Presentation (CTI)

OBJECTIVES: China faces crucial pressure from rare diseases in a population of up to 20 million. In response, the government has adopted the national negotiation of medicine prices for medical insurance to satisfy with patients’ needs. This study aimed to comprehensively assess the longitudinal trends in availability of nationally negotiated orphan drugs across China from 2018 to 2023, addressing critical gaps in understanding nationwide trends and policy efficacy.
METHODS: We conducted a temporal analysis and analyzed procurement data from 1,519 hospitals nationwide to examine the availability of 29 nationally negotiated orphan drugs targeting 17 rare diseases across 31 provinces in China from 2018 to 2023. Drug availability was analyzed at both the individual drug level and across multiple subgroups. Concurrently, we analyzed the impact of policies based on the drug availability trends.
RESULTS: The annual availability of 29 orphan drugs significantly increased from 1.42% in 2018 to 22.50% in 2023, with a compound annual growth rate of 4.22%. The availability of each drug increased dramatically after being included in the NRDL. In 2018, the number of available nationally negotiated orphan drugs was 7 (38.5%), while by 2023, 16 orphan drugs (55.2%) had availability exceeding 10.0%. However, regional disparities continue to exist and became significant from 2020. Eastern regions demonstrated higher drug average availability compared to other regions especially in northeastern regions (10.21% vs. 6.52% in average), demonstrating persistent regional inequality.
CONCLUSIONS: The availability of nationally negotiated orphan drugs in China has improved significantly, and the inclusion of the China’s National Reimbursement Drug List has proven to be a catalyst for this. However, persistent disparities highlight systemic inequities, underscoring the need for targeted policies to enhance equitable access in China’s evolving rare disease landscape.