Les nouveaux dispositifs d’accès précoce aux innovations thérapeutiques: New early access program for drug innovation

Abstract

The Marketing Authorisation (MA) procedure for a medicinal product in Europe may be centralised at European level or applied in each member state. On the other hand, access to the market for a medicinal product that has been granted marketing authorisation is a different process in each member state and in France, subject to the HAS commissions – the Transparency Commission and the Commission for Economic Evaluation and Public Health – from which the medicinal product will be reimbursed by the health insurance scheme. To speed up the availability of medicines for certain serious diseases, the so-called early access reform in 2021 has modified access to innovative medicines through compassionate access, early access and the compassionate prescription framework. During the transitional period, these drugs are reimbursed. The HAS evaluates drugs whose manufacturers request early access according to the methodology used to evaluate drugs for marketing authorisation submissions. However, in rare diseases, such as certain cancers with rare mutational alterations, it is difficult to assess the efficacy of a drug by means of a randomised controlled clinical trial, which has led to the development of alternative trials (basket, single-arm or quality of life assessment trials), whom methodology has been recommanded by the Health Technology Assessment body.1877-1203/© 2023 SPLF. Published by Elsevier Masson SAS. All rights reserved.