Last week, I looked at a consultation in the UK concerning access to unlicensed medicines. The 12-week consultation, launched by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), seeks opinions on giving physicians the option to prescribe seriously ill patients certain medicines that are still progressing through Phase II and Phase III clinical trials. The blog is available here.
The scheme forms a key part of the UK government’s Strategy for UK Life Sciences, which aims to support innovation and give the UK a leading role in life sciences globally. Included in this is a provision to provide patients with better access to innovative medicines.
The MHRA has conducted a lot of research on the subject of early access to medicines and claims there is broad support for such a scheme. One of the main points emphasised in the agency’s research is that the potential risks of taking an unlicensed medicine will need to be fully and honestly communicated to the patient. There were some concerns that desperation may cloud decision making, but it was generally felt that earlier access to medicines would benefit those with “little else to lose.”
For patients with debilitating or chronic conditions, such as rheumatoid arthritis, views were mixed on the idea of early access. In cases where patients are not in immediate danger, such as suffering from high-blood pressure, it was felt that early access to medicine should not be given.
Who’s responsible? And who will pay?
One of the big questions if the scheme is implemented will be what happens if something goes wrong? For example, what if a patient dies from severe side effects? Participants in the MHRA’s research felt that liability should lie principally with the pharmaceutical company and the clinician who offered the treatment. At the same time, though, no one person or body can be made fully liable as patients must personally accept the risks of taking an unlicensed drug.
Another question will be how early access to medicines will be funded, and how it could affect other care needs. In general, the research showed that funding could be justified if early access delivers long-term cost savings for the NHS. However, the brunt of the cost should be the responsibility of the pharmaceutical manufacturer, which will be benefitting from patients volunteering for the medicines.
I wonder what the pharma industry will think of that? Companies will have to pay to submit their medicines to the scheme but it seems as if the benefits of getting medicines to patients earlier may outweigh the costs. However, competition may be tight as the MHRA expects only one or two medicines a year to be approved for early access.
The scheme has been welcomed by several industry associations, including the UK’s BioIndustry Association (BIA), although BIA hopes that more medicines will be approved under the scheme.
“This is a step towards facilitating the availability of promising medicines to patients at an earlier stage in their development lifecycle,” said Steve Bates, BIA Chief Executive Officer, in a press statement. “However, the proposal to limit the scheme to only one or two medicines a year, targeted at the end of phase III trials, does not, in the BIA’s view, meet the ambition the UK government set out in its Strategy for UK Life Sciences. This included an option to fully discuss the review of innovative medicines at an earlier stage of development, where supported by suitable data.”
The proposed scheme is open for comments until Friday 5 October. Comments can be posted or emailed to the MHRA. Full details are available on the MHRA website.
The agency is aiming to make a decision on implementing the scheme by the end of this year.