PP142 Health Technology Assessment – A Major Bottleneck In Patient Access?

Abstract

IntroductionConditional marketing authorization (CMA) and accelerated assessment (AA) have been introduced to expedite the development of and access to therapies in Europe. However, to reach patients medicines must also be publicly reimbursed. This research evaluated the reimbursement of therapies which have received European CMA or underwent AA.MethodsMedicines that received CMA or underwent AA between January 2012 and December 2017 were identified. Appraisals of these medicines conducted by major European payer bodies were obtained from relevant websites and key data were extracted.ResultsOut of the 38 medicines that received a CMA, 83 percent (19/23) were assessed by the National Institute for Health and Care Excellence (NICE) and received positive decisions, compared with 57 percent (16/26) by the Scottish Medicines Consortium (SMC) (defined as recommended/restricted), 74 percent (14/19) by Gemeinsamer Bundesausschuss (G-BA) (defined as any level of additional benefit), and 29 percent by Haute Autorité de Santé (HAS) (amélioration du service médical rendu I-III). The median delay between CMA approval and positive health technology assessment (HTA) outcome was 13 months for NICE, 11 months for SMC, 7 months for G-BA, and 5 months for HAS. Thirty-two medicines underwent AA. Of these, 68 percent (17/25) were appraised by G-BA and received positive outcomes, compared with 29 percent (7/24) by HAS, 90 percent (19/21) by SMC, and 86 percent (18/21) by NICE. The median delay between AA approval and positive HTA outcome was 7.4 months for G-BA, 7.9 months for HAS, 11.7 months for SMC, and 11.8 months for NICE.ConclusionsCMA has expedited regulatory approval for products that address severe unmet needs. However, many of these products fail to gain public reimbursement, and even when they do there is a significant delay. AA provides market authorizations two months earlier than standard centralized assessment. Although high rates of positive payer outcomes have been achieved, the products typically experience substantial additional delays in securing public reimbursement. A parallel, cooperative approach among regulatory and HTA bodies across Europe is required to truly expedite patient access.