Characteristics and Accessibility of Orphan Drugs Through 2017-2024 National Drug Price Negotiation in China
Author(s)
Zhihao Zhao, Bachelor, Pei Wang, Master, Ziqi Zhao, Bachelor, Yu Chen, Master, Wei Huang, Bachelor, Ming Hu, PhD.
West China School of Pharmacy, Sichuan University, Chengdu, China.
West China School of Pharmacy, Sichuan University, Chengdu, China.
OBJECTIVES: This study aimed to characterize orphan drugs included in the National Reimbursement Drug List (NRDL) through National Drug Price Negotiation (NDPN) and evaluate their accessibility (price reductions, affordability, and availability) based real-world data of 2017-2024 NDPN.
METHODS: A retrospective database was constructed by integrating data from government portals, Pharmacube and YaoZH Databases. The rare diseases were defined as those listed in the first and second batches of China’s National Rare Disease Catalog. Accessibility was evaluated by referring to the WHO/HAI accessibility calculation methodology. Availability ratios were calculated as the proportion of healthcare institutions/retail pharmacies stocking rare disease drugs relative to national coverage. Affordability ratios were defined as out-of-pocket costs of each patient divided by China’s 2024 minimum daily wage. Descriptive statistical analysis was applied to key characteristics, and the χ² test was utilized to assess associations between characteristics and accessibility.
RESULTS: 71 listed orphan drugs were analyzed, covering 48 rare diseases indications and 21 treatment categories. The annual inclusion of orphan drugs increased from an average of 6 (2017-2021) to 13 (2022-2024) annually. Multiple sclerosis and idiopathic pulmonary arterial hypertension had the highest number of orphan drugs (7 each). Among these listed drugs, 40 (56.34%) were innovative drugs, 18 (25.35%) were biological products, and 22(31.00%) had both rare and common indications. The mean (SD) time from market approval to NRDL inclusion was 29.24 (40.84) months. Average price reduction reached 54.15%, while hospital and pharmacy availability remained low (3.22% and 4.87%, respectively). The number of affordable drugs (the ratio ≤1 ) for resident patients increased from 19 (26.76%) to 36 (50.70%) after reimbursement, but innovative drugs remained less affordable (χ²=7.66, p<0.05).
CONCLUSIONS: The NDPN mechanism accelerated rare disease drug inclusion, reduced prices, and improved accessibility. However, institutional availability gaps and affordability disparities for innovative drugs persist.
METHODS: A retrospective database was constructed by integrating data from government portals, Pharmacube and YaoZH Databases. The rare diseases were defined as those listed in the first and second batches of China’s National Rare Disease Catalog. Accessibility was evaluated by referring to the WHO/HAI accessibility calculation methodology. Availability ratios were calculated as the proportion of healthcare institutions/retail pharmacies stocking rare disease drugs relative to national coverage. Affordability ratios were defined as out-of-pocket costs of each patient divided by China’s 2024 minimum daily wage. Descriptive statistical analysis was applied to key characteristics, and the χ² test was utilized to assess associations between characteristics and accessibility.
RESULTS: 71 listed orphan drugs were analyzed, covering 48 rare diseases indications and 21 treatment categories. The annual inclusion of orphan drugs increased from an average of 6 (2017-2021) to 13 (2022-2024) annually. Multiple sclerosis and idiopathic pulmonary arterial hypertension had the highest number of orphan drugs (7 each). Among these listed drugs, 40 (56.34%) were innovative drugs, 18 (25.35%) were biological products, and 22(31.00%) had both rare and common indications. The mean (SD) time from market approval to NRDL inclusion was 29.24 (40.84) months. Average price reduction reached 54.15%, while hospital and pharmacy availability remained low (3.22% and 4.87%, respectively). The number of affordable drugs (the ratio ≤1 ) for resident patients increased from 19 (26.76%) to 36 (50.70%) after reimbursement, but innovative drugs remained less affordable (χ²=7.66, p<0.05).
CONCLUSIONS: The NDPN mechanism accelerated rare disease drug inclusion, reduced prices, and improved accessibility. However, institutional availability gaps and affordability disparities for innovative drugs persist.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD107
Topic Subcategory
Health & Insurance Records Systems
Disease
SDC: Rare & Orphan Diseases