罕见病病种遴选与优先主题确定方法的思考

Thoughts on Selection of Rare Diseases and Prioritized Research Topics

本文通过对罕见病病种遴选和优先主题确定全流程的梳理和总结,对罕见病病种的申报和初步审查、病种主题信息的标准化、病种主题优先级遴选的证据梳理及罕见病主题确定和病种遴选的评价方法等多个环节内容进行了深入分析。以期为后续开展罕见病病种遴选工作、提升罕见病病种遴选的公平合理性和科学性,并进一步推动中国罕见病相关领域的研究和决策提供参考和借鉴。

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 decisionmaking in China′s rare diseaserelated fields.