PSY31 - Europe-China Comparison of Orphan Drugs Approvals

Abstract

Rare diseases, today, constitute a public health issue. Specific legislations to incentivize the development of orphan drugs were adopted in Europe in 1999, whereas no specific legislation was established in China until now. Therefore, our study aimed to assess the availability of orphan drugs in China compared to Europe and to discuss potential challenges facing orphan drugs in China. The official websites of European Medicine Agency (EMA) and China Food and Drug administration (CFDA) were searched to identify approved orphan drugs up until 01/03/2018 in Europe and China, respectively. The delays in approval were calculated and delay rationales were discussed. A total of 102 orphan drugs were approved in Europe. Among these 102 orphan drugs, 15 (14.7%) were approved in China. Only the drug Dacogen® was approved earlier in China than in Europe, with a time interval of 3.98 years. The median delay was 3.29 years. The shortest and longest time lags were 55 days and 14 years, for the drugs Nexavar® and Zavesca®, respectively. Delays in approval may be due to several reasons: 1) the lack of a clear definition of rare disease in China, 2) the requirement of completing a phase 1 study outside China before implementing clinical trial in China, 3) manufacturers’ decision because of complex procedures, concerns regarding the profitability and uptake in the market 4) other reasons include staff shortage in drug evaluation center and inadequate intellectual-property protection in China. Special approval procedure for priority review of orphan drugs has been established as of 2017 in China. In addition, orphan drugs may also achieve faster reimbursement by national drug price negotiation. Improvement is expected for orphan drugs access. However, more efforts and a specific legislation for orphan drugs are still needed in China to ensure rapid patient access to orphan drugs.