PRIME: Does Accelerated Marketing Authorization Translate Into Expedited Reimbursement and Patient Access?

OBJECTIVES: The EMA Introduced PRIority Medicines (PRIME) to support the development of medicines that can potentially address unmet medical needs. PRIME medicines may also be eligible for accelerated assessment. This research examines medicines with PRIME designations that received marketing authorization (MA), and their corresponding reimbursement outcomes in France, Germany and England.

METHODS: Medicines previously granted PRIME designations which are now authorized for use in the EU identified from EMA’s website, and key information of HAS, G-BA and NICE assessments extracted from their respective websites (as of 13-May-2022).

RESULTS: 20 medicines that had PRIME support are currently authorized for use in the EU, 3(15%) of which went through accelerated assessment. 11(55%) received full MA and 9(45%) received conditional MA (CMA). 7(35%) were advanced-therapy medicinal products (ATMPs). Mean delay from MA to HTA was 7.4 mos (range:0.4–20.8). In France, 9(45%) were reimbursed to label and 6(30%) were restricted to subpopulations. ATMPs received more favourable outcomes (4[57%] reimbursed to label). Most ASMR ratings were III (9[56%]), followed by V (3[19%]) and IV (2[13%]). In Germany, 10(50%) obtained a non-quantifiable added benefit (AB) and 2(10%) received a no AB. Proportion of non-quantifiable AB was 78% for medicines with CMA and 71% for ATMPs. In England, 67% of medicines assessed by NICE were under the CDF and/or with MAA, with a higher proportion for medicines with CMA (75%) and for ATMPs (100%).

CONCLUSIONS: PRIME medicines received mixed HTA outcomes across France, Germany and England, with a considerable delay from MA to HTA. Beyond PRIME, integration and engagement with payers will be important. With joint HTA expected to roll out in 2025, there may be opportunities for EMA and the HTA network to collaborate and better support patient access.