might be a new medicine; a machine to help diagnose or treat an illness; or a new way of doing medical assessments or a new type of surgery.

Scientists spend many years collecting evidence about new health technologies through clinical and laboratory research to show they work and are suitable to use in people. However, showing that a health technology works is only part of the story!

Consider bowel cancer screening as an example…

Bowel cancer is the second biggest cancer killer in Europe (after lung cancer) affecting more than 228,000 people per year.1 If it is detected at its earliest stage, more than 9 out of 10 people can be cured.2 If it is detected at a late stage (after it has spread to other parts of the body), only 1 out of 10 people will survive for 5 years.2

Early bowel cancer has few obvious symptoms, so the best way to detect it is by running screening programs in healthy people. Screening is usually done by looking for blood in a stool sample, or by a medical procedure called a sigmoidoscopy, where a thin flexible tube with a camera is used to look inside the bowel.3

In most countries, screening is only offered to certain people who are known to have a higher than normal risk of developing the disease (e.g. older people or those with a family history of bowel cancer).3-6 If early detection of bowel cancer is so important, why don’t we screen everyone to find the few who are affected?

Screening programs need to balance the benefits of detecting bowel cancer early, against the risks and costs of the screening procedure, which may include:4-6

  • Inconvenience and discomfort for people coming into the clinic for screening
  • Doctors’ and nurses’ time to arrange the screening and analyze the results
  • Cost of buying and maintaining screening equipment
  • A chance of getting misleading screening results, causing patients to worry unnecessarily
  • A chance of missing cancer at an early stage, despite screening
  • A small chance of harm from screening, such as bleeding or tearing of the bowel

By considering all of these factors in a logical, structured way, it is possible for independent researchers to advise doctors and governments about the best way to design screening programs for bowel cancer. This is an example of a health technology assessment.

What is involved in a health technology assessment?

A health technology assessment is a type of research that looks at medical, social, financial, and ethical information to find the best way to solve a specific health problem.7 The assessment involves asking questions about how to use the health technology at the right time, in the right way, for the right group of patients. It also looks at whether the benefit gained from the new technology is worth the possibility of side effects and the costs associated with using it.7-9

Researchers conducting a health technology assessment use evidence from a wide range of sources. These usually include the results of clinical research and laboratory studies, as well as financial assessments done by specialists called health economists.9

Patients and patient groups play an essential part in health technology assessments.10 From the start of the research process, patients can help to define what a meaningful improvement in their disease would feel like to them, and how it could be measured.11 Patients may also be asked to talk to health technology assessors about their own experiences of healthcare, which can help to identify factors that should be considered during the assessment.12 For example, if patients say that they find it difficult to attend hospital for regular injections, the assessors may place greater value in an equally effective new medicine that can be given at home.

All of this information is used to calculate how many people are likely to benefit from the new health technology, and how big the impact on their length or quality of their life may be.9

Can health technology assessments improve the practice of medicine?

Health technology assessments influence the decisions of governments, doctors, insurance companies, and the general public. Their main purpose is to inform people who set health policies and decide how a country’s or a company’s health budget is spent.13 Overall, they aim to achieve the best outcomes for the highest number of people within the health budgets available.13

Health technology assessments often form the basis for published national or international guidelines that doctors can consider when treating their patients. Guidelines are intended to make it easier for doctors to make treatment decisions based on the latest evidence. Some guidelines include recommendations on the best order or sequence to use different available medicines or treatments for a particular disease.9 They also help to ensure that people in different parts of a country or region receive a similar standard of healthcare.9

To ensure healthcare continues to be accessible and affordable for as many people as possible, decisions about how to use new medical technologies are vital.

People who plan health budgets and research spending may use health technology assessments to identify what they think will be the most important future developments in medicine. This allows them to calculate how much money should be set aside for future costs of healthcare.14 These types of analyses may also be used by researchers to find gaps in evidence or new technologies that can be developed to address unsolved health problems.13,14

In many countries around the world, money and time allocated for spending on healthcare is limited.15 To ensure healthcare continues to be accessible and affordable for as many people as possible, decisions about how to use new medical technologies – that are often expensive – are vital. By systematically considering the evidence, health technology assessments can help us to understand how the best value can be gained from available medicines and procedures to improve the lives of patients.15

Sources:

  1. GLOBOCAN 2012 Estimated Cancer Incidence and Mortality Worldwide in 2012. International Agency for Research on Cancer 2012. Available from: http://globocan.iarc.fr/Pages/fact_sheets_population.aspx
  2. Bowel Cancer Survival Statistics. Cancer Research UK 2012. Available from: http://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/bowel-cancer/survival#heading-Three
  3. Screening for Bowel Cancer. National Health Service, Sept 2014. Available from: http://www.nhs.uk/conditions/Cancer-of-the-colon-rectum-or-bowel/Pages/Screeningforbowelcancer.aspx
  4. Health technology assessment (HTA) of a population-based colorectal cancer screening programme in Ireland. Health Information and Quality Authority Mar 2009. Available from: http://www.ncri.ie/sites/ncri/files/pubs/HTA_population_based_colorectal_cancer_screening_programme.pdf
  5. Gutiérrez-Ibarluzea I, Asua J, Latorre K. Policies of screening for colorectal cancer in European countries. Int J Technol Assess Health Care. 2008;24(3):270-6.
  6. What is Bowel Cancer Screening? Macmillan Cancer Support UK. Available from: http://www.macmillan.org.uk/information-and-support/diagnosing/how-cancers-are-diagnosed/bowel-screening/what-is-bowel-cancer-screening.html#tcm:9-3769
  7. Medical Devices: Health Technology Assessment. World Health Organisation. Available from: http://www.who.int/medical_devices/assessment/en/
  8. Policy on Health Technology Assessment. European Commission. Available from: http://ec.europa.eu/health/technology_assessment/policy/index_en.htm
  9. Technology Appraisal Guidance. National Institute of Health and Care Excellence. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance
  10. Facey K, Boivin A, Gracia J, et al. Patients’ perspectives in health technology assessment: a route to robust evidence and fair deliberation. International Journal of Technology Assessment in Health Care 2010; 26(03): 334-340.
  11. Patient involvement in the HTA decision-making process. European Patients’ Academy. Available from: https://www.eupati.eu/patient-involvement-in-the-hta-decision-making-process/
  12. Glossary. National Institute of Health and Care Excellence. Available from: https://www.nice.org.uk/article/pmg19/chapter/glossary#patient-expert
  13. Scott NA, Moga C, Harstall C, et al. Using health technology assessment to identify research gaps: an unexploited resource for increasing the value of clinical research. Healthc Policy. 2008; 3(3): e109–e127.
  14. Sullivan SD, Watkins J, Sweet B, et al. Health technology assessment in health-care decisions in the United States. Value Health. 2009; 12(2): S39-44.
  15. Health technology assessment (HTA). International Alliance of Patient Organizations. Available from: https://www.iapo.org.uk/health-technology-assessment-hta

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Medicines for mothers-to-be: research during pregnancy - Patient Research Exchange

Medicines for mothers-to-be: research during pregnancy

It takes an average of 27 years until a new medicine can be recommended for use by pregnant women. Why is that?

The safety of unborn babies has long been considered one of the most serious and controversial ethical issues when researching new medicines. Because of the unknown risks, pregnant women are very rarely included in clinical research studies, and women taking part in research are usually asked to avoid becoming pregnant.1 As a result, when a new medicine is approved, very little is known about its suitability for use during pregnancy.1,2

Information about use of new medicines in pregnancy is built up carefully, over many years, after the medicine becomes widely available. It takes an average of 27 years until a new medicine can be recommended for use by pregnant women.3 This means that women sometimes face a difficult choice between managing their health without the latest medicines, or taking a medicine when information about its use in pregnancy is limited.2,4,5

Why is it important to carry out research in pregnant women?

Like everyone else, pregnant women sometimes need to take medicines – they may be managing a long-term condition such as diabetes or epilepsy; treating an infection; or perhaps battling a life-threatening illness like cancer.6 They may also have medical needs related to their pregnancy, such as morning sickness. It is estimated that 6 out of every 10 women will need to take at least one prescription medicine during their pregnancy.7,8

A pregnant woman who takes a medicine needs to consider its possible effects on her unborn child. The mother’s blood and the baby’s blood are connected through the placenta and some (but not all) medicines that the mother takes can pass into the baby’s blood. There is a risk that certain medicines that are harmless to adults can affect the health or development of an unborn baby – sometimes irreversibly.9 For this reason, everyone involved in a clinical study – the research team, the participants and the committees that approve the study – are a lot more cautious than usual.

Clinical research in pregnant women will allow doctors and patients to make better decisions about the use of medication during pregnancy.

The other consideration is the potential effect of a medicine on the pregnant woman herself. Women’s bodies also change during pregnancy: they have extra fluid in their blood (an additional 30-50%), their hearts beat faster and more strongly, their kidneys work harder, and their stomach releases food more slowly. All of these things mean that pregnant women process medicines in a different way from other adults.9,10 Because for many drugs there haven’t been any or not enough clinical studies in pregnancy, the usual advice for pregnant women is to check with their pharmacist, midwife or doctor before taking any new medicine. If there is only limited information about its use during pregnancy, the woman and her doctor will need to weigh up any available evidence and decide what is right for her. Women who are managing a long-term health problem are advised to discuss their treatment with a specialist ideally before they try to conceive but at least as soon as they find out they are pregnant.11,12

How does research on medicines during pregnancy work?

