OBJECTIVES: Recently, we found that about 95% of the 76 personalized medicines approved by the FDA between 2017 and 2020 was granted at least one regulatory tool to accelerate market access. The EMA has similar regulatory tools available, but the requirement to get subsequent reimbursement approval will delay patient access in several European countries. In this study, we investigated the time between EMA approval and reimbursement decisions by NICE, SMC and HAS for personalized medicines.

METHODS: Personalized medicines were identified through FDA Progress and Outlook Reports from 2017 to 2020. EMA approval details for these drugs were extracted from the EMA website. Reimbursement details were extracted from NICE, SMC and HAS websites. Descriptive analyses on the time to reimbursement were performed (overall and stratified on whether EMA submissions were for orphan drugs or based on single arm trial data).

RESULTS: Of the 62 FDA and EMA-approved personalized medicines, reimbursement decisions were available from NICE for 40 drugs, from SMC for 37 drugs and from HAS for 57 drugs. Positive recommendation (with or without restrictions) was 100% for NICE and SMC, and 88% for HAS. The average time until decision was 227 days for NICE (median 219; range -36 to 878) and 372 days (median 294; range 81-1419) for SMC, and 216 days for HAS (median 177; range 76-663). The average time to reimbursement decision was longer for orphan drugs in the UK (NICE, n=19, 273 days; SMC, n=18, 443 days). There were no major differences in the average time until decision for EMA submissions using single arm data.

CONCLUSIONS: The average times until reimbursement decision by NICE, SMC or HAS were shorter for personalized medicines than those reported between 2015 and 2018 by EFPIA (England 349 days; Scotland 425 days; France 566 days) but still considerable, especially for orphan indications.