ACCESSIBILITY OF FULL VERSUS PARTIAL ORPHAN DRUGS IN CHINA: AN ANALYSIS BASED ON THE 2017-2024 NATIONAL DRUG PRICE NEGOTIATION
Author(s)
Siyuan Chen, Bachelor1, Miao Lin, PhD2, Zhihao Zhao, Bachelor1, Yuanhao Zhu, Bachelor1, MING HU, PhD1;
1West China School of Pharmacy, Sichuan University, Chengdu, China, 2Department of Pharmacy, Naval Medical University, Shanghai, China
1West China School of Pharmacy, Sichuan University, Chengdu, China, 2Department of Pharmacy, Naval Medical University, Shanghai, China
OBJECTIVES: This study aimed to compare the characteristics and accessibility between Full Orphan Drugs (only orphan indications) and Partial Orphan Drugs (orphan and non-orphan indications) incorporated through China’s National Drug Price Negotiation from 2017 to 2024.
METHODS: A retrospective database was constructed using public data from government portals, Pharmcube and YaoZH. Drugs with indications covering diseases in China’s First and Second National Rare Disease Catalog were included. Accessibility was assessed in accordance with the WHO/HAI methodology for measuring availability and affordability. Availability was calculated as the proportion of healthcare institutions or retail pharmacies stocking the relevant rare disease medications relative to their national representation. Affordability was expressed as the ratio of daily out-of-pocket (OOP) expense to China's 2025 minimum daily wage. Descriptive statistics were used to summarize key characteristics, and Mann-Whitney U tests were employed to compare accessibility between the two groups.
RESULTS: 71 orphan drugs were identified, including 47 Full Orphan Drugs and 24 Partial Orphan Drugs. Those identified drugs covered 44 rare diseases, with multiple sclerosis and idiopathic pulmonary arterial hypertension (7 each) being the most common for Full Orphan Drugs, while melanoma (4) for Partial Orphan Drugs. Partial Orphan Drugs received higher rates of expedited approval policy support (83.3% vs. 76.6%). Full Orphan Drugs consistently exhibited higher mean affordability ratios both before (3.863 vs. 2.77) and after reimbursement (1.159 vs. 0.831),but the difference was not statistically significant (p=0.3159). The hospital availability in Partial Orphan Drugs was significantly higher (mean 0.065 vs. 0.026, p<0.05), whereas retail pharmacy availability did not differ significantly.
CONCLUSIONS: Partial Orphan Drugs demonstrated better hospital availability, likely due to their broader indications. However, Full Orphan Drugs remained less affordable and faced greater access barriers in healthcare settings. Policies should prioritize enhancing institutional supply and affordability support for Full Orphan Drugs.
METHODS: A retrospective database was constructed using public data from government portals, Pharmcube and YaoZH. Drugs with indications covering diseases in China’s First and Second National Rare Disease Catalog were included. Accessibility was assessed in accordance with the WHO/HAI methodology for measuring availability and affordability. Availability was calculated as the proportion of healthcare institutions or retail pharmacies stocking the relevant rare disease medications relative to their national representation. Affordability was expressed as the ratio of daily out-of-pocket (OOP) expense to China's 2025 minimum daily wage. Descriptive statistics were used to summarize key characteristics, and Mann-Whitney U tests were employed to compare accessibility between the two groups.
RESULTS: 71 orphan drugs were identified, including 47 Full Orphan Drugs and 24 Partial Orphan Drugs. Those identified drugs covered 44 rare diseases, with multiple sclerosis and idiopathic pulmonary arterial hypertension (7 each) being the most common for Full Orphan Drugs, while melanoma (4) for Partial Orphan Drugs. Partial Orphan Drugs received higher rates of expedited approval policy support (83.3% vs. 76.6%). Full Orphan Drugs consistently exhibited higher mean affordability ratios both before (3.863 vs. 2.77) and after reimbursement (1.159 vs. 0.831),but the difference was not statistically significant (p=0.3159). The hospital availability in Partial Orphan Drugs was significantly higher (mean 0.065 vs. 0.026, p<0.05), whereas retail pharmacy availability did not differ significantly.
CONCLUSIONS: Partial Orphan Drugs demonstrated better hospital availability, likely due to their broader indications. However, Full Orphan Drugs remained less affordable and faced greater access barriers in healthcare settings. Policies should prioritize enhancing institutional supply and affordability support for Full Orphan Drugs.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR14
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Insurance Systems & National Health Care, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases