Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

Abstract

Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022.Methods: A survey was distributed in July 2022 to representatives of 46 countries.Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs.Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe’s Access to Novel Medicines Platform.

Advanced-Therapy Medicinal Products: Challenges for Implementation in Pediatric Clinical Practice

“…Вместе с тем разработка и внедрение в клиническую практику как разрабатываемых, так и зарождающихся медицинских технологий сопряжены с высокими рисками и для разработчиков, и для пациентов. Так, сложность демонстрации клинической ценности ЛППТ для плательщиков, в том числе ввиду реализации процедуры ускоренной регистрации [26], небольшие фрагментированные рынки, а также производственные и логистические трудности уже привели к отзыву (не продлению) ранее выданных регистрационных удостоверений их держателями [27]. Кроме того, в систематическом обзоре J. Meng и соавт.…”