Title: Access to Orphan and Ultra-Orphan Drugs in China

Abstract

Objectives: Orphan drugs face a number of access challenges in China: the absence of official legislation, slow market authorization combined with high price control, limited funding and a high rate of under-diagnosis. The objective of this analysis was to identify and synthesize the evolution of access for orphan and ultra-orphan drugs in China and identify opportunities for access in certain rare disease areas. Methods: The orphan disease and drug legislation environment in China was compared with that in USA, EU countries, Korea and Japan. In addition current policies with indirect impact on orphan diseases in China were uncovered. The pathways of funding for Orphan drugs was developed and a list of relevant products based on the products included on the provincial “Reimbursement Drug Lists” (RDL) were identified. Results: Of the 203 total FDA orphan designations approved since 2007 by the time of the analysis, 33 of them were found to be registered with the CFDA (some products were approved for multiple orphan indications by the FDA). Overall, the products approved earlier by the FDA appeared to be more likely to be registered with the CFDA. Most of the orphan analogues were seen to have some form of coverage, either from provincial insurance or city insurance. Provincial insurance coverage was determined to be the most feasible option for reimbursement of orphan drugs in China. Conclusions: Given the continued evolution in health insurance, monitoring patient access to orphan drugs and how outstanding policies are implemented should be monitored. It remains to be seen if the passing of new public legislation will impact broader patient access to such rare disease treatments in China.