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Risks and benefits in new medicines. Who should decide?

Risks and benefits in new medicines. Who should decide?

Alastair Kent: The pursuit of safety when granting a licence to a new medicine is obviously important. Given that there is no such thing as a completely safe drug, the issue becomes one of establishing whether or not the anticipated health gains for patients are sufficient to outweigh the risks inevitably associated with prescribing a powerful (and potentially somewhat toxic) medicine to a patient with a serious and possibly life limiting disease.

Traditionally the evaluation has been made by committees of experts - scientists, ethicists, clinicians etc sitting without patient and family input to their processes in order to reach a conclusion about whether or not patients can be allowed to take the risk. While it is clear that these experts have an important contribution to make, patients and families are increasingly demanding a say in this decision making process.

Until recently though, these voices were seen as being anecdotal in nature, and so difficult to take account of in what was seen to be a rigorous, science based process. In order to turn anecdote into a trend and hence into evidence that could feed in to decisions as to whether or not an innovative medicine should be granted a Marketing Authorisation by the European Medicines Agency (EMA) or National Competent Authority; Genetic Alliance UK, which is one of the largest alliances of patient support organisations in the UK, decided to convene a Citizens’ Jury.

Working in partnership with the University of Glamorgan, we brought together a jury of patients and carers living with the daily consequences of a serious, chronic and life limiting conditions. Unlike most citizens’ juries this was not a cross section of society. Rather it was a panel representative of those with most to gain or lose from the decision to grant or withhold Marketing Authorisation.

The Jury met over a period of six days to cross examine witnesses and discuss evidence about how the process operates today. At the end of this period they produced a report. This recognised the importance of efficacy as a key measure - no patient has any interest in taking drugs that don't work, but jurors felt strongly that they want to be involved in deciding whether the benefit to be had is worth the potential risks associated with the new drug. As one juror wrote: "If I was in a plane and offered a parachute with a 90% chance of failing I would refuse it, unless that plane was spiralling out of control towards the ground, in which case I might well be glad of it".

Jurors also wanted greater emphasis on the psychological and social aspects of their disease to be factored in to regulatory decisions. They also wanted a say in the design of trials so the research fitted their lives better, rather than trying to fit their lives to the research, and support in taking novel therapies so they could work out the best concordance regime when problems arose. You can access both the full summary of recommendations by the citizens’ jury for free here:
What next? Further work, to quantify the Jurors conclusions and to see to what extent they resonate with non UK patients is in hand. Watch our website:  for further developments in the coming months.

About Genetic Alliance UK
Genetic Alliance UK is the national charity of 154 patient organisations supporting all those affected by genetic conditions. We aim to improve the lives of people affected by rare genetic conditions by ensuring high quality services and information are available to all who need them. We provide a united voice for all those affected by rare genetic conditions, enabling us to work together towards a common goal of making life better for patients, their families and carers.

Alastair Kent is Director of Genetic Alliance UK.


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