How Far Have We Come? Challenges to Orphan Drug Access in China, 2011-2017.

Rare diseases are an important global public health issue. One significant challenge is to ensure the access to orphan drugs for patients with rare disease. This study aims to evaluate the accessibility of orphan drugs in China. Information pertaining to the availability and costs of each orphan drug in each hospital was obtained from the Chinese Medicine Economic Information database during 2011-2017. We measured the accessibility of orphan drugs from 3 aspects: availability, daily costs, and affordability to patients.The market availability rate of orphan drugs in China was 28.8% by June 30, 2017. The median availability rate at the hospital level was less than 15% but was increasing over time. The cost of a defined daily dose of orphan drugs varied significantly with a decreasing trend in all areas. Less than half of all surveyed orphan drugs had a cost of a defined daily dose no more than residents' average daily income.This study reveals the challenges of access to orphan drugs in China. The availability of marketed orphan drugs in China was relatively low and most orphan drugs placed a heavy financial burden on patients with rare disease. It is necessary to develop legislation for orphan drugs and encourage domestic generics. [ABSTRACT FROM AUTHOR]