Vella Bonanno Patricia, Ermisch Michael, Godman Brian, Martin Antony P, Van Den Bergh Jesper, Bezmelnitsyna Liudmila, Bucsics Anna, Arickx Francis, Bybau Alexander, Bochenek Tomasz, van de Casteele Marc, Diogene Eduardo, Eriksson Irene, Fürst Jurij, Gad Mohamed, Greičiūtė-Kuprijanov Ieva, van der Graaff Martin, Gulbinovic Jolanta, Jones Jan, Joppi Roberta, Kalaba Marija, Laius Ott, Langner Irene, Mardare Ileana, Markovic-Pekovic Vanda, Magnusson Einar, Melien Oyvind, Meshkov Dmitry O, Petrova Guenka I, Selke Gisbert, Sermet Catherine, Simoens Steven, Schuurman Ad, Ramos Ricardo, Rodrigues Jorge, Zara Corinne, Zebedin-Brandl Eva, Haycox Alan
Department of Pharmacoepidemiology, Strathclyde Institute of Pharmacy and Biomedical Sciences, University of StrathclydeGlasgow, United Kingdom.
Pharmaceutical Department, National Association of Statutory Health Insurance FundsBerlin, Germany.
Front Pharmacol. 2017 Aug 23;8:497. doi: 10.3389/fphar.2017.00497. eCollection 2017.
Medicines receiving a conditional marketing authorization through Medicines Adaptive Pathways to Patients (MAPPs) will be a challenge for payers. The "introduction" of MAPPs is already seen by the European Medicines Agency (EMA) as a fait accompli, with payers not consulted or involved. However, once medicines are approved through MAPPs, they will be evaluated for funding by payers through different activities. These include Health Technology Assessment (HTA) with often immature clinical data and high uncertainty, financial considerations, and negotiations through different types of agreements, which can require monitoring post launch. Payers have experience with new medicines approved through conditional approval, and the fact that MAPPs present additional challenges is a concern from their perspective. There may be some activities where payers can collaborate. The final decisions on whether to reimburse a new medicine via MAPPs will have more variation than for medicines licensed via conventional processes. This is due not only to increasing uncertainty associated with medicines authorized through MAPPs but also differences in legal frameworks between member states. Moreover, if the financial and side-effect burden from the period of conditional approval until granting full marketing authorization is shifted to the post-authorization phase, payers may have to bear such burdens. Collection of robust data during routine clinical use is challenging along with high prices for new medicines during data collection. This paper presents the concept of MAPPs and possible challenges. Concerns and potential ways forward are discussed and a number of recommendations are presented from the perspective of payers.
通过“患者用药适应性途径”（MAPPs）获得有条件上市许可的药物，对医保支付方来说将是一项挑战。欧洲药品管理局（EMA）已将MAPPs的“引入”视为既成事实，未与医保支付方协商或让其参与。然而，一旦药物通过MAPPs获批，医保支付方将通过不同活动对其进行资金评估。这些活动包括健康技术评估（HTA），而评估时临床数据往往不成熟且不确定性高，还包括财务考量以及通过不同类型协议进行谈判，这可能需要在上市后进行监测。医保支付方对通过有条件批准获批的新药有经验，从他们的角度来看，MAPPs带来额外挑战令人担忧。在某些活动中，医保支付方可能会进行合作。与通过传统流程获批的药物相比，关于是否通过MAPPs报销新药的最终决定会有更多差异。这不仅是因为通过MAPPs获批的药物不确定性增加，还因为成员国之间法律框架存在差异。此外，如果从有条件批准到获得全面上市许可期间的财务和副作用负担转移到批准后阶段，医保支付方可能不得不承担此类负担。在常规临床使用期间收集可靠数据具有挑战性，而且在数据收集期间新药价格高昂。本文介绍了MAPPs的概念以及可能面临的挑战。讨论了相关担忧和可能的前进方向，并从医保支付方的角度提出了一些建议。
