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细胞和基因治疗监管、定价和报销框架:以韩国和欧盟为例。

Cell and gene therapy regulatory, pricing, and reimbursement framework: With a focus on South Korea and the EU.

机构信息

Center for Growth Engine Industries, Korea Institute for Industrial Economics and Trade (KIET), Sejong, Republic of Korea.

College of Pharmacy, Chung-Ang University, Seoul, Republic of Korea.

出版信息

Front Public Health. 2023 Feb 24;11:1109873. doi: 10.3389/fpubh.2023.1109873. eCollection 2023.

Abstract

Ever since relevant bioengineering technologies have sufficiently matured to the platformizable commercialization stage, a slew of money has flocked to the cell and gene therapy market over the last few years, resulting in an abundance of clinical studies in the field. Newer modalities have brought up a string of regulatory and legislative tasks, such as developing guidelines and legislative rules to systematically regulate newer pharmaceutical products. Accordingly, another layer of legislation and guidelines tailored for cell and gene therapies has been introduced and is expected to evolve on par with technological progress. Furthermore, authorities have shifted to pricing and reimbursement policies that can share risks for cost and outcome among stakeholders altogether, such as developers and the government, while expanding the accessibility of patients to innovative cell and gene therapies. This review attempts to capture the salient regulatory features of the cell and gene therapy market in the context of South Korea and the European Union and points out where two sovereign entities currently stand on each policy element and how each tackles regulatory challenges. We can observe the converging trend where regulatory, pricing and reimbursement rules of adjoining countries in the supranational union or member countries of a consortium are getting more aligned. Evidently, concerted efforts to share regulatory science knowledge and embrace reference pricing have played their parts. The authors argue that policy priorities should be placed on initiatives to harmonize with other medical authorities to better the rights of patients and clear out the uncertainties of developers, ultimately to share and advance regulatory science and layout forward-looking policies at opportune times.

摘要

自相关的生物工程技术充分成熟到可平台化商业化阶段以来,近年来大量资金涌入细胞和基因治疗市场,导致该领域有大量的临床研究。更新的治疗方法带来了一系列监管和立法任务,例如制定指导方针和立法规则,以系统地监管更新的药物产品。因此,又引入了另一层针对细胞和基因治疗的立法和指导方针,并有望与技术进步齐头并进。此外,当局已转向定价和报销政策,可以在利益相关者(如开发商和政府)之间共同分担成本和结果的风险,同时扩大患者获得创新细胞和基因疗法的机会。本综述试图在韩国和欧盟的背景下捕捉细胞和基因治疗市场的监管特征,并指出两个主权实体目前在每个政策要素上的立场,以及各自如何应对监管挑战。我们可以观察到趋同的趋势,即超国家联盟或财团成员国的毗邻国家的监管、定价和报销规则越来越协调一致。显然,共享监管科学知识和采用参考定价的协同努力发挥了作用。作者认为,政策重点应放在与其他医疗当局协调一致的举措上,以改善患者的权益,消除开发商的不确定性,最终共享和推进监管科学,并在适当的时候制定前瞻性政策。

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