OBJECTIVES: The European Medicines Agency (EMA) can grant a Conditional Medicines Authorization (CMA) for medicines that treat severe diseases based on less comprehensive clinical data than usual, pending the collection of more comprehensive data post-authorization. This research evaluates how the number of CMAs have evolved over time and their conversion to successful reimbursement.

METHODS: All publicly-available EMA CMAs were identified (01-JAN-2006–31-DEC-2022), alongside any corresponding NICE evaluation over the past 5 years (01-JAN-2018–29-JUN-2023) and key information was extracted.

RESULTS: 81 EMA CMAs were identified, representing 6.0% of all EMA medicine authorizations (range:0.9%[2009]–14.1%[2021]). There is a clear trend towards these becoming more common over time: CMAs represented over 10% of all EMA approvals every year since 2019, but was under 10% in every year before 2009. However, only a proportion of these have been converted to successful NICE reimbursement recommendations over the past 5 years, with 14% recommended, 14% optimized, 18% recommended through CDF, 9% not recommended, 9% terminated/discontinued, 18% ongoing, and 18% N/A (primarily COVID vaccines). This trend also appears to be substantially more negative over time, with 68% of NICE appraisals being ‘Recommended’/’Optimized’/’CDF’ prior to 2021 but the corresponding figure has fallen to 23% from 2021 onwards.

CONCLUSIONS: EMA CMAs are becoming an increasingly common route to market for new medicines, representing over 10% of new EMA approvals since 2019. We have shown that only a proportion of medicines translated this expedited marketing authorization into successful reimbursement outcomes in the UK, and this trend is becoming more negative over time. This is despite the UK being one of the few payer bodies with a dynamic reimbursement pathway that allows temporary reimbursement whilst additional data is collected (Cancer Drugs Fund and Innovative Medicines Fund). Further research can elucidate if this trend is mirrored across other major European markets.