The 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.
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.
Patients Must Help Design Clinical Trials
Dr Jon Stamford BSc PhD DSc
The Cure Parkinson's Trust & Parkinson's Movement
It is a truth universally acknowledged that clinical trials are the bedrock upon which all medical progress is based. Without successful trials new medicines are not introduced, new treatments are not developed and new understanding is not gleaned. The furtherance of knowledge hinges upon the successful design, execution and interpretation of clinical trials.
It sounds simple when you put it like that. Yet too many trials fail and poorly designed studies, with little consideration to real world concerns, are doomed to failure. One of the most commonly cited reasons for failure of a clinical trial is patient recruiting and/or compliance. Patients drop out of studies, for a variety of reasons. And many a researcher bemoans the high dropout rate in some more expensive and extensive trials. Often the blame is explicitly placed firmly at the feet of the patients, and patient culpability can be assumed.
This is a fallacious argument. Lord Nuffield once famously said "if your experiment needs statistics, you should have designed a better experiment". The same applies to clinical trials. To paraphrase Lord Nuffield, if your trial needs better patient compliance than was attainable, you should have designed a better trial. If a clinical trial design flows to reflect real-world patient concerns, it is the fault of the trial designers not patients.
The solution is breathtakingly simple. Involve patients. Involve patients in every step of the process from conception to execution. Only when patients are involved in protocol design will the outcome be a clinical trial that is realistic, pragmatic and attainable.
Yet despite recognition of this obvious prerequisite, trials are still being designed without patient input. And it is hard to see the reasons why this practice should persist. To exclude from the design process the principal stakeholder group is either an act of wilful neglect or scientific arrogance. The first is, at best, an act of incompetence. The latter is perhaps more understandable -- if no less culpable -- as the death throes of the old order of patrician healthcare and research.
All of this has to change. If we want to have genuine participatory medicine, we have to acknowledge that this cannot be confined to the consulting room. It cannot simply be a change in individual healthcare. And while the notion of patient-physician partnerships began as a means of devolving decision-making more to patients, we cannot expect the concept to be confined solely to the consulting room.
Medicine is about research. And just as patients are key stakeholders in their own healthcare, so must they be key stakeholders in healthcare for the next generation. And perhaps here, we patients are to blame. Perhaps we have been insufficiently vocal. Perhaps we have failed to speak up when opportunities arose to influence these clinical practices. Perhaps our silence was taken as tacit acceptance of the status quo.
If we as patients want successful clinical trials, and I think we can assume that we do, it behoves us to help our clinical colleagues. It behoves us to insist that our views are heard, understood and actioned. We have to help our clinical colleagues understand that a better understanding of patients and a better understanding of science are one and the same.
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