OBJECTIVES

: In the past 2 decades, high priority was given to boost the development of orphan drugs (ODs) for rare diseases worldwide. In 1999, Europe established specific ODs legislation with a range of financial and non-financial incentives to encourage involvement and promote ODs approval. In contrast, guidelines to regulate ODs are just beginning to emerge in China in recent years. This study aimed to compare the approval of ODs in Europe versus China.

METHODS

: The website of European Medicine Agency (EMA) was searched to identify ODs approved up until 01/02/2020 in the European Union (EU). China National Medical Products Administration (NMPA) website was searched to investigate whether the identified ODs were approved in China.

RESULTS

: A total of 150 ODs were approved in Europe, among them, only 22 (14.7%) ODs were also approved in China. Majority of ODs were approved in China after Europe, with a median delay of 3.3 years. Encouraging news was that China began to realize the urgency for the timely market availability of innovative ODs for Chinese patients. Accordingly, a number of national policies were launched in China to reduce the gaps in ODs approval compared with developed countries. For example, a total of 37 ODs was designated as ‘overseas new drugs urgently needed in clinical settings’ until February 2020, suggesting that these ODs could be eligible for specialized and expedited approval pathways if ODs have been approved either in EU, United States (US), or Japan. This policy supported the fast approval of 4 ODs (Spinraza^®, Vimizim^®, Fabrazyme^® and Darzalex^®) with significant reduced review time in China in the last year.

CONCLUSIONS

: China is lagging behind EU in the approval of ODs. Thanks to the release of national initiatives to standardize and streamline the approval of ODs, improvements in market access of ODs in China are expected in the future.