Information about the safety and effectiveness of medicines for pregnant women is collected in special types of studies that are designed to protect the health of the mother and the baby. Often the earliest information about the effect of a medicine during pregnancy comes from studies on animals or animal cells. This is done as part of basic research on the new medicine, to find out if it might have a harmful effect on growing or developing tissues.3 Scientists may also look at the structure of the medicine to see whether it is likely to cross the placenta from the mother into the baby.3 Studies in animals can be helpful for ruling out medicines that appear to have too high a risk of causing harm, but it is still necessary to carry out research in pregnant women to find out if the medicine is truly safe and effective in pregnancy. Sometimes a small number of pregnant volunteers agree to have blood tests after they have taken an investigational medicine in carefully controlled conditions. The blood tests show how pregnant women process the medicine, and how it affects their bodies.13

Another common way to look at the effect of medicines in pregnancy is through observational studies (sometimes called pregnancy registries).14,15 This type of study collects health information from pregnant women after they have personally chosen (or been advised) to take a particular medicine for their own benefit. They are not given the medicine as part of the research. Cohort studies are a type of observational study that typically involve a large number of women, who may or may not have taken the medicine as part of their routine treatment. This type of study relies on medical records or questions answered by the pregnant woman to generate information. The information is analyzed to look for possible links between medication use and health effects on mothers or babies.14,15

What does taking part in an observational pregnancy study involve?

Observational studies usually do not require the mother-to-be to take any medicines or have any medical treatment that she wouldn’t otherwise have. Instead, she is asked to provide information about her use of the medicine, as well as information about her health and what is known about the health of her baby. In many cases, the study only requires permission for the researchers to look at the mother’s and baby’s medical records. In other cases, additional tests and assessments may be done, such as blood tests or physical examinations. Researchers may also keep track of the baby’s mental and physical development for the first few years of life in case a medicine taken during their mother’s pregnancy turns out to have long-term effects.1,4,5

However the studies are done, clinical research in pregnant women means that doctors and patients will ultimately be able to make much better decisions about taking a particular medicine during pregnancy.1

Sources:

  1. Lyerly AD, Faden RR. Mothers Matter: Ethics and Research during Pregnancy. Virtual Mentor 2013;15(9): 775-778.
  2. Thall Bastow, BD. Teratology and Drug Use During Pregnancy. 2016. Available from: http://emedicine.medscape.com/article/260725-overview
  3. Adam NP, Polifka JE, Friedman JM. Evolving knowledge of teratogenic risk of medications in human pregnancy. Am J Med Genet C Semin Med Genet. 2011;157(3):175-182.
  4. Foulkes MA, Grady C, Spong CY, Clinical Research Enrolling Pregnant Women: A Workshop Summary. J Womens Health (Larchmt) 2011; 20(10): 1429–1432.
  5. Blehar MC, Spong C, Grady C, Enrolling Pregnant Women: Issues in Clinical Research. Womens Health Issues. 2013; 23(1): e39–e45.
  6. Pregnancy and Chronic Health Conditions. National Health Service. Available from: http://www.nhs.uk/conditions/pregnancy-and-baby/pages/pregnant-chronic-health-condition.aspx
  7. Daw JR, Mintzes B, Law MR, et al. Prescription drug use in pregnancy: a retrospective, population-based study in British Columbia, Canada (2001-2006). Clin Ther 2012;34:239–249.
  8. Ethical Considerations for Including Women as Research Participants. The American College of Obstetricians and Gynecologists. Nov 2015. Available from: http://www.acog.org/Resources-And-Publications/Committee-Opinions/Committee-on-Ethics/Ethical-Considerations-for-Including-Women-as-Research-Participants
  9. Sachdeva P, Patel BG, Patel BK. Drug Use in Pregnancy; a Point to Ponder! Indian J Pharm Sci. 2009; 71(1): 1–7.
  10. Williams DJ. Physiological changes of normal pregnancy. Available from: http://oxfordmedicine.com/view/10.1093/med/9780199204854.001.1/med-9780199204854-chapter-1401
  11. Best Use of Medicines in Pregnancy. UK Teratology Information Service. Available from: http://www.medicinesinpregnancy.org/Medicine—pregnancy/
  12. Medicines in Pregnancy. National Health Service. Available from: http://www.nhs.uk/conditions/pregnancy-and-baby/Pages/medicines-in-pregnancy.aspx
  13. Guidance for industry: Pharmacokinetics in pregnancy. Food and Drug Administration. 2004. Available from: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072133.pdf
  14. Cardonick E. Usmani A. Ghaffar S. Perinatal outcomes of a pregnancy complicated by cancer, including neonatal follow-up after in utero exposure to chemotherapy. Results of an international registry. Am J Clin Oncol. 2010;33:221–228.
  15. Donegan K, Safety of pertussis vaccination in pregnant women in UK: observational study. BMJ 2014;349:g4219.

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From molecule to medicine: How are new drugs developed? - Patient Research Exchange

From molecule to medicine: How are new drugs developed?

Medical research requires many different steps and stakeholders. We are taking a closer look at these.

Edward Jenner was a doctor who lived in England near the end of the 18th century. At that time, a serious and often fatal disease called smallpox was common.1,2 Dr Jenner noticed that milkmaids, who were exposed to a related but milder disease called cowpox, rarely developed smallpox. He began an experiment to show that intentionally giving a patient cowpox could protect against smallpox. He took the bold step of injecting a young boy with pus from a cowpox blister. A little while later, when exposed to smallpox, the boy was found to be protected. Jenner had discovered vaccination – a hugely important step forward in medicine. As a result of vaccination, smallpox no longer exists anywhere in the world and many other dreaded diseases have become rare.3

Patients receiving new medicines can only do so thanks to people around the world who contributed to medical research.

Doctors and scientists developing today’s new medicines also start with an idea and explore that idea through research. Today, the safety and welfare of the people who take part in research is closely monitored by independent ethics committees and by government agencies called medicines regulators. Well-known examples of medicines regulators are the Food and Drug Administration (FDA) in the United States4 and the European Medicines Agency (EMA) in Europe.5 In the mid-20th century, the regulators introduced some important new laws: before new medicines could be approved for use by the public, the manufacturer had to collect evidence to prove that the medicine was safe and effective.4 Turning a scientific idea into a new medicine usually takes 15 to 20 years… so what actually happens during medicine development, and why does it take so long? 6

What happens in medical research?

Research is all about answering questions. These questions are asked in several stages to carefully build up information about a potential new medicine. Many scientists are employed in researching diseases and looking for new molecules that might help.7 If they find a new molecule, they ask some basic questions. What is the structure of the molecule? How might it interact with natural chemicals in the body? Is it similar to any medicines we already know or use? By answering these questions, the scientists get clues about how the new molecule might be useful in humans. This is called basic or discovery research.8

If the scientists think the molecule could be useful, they begin pre-clinical research.8 This helps to find out if the molecule is harmful to living cells, which is important to do before giving it to humans. In some cases, scientists can answer these questions using cells grown in a dish. But research involving living animals such as mice or rats is often needed as well. The animals are protected by strict rules to ensure their welfare, and other research methods are being explored to minimize the use of live animals in future.9

Throughout the development of a new molecule, manufacturers also consider whether new medicines are really needed for a particular disease, and whether they could be provided to patients on a large scale if approved.10 This is called translational research, because it looks at whether something that seems promising in the lab can be translated into actual medicine.11, 12

Medical studies in humans are called clinical research.13, 14 Clinical research usually happens in stages or phases, during which some very important questions are answered under close medical supervision:

  • Phase 1 studies – to find out how the medicine behaves in the human body, how much should be given (the dose), and whether it is well tolerated (i.e. whether it causes other troublesome health problems). The first few people to receive the potential new medicine are usually (but not always) healthy adult volunteers.
  • Phase 2 studies – to look at whether the medicine may be helpful for a particular medical condition. These studies vary in length and size, but they usually involve up to a few hundred people, and last for weeks or months.
  • Phase 3 studies – to learn more about whether the medicine works and is safe to use in a particular medical condition, and to look at whether the medicine is better than currently available treatments. These studies tend to be large, involving several hundred or a few thousand people. They may last for a year or more.

The collected evidence from basic, translational, pre-clinical and clinical studies (called ‘study data’) is presented to medicines regulators (e.g. the FDA or EMA). If the regulators decide that the evidence proves the new medicine is useful, it will be approved for wider use. Even then, the research doesn’t stop. Manufacturers and medicines regulators continue to ask for information from people who are prescribed the medicine by their own doctors. Rare side effects, for example, may only be seen after the medicine is given to a large number of people.15 Phase 4 studies, also called post-marketing studies, are the final phase of research. They look at how the medicine works outside clinical studies, and whether it has any other possible uses in human medicine.15

What is the role of patients in research?

Millions of people take part in clinical research each year.16 These people are all volunteers and are helping to increase our understanding of diseases and medicines. As a patient or a member of the public, there are many ways to get involved in research. You could donate blood or genetic (DNA) samples to help researchers learn about diseases and possible treatments.17-19 You could also consider taking part in a clinical research study, either as a healthy volunteer, or as a person with a particular medical condition. Your doctor may already be involved in a suitable study, or may be able to refer you to one in your local area. Patient spokespeople are also increasingly involved in committees that design studies or review research.20

In 2015, 39 new drugs completed testing and were approved for use in Europe.21 As a result, new treatment options are available for people with cancer, diabetes, heart disease and many other conditions.22 The patients receiving these new medicines can only do so thanks to people around the world who contributed to medical research.

