On February 6, 2018, the China Food and Drug Administration (CFDA) and its Center for Medical Device Evaluation (CMDE) issued a draft guidance entitled “Guidance on the Review of Medical Devices for Preventing and Treating Rare Diseases.” The draft guidance is based on the reform measures recently outlined in the government’s broad blueprint for drug and device reform—the Opinion on Strengthening the Reform of the Drug and Medical Device Review and Approval Process to Encourage Drug and Medical Device Innovation (“Opinion 42”).

The draft guidance provides regulatory expectations for medical device companies to conduct preclinical studies and clinical trials for medical devices intended to prevent or treat rare diseases, i.e., orphan medical devices. The draft guidance also proposes expedited programs for orphan medical devices, including a conditional approval program and a possible exemption from clinical trials, which were previously outlined in the Opinion 42.

The draft guidance applies to all medical devices, including in vitro diagnostics (IVDs), intended to prevent or treat rare diseases. The term “rare diseases” has not been defined under the current laws. However, according to the Opinion 42 and China’s primary healthcare regulator, the National Health and Family Planning Commission (NHFPC), the agency will soon release a list of qualifying orphan diseases.

This draft guidance would pave the way for successful development, review and approval of the devices intended to treat or prevent rare diseases. Specifically, the draft guidance sets forth the requirements for preclinical studies and clinical trials for companies to efficiently develop devices for treating, diagnosing, and screening rare diseases. For each intended use category, CMDE articulates expectations as to the necessary supporting preclinical and clinical data, including detailed information related to the rare disease (such as causes, symptoms, diagnosis, and available treatment), a comprehensive evaluation of the safety risks associated with the device, and a comparison with a similar product currently on the market.

Further, as set forth in the Opinion 42, the draft guidance announces the availability of a conditional approval pathway for orphan devices that (1) meet urgent clinical needs, such as devices treating serious, life-threatening diseases where no effective therapies exist, and (2) demonstrate an effect on a surrogate, or intermediate endpoint that is reasonably likely to predict clinical benefits. In those cases, CFDA may approve the product earlier under the condition that the applicant conduct an appropriate post-approval confirmatory study.

The draft guidance also states that if there is a similar medical device in the same category on the market, CFDA would allow applicants to choose to conduct a clinical evaluation (potentially relying on literature reviews, clinical experience, or clinical trial data of similar products), in lieu of conducting a new clinical trial. In accordance with the Opinion 42, the draft guidance also confirms that applicants can use the data from overseas trials to support the medical device application in China, provided that the data satisfy the requirements in CFDA’s Guidance on Acceptance of Foreign Clinical Data of Medical Devices, which CFDA has finalized last month.

Finally, the draft guidance states that CMDE will accept pre-application meeting requests for applications for orphan medical devices. Applicants should follow CMDE’s meeting request process and request a meeting with the review division under CMDE to discuss any major technical issues, safety issues, and clinical trial protocols prior to submitting a marketing application.

These are important developments that have the potential to significantly reduce the time to market orphan medical device in China. The draft guidance is open for public comments until March 5, 2018. Stakeholders should consider submitting comments and continue to follow the development of the draft guidance.