Between September and December 2011 a citizens' jury, convened by the University of Glamorgan and supported by Genetic Alliance UK, met to discuss these questions and to deliberate between two opposing arguments: Making a case for change (regulators should be more permissive); and defending the status quo (regulators should not be more permissive).
The 12 jurors were either patients with serious and/or rare conditions, or family members of someone with a serious and/or rare condition. They paid particular attention to how patients with rare and/or serious conditions perceive the risks and benefits of new medicines; how permissive regulators should be with marketing authorisations; and how involved patients should be in regulatory decision making.
After five days of debate the jury reached its verdict and voted for the case for change, and proposed four recommendations.
First, jurors argued that regulators give insufficient weight to psychosocial factors in their decision making. Applications for new medicines are judged primarily on biomedical evidence and clinical outcomes, but for patients it is likely that psychological and social factors will be equally important.
Jurors would like to see greater weight placed on the psychosocial aspects of serious and/or rare conditions, and on the potential for new medicines to alleviate (or exacerbate) them.
They stated: 'The benefits should be wider than a clinical/medical perspective and also include psychological and social benefits... [for example] ability to work (paid or unpaid), ability to socialise, maintain relationships, contribute, quality of life, well being'.
Second, regulators should be more permissive for treatments for people with rare and/or serious conditions. Because such patients often have few, or no, effective treatments available to them they may be willing to take greater risks than the system currently allows. They should be given that choice.
When making marketing authorisation decisions, regulators must consider whether the advantages of the medicine outweigh the disadvantages, and whether the side effects are acceptable. In the case of serious and/or rare conditions, regulators should lower the threshold of what they consider to be acceptably safe, giving more weight to psychosocial benefits and involving patients in the decision making.
One of the jurors said: 'For anyone with a terminal illness...such as myself, I would even take a one in three chance, or one in two chance, that a drug could cause [me] harm...versus it will halt the progression of your disease. If there is a last chance for you to fend off a terminal illness, surely the patients (not regulatory bodies) should decide if they want to take the risks associated with it'.
Third, patients should be more involved in all stages of the process, from setting the research agenda, to post-marketing authorisation decisions.
Patients' experiences and preferences should be represented in all the processes leading to the development of new medicines. This would ensure the benefits that really matter to patients, and the levels of risk they are prepared to tolerate, are considered.
This is particularly important for serious and/or rare conditions, where the stakes are so high. Patient representatives (such as patient group members) should be supported as joint decision makers, alongside clinical experts, throughout the process.
Fourth, patients should be better supported to make their own decisions. Patients wish to decide which medicines they take, reflecting their individual circumstances, beliefs and preferences. The result of the above recommendations will be that patients with serious and/or rare conditions will be faced with more choices in future. Such decision-making is challenging, but possible for most patients.
During the discussion, one patient said: 'The choices I have made now, my consultant feels really guilty... about the medication regime I'm on... I say don't be so paternalistic, I made the decision... I know the risks I am facing.... that is my choice'.
However, they will need help from their clinical team and a variety of other sources. This includes relevant, credible, and understandable information about the potential risks and benefits of the new medicines. As part of this, jurors generated a list of questions to help guide patients when deciding on their own treatment options.
Overall, these findings demonstrate that patients are willing to take great risks for the potential cure or improvement of their condition. The jurors here recommend that regulators involve them in their decisions, allowing for the more appropriate development and licensing of medicines for patients with rare and/or serious conditions.