Sources:

  1. Brought to life: Exploring the history of medicine - Edward Jenner (1749-1823). The Science Museum. Available from: http://www.sciencemuseum.org.uk/broughttolife/people/edwardjenner
  2. Smallpox fact sheet. Centers for Disease Control and Prevention, 2007. Available from: http://emergency.cdc.gov/agent/smallpox/overview/disease-facts.asp
  3. Emergencies preparedness, response: Smallpox. World Health Organisation. Available from: http://www.who.int/csr/disease/smallpox/en/
  4. About FDA: Summary of NDA Approvals & Receipts, 1938 to the present. U.S. Food and Drug Administration, 2013. Available from: http://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/SummaryofNDAApprovalsReceipts1938tothepresent/default.htm
  5. About us. European Medicines Agency. Available from: http://www.ema.europa.eu/
  6. Frequently Asked Questions: Research. The Alzheimer’s Society. Available from: https://www.alzheimers.org.uk/site/scripts/faqs.php?categoryID=200422&faqID=92
  7. Atanasov AG, Waltenberger B, Pferschy-Wenzig EM, et al. Discovery and resupply of pharmacologically active plant-derived natural products: A review. Biotechnol Adv. 2015;33(8):1582-614.
  8. Regulatory Roadmap for the development of human medicinal products (Europe focus). The RQA. Available from: http://www.therqa.com/regulatory-roadmap/
  9. About us. European Society for Translational Medicine. Available from: https://eutranslationalmedicine.org/about-us/
  10. Working to reduce the use of animals in scientific research. Department of Health, 2014. Available from: https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/277942/bis-14-589-working-to-reduce-the-use-of_animals-in-research.pdf
  11. Avorn J. The $2.6 Billion Pill — Methodologic and Policy Considerations. N Engl J Med 2015; 372:1877-1879.
  12. What is translational research? Wellcome Trust. Available from: http://www.wellcome.ac.uk/funding/Innovations/wtd027704.htm
  13. Clinical trials and medical research - Phases of trials. National Health Service. Available from: http://www.nhs.uk/Conditions/Clinical-trials/Pages/Phasesoftrials.aspx
  14. Clinical Trial Phases. U.S. National Library of Medicine. https://www.nlm.nih.gov/services/ctphases.html
  15. Suvarna V. Phase IV of Drug Development. Perspect Clin Res. 2010; 1(2): 57–60.
  16. Big rise in volunteers for medical trials. BBC News, May 2013. Available from: http://www.bbc.co.uk/news/health-22594635
  17. Donations for Laboratory Research Use. National Institutes of Health Blood Bank. Available from: http://clinicalcenter.nih.gov/blooddonor/donationtypes/lab_research.html
  18. Donating for Research. Human Fertilisation and Embryology Association, July 2015. Available from: http://www.hfea.gov.uk/donating-for-research.html
  19. Donating your tissue for research FAQs. Human Tissue Authority. Available from: https://www.hta.gov.uk/faqs/donating-your-tissue-research-faqs
  20. Public involvement in clinical trials: Supplement to the briefing notes for researchers. National Institute for Health Research, 2015. Available from: http://www.nihr.ac.uk/get-involved/INVOLVEpublicinvolvementinclinicaltrialsBriefingnotes2012.pdf
  21. Mullard A. EMA recommended 39 new drug approvals last year. Nature Reviews Drug Discovery 2016;15:77.

Other Stories

April 12, 2016
Their future in our hands: clinical research in children

Their future in our hands: clinical research in children

Children are not ‘mini adults’. That's why it is so important to conduct clinical research in children.

by Patient Research Exchange
March 1, 2016
Planning for our future: participation of the older population in clinical trials

Planning for our future: participation of the older population in clinical trials

Why are older people so often excluded from clinical trials? Is this a cause for concern?

by Patient Research Exchange
January 18, 2016
Precision Medicine Without ‘Precision Trials’?

Precision Medicine Without ‘Precision Trials’?

Find out why and how clinical trials have to change to allow advances in precision medicine.

by Durhane Wong-Rieger
How to find a perfect match – Empowering patients to find their way through clinical trials - Patient Research Exchange

How to find a perfect match – Empowering patients to find their way through clinical trials

Navigating through the clinical trial process can be challenging but there are tools which offer help.

Clinical trials are at the heart of all medical advances, and with them, researchers are looking for new ways to prevent, treat and detect diseases. Clinical trials evaluate the safety and efficacy of new treatments, compare whether or not those new treatments are better than an existing one, and overall, focus on measuring the impact and improvement new treatments might have on a patient and his/her quality of life1.

While clinical trials make a great contribution to medical knowledge and are vital to achieving better healthcare outcomes, researchers struggle to find volunteers and patients to take part in their trials2. Why is that? Obviously, there is no single answer to this question; it depends on many factors and it can vary depending on each individual. When considering treatment choices, many patients don’t even think about participating in clinical trials, possibly because they simply don’t know anything about them or because their physicians don’t see them as a possible option3. On the other hand, patients often have misconceptions about what these trials entail and are concerned about possible side effects. However, the main issue arises when trying to get information and finding that it is not usually presented in a user-friendly way, but rather full of complicated scientific jargon that makes the entire process even more difficult and intimidating. Furthermore, with a poll revealing that 75% of the public has little to no knowledge of clinical studies4, and 85% of trials being delayed because of the lack of participants5, it is no wonder that many trials fail before they even begin due to not reaching their recruitment goals.

We must empower patients to understand and navigate the clinical trial process in order to keep improving treatments and health outcomes.

For this reason, many have taken the initiative to create and develop tools that simplify the process for people trying to find information about clinical trials. These nonprofit clinical trial matching services use patient-friendly language to present information, making it understandable and accessible for patients, their physicians and their families. In addition to this, tools like these also provide assistance when it comes to finding the right trial for each individual based on their condition, eligibility criteria, location and several other variables. Ultimately, the aim of these matching services is to encourage people to “consider clinical trials as an option for care when making treatment decisions”.6

Let’s use Metastatic Trial Search as an example. This tool is dedicated to help women looking for metastatic breast cancer clinical trials, taking into consideration their individual diagnosis and needs. Given that there are no cures available for this type of cancer at the moment, making information accessible faster to the people who need it the most is crucial7, and advances in research are pivotal for better results in the future.

Available in the US, BreastCancerTrials.org offers a matching tool service to personalize patients’ search by sorting through hundreds of recruiting trials and selecting the best options for each person based on their health history; if the patient prefers, she can also skip the matching tool option and get access to information about all open trials filtering by tumor type or trial type8.

Another website called TrialReach.com provides standardized information in plain English, facilitating patients’ access to trial information by just answering a few questions that will help them get a trial tailored to their condition. At the moment, TrialReach.com has a database of thousands of clinical studies from several sources; for diabetes an advanced search is enabled, and for other conditions there is a basic search tool9.

Even though many patient-centered organizations facilitate and promote the consideration of clinical trials as a valid option for care (for example, the Michael J. Fox Foundation for Parkinson, Diabetes UK, the National Multiple Sclerosis Society, Cancer Research UK, HealthTalk and Arthritis Research UK just to name a few), the challenge still remains in making information accessible and as clear and simple as possible for the patients in a systematic way. Empowering patients and the general public to understand and navigate the clinical trials process is an imperative that we must ultimately strive to promote in order to keep improving treatments and outcomes for patients.

Sources:

  1. https://clinicaltrials.gov/ct2/about-studies/learn
  2. http://www.techipedia.com/2014/social-media-clinical-research/
  3. http://genomemag.com/clinical-trials/#.VnKMA42FMdW
  4. http://www.forbes.com/sites/zinamoukheiber/2015/04/23/drug-companies-pin-hopes-on-trialreach-to-enroll-patients-in-clinical-trials-faster/ & http://www.centerwatch.com/about-centerwatch/news/
  5. http://www.techipedia.com/2014/social-media-clinical-research/
  6. http://www.eurekalert.org/pub_releases/2015-10/kc-blm100615.php
  7. http://www.eurekalert.org/pub_releases/2015-10/kc-blm100615.php
  8. https://www.breastcancertrials.org/BCTIncludes/FindATrial/HowMatchingWorks.html
  9. http://trialreach.com

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August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
September 22, 2015
Crowdsourcing Clinical Trials

Crowdsourcing Clinical Trials

The notion that too many people involved in a project can ruin it is quite common.

by Editorial Team
May 13, 2015
Towards Patient-Engaged Research

Towards Patient-Engaged Research

Research is finding answers to questions. Patients have lots of questions.

by Durhane Wong-Rieger
Listening to patients and what matters to them - Patient Research Exchange

Listening to patients and what matters to them

The use of Patient Reported Outcomes (PROs) in clinical research

Clinical trials usually rate the success of a potential new treatment based on the things the research team can check or measure. For example, in trials of potential new medicines for the skin condition psoriasis, an official rating scale called the Psoriasis Area & Severity Index (PASI) is typically used to assess three things: the redness, thickness, and scaliness of the skin patches.1 A score is given to rate these three aspects before and after study treatment.

But using an approach like this by itself has limitations. Often what matters most to people with psoriasis is how disfiguring or embarrassing they perceive their skin problem to be, how it affects their self-confidence and whether having the condition stops them from doing the things they would normally do.2 In a similar way, people with life-threatening conditions like advanced cancer are sometimes mainly concerned with pain management and whether they feel comfortable and calm, rather than whether their lifespan is extended by a few more weeks or months.3

By including patient reported outcomes in clinical studies, the true value of a potential treatment – according to patients as well as to doctors – is more likely to be shown.

In recent years, researchers have come to realize that testing new treatments based on things that are meaningful and important to patients – in addition to the things that the research team can measure– may give a much more complete picture of how useful the potential new treatment truly is. Sometimes a change in a score on an established rating scale may not translate into a noticeable difference in how the person feels. These realizations have led to the development of Patient Reported Outcomes (PROs).

What are Patient Reported Outcomes (PROs)?

The US Food and Drug Administration (FDA) defines a patient-reported outcome (PRO) as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.”4

The European Medicines Agency (EMA) uses a very similar definition: “A patient reported outcome (PRO) is an umbrella term that can be defined as a measurement based on a report that comes directly from the patient about the status of disease and treatment without amendment or interpretation of the patient’s response by a clinician or anyone else.”5

In order to receive these reports from patients in a way that is ‘standardized’ (i.e. in the same format for every patient in a clinical study), research teams have worked to devise detailed patient questionnaires that are valid for the condition being studied. The questionnaires focus mainly on what is known as ‘quality of life’ – i.e. how the condition and the treatment affect someone’s daily life physically, mentally, emotionally, practically and socially. Over the last two to three decades, hundreds of PRO questionnaires have been developed for many different conditions6 and they are increasingly being used in clinical trials and also to some extent in routine clinical practice.

Some questions may have ‘yes / no’ answers but in most cases patients will be asked to choose amongst a selection of answers. Each response will then be given a score and the total score calculated.7

How are PROs developed?

