Clinical benefit, price and epidemiology of orphan drugs approved in China: a cross-sectional analysis of orphan drug policy (ODP) in China.

objectivesTo evaluate the regulatory approval, epidemiology, clinical benefits and pricing of orphan drugs approved by the National Medical Products Administration (NMPA) in China.

designCross-sectional analysis. STUDY SAMPLE: Orphan drugs approved by the NMPA for rare disease indications between 1 January 2005 and 31 December 2022. OUTCOME MEASURES: The outcomes included regulatory approval lag time, National Reimbursement Drug List (NRDL) coverage, monthly treatment costs, clinical benefit (SMR, Service Médical Rendu) and additional therapeutic value (ASMR, Amélioration du Service Médical Rendu) ratings from the French National Health Authority (HAS, Haute Autorité de santé). Data were analysed at the drug indication level. The approval time lag was defined as the interval between the earliest global marketing authorisation and subsequent approval by China's NMPA.

resultsA total of 59 orphan drugs were approved by the NMPA for 32 rare disease indications in 2005-2022. Of the 24 indications with epidemiological data, the incidence ranged from 0.098 to 9.62 cases per 100 000 persons and the prevalence ranged from 0.017 to 25.7 cases per 100 000 population. A total of 51 drug-indication pairs had SMR ratings: 40 (63.5%) were rated as important, 2 (3.2%) as moderate, 6 (9.5%) as weak and 5 (7.9%) as insufficient. For the 61 indications with pricing data, the median annual treatment price was US$18 259.8 (IQR: US$6504.6 to US$184 011.9), while negotiated drugs, those included in the NRDL through price negotiation, had significantly lower prices than non-negotiated drugs (US$13 677.8 vs US$62 870.0, p=0.015). The ASMR ratings indicated that 5 (7.9%) were rated as important (II), 10 (15.9%) as moderate (III), 11 (17.5%) as minor (IV), and 25 (39.7%) as no improvement (V).

conclusionsThe orphan drug policy in China has improved access to and affordability of orphan drugs, particularly through priority reviews and NRDL price negotiations. However, substantial barriers remain in accessing orphan drugs, including limited definitions of rare diseases, the unavailability of overseas-approved orphan drugs and the high costs of non-NRDL drugs. Therefore, 'Three Medical Linkage' healthcare reform, which integrates health insurance, medical service delivery and pharmaceutical regulation, should continue to incentivise the development of orphan drugs and improve patient accessibility and affordability.