China Initiates a New Program for Rare Diseases

In late May, China’s CDE under the NMPA, promulgated a new program for rare diseases called the – Patient-Centered Action for Rare Disease Encouragement (better known as the Care Plan). This pilot program focuses on the “rare disease patient” and is geared toward including the patient’s voice in the rare disease drug process. Rare disease patients should have more chances to include their own experience in the scientific development of rare drugs and increased interaction with sponsors, regulators, and other relevant parties.

The CDE will pick rare disease projects in different stages of drug development for their pilot program. Drug sponsors who want to submit applications for this Care Plan must submit an application outlining their drug and the specific rare disease it addresses, the clinical plan and how appropriate patients will be selected, etc. The applications will be submitted on the CDE website. The benefits of the Care Plan include – increased dealings with CDE officials, products currently not on China’s rare disease list can be considered with KOL support, etc. In short, China is expanding its efforts in the rare disease space.


Written by: Ames Gross – President and Founder, Pacific Bridge Medical (PBM)

Mr. Gross founded PBM in 1988 and has helped hundreds of medical companies with regulatory and business development issues in Asia. He is recognized nationally and internationally as a leader in the Asian medical markets. Mr. Gross has a BA degree, Phi Beta Kappa, from the University of Pennsylvania and an MBA from Columbia University.