When developing a PRO for a particular condition, a team of healthcare professionals usually meet to discuss it. The members of the team will differ but may include specialist doctors, primary care doctors (i.e. GPs or family physicians), nurses with experience in the condition and medical statisticians (experts in the mathematical calculations needed to judge how well a treatment does in a clinical trial). These days, patients themselves – or people who work for patient support groups related to the condition – are often also invited to join PRO development teams and their inclusion is increasingly viewed as important.8

Once a PRO questionnaire has been drafted, there are a number of ways that it has to be validated (tested) in people with the relevant condition to ensure it is reliable and appropriate to use in a clinical study.8 To be useful, a PRO questionnaire also needs to be easily understood by the people who will complete it and must not take up too much time.8 Although many PRO questionnaires already exist, new ones are constantly being developed as researchers learn more about diseases, treatments and the important things to ask patients.

How are PROs used as measures in clinical trials?

There are some conditions where using PROs is obviously needed, like assessing pain in studies of potential new painkillers or assessing mood in studies of potential new treatments for depression. However, some experts now think that PROs should be included in all clinical studies, even where the main thing to judge is something only doctors can detect.9 This is because patients’ opinions, thoughts, beliefs and perceptions about their condition and the treatment being tested should always count. In the past, PROs were included in clinical studies as ‘just another thing to do’ and were not really central to the study design. However, these days, more studies are giving PROs greater importance and some are even making a PRO the main thing that is assessed (called the primary endpoint).10

What are the benefits and challenges of using PROs in clinical research?

The main difficulty with PROs is that they are not an exact science. Different people rate things differently: for example, one person’s idea of ‘the worst pain you can imagine’ might not match another person’s. The validation testing is designed to ensure this variability is accounted for but it can be challenging. Then there is the problem of incomplete questionnaires. Sometimes analyzing PRO questionnaires can also be time-consuming for study teams. But PROs are being refined and improved all the time. By including PROs in clinical studies, the true value of a potential treatment – according to patients as well as to doctors – is more likely to be shown.

Sources:

  1. Feldman SR, Krueger GG. Psoriasis assessment tools in clinical trials Ann Rheum Dis 2005;64: ii65-ii68.
  2. McKenna SP, Cook SA, Whalley D et al. Development of the PSORIQoL, a psoriasis-specific measure of quality of life designed for use in clinical practice and trials. Br J Dermatol 2003; 149(2):323-31.
  3. Basch E, Geoghegan C, Coons SJ et al. Patient-reported outcomes in cancer drug development and US regulatory review perspectives from industry, the Food and Drug Administration and the patient. JAMA Oncol 2015; 1(3):375-379.
  4. Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. U.S. Department of Health and Human Services Food and Drug Administration Dec 2009. Available from: http://www.fda.gov/downloads/Drugs/.../Guidances/UCM193282.pdf
  5. Reflection paper on the use of Patient-Reported Outcomes. European Medicines Agency June 2014. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2014/06/WC500168852.pdf
  6. Nelson EC, Eftimovska E, Lind C et al. Patient reported outcome measures in practice. Brit Med J 2015; 350: g7818.
  7. FACIT.org questionnaires. Available from: http://www.facit.org/FACITOrg/Questionnaires
  8. Trujols J, Portella MJ, Ioseba Iraurgi I et al. Patient-reported outcome measures: are they patient-generated, patient-centred or patient-valued? J Mental Health 2013; 22(6): 555-562
  9. Denniston AK, Kyte D, Calvert M et al. An introduction to patient-reported outcome measures in ophthalmic research. Eye 2014; 28: 637–645.
  10. Calvert M, Blazeby J, Altman DG et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA 2013; 309 (8):814-22.

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January 18, 2016
Precision Medicine Without ‘Precision Trials’?

Precision Medicine Without ‘Precision Trials’?

Find out why and how clinical trials have to change to allow advances in precision medicine.

by Durhane Wong-Rieger
May 13, 2015
Towards Patient-Engaged Research

Towards Patient-Engaged Research

Research is finding answers to questions. Patients have lots of questions.

by Durhane Wong-Rieger
April 21, 2015
Patients Becoming Partners in Research

Patients Becoming Partners in Research

When it comes to clinical trials, everyone knows the drill: experts create and patients participate. Right?

by Editorial Team
Their future in our hands: clinical research in children - Patient Research Exchange

Their future in our hands: clinical research in children

Children are not ‘mini adults’. That's why it is so important to conduct clinical research in children.

Why is it so important to conduct clinical research in children?

In the past, it was considered too risky to do clinical research in children.1 We all want to protect children: they are vulnerable, they depend on adults, and often, they cannot speak for themselves. But this has meant that doctors often had to give children medicines that had only been tested in adults.2, 3, 4 Why is this a problem? Children are not ‘mini adults’. Medicines are digested and absorbed into a child’s bloodstream at different rates and in different amounts compared to what happens in adults. Also, children are different from adults in regards to how much of the medicine gets to where it is needed and how quickly or slowly it leaves the body.1, 5, 6 These differences can mean that a medicine proven to be helpful in adults may have a different effect if given to a child – or it could even be harmful.7 Of course childhood is a period of change, including significant growth and development, so young children can be very different than older children, and therefore react differently: e.g., a four-year-old child might not react in the same way to a medicine compared to a twelve-year-old.7 We may think we can predict responses to medicines in children, but really we can’t without properly studying them.4 So… in our eagerness to protect children from untested drugs (which they would be exposed to as part of a clinical trial), we actually may have left them at greater risk in everyday medical practice from drugs that have not been tested in children. This means that doctors have had to guess what dose to give them (often by cutting up tablets or opening capsules)6 – and then just hoped for the best.4, 8 Scientists in the US have shown that if we rely on adult research findings to guide how to give medicines to children, we will be wrong almost half the time.9 There are also some diseases directly related to childhood that of course are not seen in adults. For many of these diseases no medicines are available at all because of reluctance to involve children in clinical research.1 A UK survey of 1000 people found that nearly 90% of them did not know that many medicines given to children are ‘unlicensed’ (not tested or approved to give to children), and once they found out about this, over 60% found this worrisome.10

We may think we can predict responses to medicines in children, but really we can’t without properly studying them.

How have attitudes to clinical research in children changed?

In 1964, a very important announcement called the Declaration of Helsinki made it clear that clinical research in children was ‘ethical’ – in other words, it was not wrong, uncaring or unfair, as long as there was a clear need for it and it was likely to make a useful contribution to medical knowledge.1, 6 But progress was very slow. It was not until 2002 that new laws came into force in the US making it much harder for drug developers to miss out studying their medicines in children. The European Union introduced similar laws a few years later. Then in 2006 the World Health Organization (WHO) joined forces with the big children’s charity UNICEF to launch a number of campaigns intended to encourage research in children. They drew attention to ‘missing’ medicines for childhood illnesses and to situations where existing medicines needed to be adapted for use in children (‘Make Medicines Child Size’).6 This included finding the right dose and developing ways of giving the medicine that were not unpleasant for children to take (e.g. flavoured syrups or tablets that dissolve in the mouth without water).6, 11 In 2008, The International Federation of Pharmaceutical Manufacturers and Associations (a worldwide organization that represents drug developers) set up a special team to work with the WHO in this area.6 There are many examples of the benefits of research in children. A particular success story is for childhood leukaemia. Figures from the UK show that, in 2005, over 80% of children diagnosed with leukaemia survived for at least 10 years, compared with only 27% in 1975 – a direct result of clinical research.7

How are proposals for studies of children decided upon?

All clinical trial proposals are considered in detail by an independent group called an ethics committee before they are allowed to go ahead. The committee – which includes general members of society as well as doctors and scientists – discusses whether the study may cause an unacceptable level of pain, worry, fear, stress, upset or risk to participants. Not surprisingly, studies for children are particularly carefully considered, but at the same time committee members know that they need to remember the potential value of the research for future generations.7 Research teams and ethics committees are now also beginning to invite children, teenagers and parents to take part in meetings and offer their personal opinions on proposed studies for children. Also, families are asked to give their feedback to study information materials like booklets and videos.7 Getting the input of young people early on is a valuable way to gain the perspective of the sort of people who will ultimately take part in a study. In fact, many countries have now set up networks of young people who can advise on planned studies for children.7

Action to protect the health of children has progressed more in the last ten years than in the previous fifty, giving us an optimistic view of the future.

What about consent?

Every clinical trial participant has to agree to take part via a process called ‘informed consent’. This means that the study is clearly and thoroughly explained to them and they decide to join of their own free will once they have understood what the study is aiming to achieve and exactly what will be involved. This is where things get complicated when it comes to research in children. Many children will not be mature enough to understand something as complex as a clinical study and will be unable to make choices on their own. Of course most teenagers will understand a lot more than young children and some may have fully formed views and preferences.7 From the point of view of the law, however, everyone under 18 is considered unable to make their own decisions; so, official informed consent is required from a parent or legal guardian to join a clinical trial.4 Still, involving the child in the decision-making process is very important. Getting the child’s agreement is a more informal process known as ‘assent’.1, 7, 8 Many clinical study teams now offer booklets, videos or websites that explain their study in simple, child-friendly language, sometimes with cartoons, drawings, quizzes and other techniques designed to appeal to children or adolescents.

