How Do Post-Registration Studies Impact Drug Evaluation by the Transparency Committee?

OBJECTIVES: Post-registration studies (EPI) may be requested by the Transparency Committee (TC) when it issues an opinion on a drug and is faced with uncertainties about its use in current practice, its clinical benefit or its safety profile. The results of these studies contribute to reassessments by the TC. Our aim was to determine a package profile leading to a request and the consequences on the reimbursement.

METHODS: We identified TC opinion including an EPI requested or results, from 2016 to 2024. For these products, we collected TC opinion date, level of clinical benefit (SMR) and clinical added value (ASMR), role in therapeutic strategy, clinical package, expected reassessment timeline and the actual one.

RESULTS: A total of 106 TC opinions included an EPI requested and 81 opinions EPI results between 2016 and 2024. The EPI requests concerned drugs with improved clinical benefits in 57% of cases and asked for results within a 5-year delay. The majority of EPI confirmed the SMR level (89%), ASMR level (94%) and place in the therapeutic strategy (91%) attributed to the product.

CONCLUSIONS: The relatively small number of requests for additional data, compared with the number of drugs evaluated by the TC, indicates that these requests are targeted and are therefore part of a specific evaluation context. However, this number remains considerable, given the internal resources required to set up and monitor these EPIs, which often take several years to complete.