中国罕见病药品审评审批的改革与发展

Reform and Development of Rare Diseases Drug Evaluation and Approval in China

黄果 ¹杨志敏 ²唐凌 ²张杰 ²艾星 ²色日格楞 ²赵田²

扫码查看

作者信息

1. 国家药品监督管理局,北京 100076
2. 国家药品监督管理局药品审评中心,北京 100076
折叠

摘要

罕见病对社会、经济、乃至医疗体系构成了不容忽视的深远影响.罕见病药物的研发之路尤为艰难.由于患者群体小、市场需求有限,医药企业在药物研发上往往缺乏足够的动力与资源投入,加之研发周期长、成本高、风险大,使得许多潜在的治疗药物在研发初期便夭折.本文总结了国家药品监督管理局针对罕见病这一重要的公共卫生问题所采取的一系列鼓励政策,以及近年来罕见病药物审评审批的成果.正是这些政策加速了药监部门的审批速度,在确保药品安全有效的前提下,为罕见病患者带来了更多的治疗选择与希望.随着政策环境的不断优化与研发技术的不断进步,未来中国药监部门将继续围绕罕见病临床需求,实践"以患者为中心"的药物研发理念,为罕见病药物研发注入新活力,为罕见病患者带来更加精准、有效的治疗选择.

Abstract

Rare diseases have a significant and profound impact on society,the economy,and the health-care system.The path to developing drugs for rare diseases is particularly arduous.Due to the small number of pa-tients and limited market demand,pharmaceutical companies don't have enough incentives and resources to invest in drug research and development.Additionally,the long development cycles,high costs,and high risks have led to a number of potential therapeutic drug failures at the early stages of development.This article summarizes a se-ries of encouraging policies adopted by the National Medical Products Administration for rare diseases,which is an important public health issue,as well as the achievements in the review and approval of rare disease drugs in recent years.These policies have accelerated the approval process.Meanwhile,the policies ensure the safety and effectiveness of drugs and offer more treatment options and hopes to patients with rare diseases.With the continu-ous effort at optimizing the policy environment and the advancement of research and development technologies,China's drug regulatory authorities will continue to focus on the clinical needs of rare diseases,to implement"patient-centered"approach to drug development,inject new vitality into the research and development of drugs of rare diseases,and offer more precise and effective treatment choices for patients with rare diseases.

关键词

罕见病/药品监管/药物研发/审评审批

Key words

rare diseases/drug supervision/drug development/evaluation and approval

引用本文复制引用

出版年

2024

罕见病研究

CSTPCD

ISSN：

段落导航