Presentation (CTI)

OBJECTIVES: The Early Access to Medicines Scheme (EAMS) was launched in 2014 to provide UK patients with pre-authorisation access to medicines addressing a clear unmet need. While oncology dominated early uptake, rare disease assets have featured more prominently in recent years. This research characterises the use of EAMS in rare diseases and explores its impact on subsequent HTA outcomes in England and Scotland.
METHODS: A review of the GOV.UK website was performed in June 2026 to identify all active and expired EAMS scientific opinions. Corresponding HTA outcomes were captured from the NICE and SMC websites.
RESULTS: Of 53 EAMS scientific opinions issued by the MHRA, twelve (22.6%) were for rare disease assets, of which two remain ongoing. Rare disease assets spent substantially longer in EAMS than non-rare disease assets (13.1 vs. 4.6 months). Of the ten rare disease assets with published NICE guidance, eight (80%) received a full or optimised recommendation and two (20%) were discontinued. SMC outcomes were similarly positive: seven (70%) accepted, one (10%) not recommended, and two (20%) non-submissions. Outputs from EAMS were presented in HTA submissions to provide qualitative (often anecdotal) and quantitative information regarding real-world outcomes, trial generalisability, dosing frequency, discontinuation rates, and incidence of adverse events. However, the influence of EAMS outputs on HTA decision-making appears limited; only three appraisals explicitly referenced EAMS in final NICE guidance and only two in final SMC guidance.
CONCLUSIONS: Rare disease assets typically spent longer in EAMS than non-rare disease assets and mostly achieved positive NICE and SMC recommendations. However, despite its potential to strengthen the evidence package in rare diseases, the influence of EAMS outputs on NICE and SMC decision making appears limited. This suggests either that evidentiary contribution from EAMS is underacknowledged in decision-making, or that data collection during EAMS is often inadequate to meaningfully support HTA in rare diseases.