从利司扑兰浅谈我国罕见病药物的审评审批

Regulatory evaluation of drugs for the treatment of rare disease by taking Risdiplam as an example

利司扑兰是基于我国罕见病监管政策快速获批的脊髓性肌萎缩症(SMA)治疗药物,其注册上市有效解决了我国患者未满足的临床需求和药物可获得性问题。本文结合SMA的疾病特征和未满足临床需求,简要回顾了利司扑兰的研发过程,以及申请人在早期临床研究期间与国家药品监督管理局药品审评中心(CDE)之间的沟通交流,进而阐述了CDE对利司扑兰关键性临床研究的评估,着重介绍了有关地区间差异问题的数据分析和审评考量。这个案例不仅展现了我国监管机构从患者的临床需求出发、积极解决罕见病药物可及性的价值导向,和对罕见病人群的人文关怀,另一方面也彰显了以数据为基础、以科学为依据的审评原则。

Risdiplam is a novel drug intended for the treatment of spinal muscular atrophy(SMA).Thanks to the support of China’s regulatory policy on rare diseases,the approval of Risdiplam registration by the Center for Drug Evaluation(CDE)of China National Medical Product Administration was relatively fast,which provided effective solution to the problems of unmet medical need and clinical availability for SMA patients in China.This manuscript introduced the disease characteristics of SMA and its unmet medical needs,then briefly reviewed the development process of risdiplam,during which there were sufficient communications between the applicant and CDE.This article elaborated the evaluation of the pivotal studies with risdiplam by CDE reviewers and emphasized on the assessments and conclusions of CDE regulators regarding potential inter-regional differences implied by the trial data.This case not only demonstrates the mindset of China’s regulatory agency towards unmet medical needs and drug availability in the context of rare diseases but also presents humanistic care for patients suffering from rare diseases as well.On the other hand,this case also reflects the science-based,data-oriented review principles of China’s regulatory agency.