我国罕见病用药可及性现状分析

Analysis of the rare disease drugs availability in China

罕见病是一类具有单病种发病率低、发病机制复杂等特点的疾病,其多数为慢性疾病,儿童期就发病,且疾病常常危及生命。全球已知的罕见病约有7000多种,疾病数目庞大。本文通过梳理我国现有罕见病治疗药物的相关政策,分析罕见病治疗药物政策措施及现有成效和可及性现状,为我国罕见病用药可及性发展方向提供思路与参考。

Rare diseases are characterized by low incidence of single disease and complex pathogenesis.Most of them are chronic diseases that start in childhood and are often life-threatening.There are more than 7,000 known rare diseases in the world.This article combs through the relevant policies of rare disease drugs in China,analyzes the policies and measures of rare disease drugs,and the current effectiveness and availability status,so as to provide references for the development direction of rare disease drugs accessibility in China.