Early Access to Medicines Scheme - where are we now?
It’s been 6 months since the launch of the Medicine and Healthcare Regulatory Agency’s (MHRA) Early Access to Medicines Scheme (EAMS) in April 2014. Some might not expect too much to have happened in that time since, after all, companies needed time to review the scheme, the detailed guidance from MHRA, and consider the pros, cons and real costs of taking part. The first slots for submissions from companies only opened in May too.
But if we step back and reflect on the time that EAMS has been discussed, developed, refined, and eventually implemented, perhaps we might have expected more given it had its beginnings in Sir David Cooksey’s 2006 Review of UK health research funding so companies have known that such an option was likely to come to fruition for some time.
A reminder of EAMS
Perhaps the most crucial element of the scheme is that it could mean faster access for patients by perhaps a year or even more.
The scheme is voluntary and companies can put their product forward if they meet the MHRA criteria:
• The condition is life-threatening or seriously debilitating and there is a high unmet need
• The product is likely to offer major advantage over methods currently in use in the UK
• Reasonable expectation of a positive benefit risk balance based on preliminary evidence
The scheme has two key stages with the regulator:
• Step I: MHRA considers whether to award a Promising Innovative Medicine (PIM) designation based on early data
• Step II: An EAMS scientific opinion to describe the benefits and risks and if positive, a Public Assessment Report (PAR) and an EAMS treatment protocol to support prescribing decisions before licensing
Companies pay fees to the MHRA for both Step I and Step II of £4,027 for applying for a PIM, £29,000 for the scientific opinion. They must also collect real world evidence to inform the future licensing decision.
Companies are expected to provide their product at no charge to the NHS whilst part of EAMS. However, fast assessment (known as Step III) by the National Institute for Health and Care Excellence (NICE) and commissioning by NHS England (NHSE) is expected if all decisions are positive.
Companies can access a whole host of guidance from the MHRA on the scheme through their dedicated EAMS webpage.
Interest in EAMS
MHRA has told me that they’ve had 15 enquiries from companies, consultants and Universities about EAMS so there is interest, and from a range of people too.
As at 22nd October, there have been two PIMs awarded; one in September, and the other this month. A PIM designation is confidential unless the company itself wishes to make it public. Only one of the companies who have secured a PIM have made that public; Northwest Biotherapeutic secured the first PIM for their product, DCVax-L for Glioblastoma multiforme (GBM). GBM is the most lethal form of brain cancer and only 30% of patients are still alive a year after diagnosis.
No positive EAMS scientific opinions…yet
The two positive PIMS have not yet translated into EAMS positive scientific opinions from MHRA. However, in fairness it may simply be too soon, as there is a 75 to 90 day timeline (or longer at the request of the company) post PIM, to receive the full EAMS scientific opinion. We may yet see the first positive EAMS in a matter of weeks, if companies took up the chance to begin the process in the earliest slots from MHRA in May of this year.
It’s too early to either declare success or failure. This is especially because the true measure of success is not in the milestones of achieving PIM, or even a positive EAMS scientific opinion, but the improved health outcomes of patients balanced against the risks of earlier access. These might be precious extensions to life, or improved quality of life or avoiding worsening health status. But we can ask questions about where we are and whether there are material issues that need to be addressed to encourage uptake of EAMS:
- Do all companies who could benefit from EAMS know about it, and how do they view it?
- Do investors know about it, and how do they view it?
- Is the lack of funding for the product whilst within EAMS putting off companies? Does this affect smaller companies more than larger companies?
- Do companies feel confident in anticipating and meeting the requirements and associated costs of taking part in EAMS?
- How do prescribing clinicians, and the patients that they care for, view EAMS and will they take-up the opportunity for earlier access?
These questions and more will be discussed by Empower: Access to Medicine along with a range of expert commentators including Geoffrey Guy, of GW Pharmaceuticals and Babulal Sethia, the new President of the Royal Society of Medicine, in a forthcoming breakfast meeting on 26th November.
Leela Barham is an independent health economist and policy expert