Analysis of the Current Situation of Orphan Drugs for the Treatment of Rare Diseases in Children and Their Coverage Level of National Basic Medical Insurance in China
10.3870/j.issn.1004-0781.2025.12.013
- VernacularTitle:我国儿童罕见病治疗专用药及其医疗保障现状分析
- Author:
Yu HOU
1
;
Aili REYISHAMU
;
Li ZHOU
;
Yaqin WANG
;
Liru QIU
;
Dong LIU
;
Shiwei GONG
;
Wenting ZHANG
Author Information
1. 华中科技大学同济医学院药学院,武汉 430030
- Publication Type:Journal Article
- Keywords:
Rare diseases in children;
List of orphan drugs;
ATC classification of drugs;
Drug price level;
Drug coverage rate of national basic medical insurance
- From:
Herald of Medicine
2025;44(12):1962-1970
- CountryChina
- Language:Chinese
-
Abstract:
Objective To establish a pediatric rare disease catalog,analyze the current status of therapeutic drugs and their coverage of the medical insurance in China,and propose strategies to enhance drug accessibility.Methods Pediatric rare diseases were identified from China's two national rare disease catalogs combined with the EU Orphanet database,US FDA orphan drug database,and the Diagnosis and Treatment Standards for Rare Diseases in Children.We created a specialized drug catalog for pediatric rare diseases,then analyzed drug types(ATC classification),pricing,and medical insurance coverage using descriptive statistics based on Yaozhi.com drug bidding prices and the 2024 Drug of List National Basic Medical Insurance(NBMIDL).Drug affordability was assessed through annual treatment cost calculations.Results The national catalogs included 151 pediatric rare diseases(72.95%of listed conditions),spanning 13 disease systems.We identified 94 dedicated orphan drugs(by generic name)for these conditions,among which 43 were approved internationally but unavailable in China.The average unit price per package was 6 113.53 yuan.Overall NBMIDL coverage was 68.83%,but drugs priced above 7 000 yuan per unit had only 7.69%coverage.Annual treatment costs reached 4.54 million for laronidase(mucopolysaccharidosis).Conclusions Critical gaps persist in China's pediatric rare disease treatment landscape,including catalog deficiencies,inadequate coverage for high-cost drugs and insufficient domestic innovation.It is recommended to establish a list of orphan drugs for pediatric rare diseases,accelerate the import of foreign drugs and the local innovative drugs through policy incentives,optimizing medical insurance reimbursement mechanisms for pediatric rare disease drugs to comprehensively improve therapeutic accessibility.
