OBJECTIVES: Many countries have different pricing and reimbursement (P&R) processes for prescription drugs, hospital drugs, medical devices, and procedures. Newer transformational technology classes such as CAR-T, cell- and gene-based therapies may not be classified as drugs and thus may be subject to distinct P&R processes. This research examines how advanced therapy medicinal products (ATMPs) have been reimbursed in major European markets.

METHODS: European Commission (EC)-approved ATMPs were identified alongside their HTA decisions in the UK (NICE, SMC), France (HAS), Italy (AIFA), Spain (AEMPS) and Germany (G-BA, IQWiG) (to 15/01/2019).

RESULTS: 13 ATMPs have received EC-approvals to date, with the first four being withdrawn from the market for commercial reasons. In total, 22 HTA appraisals were identified for the nine currently marketed ATMPs; each HTA agency assessed a mean of 3/9 nine available ATMPs (ranging from 7/9 [NICE] to 1/9 [SMC]). Positive appraisals were received in 48% of HTA submissions, with significant variability noted between agencies (AIFA recommended 100% [3/3] of submissions, whereas AEMPS rejected 100% [2/2] of submissions). The mean time to a positive decision was 367 days, with the mean for HTA agencies ranging from 113 by IQWiG to 418 by AIFA. In Germany, one of the nine approved ATMPs (Holoclar®) was classified as a procedure achieving NUB-Status 4; two ATMPs withdrawn from the market were also classified as procedures.

CONCLUSIONS: Many ATMPs remain unassessed by HTA bodies in key European markets despite achieving EC marketing authorisation, and those that have been assessed have a low rate of positive decisions. In some markets ATMPs are assessed as devices, or procedures, with significant implications to their evidentiary requirements and pricing and reimbursement. Because of these variations, strategic payer evidence plans and communications require forethought and planning to ensure alignment with different national processes to optimize commercial success.