SFL Regulatory Affairs and Scientific Communication GmbH, Basel, Switzerland.
Handb Exp Pharmacol. 2024;284:367-387. doi: 10.1007/164_2023_648.
In the European Union (EU), advanced therapy medicinal products (ATMPs) undergo evaluation by the European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT) to obtain marketing authorization under the centralized procedure. Because of the diversity and complexity of ATMPs, a tailored approach to the regulatory process is required that needs to ensure the safety and efficacy of each product. Since ATMPs often target serious diseases with unmet medical need, the industry and authorities are interested in providing treatment to patients in a timely manner through optimized and expedited regulatory pathways. EU legislators and regulators have implemented various instruments to support the development and authorization of innovative medicines by offering scientific guidance at early stages, incentives for small developers and products for rare diseases, accelerated evaluation of marketing authorization applications, different types of marketing authorizations, and tailored programs for medicinal products with the orphan drug designation (ODD) and the Priority Medicines (PRIME) scheme. Since the regulatory framework for ATMPs was established, 20 products have been licenced, 15 with orphan drug designation, and 7 supported by PRIME. This chapter discusses the specific regulatory framework for ATMPs in the EU and highlights previous successes and remaining challenges.
在欧盟（EU），先进治疗药物产品（ATMPs）需要经过欧洲药品管理局（EMA）的先进治疗药物委员会（CAT）评估，才能通过集中程序获得上市许可。由于 ATMPs 的多样性和复杂性，需要采用量身定制的监管程序，以确保每个产品的安全性和有效性。由于 ATMPs 通常针对具有未满足医疗需求的严重疾病，因此行业和监管机构有兴趣通过优化和加速监管途径，及时为患者提供治疗。欧盟立法者和监管机构已经实施了各种工具，通过在早期提供科学指导、为小型开发商和罕见病产品提供激励、加速评估营销授权申请、提供不同类型的营销授权以及为具有孤儿药指定（ODD）和优先药物（PRIME）计划的药物制定量身定制的方案，来支持创新药物的开发和授权。自 ATMPs 的监管框架建立以来，已有 20 种产品获得许可，其中 15 种具有孤儿药指定，7 种获得 PRIME 支持。本章讨论了欧盟 ATMPs 的具体监管框架，并强调了以前的成功和仍然存在的挑战。
