Public Health Program, University of Brasília, DF, Brazil.
Genomic Sciences and Biotechnology Program, Catholic University of Brasília, DF, Brazil.
Clin Ther. 2021 May;43(5):e103-e138. doi: 10.1016/j.clinthera.2021.02.006. Epub 2021 Apr 21.
The field of human medicine is in a constant state of evolution, developing and incorporating technological advances from diverse scientific fields. In recent years, cellular and gene therapies have come of age, challenging regulatory agencies to define the path for commercial registration. Approval necessarily demands robust evidence for safety and efficacy, but these exigencies must not be such that they render unviable the development and testing of the therapeutic agent. Furthermore, reimbursement strategies are required to guarantee commercial viability of these products, to avoid the risk that they will be removed from the market or become unavailable to most patients through lack of financial resources. To address such challenges, several countries have created strategies to manage advanced therapy products.
Based on official documents published by regulatory agencies worldwide, this review summarizes the current scenario in the United States, Europe, Brazil, Japan, South Korea, and China in this regard, discussing the harmonized and dissonant aspects of the regulatory framework in different regions of the world and exploring perspectives for the future.
The technical aspects of advanced therapies are increasingly complex, bringing challenges for high mass commercialization and demanding specific regulation. The regulatory framework of the analyzed regions is mainly recent and discordant, but many harmonizing initiatives were observed.
The comparative analysis of regulatory frameworks in different parts of the world is informative, as scientists must be aware of the rationale of regulators to assertively develop new technology and products that will be commercialized. The comparative analysis also provides insight into the main dissonances that must be addressed, fostering the harmonization of local regulatory frameworks. Many unanswered questions still lie ahead for the field of advanced therapies, and empirical evidence will be the most effective way to separate hype from hope and to establish the most sustainable mechanisms to regulate and finance such products in each part of the world.
人体医学领域处于不断发展和演变之中，从多个科学领域吸收并应用技术进步。近年来，细胞和基因治疗已日趋成熟，这给监管机构带来了挑战，需要它们为商业化注册制定发展道路。批准必然需要安全和疗效的有力证据，但这些要求不应使治疗剂的开发和测试变得不可行。此外，需要报销策略来保证这些产品的商业可行性，以避免因缺乏资金而使这些产品从市场上撤出或使大多数患者无法获得这些产品的风险。为应对这些挑战，一些国家制定了管理先进治疗产品的策略。
基于世界各地监管机构发布的官方文件，本文综述了美国、欧洲、巴西、日本、韩国和中国在这方面的现状，讨论了世界不同地区监管框架的协调和不和谐方面，并探讨了未来的发展方向。
先进疗法的技术方面日益复杂，为大规模商业化带来挑战，需要特定的监管。分析区域的监管框架主要是最近制定的，且存在差异，但也观察到了许多协调举措。
对世界不同地区监管框架的比较分析具有启发性，因为科学家必须了解监管机构的基本原理，以积极开发将商业化的新技术和产品。比较分析还提供了对必须解决的主要不和谐问题的深入了解，促进了地方监管框架的协调。先进疗法领域仍有许多悬而未决的问题，实证证据将是区分炒作与希望、建立监管和为世界各地区此类产品提供资金的最可持续机制的最有效方式。
