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.
- 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.
- 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.
- Sharma NS. Patient centric approach for clinical trials: current trends and new opportunities. Perspect Clin Res 2015; 6(3): 134–138.
- National Institute of Health Research INVOLVE http://www.invo.org.uk/frequently-asked-questions/
- 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).
- 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).
- Domecq JP, Prutsky G, Elraiyah T et al. Patient engagement in research: a systematic review. BMC Health Serv Res 2014; 14:89.