Accessibility and Reimbursement Coverage of Rare Disease Drugs in China: Insights From the China's Lists of Rare Diseases

OBJECTIVES: To assess the accessibility and reimbursement coverage of drugs for the treatment of rare diseases listed by the China’s First and Second List of Rare Diseases, which were released in May 2018 and September 2023, respectively.

METHODS: As of June 21, 2024, we conducted a quantitative analysis of the rare disease drugs approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and investigated their marketing approvals and reimbursement coverage in China.

RESULTS: Out of the 207 diseases included in the China’s List of Rare Diseases, 132 had therapeutic drugs with global access. Among the diseases with available drugs, 92 (75.0%) had approved indications in China, and 58 (43.9%) had therapeutic drugs included in the China’s National Reimbursement Drug List (NRDL). Comparatively, among the rare diseases listed in China in 2019, 21 (55.3%) had their therapeutic drugs launched in China, out of the total 38 with globally accessible drugs. In addition, 5 unapproved rare disease drugs were piloted in the Hainan Free Trade Port, allowing the utilization of these drugs in designated hospitals. Encompassing 40 rare disease drugs (out of a total of 73 drugs)，three batches of Lists of Overseas New Drugs Urgently Needed in Clinic were issued, in response to the release of the China’s First List of Rare Diseases. Notably, among these drugs, 33 (54.8%) had approved indications from the China’s First List of Rare Diseases, 20 (27.4%) underwent expedited approval, and 13 (17.8%) were included in the NRDL.However, 40 listed rare diseases still lack available therapeutic drugs in China, and 34 approved rare disease drugs are not covered by health insurance.

CONCLUSIONS: The release of the China’s List of Rare Diseases has significantly improved the accessibility and reimbursement coverage of rare disease medicines, although further improvements are needed.