Action to protect the health of future generations of children has progressed more in the last ten years than in the previous fifty, giving us an optimistic view of the future. Today we can see how the situation has started to change when it comes to childhood cancers for example, where clinical trials are the standard of care rather than the exception, and 60 % of cancer patients under the age of 29 are enrolled in a clinical study. 12

Sources:

  1. Medical Research Council. MRC Ethics Guide: Medical Research in Children 2004. Available from: www.mrc.ac.uk/documents/pdf/medical-research-involving-children/
  2. World Health Organization. Clinical Trials in Children. 2016 www.who.int/ictrp/child/en/
  3. National Institutes of Health. NIH Clinical research trials and you. 2016 www.nih.gov/health-information/nih-clinical-research-trials-you/parents-children
  4. U.S. Food and Drug Administration. Should your Child Be in a Clinical Trial? 2016 www.fda.gov/forconsumers/consumerupdates/ucm048699.htm
  5. Lu H, Rosenbaum S. Developmental Pharmacokinetics in Pediatric Populations. J Pediatr Pharmacol Ther 2014;19(4):262–276
  6. Finney E. Children’s Medicines, a Situational Analysis 2011. Available from: www.who.int/childmedicines/progress/CM_analysis.pdf
  7. Nuffield Council on Bioethics (2015). Children and clinical research: ethical issues. Available from: nuffieldbioethics.org/publications/
  8. Yeung V. Clinical Trials in Children. Chapter 6 in Paediatric Drug Handling ISBN 978 0 85369 686 5. Available from www.pharmpress.com/files/docs/paeditaric_sample_chapter.pdf
  9. NIH Medline Plus: The Importance of Children in Clinical Trials. Available from: www.nlm.nih.gov/medlineplus/magazine/issues/winter12/articles/winter12pg6-7.html
  10. Mukattash T, Millership J, Collier P et al. Public awareness and views on unlicensed use of medicines in children Br J Clin Pharmacol 2008; 66(6): 838-45
  11. WHO Expert Committee on Specifications for Pharmaceutical Preparations. Annex 5: Development of paediatric medicines: points to consider in formulation. WHO Technical Report Series no.970: 2012. Available from: apps.who.int/medicinedocs/documents/s19833en/s19833en.pdf
  12. St. Baldrick’s Foundation. The Road to Success: Clinical Trials and the Children’s Oncology Group. www.stbaldricks.org/blog/post/the-road-to-success-clinical-trials-and-the-childrens-oncology-group/

Other Stories

March 1, 2016
Planning for our future: participation of the older population in clinical trials

Planning for our future: participation of the older population in clinical trials

Why are older people so often excluded from clinical trials? Is this a cause for concern?

by Patient Research Exchange
January 18, 2016
Precision Medicine Without ‘Precision Trials’?

Precision Medicine Without ‘Precision Trials’?

Find out why and how clinical trials have to change to allow advances in precision medicine.

by Durhane Wong-Rieger
November 16, 2015
Gender equality in clinical research – are we already there?

Gender equality in clinical research – are we already there?

One would think that gender equality in medical research has been a reality for a long time, right? Let's have a look at the facts.

by Durhane Wong-Rieger
Planning for our future: participation of the older population in clinical trials - Patient Research Exchange

Planning for our future: participation of the older population in clinical trials

Why are older people so often excluded from clinical trials? Is this a cause for concern?

The world is ageing. By 2050, the number of people aged 65+ will be 16% of the global total, coming from only 8% in 2010.1 While this trend can be considered a triumph of medical, social and economic advances, it also causes big challenges. We have often heard about how the ageing population strains insurance and pension systems. But there are also other consequences which may be less well known. One of these is related to the testing and use of medicines. People over 65 make up the majority of patients for many medications intended to treat chronic conditions, but typically those who are over age 65 are excluded from participating in clinical trials. Should we be worried about this?

Twenty years ago, an important global event called the International Conference on Harmonisation (ICH) laid out a compelling case for including people aged 65+ in all clinical trials for treatments aimed at adults. It argued that results from trials studying only younger adults may not be relevant to older people, whose bodies are quite different.2 Yet this is exactly what is still happening in daily medical practice today: clinical decisions for older adults are routinely based on data from studies of younger people. In these situations, doctors are left to treat older patients without knowing how well the chosen drug works in this population, how it behaves in their bodies, what side-effects it may cause, what doses are best, and to what extent it may interact with other drugs being taken.

The world is ageing. While this trend can be considered a triumph of medical, social and economic advances, it also causes big challenges.

The issue of side effects is a good example. Many studies show that the older you get, the more likely you are to experience side-effects from drugs.3 The reasons for this are complicated but may be attributed to the fact that the way drugs pass through the bloodstream, reach the tissues and are excreted (termed ‘pharmacokinetics’) changes with ageing.4 People also tend to take more medicines as they get older.3 A US article reports that about 50% of the over-65s take at least five different drugs.5 And unfortunately, the side-effects of one drug can sometimes boost the side effects of another.3

So how bad is the problem of under-representation of people aged 65+ in clinical trials? Here is one example: only about 25% of participants in cancer trials are older people, despite the fact that two-thirds of cancer patients are 65 or older.6 This one is even more surprising: older people are consistently under-represented in trials for Alzheimer’s disease, even though it’s well known that this is nearly always a disease that affects older people.6

A study looking at trials published from 1994 to 2006 found that in nearly 40% of those trials, people over the age of 65 had explicitly not been deemed eligible to participate.7 And even when older people aren’t excluded because of their age, they get left out for other reasons. More than 45% of the trials that didn’t have age limits, excluded people for reasons like taking other medications, having other illnesses or physical disabilities, or even for living in a nursing home – all restrictions that tend to remove older people from the clinical study population.7

Admittedly there have been good reasons why people older than 65 have been excluded from trials:

  • As mentioned before, older people are at greater risk of drug side-effects, which can be serious. When this happens in a clinical trial, the patient may have to drop out of the trial which is always a problem for research teams.
  • When a patient has several medical conditions – as older adults often do – this can make trial findings ‘messier’ and more difficult to interpret, which means the trial may end up being less valuable.
  • There are also various practical challenges: such as supporting older people with transportation to the study clinic, and the need for carers to be involved in helping the patient to comply with the trial requirements.

But these issues should be seen as challenges, not excuses. It will also become harder to side-line the older population in clinical research because Regulatory Authorities (the authorities who license new medications, such as the US Food and Drug Administration (FDA)) are tightening up. Some people think drugs for chronic diseases that are not adequately tested in people over 65 years old may soon not make it through the approval process.8

A New Definition of ‘Older People: 75 Is the New 65

Of course, older people are not the only group to have been consistently under-represented in clinical trials. A similar situation existed with women and also with children. However, the FDA has announced its position has “evolved from a view that we must protect children from research to a view that we must protect children through research.” 9

Many experts and policymakers recognize a need to adjust the definition of ‘older person’ from 65+ to 75+ to reflect the ageing of society.10 By continuing to exclude the over-65s, those who design and fund clinical trials could be living in the past.

Sources:

  1. https://www.nia.nih.gov/research/publication/global-health-and-aging/humanitys-aging
  2. ICH. Guidance for Industry: E7 Studies in Support of Special Populations: Geriatrics. Questions and Answers. February 2012, ICH.
  3. Routledge AP, O’Mahony MS, Woodhouse KW. Adverse drug reactions in elderly patients. Br J Clin Pharmacol 2003, 57(2): 121-6.
  4. http://www.nps.org.au/topics/ages-life-stages/for-individuals/older-people-and-medicines/for-health-professionals/ageing-related-changes
  5. http://www.americannursetoday.com/preventing-polypharmacy-in-older-adults/
  6. Herrera AP, Snipes SA, King DW et al. Disparate inclusion of older adults in clinical trials: priorities and opportunities for policy and practice change. Am J Public Health 2010; 100(Suppl 1): S105–S112.
  7. http://newoldage.blogs.nytimes.com/2011/08/19/clinical-trials-neglect-the-elderly/?_r=0
  8. http://www.outsourcing-pharma.com/Clinical-Development/Clinical-trials-should-involve-the-elderly-more-say-experts
  9. http://contemporarypediatrics.modernmedicine.com/contemporary-pediatrics/news/modernmedicine/modern-medicine-news/fda-addresses-guidelines-pediatric-
  10. www.who.int/healthinfo/survey/ageingdefnolder/en/

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January 18, 2016
Precision Medicine Without ‘Precision Trials’?

Precision Medicine Without ‘Precision Trials’?

Find out why and how clinical trials have to change to allow advances in precision medicine.

by Durhane Wong-Rieger
November 16, 2015
Gender equality in clinical research – are we already there?

Gender equality in clinical research – are we already there?

One would think that gender equality in medical research has been a reality for a long time, right? Let's have a look at the facts.

by Durhane Wong-Rieger
April 21, 2015
Patients Becoming Partners in Research

Patients Becoming Partners in Research

When it comes to clinical trials, everyone knows the drill: experts create and patients participate. Right?

by Editorial Team
Patient Research Exchange - Patient Research Exchange

Patient Research Exchange

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Precision Medicine Without ‘Precision Trials’? - Patient Research Exchange

Precision Medicine Without ‘Precision Trials’?

Find out why and how clinical trials have to change to allow advances in precision medicine.

A pipe dream or nearly a reality? How close are we to a medicine that is precisely matched to your unique genetic makeup, environment, and lifestyle? Such a highly personalized medicine would not only give you the best possible clinical outcomes but would likely also cause less side effects. The reality is that such medicines are not yet standard but they will “sooner than later” replace the “large population” drugs that have dominated drug development for the past 40 years. Those blockbuster drugs represented breakthroughs in treating so many common conditions, including cancer, cardiovascular disease, neuromuscular conditions, inflammatory diseases, and immune disorders. But because they were developed for use in patients highly diverse in other characteristics, the benefits and adverse effects often varied significantly from one patient to another. In contrast, the new generation of personalized, precision, or targeted medicines designed to treat specific subsets of patients not only produce superior desired outcomes but also less unwanted effects. A future where highly specialized therapies will be the “new norm”, even for the most common conditions, is definitely within sight and indeed gaining momentum thanks to initiatives such as the Precision Medicine Initiative launched by US President Barack Obama1, the Canadian Institute for Health Research Personalized Medicine Initiative2, and European Commissions on personalized medicine pilot projects.3

Given the seemingly haphazard results from many of the current blockbuster drugs – some have been shown to only improve the condition in as few as 1 in 20 persons4 – the impetus toward personalized therapies is not only desirable but highly overdue. According to the US National Institutes of Health, precision medicine is ‘an emerging approach for disease prevention and treatment that takes into account people’s individual variations in genes, environment and lifestyle’.5 BUT … in order to develop and approve drugs that are tailored to specific people, the entire clinical trial process needs to follow a personalized approach.

