Alice Cheng and Zhi Xie of the Rare Genomics Institute have published a open-access paper, "Challenges in orphan drug development and regulatory policy" in the Orphanet Journal of Rare Diseases.
Orphan drugs are pharmaceutical treatments developed to treat specific rare diseases. They aren't profitable for pharmaceutical companies to produce due to their extremely specific nature. Regulatory policies on orphan drug development are well-defined in the United States and European countries, but rare disease policies in China are still fluctuating. Pharmaceutical companies in China are de-incentivisted to pursue drug development for rare diseases due to a lack of clear definition and regulatory approval process. As a result, many rare disease patients in China pay out of pocket for international treatments.
Many grassroots movements have begun to support rare disease patients and facilitate research for the development of orphan drugs. The Chinese FDA has recently set new regulatory guidelines for drugs being developed in China, including an expedited review process for lifesaving treatments.
Cheng and Xie's paper compares orphan drug development and regulatory policy in China and the US. They find that due to political, economic, and cultural differences, China cannot simply base its policies on the American model. China's public healthcare system has the opportunity to take advantage of available data to create aggregated databases for diseases and genomic information, assisting epidemiology research.
The authors advocate for the five suggestions proposed by the National People's Congress and Chinese People's Political Consultative Conference of 2009:
- Establish a definition for rare diseases.
- Develop an orphan drug reimbursement system.
- Propose a clear and simple approval pathway for imported orphan drugs.
- Promte rare disease research through policy.
- Develop government-supported programs for rare disease patients.