Taking part

Jon StamfordThe quiet revolution

I think we all accept the notion that clinical trials are central to the development of new medicines and new treatments for Parkinson’s. And it’s also true that the majority of these will be conducted by the pharmaceutical industry or academic units. Both have a strong vested interest in the success of trials. For the pharmaceutical industry, the outcome can be caged in very simple terms as profit and loss. In academia, promotion and academic standing are at stake. Yet neither have a greater vested interest in the outcome of clinical chance than the patients themselves. For us, the success or failure of a trial can be gauged much more directly and personally. We do not put profits or promotion on the line. We put our lives on the line. It really is as simple as that. Without our involvement as patients, there can be no trials, no discoveries, no progress.

And with that pivotal position comes equal responsibility. The gains for us as patients are huge. A successful major clinical trial is a headline event, its implications felt like ripples throughout the community. And the failure of a promising drug in late stage clinical trial is a cause for more than mere disappointment. It is the extinguishing of hope, or at least one fragment thereof. And when a drug trial fails because of something as simple as inadequate recruitment, retention or compliance, there is the sense that we have failed as a community. By community, I include physicians, scientists, patients and carers. Yet trials do still fail for reasons that are not linked to lack of efficacy of the drug. Often overoptimistic recruiting targets, unrealistic patient retention estimates and inadequate follow-up contribute to failures.

These are entirely avoidable but they require planning. Above all, they require patient involvement in the design process of the clinical trial to establish what is realistically attainable and what is not. A decade ago, it would have been difficult to write such a sentence. The idea that patients could be anything other than human guinea pigs would simply not have been understood. A patrician medical profession would have struggled to understand the concept or to see its importance.

But over the last few years, a quiet revolution has been taking place. Patients are now increasingly becoming part of clinical trials at all levels. Moreover, in the better centres, patient input is actively solicited by the clinical representatives of the trial design team. At its most enlightened, patient views are solicited from inception of the trial onwards.

And this has huge implications for patient involvement as subjects. Ask yourself this – would you be more comfortable entering a clinical trial designed entirely by physicians or one where the trial had been designed by a partnership of physicians and patients? I think that’s a no-brainer. My anticipation of a clinical trial designed by such a team would be extremely positive. I would be confident that any procedures would not be arduous. I would be confident that my best interests were being catered for. And I would be confident that the trial would balance the needs of all participants.

volunteer sign up

Ten years ago, you would struggle to find any clinical trials in which patients contributed to the genesis of a project. Now, whilst not yet universal, it is increasingly adopted and we anticipate that this will lead to greater success in clinical trials.

So if you have not already taken part in clinical trials, please consider doing so. Because you do so in the knowledge that the trials will, as likely as not, have been designed in consultation with patients – other people like you and me. To paraphrase Neil Armstrong, this is one small step for patients, one giant leap for patientkind.

Dr Jon Stamford

Read Jon’s blog, published in the BMJ about why patients must be involved in trial design

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