The impact of national reimbursement drug negotiation on hospital procurement of rare disease drugs: an interrupted time series analysis

Growing recognition has established rare diseases as a public health priority. With over 20 million rare disease patients in China, establishing a robust medical security system is crucial for advancing the Healthy China initiative. The National Reimbursement Drug Negotiation (NRDN) has emerged as a key pathway to incorporate rare disease drugs into the National Reimbursement Drug List (NRDL), aiming to improve drug accessibility and affordability. This study aims to evaluate the impact of the NRDN policy on rare disease drugs. Monthly drug procurement data were obtained from the China Hospital Pharmaceutical Audit Report database covering 2562 sample hospitals between January 2016 and December 2024. We determined the monthly procurement amount, procurement volume, daily cost and affordability of 19 rare disease drugs that were included in the NRDL via NRDN between 2017 and 2022. Single-group interrupted time series analysis (ITSA) was performed to assess the impact of the NRDN policy on drugs. The NRDN policy led to a significant increase in the procurement volume and procurement amount of 19 rare disease drugs both at instant and trend level, while daily costs had immediate drops but a long-term upward trend. Long-marketed drugs like Eptacog Alfa (Activated) and Bosentan showed no significant procurement changes after policy implementation. For 3 drugs that underwent renewals, variations were observed in procurement changes, while daily costs experienced significant immediate decreases and were further reduced by renewals. Affordability ratios declined substantially, with 9 drugs falling below the 40% threshold, though 10 still exceeded it. The NRDN policy is associated with increased procurement volumes and amounts of the 19 rare disease drugs, significantly reducing their daily costs, while improving affordability and validating the “price-for-volume” strategy. However, the overall affordability of rare disease drugs remains limited. Future efforts should be made to further enhance drug accessibility and alleviate patients' financial burdens.