Presentation (CTI)

OBJECTIVES: To improve access to orphan drugs for rare diseases, a critical public health issue in China affecting an estimated 20 million individuals, the government implemented a national medicine insurance price negotiation policy. However, evidence of its impact remains limited. This study assessed longitudinal trends in the availability and affordability of nationally negotiated orphan drugs across China from 2020 to 2022 to evaluate policy efficacy.
METHODS: Procurement data from a national-level database were analyzed to assess the utilization of 29 nationally negotiated orphan drugs covering 17 rare diseases across 31 provinces in China from 2020 to 2022. Changes in procurement volume, defined daily doses (DDDs), and defined daily dose cost (DDDc) at both national and provincial levels were examined to reflect shifts in drug availability and affordability.
RESULTS: The total procurement volume of 29 orphan drugs increased from USD 169.91 million in 2020 to USD 419.55 million in 2022, with a relative growth of 146.92%. The DDDs of all drugs increased from 1,480.78 in 2020 to 4,476.08 in 2022, reflecting a 202.28% relative increase. Most provinces demonstrated an upward trajectory in total DDDs; however, regional disparities continue to exist. Shanghai exhibited the highest absolute increase (ΔDDDs = 96,659.23), and Jiangsu had the most substantial fluctuation in DDDc. Eastern region exhibited the largest absolute changes in DDDs and DDDc, compared to other regions especially in northeastern regions, demonstrating persistent regional inequality.
CONCLUSIONS: The availability of nationally negotiated orphan drugs in China has improved significantly. However, persistent disparities highlight systemic inequities, underscoring the need for targeted policies to enhance equitable access in China’s evolving rare disease landscape.