A pharmaceutical industry-funded Early Access to Medicines Scheme (EAMS) is to launch in the UK in April, offering severely-ill patients the opportunity to try ground-breaking new medicines “years” before they would normally reach them, Ministers have announced.
The Scheme will enable patients with life-threatening and seriously debilitating conditions to receive innovative and promising new drugs as soon as the Medicines and Healthcare products Regulatory Agency (MHRA) has signalled that the benefits outweigh the risks, through a two-stage evaluation process.
In stage one, if the MHRA considers a product to be a possible candidate for EAMS, it will issue a Promising Innovative Medicines (PIM) designation, based on early clinical data. In stage two, the Agency will issue an EAMS Scientific Opinion if the quality, safety and efficacy data provided in support of the application is sufficient to support a positive benefit/risk balance and added clinical value.
EAMS will be complemented by a new National Institute for Health and Care Excellence (NICE) technology appraisal and NHS England commissioning process.
Because the Scheme is to be funded by pharmaceutical companies, patients will benefit from world-class breakthroughs at no cost to the NHS, says the Department of Health. And drugmakers will gain from being able to obtain experience of their medicines being used in the NHS and work closely with regulators to look at the value of their products, gaining guidance and advice much earlier in the regulatory process.
“Companies are expected to invest more in the UK as a result because of their increased confidence that they can work with doctors and patients to get experience of the new medicines and bring drugs to patients quicker. We expect charities and small businesses to also work together to develop new approaches to treating rare and life-threatening diseases, which could include cancer, muscular dystrophy and dementia,” the Department adds.
Health Secretary Jeremy Hunt commented that making Britain the best place in the world for science, research and development is a central part of the government’s long-term economic plan. “This ground-breaking scheme will provide cutting-edge medicines earlier, give hope to patients and their families and save lives. And as part of our strategy for life sciences, it will create more jobs and opportunities for people, helping secure a better future for our country,” he said.
Patient groups, charities and industry have broadly welcomed the EAMS, pointing out that a similar approach is working well in the US through the Food and Drug Administration (FDA)’s Breakthrough Therapy Designation.
“Companies use it to secure investment and increase the credibility of the medicines in the scheme, particularly in other countries,” noted Peter Butterfield, chair of the Smaller Companies Forum at the Association of the British Pharmaceutical Industry (ABPI), and he said the UK initiative will provide “an exciting opportunity for smaller companies.”
However, the lack of central funding for the scheme will be an issue for some companies, as it means they will have to bear the risk associated with the upfront investment required to participate in the scheme, noted Paul Catchpole, director of value access at the ABPI.
The Association is calling for a review of the Scheme, one year on, “so that we can appraise its first year and potentially review funding options,” said Mr Catchpole.
And Steve Bates, CEO of the BioIndustry Association (BIA), warned that, without centrally-funded reimbursement, the Scheme runs the risk of being underutilised. “The BIA remains committed to working in partnership with all stakeholders to ensure that this is not the case and the Scheme remains globally competitive,” he said.
Alongside the EAMS, the Department has launched the BioResource project, a bank of nearly 75,000 medical research volunteers, funded by the National Institute for Health Research (NIHR) and led from Addenbrookes Hospital in Cambridge, which it says should make it much easier for researchers to recruit into research and trials people with specific conditions and a family history of conditions.
BioResource will focus on heart disease, dementia, infections and rare diseases, and “could speed up the development of new treatments and attract international investment as a result of companies having access to a growing bank of tens of thousands of volunteers from across the country who are keen to help medical research,” says the Department.
- Earlier Access to New Medicines is the subject of PharmaTimes Directors’ Club (PDC)’s spring meeting, to be held on April 15 at Royal Mint Court in central London. Discussing the issues will be senior figures from NICE, MHRA, NHS England and McKinsey & Co.
For further information on the meeting and to book your place, click on: www.pharmatimes.com/Events/PharmaTimesDirectorsClub.aspx