What next for the Early Access to Medicines Scheme?
The Early Access to Medicines Scheme was launched in 2014 to help patients in need get faster access to new treatments. Four years in, is the initiative going as planned? The House magazine recently hosted a roundtable with leading figures from the healthcare industry to discuss.
Severe atopic dermatitis is a uniquely vexing affliction. Sufferers of this skin condition, better known as eczema, experience intense itching to the point of bleeding, and most would probably say the steroid creams they’re prescribed aren’t effective. There’s always the option of immunosuppressives – eczema flare-ups are a manifestation of the immune system in overdrive – but these can bring their own train of side effects, including reduced function of the kidneys and raised blood pressure. Many patients are thus stuck with a choice between two evils: endure the eczema symptoms, or deal with the unwanted secondary effects of drugs.
That was the dilemma until dupilumab, a potential alternative to immunosuppressives developed by the global pharmaceutical companies Sanofi and Regeneron, sprang onto the scene. In 2015, dupilumab was awarded Promising Innovative Medicine (PIM) status by the Medicines and Healthcare products Regulatory Agency (MHRA), and last year was added to the Early Access to Medicines Scheme (EAMS), effectively giving patients access to this as-yet unlicensed drug.
EAMS was launched in 2014 to help patients in need get faster access to new medicines. Since then, MHRA has issued 51 PIM designations – which offer an indication of future potential – and 19 ‘positive scientific opinions’, which grant treatments full EAMS status. Now four years in, the question at a recent roundtable discussion hosted by The House magazine in partnership with Sanofi is whether the scheme is going as planned, or whether it can be improved.
The scheme of things
Jasmin Hussein, Sanofi’s Head of Dermatology and Respiratory Franchise, counts herself as both a proponent and a sceptic. She’s grateful to MHRA for how diligently they worked through the dupilumab process. “Kudos for your hard work,” she says directly to Dr Ian Hudson, MHRA’s CEO. “Even nearing Christmas all of our questions were answered.” But she and others feel that the EAMS process can at times be a logistical nightmare, and doesn’t fully involve all the relevant stakeholders. “As a result we waited until this month to get our first appraisal committee meeting.”
Dr Hudson sees things differently. “The scheme from our point of view has gone very well,” he says. “We had a ministerial strategy group consider early access in the mid-2000s, and now we’ve had 51 PIM designations and 19 opinions.” Hudson says that there is a lot of ongoing activity to refine the scheme, but for the most part it seems to work. “From a purely regulatory point of view, the scheme has been going really well,” he says.
So, is EAMS working? Because the scheme has multiple phases – from the application for PIM designation all the way through to NHS funding – it may be that some parts of the process work well while others don’t. Henry Featherstone, Sanofi’s Director of Public Affairs, thinks the regulatory side of things is fine, but that the reimbursement stage (when NICE appraises the licensed drug) needs work. Paul Catchpole, Value & Access Director at the Association of the British Pharmaceutical Industry, offers up the same diagnosis. “For EAMS to work, it requires a multi-agency response,” he says. “The MHRA has done a great job at what needs to happen at the front-end of the process. It’s how the other agencies respond down the line that needs fixing.” Catchpole argues that a “break in the pathway” at any stage of the process ultimately robs the patient of potential benefit.
But more important to the process itself is the number of stakeholders involved – patient groups, pharmaceuticals, regulators, clinicians – some of whom may benefit more than others. For example, Jayne Spink, CEO of Genetic Alliance UK, emphasises how important it is for schemes like EAMS work as seamlessly as possible with commissioning decision making processes in order to encourage the research and development community to get fully onboard. “We have seen too many instances of delays post-license,” she says, referring, like Featherstone, to the period between when a drug is licensed and when it’s reviewed by NICE or NHS England. “I suspect that these delays are a factor in companies deciding not to seek EAMS approvals for rare disease medicines.”
All hands on deck
One of the most significant perspectives is that of the rare disease patient, who is uniquely poised to benefit from EAMS since the vast majority of the roughly 7,000 known rare diseases have scarcely any clinical research and no licensed medicine. DUP MP Jim Shannon is concerned about whether they’re being properly considered. “Every time I meet medical bodies, they always say to me that the process has not taken forward for rare diseases,” he says. Spink adds that there’s a “huge unmet need” when it comes to rare diseases, but EAMS has yet to deliver for rare disease patients. “Currently there are only two EAMS approvals for rare disease medicines,” she says. One of them, a drug to treat Familial Chylimicronaemia Syndrome (FCS) has only very recently been approved. The other, which slows respiratory decline in patients with Duchenne Muscular Dystrophy, has had slower uptake than expected. Spink seems to suggest that EAMS has been only moderately successful for rare disease drugs, which isn’t enough to convince the research community to make big investments.
The SME angle is another that tends to be neglected, according to Rachael Mann, Policy and Public Affairs Manager at the BioIndustry Association. “Schemes like this are important to SMEs,” she says. “For small companies that concentrate on innovative projects and small data sets, early access schemes are really important.” Mann says that if a large pharmaceutical like Sanofi finds EAMS difficult, an SME might find it impossible. “Things like MHRA and NICE decisions are seen as strong signals for a company and whether people will invest in it,” she says. “For SMEs especially, we need to de-risk the scheme if we want them to get involved.”
An apparent drawback of EAMS that seems to cut across all parties is a simple lack of awareness. Mann cites a poll in which only 20% of NHS front-line staff say they’re aware of the scheme. “If clinicians don’t know about this, how could patients?” she asks. It’s a point echoed by Sanofi’s Harry Thurston-Smith, who says central government “needs to do a better job” raising awareness of the scheme. Clinicians, patients, patient group, either don’t even know about EAMS, or if they do they don’t know how to interact with that system,” he said. “If it is a successful scheme the should be shouting about it, and encouraging people to go into it. That’s something that MPs can definitely help with.”
Gary Jones, Senior Government Affairs Manager at AbbVie, says that there’s a lack of involvement from devolved administrations. “It’s just much more challenging to get that engagement with them,” he says. Both of these perspectives elicit nods from Alexander Davison, Policy Advisor at the Department of Health and Social Care and the Department of Business, Energy, and Industrial Strategy. Part of Davison’s job is to help coordinate the EAMS government-industry stakeholder task group, which brings together all these disparate parties to inform the scheme’s development. “Both devolved administrations and greater engagement with clinicians is something we’re working on,” he says. “The next step is to create [educational] materials, which will require input from across the board.”
All parties around the table agreed on the importance of continuing the conversation, and plan to meet with the government task group later in the year to assess progress.
There’s disagreement about EAMS, which is predictable for a programme just four years old. But the disagreements are logistical, and don’t reflect the bedrock values that spurred the scheme into existence. There is consensus that EAMS and similar programmes must ultimately be about helping the patient. “When it’s someone you love, you’ll do anything,” says Baroness Masham of Ilton, whose brother and husband both took unlicensed medication for serious illnesses. Her sentiments reflect a sense of urgency shared by everyone to get things right for those who need it most. Beyond that is just hammering out the details.