Policy update Consultations for clinical R&D guidelines for rare disease drugs


To encourage greater investment in the research and development, and commercialization of novel drugs, on October 11th this year, the State Drug Administration Drug Review Center issued an official stakeholders’ consultation notice with regards to the "Clinical Research and Development Guidelines for Drugs to Treat Rare Diseases".

The current draft is mainly applicable to chemical and therapeutic biological products, including special considerations being applied to the R&D of rare disease drugs, clinical research and development plans, clinical trial design, safety assessment requirements, and communication etc., to further enhance the efficiency of clinical R&D in combating rare diseases.

Utilising this initiative as a method to resource-pool, gain invaluable insight, expertise and recommendations, the desired outcome will be to cater to the treatment needs of patients plagued with rare diseases, to deepen our understanding of the characteristics of rare diseases, which in turn would aid in the development of novel drugs in this field.