孤儿药研发激励制度设计与激励机制研究

Research on Incentive System Design and Incentive Mechanism for Orphan Drug R&D

目的:通过深入分析美日欧孤儿药研发激励政策的背景、激励机制以及实施效果,为我国孤儿药研发的制度设计提供建议。方法:对罕见病及孤儿药相关政策法规进行研究。结果:美日欧的孤儿药研发激励制度设计贯穿药品的研发、审评和上市后各阶段,孤儿药资格认定与激励措施相关联。孤儿药的研发激励机制以研发投入与"预期收益"相匹配为核心,在上市前给予研发资助、税收减免、沟通交流等激励;上市后给予市场独占期保护,从而最大限度地鼓励研发。结论:建议我国从罕见病患者健康权角度出发,尽快对罕见病和孤儿药研发进行单独立法,建立系统的研发激励机制。

Objective: To provide recommendations for the design of orphan drug development policy in China through an in-depth analysis of the background, incentive mechanism and implementation effects of orphan drug R&D policies in the US, Japan, and the Europe. Methods: Policies and regulations related to rare diseases and orphan drugs were studied. Results: The design of the orphan drug R&D incentive system in the US, Japan and Europe works throughout the drug development, review and post-marketing phases, and the qualification of orphan drugs is related to the incentive policies. The R&D incentive mechanism focuses on matching R&D investment with prospective return of the company. There several incentive policies such as R&D funding, tax reduction, communication with the Agency during clinical trial and review, and marketing exclusivity after the market launch, so as to encourage R&D to the maximum extent. Conclusion: It is suggested that China should enact separate legislation on rare diseases and orphan drugs as soon as possible from the perspective of the right to health of patients with rare diseases, and establish a systematic R&D incentive mechanism.