Abstract
The Early Access to Medicines Scheme (EAMS) was launched in the UK in April 2014 following a public consultation and government response, which outlined the scope of a potential scheme. The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for two pivotal milestone decisions in the EAMS process: the Promising Innovative Medicine (PIM) designation and the EAMS scientific opinion, described in an EAMS public assessment report and 3 EAMS treatment protocols. In the 3 years since its launch, hundreds of patients with life-threatening or seriously debilitating conditions have benefited from EAMS medicines in a variety of mainly oncology indications. This editorial describes some of the achievements of the scheme over the past 3 years and provides insight into how the scheme may evolve over the coming years.
Keywords
Early Access to Medicines Scheme - 3 years on
The Early Access to Medicines Scheme (EAMS) was launched in the UK in April 2014, following a recommendation from the Ministerial Industry Strategy Group for an early access scheme, with additional input into the scheme's design via a public consultation and an expert group on the innovation in the regulation of healthcare under the Prime Minister's Strategy for UK Life Sciences (1–5).
The first Promising Innovative Medicine (PIM) designation was awarded in September 2014. A PIM designation is an early indication that a particular medicine is a candidate for the EAMS, with the potential to address an unmet medical need. The 3 PIM criteria include (i) a documented life-threatening or seriously debilitating condition and with high unmet clinical need; (ii) the medicine is likely to offer a major advantage over methods currently used in the UK; and (iii) the potential adverse effects of the medicinal product are likely to be outweighed by the benefits. To date, there have been 30 PIM designations awarded. The MHRA does not publish the PIM designation, although the company in receipt of a PIM is free to do so. Of importance, to facilitate interaction between a PIM designation holder and other stakeholders involved in patient access, the notification of a PIM designation by the MHRA includes contacts in UK National Health Service (NHS) and health technology assessment bodies. These contacts are also confidentially informed of the designation.
The first EAMS scientific opinion was approved in March 2015. The scientific opinion describes the positive benefit risk profile of the medicine, and supports the prescriber and patient in making a decision on whether to use the medicine before its licence is approved. The EAMS criteria build on those for the PIM designation, with the additional aspect that the company should supply a medicine manufactured to a consistent quality standard. During the assessment process (a day 75 or a day 90 timetable), a day 45 notification has been built into the scheme post launch, in order to facilitate the communication of a pending potentially positive EAMS scientific opinion. This notification flags to the NHS systems in the four UK countries that an EAMS medicine is likely to become available in a specific condition, so that necessary preparations might be brought forward, as appropriate.
A case study regarding the experiences and lessons learned from the first EAMS product, pembrolizumab, was published by the MHRA (Innovation: over 500 UK patients gain early access to new skin cancer treatment). Here, the company stated that “EAMS undoubtedly accelerated access to pembrolizumab for patients with advanced melanoma and demonstrates a world-leading example of how healthcare agencies and industry can work together to get treatments to patients more quickly” (6). From an MHRA perspective, the application process with pembrolizumab helped to identify and resolve some practical issues of running the scheme. These issues included the structure of the data requirements (including pharmacovigilance aspects), appropriate labelling, and further optimizing timelines. In this regard, the MHRA will be updating their guidance documents in the near future to reflect the lessons learned from the first 3 years of operation, strengthening the current procedures, and providing more comprehensive guidance for potential applicants.
To date, there have been 11 scientific opinions published, with the vast majority of opinions being in the oncology field (Tab. I). This is reflected in the PIM designations, where oncology has been a very strong focus. However, as the scheme has matured, applications for PIM have been received for a variety of conditions outside of cancer – including rare diseases – and it is expected that scientific opinions will broaden and cover other disease areas.
Table of EAMS scientific opinions (first three years since launch)
Evolving EAMS
Since its launch, the EAMS Government-Industry Stake-holder Task Group has regularly brought together key stakeholders from the bio-pharmaceutical industry, the NHS, and arm's-length bodies to inform the development of EAMS. This stakeholder group has produced EAMS “principles” and operational guidance describing the relationships between the MHRA, the National Institute for Health and Care Excellence (NICE), and NHS England (7). Ongoing work at the task force includes a focus on the opportunities and challenges of collecting real-world data in the NHS during the EAMS scientific opinion. The ability to collect such data offers an added benefit to companies involved in the scheme, with the potential to augment data collected from clinical trials. In this regard, the Office for Market Access at NICE offers companies the opportunity to have a supplementary meeting (NHS England are also invited) to discuss data collection plans during the EAMS period, where data may be generated to address uncertainties (8). It is expected that data collection during the EAMS scientific opinion will evolve alongside the already established pharmacovigilance aspects, providing a valuable opportunity to better understand the medicines' profile in the real-world setting, and complementing data derived from sources, such as clinical trials.
The UK Government's Accelerated Access Review (AAR) aims to speed up access to innovative drugs, devices, and diagnostics for NHS patients (9). The review was launched in March 2015, with a final report published in October 2016. The review makes recommendations to the government on reforms to accelerate access, and includes EAMS as a term of reference. The report builds on an independent review of EAMS (10). Statements in the report that specifically relate to EAMS include:
Determining which products should receive a transformative designation will be of critical importance and should build on schemes such as Priority Medicine (PRIME) in Europe and EAMS in the UK;
Small and medium-sized enterprises and not-for-profit organizations with products on the EAMS pathway should, in some cases, receive some level of funding prior to NICE assessment;
EAMS will be an integral part of the accelerated access pathway, providing prelicence access for strategically important products.
The government response to the AAR report is currently awaited.
Where to next?
The UK EAMS addresses a public health need to improve access to important innovative medicines, and has successfully facilitated the access to new medicines and new indications for hundreds of patients with unmet medical need. A number of proposals to strengthen the scheme since its launch have been implemented in the last 3 years. Exciting developments are to be expected as the scheme matures and the recommendations from the EAMS task force and the government response to the AAR are applied, which will further benefit patients and support innovation.
Footnotes
Financial support: No grants of funding have been received for this study.
Conflict of interest: None of the authors has financial interest related to this study to disclose.