Thoughts on Selection of Rare Diseases and Prioritized Research Topics

Kexin LI; Jingdan Chen; Dingding Zhang; Wudong Guo; Jiayin Zheng; Linkang LI; Kun Zhao; Shuyang Zhang

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Thoughts on Selection of Rare Diseases and Prioritized Research Topics

VernacularTitle:罕见病病种遴选与优先主题确定方法的思考
Author: Kexin LI ¹ ; Jingdan CHEN ² ; Dingding ZHANG ³ ; Wudong GUO ⁴ ; Jiayin ZHENG ⁵ ; Linkang LI ¹ ; Kun ZHAO ⁴ ; Shuyang ZHANG ⁶
Author Information

1. China Alliance for Rare Diseases, Beijing Society of Rare Disease Clinical Care and Accessibility, Beijing 100022, China.
2. Institute of Clinical Medicine, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China，Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, National Health Commission Key Lab of Health Economics and Policy Research, Center for Health Preference Research, Shandong University, Jinan 250012, China.
3. Institute of Clinical Medicine, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
4. China National Health Development Reasearch Center, Beijing 100032, China.
5. China Alliance for Rare Diseases, Beijing Society of Rare Disease Clinical Care and Accessibility, Beijing 100022, China，Insititute for Health China, Tsinghua University, Beijing 100080, China.
6. Department of Cardiology, State Key Laboratory of Complex Severe and Rare Diseases, National Rare Disease Medical Quality Control Center, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
Publication Type:Journal Article
Keywords: rare diseases; disease selection; prioritized topics; multi-criteria decision analysis
From: JOURNAL OF RARE DISEASES 2024;3(2):269-274
CountryChina
Language:Chinese
Abstract:
This article combs and summarizes the entire process of rare disease selection and priority theme determination, including the application and preliminary review of rare diseases, standardization of disease theme information, the evaluation methods of evidence sorting and disease selection for priority selection of disease themes, and other aspects of the content were analyzed in depth. It is expected to provide reference for the subsequent selection of rare diseases, improve the fairness, rationality and scientificity of rare disease selection, and further promote research and decision-making in China′s rare disease-related fields.