A review of China's rare disease drug policies and their implementation effectiveness

CUI Na; LIU Kai; DONG Quan-liang; TIAN Li-juan

doi:10.20251/j.cnki.1003-3734.2025.19.002

Abstract：Objective:To sort out the policies related to rare disease drugs in China,exploring their incentive and promotion effects on the marketed entry of rare disease drugs.Additionally,the implementation effects and shortcomings of the policies were analyzed,and suggestions to improve the policy of rare disease drugs were put forward.Methods:China's policies on R&D registration and medical security of rare disease drugs and approved rare disease drugs from 2018 to 2024 were summarized and analyzed.Results:China's rare disease drug policies currently focus on the research and registration of drugs prior to market approval,as well as the construction of post-market medical security systems.Under the impetus of policies,the difficulties on research and development of domestic rare disease drug in China has decreased,the time to market has been shortened.Also,the number of drugs in the pipeline and those already in the market has continued to grow.While ensuring the accessibility of rare disease drugs for patients,medical security policies have also significantly reduced the financial burden on patients.Flexible clinical research and development strategies have accelerated the domestic market entry of foreign rare disease drugs,and most domestic research and development pipelines have also accelerated their progress under the guidance and effective combination of multiple policies.Conclusion:In the future,China should accelerate the formulation of guiding documents and continuously improve the establishment of comprehensive policy system and management mechanism for rare diseases.This will encourage domestic pharmaceutical enterprises in China to enhance their independent innovation and R&D investment,and support the research and development and market entry of innovative and generic drugs for rare diseases,so as to accelerate the development of China's rare disease industry.