The release of the rare diseases list, along with the allowance of foreign clinical data in new drug applications, is expected to substantially shorten the timeline and streamline the process for the approval of new orphan drugs, including imported drugs, and as a result encourage more Western orphan drug developers to bring more new drugs into the country.

Five Chinese governmental authorities, including the newly renamed State Drug Administration (SDA) and the National Health Commission (NHC), jointly issued on May 11 The List of Rare Diseases (First Batch) (the First List). This is the first time China has recognized rare diseases on a national level. The First List covers 121 rare indications. (See here for the full text of the First List.) It is expected that more indications could be added in the second and third batches of the same list to be issued in the future.

The SDA and the NHC on May 24 jointly published the Announcement on Optimization of Drug Review and Approval (the Announcement). Under the Announcement, the study sponsors for treatments for life-threatening indications without any effective therapy and rare diseases can file a new drug application (NDA) using clinical data obtained from outside China, on condition that the sponsor shows evidence that no racial differences exist. In addition, the Announcement further facilitates the review and approval process for clinical trial applications (CTA) and NDA for rare diseases on the basis of the priority review and approval process under the CFDA Opinions (defined below).

The SDA on July 6 issued the Technical Guidance for Accepting Drug Clinical Trial Data Obtained Overseas (Technical Guidance). The Technical Guidance attempts to further clarify the requirements on using clinical trial data obtained overseas for CTA and NDA in China. Under the Technical Guidance, with respect to NDAs for rare diseases, even though the clinical trial data obtained overseas is considered partially acceptable due to concerns of potential racial differences, SDA may still accept such clinical trial data with conditions, and require the registration applicant to collect supplementary data regarding safety and efficacy of such drugs after market authorization.

It should also be noted that prior to the release of the above new regulations, the then CFDA (now SDA) also issued Opinions on Priority Review and Approval Process to Encourage Drug Innovation (CFDA Opinions) on December 28, 2017. According to the CFDA Opinions, CFDA would reduce or waive clinical trials for foreign drug makers that file NDAs for rare disease drugs that have yet to be launched in China, and rare disease drugs with significant clinical advantages can submit the application to the Center for Drug Evaluation (CDE) for priority review and approval process during CTA and NDA. CDE will decide whether any further testing is necessary and notify the sponsors if they can proceed, with a waiver granted if the CDE deems this is not required. The CDE will also oversee the re-registration process for imported drugs.