摘要目的 探索提高我国罕见病患者用药可及性的管理策略.方法 通过对我国近年典型罕见病用药事件进行分析研究、对医院管理和临床医疗专家进行访谈式问卷调查,以及对我国目前罕用药医疗保险政策进行分析研究,确定药品可及性的含义,明确影响罕见病患者用药可及性的障碍因素.结果 我国医药卫生系统目前主要在科技、供应、信息传递和医疗保障能力四个方面存在主、客观障碍因素,影响罕见病患者用药的可及性,患者呈现出用不到药、用药不及时和用不起药的特点.结论 提高我国罕见病患者用药的可及性,应明确罕见病与罕用药的基本概念和激励机制,设计提高医药卫生系统罕用药供应的反应性机制,构建政府、企业、患者三维主体协作模式,降低患者的经济负担.

abstractsObjective To probe into the strategies for improving the accessibility to orphan drugs for patients of rare diseases in China. Methods Analysis of typical cases of orphan drug use in recent years in China, interviews of hospital administrators and clinical doctors, and analysis of the present health insurance policies in China for orphan drugs, definition of the concept of drug accessibility, clarification of the factors hindering orphan drug accessibility. Results Four factors are found to hinder orphan drug accessibility in such aspects of science and technology, supply, information transfer and medical assurance in China's medicine and healthcare system. These subjective and objective factors affect drug accessibility of the patients of rare diseases, denying them of drug accessibility, of drug use in time, and of affordability of such drugs. Conclusion To raise the orphan drug accessibility in China, it is necessary to define basic concepts and incentive mechanism of rare diseases and orphan drug, design and raise the response mechanism of the medicine and health system in orphan drug supply, build a three dimensional cooperation model between such parties as the government, enterprise and patient, and reduce patients' economic burdern.