Current Status of Drug Development and Implementation for Diseases Included in the Second Catalog of Rare Diseases
10.12376/j.issn.2097-0501.2023.04.015
- VernacularTitle:《第二批罕见病目录》收录病种的药物研发和落地现状
- Author:
Kexin LI
1
;
Kun ZHAO
2
;
Jiayin ZHENG
3
;
Yuqi YANG
4
;
Linkang LI
1
Author Information
1. China Alliance For Rare Diseases, Beijing Society of Rare Disease Clinical Care and Accessibility, Beijing 100020, China.
2. Vanke School of Public Health, Tsinghua University, Beijing 100080, China.Insititute for Health China, Tsinghua University, Beijing 100080, China.
3. China Alliance For Rare Diseases, Beijing Society of Rare Disease Clinical Care and Accessibility, Beijing 100020, China.Vanke School of Public Health, Tsinghua University, Beijing 100080, China.
4. China Pharmacentical University, Nanjing 211198, China.
- Publication Type:Journal Article
- Keywords:
rare diseases;
orphan drugs;
accessibility
- From:
JOURNAL OF RARE DISEASES
2023;2(4):596-601
- CountryChina
- Language:Chinese
-
Abstract:
The National Health Commission of the People′s Republic of China and six other departments jointly released the Second Catalog of Rare Diseases, which expanded the definition of rare diseases in China. The expansion of the catalog means greater attention to rare diseases at the national level, and is also expected to accelerate the launch of relevant rare disease drugs and benefit more rare disease patients. When the new rare disease catalog released, it is necessary to quickly sort out the availability of drugs and find out the base number for the advancement of subsequent relevant measures. This paper searched official medical information websites and medical databases such as the National Medical Products Administration (NMPA), the Center for Drug Evaluation (CDE), and foreign drug regulatory agencies, and sorted out and summarized the subject categories of diseases included in the Second Catalog of Rare Diseases, the corresponding global research and development and implementation of drugs, as well as the medical insurance access situation in China, elaborates on the differences in the accessibility of orphan drugs at home and abroad. As China′s prevention, treatment and protection of rare diseases has reached a new point, plans and prospects are made for the introduction of relevant policies in the future, as well as the promotion of research and development and protection and other key tasks.