Clinical trials should focus on individual, not average, responses to therapy.

Classical clinical trials are based on findings from hundreds or even thousands of participants, collecting a limited number of parameters, often not taking into account many additional factors – like genetic makeup or lifestyle – that shape a person’s response to a certain treatment. The large number of participants is necessary in order to increase the chance of achieving statistically significant results. However, these large trials are not very good at predicting which individuals will respond positively and also which develop adverse effects. Post-trial analyses are required to determine which factors were responsible for the different outcomes in different people. Moreover, because extraneous information may not have been collected or sufficiently documented in the course of the trial, additional studies are often required to identify meaningful differentiations among potential users.

In contrast, targeted drug development is based on alternative trial designs that attempt to accommodate relevant variability among patients from the outset. Basket trials, often used for cancer research, test drugs based on their mode of action rather than a specific cancer site. Participants included in a single basket trial may have different types of cancer, for example breast cancer or colon cancer, but all have a common causative or facilitating factor, such as tumour expression of an abnormal protein, which is the target of the investigated drug.6

Umbrella trials follow an opposite strategy. In an umbrella trial, participants have the same clinical diagnosis but they vary in terms of specific genetic markers or other factors. Patients are assigned to a test therapy according to their individual profile, so several different drugs may be tested for the same disease in one trial.6

Finally, in cases where there may be considerable uncertainty as to the probable impact of an investigative therapy, researchers and regulators are collectively designing and approving adaptive trial protocols that allow modifications in interventions for a subset of or individual participants while the study is ongoing, based on participant responses.6

These alternative trial designs are a step in the right direction, but some experts claim that these studies are still not sufficiently personalized. Consider a hypothetical basket trial for KRAS-mutated tumours. Mutations in the KRAS gene may be evident in many different kinds of cancers7, and a single investigative drug against various types of cancer cells with this mutation is tested using a basket trial design. However, even if the patients’ tumours were tested positively for the presence of KRAS-mutated cells, we cannot guarantee success for all affected patients since many other factors are involved in the treatment response. Another problem is that diseases like cancer might ‘evolve’ in the course of the therapy and become resistant to a previously effective treatment.8

N-of-1 trials can give patients a sense of empowerment and more control and understanding of their condition

According to Nicholas J. Schork, director of human biology at the J. Craig Venter Institute in La Jolla, California, a better solution is ‘clinical trials that focus on individual, not average, responses to therapy’. In other words, we need to utilize N-of-1 trials.4

N-of-1 or single subject trials are not a new concept. In fact, physicians have been doing this for a long time as part of their clinical practice. In certain areas of medicine such as rare diseases, N-of-1 trials have been utilized based on necessity. However, the results from these trials, when conducted in an ad hoc fashion, are not easily extrapolated to other patients.4 A more formalized approach may be necessary to provide guidance to other clinicians and/or to lead to an expanded approved indication for the therapy.

In well-designed N-of-1 trials, different treatments – either active treatments or placebo – are evaluated in just one patient over a period of time. Despite the simplicity of this approach, N-of-1 trials require elaborate study design, including sound statistical methodology and strategies, such as patient and clinician blinding. Although the participant is the main beneficiary of these kinds of trials, combined results of many equally designed N-of-1 trials can provide medical information for subsets of the population or even the general population.9

In addition to the medical benefit, research has shown that N-of-1 trials can give patients a sense of empowerment and more control and understanding of their condition.10 By collaborating with their health care professionals and playing an active role in their treatment, patients have the chance to be study contributors rather than study subjects.

Sources:

  1. https://www.whitehouse.gov/precision-medicine
  2. http://www.cihr-irsc.gc.ca/e/43707.html
  3. http://www.permed2020.eu/_media/2014-03-27_PerMed_EC_presentation.pdf
  4. http://www.nature.com/news/personalized-medicine-time-for-one-person-trials-1.17411
  5. http://syndication.nih.gov/multimedia/pmi/infographics/pmi-infographic.pdf
  6. http://am.asco.org/clinical-trial-designs-studying-targeted-therapies
  7. http://www.hindawi.com/journals/bmri/2010/150960/
  8. http://www.ncbi.nlm.nih.gov/pubmed/16454743
  9. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3118090/
  10. http://www.effectivehealthcare.ahrq.gov/ehc/products/534/1844/n-1-trials-report-130213.pdf

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November 16, 2015
Gender equality in clinical research – are we already there?

Gender equality in clinical research – are we already there?

One would think that gender equality in medical research has been a reality for a long time, right? Let's have a look at the facts.

by Durhane Wong-Rieger
May 13, 2015
Towards Patient-Engaged Research

Towards Patient-Engaged Research

Research is finding answers to questions. Patients have lots of questions.

by Durhane Wong-Rieger
April 21, 2015
Patients Becoming Partners in Research

Patients Becoming Partners in Research

When it comes to clinical trials, everyone knows the drill: experts create and patients participate. Right?

by Editorial Team
Gender equality in clinical research – are we already there? - Patient Research Exchange

Gender equality in clinical research – are we already there?

One would think that gender equality in medical research has been a reality for a long time, right? Let's have a look at the facts.

Is the fact that women are still underrepresented in clinical research a cause for concern? Or does it make no difference? The evidence suggests that every female needs to be concerned.

Jill Becker, a senior research scientist at the University of Michigan, explained in a New York Times article: “One of the underlying assumptions has been that females are simply a variation on a theme […] that if you’ve learned about the male you’ve learned enough to deal with both males and females.” However, we know this is not always the case. In fact, women often react differently than men to the same drug.1

A study from 2001 reported that female patients have a 1.5 to 1.7-fold greater risk of developing adverse drug reactions than men. According to the authors the reasons for this increased risk included gender-related differences in pharmacokinetics as well as immunological and hormonal factors.2 It is known that psychotropic drugs like painkillers or antidepressants have gender-specific effects. Anticonvulsants might be less effective in young women because a liver enzyme, responsible for metabolizing these drugs, is especially active in this group.3 On the other hand, other drugs might have a stronger or longer lasting effect in women. For example, in 2013 the FDA recommended that women should reduce their dose of a particular sleeping pill, as it takes them longer to eliminate the substance from their bodies.4

In order to completely close the gender-gap in medical innovation, there is still a need for education for all stakeholders, from researchers in the lab to those who design late-stage clinical trials.

Despite these differences, women continue to be underrepresented in clinical trials. One reason offered by some investigators is that men are cheaper and easier to study as the female estrus cycle has to be considered in the study design; moreover, there is the fear that inclusion of women of childbearing age might endanger potential unborn children.5 Although action should be taken to avoid any complications, women can’t be excluded from clinical trials for these reasons anymore.

To address the underrepresentation, the US National Institutes of Health (NIH) established the Office of Research on Women’s Health (ORWH) in 1990. Since 1993 all NIH-founded clinical research projects are required to include women in representative numbers.6 In the European Union, the Clinical Trials Regulation states that a clinical trial has to represent the population groups that are likely to use the investigated product.7 However, despite these legislative guidelines, women continue to be underrepresented, especially in early phases of experimental drug studies.8

The gender gap differs between disease areas. In some fields it has already closed, but in others it is still a big issue and is inadequate to ensure that evidence-based sex-specific recommendations can be made.9 As reported in the Journal of Women’s Health, in the US almost half of the study participants in post-approval studies for medical devices for ear, nose and throat conditions were female. However, in cardiovascular studies only 32% were women.10 A European study came to the same conclusion and also found that approximately half of the trials did not report an analysis of the results by gender. The reason for this might be the misconception that cardiovascular disease is a male disease, although it is also a leading cause of death in women.11

The need for inclusion doesn’t just start at the clinical trial phase. In pre-clinical research, scientists often prefer single-sex studies with only male cell lines or animals because they reduce variability and make it easier to detect the effect being studied.12 As well as putting women at risk, insufficient representation of female cells and animals in experiments and inadequate analysis of data by sex may contribute to the observed rise of irreproducibility in pre-clinical biomedical research.6 Adding female cell lines and animals to experiments requires the whole study to be scaled up in order to maintain statistically representative numbers for each sex, which implies higher costs for the research teams. To address this, in 2014 the NIH announced that they would provide 10.1 million USD in grants to more than 80 scientists to ensure that sufficient women are included in their clinical trials, that the early pre-clinical research is representative of both genders and that gender differences are analyzed in the resulting data.

In clinical as well as pre-clinical research, things are moving in the right direction. However, in order to completely close the gender-gap in medical innovation, there is still a need for education for all stakeholders, from researchers in the lab to those who design late-stage clinical trials.

What is the situation in your field of expertise? Are women adequately represented? We would be happy to hear about your experience! Follow this link and join the discussion.

Sources:

  1. http://www.nytimes.com/2014/05/15/health/nih-tells-researchers-to-end-sex-bias-in-early-studies.html?_r=0
  2. http://www.ncbi.nlm.nih.gov/pubmed/11770389
  3. http://www.scientificamerican.com/article/psychotropic-drugs-affect-men-and-women-differently/
  4. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm334798.htm
  5. http://www.jci.org/articles/view/19993
  6. http://www.nature.com/news/policy-nih-to-balance-sex-in-cell-and-animal-studies-1.15195
  7. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2014_536/reg_2014_536_en.pdf
  8. http://www.ncbi.nlm.nih.gov/pubmed/23027449
  9. http://circoutcomes.ahajournals.org/content/3/2/135.full
  10. http://www.beckershospitalreview.com/supply-chain/9-statistics-on-male-female-participation-in-fda-clinical-trials.html
  11. http://www.euractiv.com/health/health-experts-urge-gender-balan-news-518335
  12. http://www.nytimes.com/2014/09/23/health/23gender.html?action=click&contentCollection=Health&region=Footer&module=MoreInSection&pgtype=article&_r=3
  13. http://www.nih.gov/news-events/news-releases/new-supplemental-awards-apply-sex-gender-lens-nih-funded-research

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September 22, 2015
Crowdsourcing Clinical Trials

Crowdsourcing Clinical Trials

The notion that too many people involved in a project can ruin it is quite common.

by Editorial Team
August 3, 2015
Clinical Trial Recruiting Tools in the Digital Age

Clinical Trial Recruiting Tools in the Digital Age

Tablets, smartphones, and even self-driving cars likely come to people’s minds when they hear about the information technology revolution affecting the way we work and play.

by Editorial Team
August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
Journal of Clinical Oncology - Patient Research Exchange

Journal of Clinical Oncology

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UC Davis Health System - Patient Research Exchange

UC Davis Health System

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Crowdsourcing Clinical Trials - Patient Research Exchange

Crowdsourcing Clinical Trials

The notion that too many people involved in a project can ruin it is quite common.

