The impact of China’s drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog

ObjectiveChina released two batches of the Rare Disease Catalog in 2018 and 2023, respectively, listing a total of 207 diseases. This study aims to analyze the accessibility of medications for diseases included in the catalog and assess the impact of drug regulatory reforms on the supply of medications for rare diseases.MethodsThis study compiled five categories of information on drugs for 207 rare diseases: basic information on drug availability, the number of drug approvals and production capa