Last week we responded to the consultation from the MHRA to introduce an Early Access to Medicines Scheme. The MHRA has been asking for opinions on their proposal to allow early access to medicines, for serious conditions, where the drugs have finished phase three trials but have not yet been licenced. We welcomed this as an important first step that allows patients access to drugs more quickly but we also outlined why the Government needs to do more to explore flexible so-called adaptive approaches to licensing new drugs. We have also asked the MHRA to involve medical research charities and their patients in future work.
This was widely trailed in David Cameron’s Life Sciences announcement back in December 2011 as a crucial development in identifying new treatments that might be given conditional authorisation, or be licensed early – so that drugs could be developed faster and patients benefit from them sooner.
What we said
The MHRA has been asking for opinions on their proposal to allow early access, for serious conditions, to drugs which have proved promising in trials but before they have completed all the stages needed to be licensed under the current processes. Whilst there were lots of questions in the consultation about the economics of possible options to assess cost effectiveness and pricing – more properly aimed at pharmaceutical companies – we confined our response to the issues of most importance to research charities. This is what we said:
- We welcome any steps that allow patients to have access to drugs more quickly – but this sort of scheme is only likely to result in one or two additional drugs a year becoming available for patients;
- When considering benefits for a cost effectiveness assessment, psychosocial factors such as the impact of a drug on the ability to work, socialize or maintain relationships for example are just as important as more traditional biomedical or pharmaceutical analyses of treatments and cures; Genetic Alliance and the University of Glamorgan set up some panels of patients to explore their views of the most important benefits to them (you can read more about that here)
- Patients who have serious and/or rare conditions often have few or even no effective treatments available to them and may be prepared to take a greater risk than the system currently allows. Involving patients in the deliberative process and taking into account their views of risk/reward must inform decisions that regulators take;
- Developments such as stratified medicines targeted to small populations mean that the current model of running clinical trials of increasing scale prior to licencing is unlikely to be workable in the future, so change to our licensing regime is needed;
- This early access system is only a very small step on the path to the more proportionate and agile regulatory system that we need to increase benefits to patients and to support the life sciences sector. It should not be seen as a substitute for the wider plans that were announced in the UK Life Sciences Strategy back in December for a more “adaptive” licensing framework where regulators, clinicians, patients, the research community and industry are jointly involved in assessing the best regulatory outcome for a medicine to encourage innovation and get a greater number of promising drugs to patients more quickly; and
- The strategy proposed an Expert Group to lead this. Charities, with their valuable access to patients and well organised patient groups can play a very important role in helping MHRA to come to a settled view on how to assess risks and benefits and are keen to be involved in that Expert Group.
The Centre for Accelerating Medical Innovations (CAMI) has been set up, led by Dr Richard Barker – former Chief Executive of ABPI – to look at how research and development and the regulatory processes around medical research can be improved to bring advances more rapidly and affordably to patients. They are looking very closely at more adaptive licensing approaches.
Alongside this, Les Halpin, who has Motor Neurone Disease, has recently started a campaign, Empower- Access to Medicine, which is working to raise awareness of these issues and what remains to be done.
AMRC is contributing to the work of CAMI and others to explore possible adaptive licencing models that might be used to accelerate access to novel drugs and what these models may mean for patients. It is likely that there will be some pilot projects which could involve research into promising therapies funded by charities and we will keep you posted as these opportunities emerge.
You can read our full response here.