Management strategy for raising the accessibility to orphan drugs in China

Abstract

Objective 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. Key words: Rare diseases; Orphan drug; Drug accessibility; Drug management