目的: 系统性评估先进治疗药品（ATMPs）因其独特技术特征在全生命周期中引发的患者可及性风险,并深入探讨以商业健康保险为核心的多元化金融解决方案在化解这些挑战中的关键作用。方法: 本研究通过剖析ATMPs从基础研究到商业化的全生命周期,归纳其高度个体化、生产供应链复杂、潜在长期影响等核心技术特征,识别与论证研发成功率、投入资金、疗效、长期安全等核心风险;梳理国际上在政府干预、金融工具及商业健康保险等方面的应对策略以及国内实践案例。结果: 商业健康保险基于ATMPs的技术特征设计定价与多维风险市场转化策略,通过研发阶段早期介入、创新支付方式等策略分担其财务与疗效不确定性风险。构建基本公共医疗保险、私人商业健康保险及创新支付协议等构成的多元化支付体系是促进市场转化的可行路径。结论: 打破碎片化的风险管理方式,构建一个政府、产业、金融及医疗机构多方协同、贯穿全生命周期的整合性风险治理框架,通过应用不同层次的支付工具构建起高效多元的ATMPs医疗支付体系,从而实现技术创新价值向患者获益的有效转化。
Abstract
Objective: The paper systematically evaluates the patient accessibility risks posed by advanced therapy medicinal products (ATMPs) throughout their entire lifecycle due to their unique technological characteristics, and explores the key role of diversified financial solutions, centered on commercial health insurance, in mitigating these challenges. Methods: This study analyzes the entire lifecycle of ATMPs from basic research to commercialization, summarizing their core technological characteristics, such as high personalization, complex production supply chains, and potential long-term impacts. Based on this, identifying and evaluating pivotal risks such as R&D success probability, financial commitment, therapeutic performance, and long-term safety. Subsequently, this study systematically reviews the response strategies of government intervention, financial instruments, and commercial health insurance throughout the entire lifecycle, and conducts case studies on domestic and international practices. Results: Based on the technical characteristics of ATMPs, commercial health insurance designs pricing and multi-dimensional risk market transformation strategies. It shares the financial and efficacy uncertainty risks of ATMPs through early intervention in the R&D stage, innovative payment methods, and other strategies. Building a diversified payment system consisting of basic public medical insurance, private commercial health insurance, and innovative payment agreements is a feasible path to promote market transformation. Conclusion: By breaking down fragmented risk management, the paper constructs an integrated risk governance framework that involves collaboration among government, industry, finance, and healthcare institutions throughout the entire life cycle. This framework should be built by applying different levels of payment tools to create an efficient and diversified ATMPs healthcare payment system, thereby effectively translating the value of technological innovation into patient benefits.
关键词
先进治疗药品 /
全生命周期 /
患者可及性 /
商业健康保险
Key words
advanced therapy medicinal products /
full lifecycle /
patient accessibility /
commercial health insurance
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