Steve Jobs and Apple showed us that a singular vision for the way something should be can create amazing, beautiful and revolutionary things. But is it always true? Some clinical trial experts don’t think so—and are working to prove otherwise.

For decades research studies were mainly designed by small groups of 10 or fewer medical professionals. Once they created the parameters and protocols only then were patients and caregivers enlisted to provide the data. This has started to shift as experts better appreciate the in-depth day-to-day knowledge of those living with conditions and see the importance of having their input earlier in the clinical trial design process.

A similar change is underway with the use of the Internet. While researchers have collaborated online for nearly two decades it’s only recently that they’re using the Web to connect with patients. Thanks to the use of crowdsourcing, many people are working together offering ideas, content and even funding to help design the best clinical trials possible.

Crucial to this new way of thinking and doing things is Protocol Builder, a proprietary clinical trial crowdsourcing tool from Transparency Life Sciences, the first clinical-stage drug development company based on open innovation. Rather than looking for other like-minded experts, Protocol Builder hopes to encourage structured input from skeptics as well as ideas the original designers might not have considered.1,2, 2a

Feedback is solicited on topics like the study’s scientific rationale, inclusion and exclusion criteria and alternative endpoints from a wide range of experts in other areas besides just the trial subject. Patients and advocates are also encouraged to submit information about what questions are most important to them. This creation and exchange has not only led to new and important ways of looking at clinical trials but also hopefully more speedy success with faster trial development and more patients knowing about them and being interested in participating.1

Crowdsourcing is a way of obtaining information, input or funding for a project or task from a large number of people, typically through the internet or social media.

Crowdsourcing has proven particularly helpful in the study of Amyotrophic Lateral Sclerosis, also known as ALS or Lou Gehrig’s disease. Because of its fast moving nature, estimating its progression is an important part of making sure the patient gets the best care and decisions are made at the correct time.3,4

Through a crowdsourced competition, 37 groups from around the world—many with no ALS experience —created tools to better predict how the disease would develop which in turn could make it easier to test new medications and their impact. Crowdsourcing brought in new ideas and different perspectives as well as providing a clear assessment of the solutions’ strengths through its design of blinded side-by-side comparisons of competing methods.3,4

Another interesting company is FasterCures, a center of the Milken Institute working to speed up the medical research process by encouraging collaboration, innovation and efficiency.4a

While these are several very specific examples of crowdsourcing’s success, many people believe there are more general benefits as well.

While researchers have the most hands-on, in-depth knowledge, they aren’t the only ones with good ideas. Many healthcare professionals on the front lines of patient care see interesting areas of potential study, but lack the time, resources, and knowledge to write the detailed protocols necessary to conduct clinical trials. The Alliance for Clinical Trials in Oncology is working to change this by offering a place online for people to submit a concept for possible further study.5,6

Another fascinating option would involve researchers being able to post details of their proposed trials online seeking comments from other experts, caregivers and patients. By putting heads together in advance, there may be fewer revisions later.5

Finally, more online information, whether it’s sharing favorites, casting votes or making suggestions, can’t hurt and it can help patients become more aware of, more knowledgeable about and hopefully more interested in participating. This would be huge given that only about 3 percent of U.S. cancer patients enroll in research studies.5

Ultimately, while some might believe more opinions equals more confusion and some must lead while others follow, clinical trial researchers are betting on a different outcome—that more ideas will equal more insight, inspiration and breakthroughs.


Reviewed and amended on September 22, 2015

Sources:

1. http://linkis.com/journals.lww.com/onc/d1TS4

2. http://transparencyls.com/how-it-works

2a. http://www.crowdsourcing.org/document/transparency-life-sciences-to-conduct-sarcoidosis-trial-for-auven-therapeutics-kiacta-using-crowdsourcing-and-telemonitoring/33841

3. http://www.nature.com/nbt/journal/v33/n1/full/nbt.3051.html

4. http://news.sciencemag.org/brain-behavior/2014/11/crowdsourcing-project-predicts-progression-neurodegenerative-disease

4a. http://www.fastercures.org/about/who-we-are/

5. http://connection.asco.org/Commentary/Article/ID/3762/Three-Ways-to-Improve-Clinical-Trials-through-Crowdsourcing.aspx

6. https://www.allianceforclinicaltrialsinoncology.org/main/public/standard.xhtml?path=%2FPublic%2FSCRC

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August 3, 2015
Clinical Trial Recruiting Tools in the Digital Age

Clinical Trial Recruiting Tools in the Digital Age

Tablets, smartphones, and even self-driving cars likely come to people’s minds when they hear about the information technology revolution affecting the way we work and play.

by Editorial Team
August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
September 22, 2015
Making the Partnership between Researchers and Patients Work

Making the Partnership between Researchers and Patients Work

From Sherlock Holmes and Dr. Watson to Abbott and Costello, we know that partnerships between opposites can lead to solved mysteries and hilarious laughs.

by Editorial Team
Making the Partnership between Researchers and Patients Work - Patient Research Exchange

Making the Partnership between Researchers and Patients Work

From Sherlock Holmes and Dr. Watson to Abbott and Costello, we know that partnerships between opposites can lead to solved mysteries and hilarious laughs.

In the case of researchers and patients joining forces in developing clinical trials, the stakes are even higher as they seek to make life-changing discoveries, together.

The trick is learning to recognize the assets each party brings to the relationship and work as a team. The roles of researcher and patient have been refined and reinforced over decades with a parent/child relationship firmly established. It’s only natural that a change in the role patients play and collaboration between patient and researcher is going to take some time to get used to. The approach is new, still evolving and models are still being tested.

Researchers, for example, have been trained to adhere to scientific methodology, often ignoring real-world urgencies for useful findings. Some may also question the value of patient involvement in the early stages and wonder if patients have the knowledge and skills necessary to contribute constructively.1

Teaming up researchers and patients can be a win-win for everyone as long as it’s treated like any relationship—with effort, enthusiasm and above all respect

Patients also have their own concerns. Some worry that researchers won’t listen or take them seriously. Many may be put off because they feel they don’t have enough scientific training, don’t understand the jargon and (most terrifying) will have to deal with statistics. Paradoxically, if the patients involved are “too skilled,” researchers may wonder if they are truly representative of others with the condition who aren’t included in the trial. Finally, patients may be apprehensive over potential conflicts of interest between the needs of the trial and their involvement with patient groups.1

Both groups may also share the concern that their collaboration could slow down the development process, delaying the trials and holding up the acquisition of knowledge and implementation of treatments.1

Fortunately past and current partner experiences have provided some useful recommendations and advice for best practices. Rather than focusing on doing what they feel has worked before, researchers from within pharmaceutical and life sciences companies do well when they are open-minded to new ways, accept patients as true partners, and value patient-centred outcomes that benefit the ultimate end user. Understanding what really matters to patients and being concerned about the patients’ benefits are crucial to presenting study findings in a way that is easily understandable and useful for patients—not just clinical researchers.2 Patients feel most valued as partners when they believe the researchers and companies are working for their same goal of finding safe, useful solutions as quickly as possible while still adhering to rigorous scientific methodology.

And the benefits of the “patient effect” can be great. The research may be more on target with patients helping refine and prioritize what is studied and also the way the trials are designed, such as how data is collected and analyzed. Patient participants may also question researchers’ assumptions and encourage thinking in new directions.1

From a practical standpoint, patient involvement may result in an increased number of study participants as they can offer recruitment suggestions, spread the word and serve as an example. They can also help with wider dissemination of findings through friends and networks and offer feedback on information’s understandability and usefulness to the general community.1

As patients move more toward directing their own healthcare they can gain untapped knowledge as well as an increased sense of self-confidence, usefulness and feelings of ownership in the process. On a larger scale, they can help encourage better understanding between patients and researchers on topics like how the trial is presented to patient recruits plus assist in directing the content of clinical trials to areas that are truly useful to people in real life.1

The key to success is finding the best ways to work together including pairing the right research project and principal investigator with the correct type of patients. The researcher should be able to interact well with regular people as part of the team while the patients need to have in depth knowledge and experience with the subject matter going in.1

Collaboration should start early with everyone’s roles clearly defined and realistic expectations set. Initial training and development for patient participants on research methods, statistics and understanding outcomes can also be helpful. Communication including feedback and acknowledgement of contributions are important too.1

Teaming up researchers and patients can be a win-win for everyone as long as it’s treated like any relationship—with effort, enthusiasm and above all respect.


