Accessibility of drugs for rare diseases in China: Policies and current situation

"Poor accessibility to drugs" is the most problematic issue for patients with rare diseases in China. In recent years, China has issued a number of policies, such as prioritizing speeding up the evaluation for rare disease drugs, publishing national rare disease lists and giving priority to treatments for severe diseases like rare diseases during annual adjustments of National Medical Insurance Medicine Catalogue to improve the accessibility of rare disease drugs. From the outcome perspective, the evaluation of rare disease drugs takes 3 months shorter than ordinary drugs, basic research projects have been started and the number of rare disease drugs included in National Medical Insurance Medicine Catalogue has increased to 50. However, the policies' effects on new drug research and development, rare disease diagnosis and treatment as well as drug pricing arc limited. It is recommended to learn the tilt policy of research and development for rare disease drugs from foreign countries and the mechanism of medical insurance funding and patient co-payments. Thus it is important to improve the availability, accessibility and affordability of rare diseases drugs based on the Chinese context.