EAMS evidence: A key player in drug value proposition and NICE approval?

Overview of EAMS 

EAMS creates the opportunity for treatment with unlicensed medicines when no other treatment options are viable and consists of a 2-step evaluation on process as detailed below. This scheme is not considered a replacement for standard licensing routes; however, if prespecified criteria are met, patients could potentially gain access to medicines up to a year prior to marketing authorisation.

The promising innovative medicine (PIM) designation 

PIM applications can be submitted when early clinical evidence demonstrates that the product conveys potential benefit for patients with life-threatening or seriously debilitating conditions with clear unmet need. If successful, the PIM designation is typically granted following review at an MHRA meeting.

The EAMS scientific opinion (SO)

Upon positive PIM designation, the MHRA will evaluate the risk/benefit ratio of the medicine based on the available data at the time of EAMS submission. This helps inform the treatment decisions of the clinician and patient before the medicine is approved. Once a positive SO has been communicated, the MHRA will release a public assessment report (PAR) along with the EAMS treatment protocol. A positive SO is subject to renewal every year.

Following these two processes, the EAMS period officially commences, where the company supplies the medicine at no cost and collects real-world evidence until marketing authorisation is granted. In parallel, the company will also prepare for the NICE technology appraisal. Patients who initiated treatment during this EAMS period will continue to receive the medicine at no cost to the National Health Service (NHS) until a positive recommendation is issued.

Retrospective analysis objective

While EAMS offers drug manufacturers a crucial opportunity to provide treatment to patients ahead of regulatory approval, enables clinicians to gain experience with the product, and begins to generate real-world evidence, the impact of a positive EAMS opinion on the probability of receiving a positive NICE recommendation remains unclear. As such, we conducted a retrospective analysis to assess all EAMS products and their NICE appraisal outcomes over the past 4 years.

All products for which the EAMS SO expired from January 2020 to December 2023 and the corresponding NICE recommendation, if available, were analysed.

Results

A total of 25 EAMS products with an expired EAMS SO within the designated time period were identified, 60% (n=15) of which were oncology treatments. Of these, the average EAMS period was 8.7 months (range: 1.2 months [remdesivir] to 39.7 months [raxone]; Figure 1). This was approximately twice as long as the average EAMS period during the 2015 to 2019 period (3.8 months). Although the root cause of this trend is unclear, the potential reason behind this delay could be related to COVID-19. The COVID-19 pandemic forced the diversion of regulatory resources to deal with the unforeseen public health crisis. The influence of Brexit and changes in the licensing pathways may lead to approval delays, potentially prolonging the EAMS period. This pattern was evident in 2021, when the MHRA authorized fewer new drugs compared to European counterparts, although none of these were EAMS products. Nevertheless, it is essential to note that Brexit could have affected other EAMS products and might continue to do so in the future. Therefore, further analyses are necessary to gain a comprehensive understanding of the impact.

Figure 1. EAMS length per product between 2020 and 2023

• Uptake supports unmet need
• Observed efficacy and safety data validate the benefits demonstrated in the trials 
• Use supports product positioning
• Because of EAMS, more centers are treating patients, reducing the time to diagnosis
• Clinical benefits were observed shortly after administration
• Confirmed infusion time 
• Clinicians who have experience with the product through EAMS can help train other clinicians
• Cost data collected during EAMS were included in the health economic models

Conclusion

While the duration of EAMS has doubled over the past four years, it remains uncertain whether this is a broader trend or if it was influenced by COVID-19-related delays. Furthermore, the duration of EAMS is highly variable, ranging from just over a month to over three years. However, when excluding the three products with the longest EAMS periods (raxone, cipaglucosidase alfa, avalglucosidase alfa), all remaining 22 products (88% of total) were available for ≤12 months, thereby drastically reducing variability regarding the length of EAMS. For raxone and cipaglucosidase alfa, the extended EAMS period was due to delays with the NICE appraisals.

This analysis also reveals that EAMS products are more likely to receive a positive NICE recommendation than products appraised via standard licensing pathways (58% vs 43%, respectively). Evidence collected during EAMS can support a product’s value proposition, even if the data are only qualitative in nature. Consequently, when preparing for EAMS, it is crucial for manufacturers to thoroughly assess the data that can be collected to address uncertainties and pre-empt future market access challenges.

Sources

Association of the British Pharmaceutical Industry (ABPI). Early Access to Medicines Scheme. Accessed April 2024. https://www.abpi.org.uk/media/dfikg1ka/early_access_to_medicines_scheme.pdf

Imperial College Business School. Post-Brexit medicine approvals: what we know. Accessed April 2024. https://www.imperial.ac.uk/business-school/ib-knowledge/health/post-brexit-medicine-approvals-what-we-know/

Medicines and Healthcare products Regulatory Agency (MHRA). Apply for the early access to medicines scheme (EAMS). Accessed April 2024. https://www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams

National Institute for Health and Care Excellence Centre for Health Technology Evaluation. Note to describe procedures at NICE to support the Early Access to Medicines Scheme. Accessed April 2024. https://www.nice.org.uk/Media/Default/About/Who-we-are/Policies-and-procedures/eams-process-jan-16.pdf

National Institute for Health and Care Excellence (NICE). Guidance, NICE advice, and quality standards. Accessed January 2024. https://www.nice.org.uk/guidance/published?sp=on

Ringger D, Wissinger E. Examining the impact of the Early Access to Medicines Scheme (EAMS) on the National Institute for Health and Care Excellence (NICE) recommendations over the past 4 years. Poster presented at: The Professional Society for Health Economics and Outcomes Research (ISPOR) 2024 Meeting; May 2024; Atlanta, GA.

This article summarises Cencora’s understanding of the topic based on publicly available information at the time of writing (see listed sources) and the authors’ expertise in this area. Any recommendations provided in the article may not be applicable to all situations and do not constitute legal advice; readers should not rely on the article in making decisions related to the topics discussed.