Progress in the research and pharmacoeconomic evaluation of drugs and devices for rare diseases in China.

The development, importation, and reimbursement of drugs and medical devices for rare diseases have become critical issues within China's healthcare system. Since 2018, China has issued two national Rare Disease Lists, covering 207 diseases. As of December 2025, 223 drugs for rare diseases have been marketed domestically, with 136 (61.0%) included in the national list for reimbursement by basic medical insurance scheme. Advances have also been made in diagnostic technologies and treatment equipment. This article also examines the issues with and factors influencing the pharmacoeconomic evaluation of rare disease therapies. Additionally, over 100 registered patient organizations contribute substantially to care, education, research, and advocacy. China has piloted multi-level healthcare security system, including national and local healthcare security systems. The introduction of a list of innovative drugs covered by commercial insurance in 2025 further supplements this system. These measures have collectively expanded reimbursement coverage. Despite progress in drug development, insurance coverage, and evaluation of drugs in terms of health economics, continued efforts are needed to enhance treatment accessibility and equity. Key measures include putting forward rare disease legislation, promoting research on health technology assessment, improving health utility measurement, encouraging domestic orphan drug development, and strengthening international collaboration. China's experience offers valuable insights for global rare disease prevention and treatment initiatives.