Presentation (CTI)

OBJECTIVES: Healthcare systems have distinct priorities impacting patient access. While the USA has historically prioritised broad, fast access—achieved via Food and Drug Administration (FDA) approval and various routes to market—other systems, like the UK, more stringently balance access against value-for-money, with public reimbursement achieved only after regulatory review and HTA assessment. Most-Favoured-Nation (MFN) pricing, if implemented, could blur these differences, with the USA importing other countries’ prices and, potentially, their value judgements. Recent analyses have compared FDA approval timelines to those in the UK and Europe. This research extends those analyses, examining how UK access is influenced by the National Institute for Health and Care Excellence’s (NICE’s) HTA process and exploring differences between FDA and European Medicines Agency (EMA) approval patterns at the indication level.
METHODS: We identified all new and supplementary FDA approvals (2019-2025) from annual reports; EMA approvals (initial and extensions) through CHMP meeting highlights (2021-2025); and indications appraised by NICE through the TA and HST programmes (2021-2025). We manually matched molecule-indication pairs across agencies, identifying where UK patients received full, partial (optimised decisions), or no access relative to the FDA and EMA, and comparing approved indications across the FDA and EMA.
RESULTS: Preliminary results indicate UK patients have access to just over 50% of EMA-approved indications, including medicines granted access outside of NICE. Among positive NICE decisions, approximately half were optimised, affording NHS access to a subset of the licensed population. These outcomes illustrate how HTA affects access, prioritising interventions offering good value-for-money. Our next work phase will relate these differences to FDA approvals and explore variations across therapeutic areas.
CONCLUSIONS: These findings highlight how a national evaluation process like HTA can impact patient access. They are particularly relevant in the context of MFN, which could risk importing other countries’ lower prices alongside their access constraints and country-specific priorities.