Proposed Changes by China’s FDA Hold Major Implications for the Early Stage Life Science Space

By James Huang, Research Analyst, LSN

The CFDA released a set of proposals on May 11th regarding major reforms in the Chinese regulatory and approval system. The series of proposals is designed to accomplish four major goals:

From the many proposals presented, two key points stand out as potentially having a major impact on life science startups outside of China:

• NHFPC will issue a rare diseases list and drugs/medical devices targeting these diseases will be eligible for a clinical trial waver and accelerated review. Drugs with orphan approval overseas will initially receive conditional approvals in China and only need minimal additional trials for full approval by the CFDA. Additionally, if investigational therapeutics or devices in the rare disease space show early clinical efficacy and predictable therapeutic benefit, they may receive conditional approval.
• Applicants who submit CTA/INDs may proceed with clinical trials if the Chinese Centre for Drug Evaluation (CDE) does not send comments or a rejection notice within 60 working days.

The first proposal regards approval and use of orphan medications in China. The CFDA is currently proposing to establish an orphan and rare disease list and, additionally, therapeutics and devices that treat life-threatening conditions on this list can receive conditional approval if early or mid-stage data can predict clinical benefit for these therapeutics and devices. Furthermore, the CFDA also proposed that conditional approval be given to orphan therapeutics and devices that have already received approval in foreign countries, even if they have no clinical trial data in China. Instead, these therapeutics and devices would have to conduct confirmatory trials as directed by the CFDA to keep the conditional approval. Fierce Biotech delves a bit more into the implications of these changes here.

The implications of these changes for both investigational and marketed therapeutics and devices in the orphan space are huge since, if approved in their present form, these proposals would make it easier to access the Chinese market, a process which has been both time consuming and financially prohibitive historically.

The second proposal focuses on the review process.  A key highlight is the proposed change to allow Phase I clinical trials in China to begin if no comments or rejection has been received from the CFDA review committee 60 working days after the application has been submitted. This change combined with the orphan therapeutics change mentioned above means that orphan therapeutics can be sure to get on a fast track to testing and on the market very quickly.

However, whether all these proposals will be approved in their current state remains to be seen. Keep your eyes peeled for the CFDA’s announcement coming in June.