Reviewed and amended on September 22, 2015

Sources:

  1. http://www.patientpartner-europe.eu/en/resources/active-involvement-in-the-process
  2. http://www.lifescienceleader.com/doc/how-to-build-real-patient-centered-pharmaceutical-companies-0001

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April 21, 2015
Patients Becoming Partners in Research

Patients Becoming Partners in Research

When it comes to clinical trials, everyone knows the drill: experts create and patients participate. Right?

by Editorial Team
August 3, 2015
Clinical Trial Recruiting Tools in the Digital Age

Clinical Trial Recruiting Tools in the Digital Age

Tablets, smartphones, and even self-driving cars likely come to people’s minds when they hear about the information technology revolution affecting the way we work and play.

by Editorial Team
August 3, 2015
Online Patient Communities Changing the Nature of Clinical Trials

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

by Editorial Team
Toolkit: Increasing Global Diversity in Clinical Trials - English (NMQF) - Patient Research Exchange

Toolkit: Increasing Global Diversity in Clinical Trials - English (NMQF)

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Toolkit: Increasing Global Diversity in Clinical Trials - Vietnamese (NMQF) - Patient Research Exchange

Toolkit: Increasing Global Diversity in Clinical Trials - Vietnamese (NMQF)

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Toolkit: Increasing Global Diversity in Clinical Trials - Spanish (NMQF) - Patient Research Exchange

Toolkit: Increasing Global Diversity in Clinical Trials - Spanish (NMQF)

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Toolkit: Increasing Global Diversity in Clinical Trials - Korean (NMQF) - Patient Research Exchange

Toolkit: Increasing Global Diversity in Clinical Trials - Korean (NMQF)

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Toolkit: Increasing Global Diversity in Clinical Trials - Chinese (NMQF) - Patient Research Exchange

Toolkit: Increasing Global Diversity in Clinical Trials - Chinese (NMQF)

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Online Patient Communities Changing the Nature of Clinical Trials - Patient Research Exchange

Online Patient Communities Changing the Nature of Clinical Trials

The Internet may have made us smarter but social media gives us new opportunities to meaningfully connect with communities

From “meeting” new people with similar interests to participating in causes we care about, Facebook, Twitter and more have expanded our relationships and involvement on a global scale. This fact has not been lost on companies and researchers looking to fill clinical trials.

Researchers around the world are conducting more studies than ever before and it’s naturally a challenge to find willing participants. Without enough qualified participants, trials can be delayed or even terminated.1

Enter the global village (aka social media)—and some innovative ways to reach it.

Digital partnerships, like the one undertaken by CureClick.com, WEGO Health and TrialResearch, hope to solicit the help of patient opinion leaders in recruitment efforts. This convergence brings together over 100,000 experts in healthcare social media, opinion leaders and a database with easy-to-understand information about clinical trials.2

Digital partnerships hope to solicit the input of patient opinion leaders in recruitment efforts.

Entrepreneurs have also realized the power of tapping into like-minded communities. PatientsLikeMe offers an online data-sharing platform for its community of people with serious medical ailments that helps individuals manage their conditions as well as assisting clinical trials in recruiting patients.1,3 Acurian provides patient enrollment often using social media and improves participation through better engagement and retention.1,4

Clinical Trials GPS makes finding and tracking studies to participate in easier than ever including taking location into account.1 The U.S. government is getting involved with the National Cancer Institute’s (NCI) app allowing users to check out the NCI clinical trials database, save their favorites and share on social media.1

While these online tools can be useful when it comes to recruitment, researchers have found it can also lead to some headaches once trials are underway especially when sharing becomes oversharing.

Many clinical trials rely on researchers and subjects not knowing if they are receiving the experimental treatment, standard therapy or a placebo. However, with online forums, blogs, Facebook groups and more at their fingertips some patients in trials for multiple sclerosis, hepatitis C and Lou Gehrig’s disease (amyotrophic lateral sclerosis) found themselves detailing their symptoms to others online in an attempt to compare and uncover whether or not they were receiving the trial medication.5

That’s not all.

Some patients have shared tips on ways to get accepted into trials even if they don’t meet all the criteria. Others, perhaps frustrated with the often lengthy timeframes involved in clinical studies, have collected and shared data during the trial to try to get a sense whether the treatment will be successful.5

It’s understandable that researchers and pharma companies would fear these types of behaviors because they could jeopardize both the trial outcome and the patient’s well-being. Encouraged by information online (that may or may not be accurate), patients might jump to conclusions as to whether or not they’re receiving the experimental treatment. If they think they are not, researchers worry that they could quit the trial prematurely and threaten its ability to be completed. Experts are also concerned that by reading about others’ experiences online patients could be influenced into reporting incorrect symptoms.5

Despite knowing the risks associated with these issues, patients aren’t about to go back to the dark ages of pre-Internet. Websites provide valuable information and social media and other forums allow important access to advice, support and community from others who know what they’re going through. A good percentage would likely quit a clinical trial before agreeing to limit their interactions online.

So what’s the answer?

Many believe it lies in education—for everyone. For researchers, this means understanding the benefits patients receive by sharing their experiences on social media and ultimately changing their expectations to fit this new normal. Patients, however, must also realize the power of their words. One person’s comments can have far-reaching and unintended consequences ranging from changing someone’s thoughts or feelings to actually spurring them to take an action that could affect the result of a clinical trial, impact the future of a treatment and perhaps most dangerously, endanger the trial participants.5

By recognizing this issue, organizations like the Center for Information and Study on Clinical Research Participation (CISCRP) are working to get the word out. Through its Speak Out, But Speak Smart website, the CISCRP provides videos educating people about how their words could negatively influence others’ interpretations of their symptoms and even affect the outcome of the trial.6

The Internet and social media’s ability to connect people is amazing. With its popularity and global reach it’s very likely its impact on clinical trials recruitment will be too.


Reviewed and amended on August 3, 2015

Sources:

  1. http://www.techipedia.com/2014/social-media-clinical-research/
  2. http://www.mmm-online.com/trial-recruiting-looks-to-tap-online-community-leaders/article/386519/
  3. http://www.patientslikeme.com/about
  4. https://www.acurian.com/acurian-solutions.html
  5. http://www.wsj.com/articles/researchers-fret-as-social-media-lift-veil-on-drug-trials-1406687404
  6. https://www.ciscrp.org/primer/index.php#about-trials

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Tablets, smartphones, and even self-driving cars likely come to people’s minds when they hear about the information technology revolution affecting the way we work and play.

In reality, however, it goes much deeper than a myriad of apps that count your steps. New technology is paving the way for discoveries and novel abilities that won’t just enrich our lives, they may actually save them.

Take clinical trials for example.

Thanks to advances in digital imaging, 3D computer modeling and cloud-based data analytics, researchers are better able to understand cancer tumors at a molecular level. This in turn has opened up whole new areas of treatment exploration.1

That’s not the only way tech is changing research studies.

By helping to get the word out about research, raising awareness among patient communities, and increasing access, making contact with people through the Internet and social media are proving helpful in solving long-standing issues of recruitment and the resulting delays or cancellations of trials.

The internet and social media are proving helpful in solving long-standing issues of recruitment and the resulting delays or cancellations of trials.

This is no small feat. In less than 10 years the number of registered studies has gone from 50,000 in 2007 to more than 180,000 in 2014.2 That’s a lot of trials and a lot of people needed to fill them. It’s no wonder 85 percent of studies have been delayed due to not enough participants.3

Experts are using a wide range of technological options from websites to social media and more in an attempt to cast the widest net possible.

The Michael J. Fox Foundation for Parkinson’s Research created Fox Trial Finder, a site to help match those with Parkinson’s to appropriate trials (as well as sign up people without Parkinson’s to be control subjects). So far, over 40,000 individuals have registered to participate.4

Another site, Smart Patients, serves as an online community where patients can connect and learn about their ailments, the latest treatments and the most current science. It also has a built-in clinical trial search engine to make it easier to find studies, track them and discuss with others.5,6

Even the U.S. National Institutes of Health has gotten involved with the launch of their website, ClinicalTrials.gov. This resource includes a registry and results database of human clinical studies from around the world. Incorporating both publicly funded and privately supported trials, the site lists over 185,000 current entries.7

Social media is another way researchers are using the Internet to help with enrollment. Pharmaceutical companies have found success partnering with PatientsLikeMe, an online forum for people with medical conditions, and using its data-sharing platform to find subjects.3 WEGO Health, a network of over 100,000 bloggers, tweeters, Facebook participants and more, offers assistance in connecting patients with study trials among other services.8

More direct-to-patient approaches are being used as well. For example, Quintiles, a clinical research organization, skips physician sites and reaches out directly to people through mobile and video ads, social media sites and patient community groups it has created.9 One such example is their Mediguard, a site designed to help people monitor their medications and alert them to recalls and drug interactions. With over 2.6 million people registered, the site also provides access to a large group of potential clinical trials subjects.9,10

Quintiles has ventured onto Facebook and created a Page entitled “I Am More Than Lupus.” In addition to being a place people can find information, support and connection, the Page specifically states it was created as a means of introducing research opportunities to the community including clinical research, observational studies and disease management programs.9,11

They aren’t the only ones trying new avenues. TrialReach, a company that works to facilitate more patient involvement in clinical trials, provides everything from self-service tools to translate study protocols into easy-to-use interactive web pages to help getting the word out about trials to assistance in tracking information.12

Still others are thinking even further outside-the-box. When Johnson County Clin-Trials didn’t get enough responses to email blasts seeking participants for a vaccine clinical trial, they contacted Mosio, a company specializing in text messaging to recruit and retain subjects. 1,541 texts were sent to people telling them they could be eligible for a clinical trial; the action resulted in 795 potential subjects and 265 ultimate trial participants. The response was five times the rate of that to emails.9,13

There’s no doubt technology has changed our lives. From constant information to instantaneous communication, things are moving faster than ever. With all these new recruiting tools available, hopefully filling clinical trials and getting results will too.


Reviewed and amended on August 3, 2015

Sources:

  1. http://www.cancer.gov/researchandfunding/areas/clinical-trials
  2. https://clinicaltrials.gov/ct2/resources/trends
  3. http://www.techipedia.com/2014/social-media-clinical-research/
  4. https://foxtrialfinder.michaeljfox.org
  5. https://www.smartpatients.com
  6. https://www.smartpatients.com/about
  7. https://clinicaltrials.gov
  8. http://www.wegohealthsolutions.com/solutions#recruitment
  9. http://www.mmm-online.com/cros-trial-blazing/article/384041/
  10. https://www.mediguard.org
  11. https://www.facebook.com/IAmMoreThanLupus/info?tab=page_info
  12. http://trialreach.com/trialmanager/
  13. https://www.mosio.com

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