Early Access to Medicines Scheme – One Year Old
Early Access to Medicines Scheme – One Year Old
The Early Access to Medicines Scheme (EAMS) is a year old this month (April 2015). Under the scheme companies voluntarily apply to have their early evidence on a new medicine assessed by the Medicines and Healthcare Products Regulatory Authority (MHRA). If the MHRA believes that the product really is promising, and with what looks to be an acceptable benefit/risk profile - and a plan to manage risk in place - they will provide early guidance to help doctors and their patients decide whether to use the product before it’s formally licensed. With a year to reflect on, and with the very first full EAMS scientific opinion published in March 2015, it’s time to take another look at progress.
Seven Promising Innovative Medicines
The first stage of EAMS is for companies to apply for Promising Innovative Medicine (PIM) designation. Companies need to have had a pre-submission meeting with the MHRA to discuss the application, fill in the application form, provide a summary of the pharmacoviligance system master file, and a risk management plan using the MHRA template. The direct cost is a fee of £4,027 to the company, but the indirect cost is the time and effort to put the package together and engage.
So far, seven companies have been successful at securing the PIM status. It’s a signal about the potential value of the product and is a pre-requisite for going on to the next stage of the scheme.
• Northwest Biotherapeutics DCVax-L for malignant gliomas with the very first PIM in September 2014
• Bristol-Myers Squibb’s Opdivo (nivolumab) for melanonma in January 2015
• Novartis’ Zykadia (ceritinib) for anaplastic lymphoma kinase positive advanced non-small cell lunch cancer in March 2015
• MSD’s Keytruda (pembrolizumab) for advanced melanoma (although MSD did not publicise the PIM, but they must have secured it by virtue of achieving the very first EAMS positive scientific opinion)
MHRA keeps details confidential about applications and the success or otherwise of them, so it’s up to companies to share. However, it seems not every company wants to share: details of just four of these seem to be in the public domain (let me know if I’ve missed them!).
One full EAMS positive scientific opinion
MSD’s Keytruda was given the first positive EAMS scientific opinion in March 2015. This means that we now know exactly what an opinion looks like: concise and clear, spelling out the evidence base, the benefits and risks and the approach to managing risk in early use in 3 pages of the Public Assessment Report. It comes too with a treatment protocol for healthcare professionals, for patients and more detail on the pharmacovigilance system.
We don’t yet know how it will affect access and the most important thing of all, what difference it will make to patients: it’s too early to say. What we do know is that MSD has spent time and money on not only the research and development to reach this point, but also to take part in the scheme (the fee for this stage is £29,000), and provide the product at no charge to the NHS before Keytruda is formally licensed.
We also don’t know yet whether the other PIMs, including the very first for DCVax-L, will progress to the full scientific opinion or how MSD beat them to it. (I did ask them, but they didn’t reply).
Not as much interest as expected?
A simple measure for success is the interest in the scheme from companies. By October 2014 - 6 months into the scheme -15 expressions of interest were made to MHRA, but only MHRA knows how many more were received in the last 6 months.
Before the scheme was launched MHRA thought perhaps 2 products a year might be granted an opinion under the scheme. That’s not quite happened for the first year, but it might for future years. Respondents to the consultation to bring the scheme in thought that perhaps be 5 to 12 products where EAMS could work. We’re nowhere near that – but perhaps that is possible if the 7 PIMS translate to positive scientific opinions later.
Just for cancer medicines?
Although it is undoubtedly good news for those with cancer that there is interest from companies in bringing these products earlier to patients, it’s perhaps a little surprising that there aren’t (in the public domain at least) other therapy areas represented. When the scheme was launched back in March 2014, muscular dystrophy and dementia, alongside cancer, were mentioned. So too was Summit plc’s work on treatments for Duchene muscular dystrophy. The company was reportedly wanting to be ‘first in line’ to apply. They may well have done, and taking time to both give MHRA the evidence it needs, and allowing MHRA the time needed to consider that (although MHRA do have a target timeline – it can be slowed at the companies request), should not be rushed. After all, we are talking about a careful balance between safety and speed. Let’s hope that it’s not just cancer medicines under consideration.
Just for big pharma?
Again, it’s hard to know for sure, but based on what seems to be out there, it looks like EAMS is of particular interest to the big pharma companies. BMS, Novartis and BMS were all in the top 15 pharmaceutical companies by global sales in 2014. Northwest Biotherapeutics is the one that stands out as a small biotech, developing cancer vaccines to treat solid tumour cancers.
The use of the scheme by the big pharma companies contrasts with the hope expressed at the outset of the scheme; George Freeman saw it as “a hugely important initiative for the smaller biotech and emerging pharma companies”.
Of course the big companies should take up these opportunities – the patients that their products treat deserve it – but is it easy enough for the smaller companies to take up the opportunity too?
Key role for MHRA to review progress given voluntary and confidential nature of the scheme
Only MHRA will know exactly who has been interested in the scheme as confidentiality is given to companies who express an interest or formally apply.
But making sure that smaller companies know about the scheme, and making sure that the process is as streamlined as possible whilst still ensuring the necessary due diligence takes place, might help encourage them to take part in the voluntary scheme. To know that, we’ll need to ask companies and their representative associations what they think about how it’s working so far.
Funding may also be an issue; providing the product at no cost is unlikely to be attractive – despite the benefits of early use. This is especially the case when there are bigger battles over how the NHS decides what drugs to pay for and whether the price is reasonable. This raises questions about what revenue a company will eventually get, even if a company can receive a PIM, a positive scientific EAMS scientific opinion, and a license. Just see the battle over translarna to get a sense of those difficulties.
EAMS is part of the much wider Innovative Medicines and MedTech Review (also known as the Accelerated Access Review or the End to End Review) looking at a host of issues about how to improve the system from bench to bedside. So watch out for the results of that review coming later in 2015.
Leela Barham is an Independent Health Economist and Policy Expert