Patient with medication

Clinical trials depend on the willingness of participants — and their involvement must be better acknowledged. Credit: Dina Rudick/The Boston Globe/Getty

When researchers at the drug giant Pfizer wanted to improve their clinical trials, the people who had taken part had a clear suggestion: researchers should say thank you.

It is a simple request, but a revealing one. When a clinical trial is completed, many participants walk away empty-handed. Most never hear from the investigators or the trial’s sponsor again. Many do not learn the results of the study in which they took part. It’s not good enough — and it indicates a deeper problem.

As we discuss in a News Feature this week, clinical-trial participants and the people who care for them are increasingly seen as partners in research. They are more informed than ever about their conditions and their medical options. And they are demanding — and receiving — more of a say in how clinical trials are designed and conducted. Some of this activity has been boosted by social media, which has allowed people with medical conditions and their carers to band together, share their experiences and advocate for change.

There has been some progress. In the late 1990s, Sharon Terry arrived for a meeting at the US National Institutes of Health to discuss a project to study a condition called pseudoxanthoma elasticum, which affects elastic fibres in some tissues. Even though Terry was the founder of an advocacy group that wanted to fund part of the study, she was told that she could not join the meeting because she wasn’t trained in biomedical research. She was eventually allowed to attend, but only if she served as an assistant to the medical director of her group. (Terry decided to pull funding for the project.)

Terry says it is hard to imagine the same scene today. Many pharmaceutical companies and medical centres now routinely consult people with a condition about clinical-trial designs, to get early feedback rather than risk launching a trial that no one wants to join. In response, trial organizers have tweaked protocols and created research programmes. In cancer studies, for example, this type of feedback has fuelled a push to find ways to combat the side effects of cancer treatment, and to improve care for survivors of cancer.

The benefits of such an approach are persuasive. Closer engagement with participants could yield clinical-trial protocols that are more effective and convenient for patients. This can translate into a trial that meets its enrolment targets more quickly, and which has a lower dropout rate.

Nancy Roach, founder of the advocacy group Fight Colorectal Cancer in Springfield, Missouri, recalls a meeting at the US National Cancer Institute about a trial in which participants would be assigned a treatment on the basis of their tumour mutations. An early proposal called for tumour samples to be characterized in three to four weeks. Roach, as well as others at the meeting who represented the participants, said it would not work: the longest they would be willing to wait before settling on a course of treatment was two weeks. After a subsequent survey of clinicians and investigators confirmed that they would also wait only two weeks before deciding on a treatment, the project team worked with pathologists at the trial sites to shorten the time it took to process the samples. The trial, called NCI-MATCH, initially had trouble meeting those goals because so many more people enrolled in the study than expected. So far, there are more than 6,000 participants.

More projects should follow this approach. As the phrase ‘patient engagement’ sweeps through medical science, it is important to make sure that it’s backed by meaningful action. It is not enough to put a potential trial participant in the room during meetings to discuss protocol designs. And it’s unacceptable that some scientists still consult people about a trial protocol only after it has been approved by a review board, when all involved are reluctant to revise it.

Engagement means offering training to participants and their carers so that they have the skills to contribute with confidence. Some say that it is intimidating to be in a room full of specialists, with the added responsibility of speaking for an entire community of people who have a medical condition. Engagement is also about researchers being willing to incorporate patient feedback. There are plenty of examples of best practice to follow, including lessons from social scientists who have studied community engagement to learn how best to achieve it.

Clinical trials depend on the willingness of participants, some of whom are critically ill. They all deserve a thank you. They rightly expect much more.