Presentation (CTI)

OBJECTIVES: Rare disease patients face a heavy economic burden, which is primarily attributable to the high costs of their treatments that often require long-term or lifelong adherence. National Reimbursement Drug List (NRDL) negotiation policy implemented in China has reduced the prices of rare disease medications, thereby alleviating the economic burden of rare diseases. This study aimed to evaluate the impact of the NRDL policy on the economic burden of rare diseases.
METHODS: We obtained economic burden data of rare disease from 208 tertiary hospitals in Central China from January 2022 to August 2025. A controlled interrupted time series design was used to evaluate changes in the total medical cost per hospitalization, and out-of-pocket cost per hospitalization among hospitalized patients with rare disease after policy intervention. Patients were grouped based on whether affected by the NRDL negotiation policy, those impacted by the 2022 negotiation NRDL policy (implemented in March 2023) were assigned to Group A, those impacted by the 2023 negotiation NRDL policy (implemented in January 2024) were assigned to Group B, and patients with rare diseases unaffected by the NRDL policy were assigned to Group C.
RESULTS: Overall, 77 rare diseases and 34,808 rare disease-related hospitalizations were included in this study. Compared with Group C, after policy intervention, patients in Group A showed significant decreases in both total medical cost per hospitalization (β = -9040.722, P = 0.002) and out-of-pocket cost per hospitalization (β = -2451.992, P = 0.005), patients in Group B also showed significant decreases in the total medical cost per hospitalization (β = -2605.56, P=0.044) and out-of-pocket cost per hospitalization (β = -1741.203, P=0.050).
CONCLUSIONS: The NRDL negotiation policy can significantly reduce the economic burden of rare diseases in China, serving as an effective measure for the Chinese government to achieve the goal of universal health coverage.