Conditional Marketing Authorization: How Do Outcomes in France, Germany, and the UK Compare?
Author(s)
Macaulay R1, Foden C2, Bear M3
1Precision AQ, Edinburgh, UK, 2Precision AQ, London, London, UK, 3Precision AQ, London, LON, UK
Presentation Documents
OBJECTIVES: The European Medicines Agency (EMA) and Medicines and Healthcare products Regulatory Agency (MHRA) can grant a Conditional Marketing 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 and cross-checked with MHRA CMAs (01-JAN-2021–31-DEC-2023), alongside any corresponding NICE, G-BA, and HAS evaluations over the past 5 years (01-JAN-2018
–29-JUN-2024). In Germany and France, negative reimbursement outcome was defined as a no added benefit rating and SMR insufficient / ASMR V, respectively.RESULTS: 89 EMA CMAs were identified, representing 6.2% 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 >10% of all EMA approvals every year since 2019 but <10% every year beforehand. However, only a proportion of these have been converted to positive reimbursement outcomes by HAS, G-BA, or NICE after 2017: 27% in France (2% ASMR I; 9% ASMR III; 14% ASMR IV // 41% ASMR V; 18% insufficient; 16% not assessed), 47% in Germany (2% minor added benefit; 42% non-quantifiable benefit // 36% no added benefit; 18% not assessed), and in the UK 56% (22% recommended; 20% CDF recommended; 17% optimized // 16% ongoing; 12% terminated; 2% discontinued; 5% not assessed).CONCLUSIONS: EMA CMAs are becoming an increasingly common route to market for new medicines. However, only around half of medicines translated this expedited marketing authorization into positive reimbursement outcomes in the UK and Germany, while France only a quarter received positive reimbursement. This emphasizes how manufacturers need to carefully assess the trade-offs and risks before leveraging expedited access pathways, risking delaying access to patients.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
HTA390
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Approval & Labeling, Decision & Deliberative Processes
Disease
Drugs, No Additional Disease & Conditions/Specialized Treatment Areas