The therapeutic landscape of inherited bleeding disorders in China

The demand for diagnosis and treatment of inherited bleeding disorders, particularly hemophilia, is increasing in China. In recent years, significant progress has been made in the country regarding the development and application of related therapeutics and the construction of the Chinese healthcare delivery system with attention to more efficient, cost-effective and convenient healthcare access to the population in both the remote/rural and urban areas. This article aims to systematically review the current therapeutic landscape of inherited bleeding disorders in China, elucidate the developmental pipeline of investigational domestic novel drugs, and discuss the establishment of a tiered healthcare system for hemophilia in China in line with the developing national healthcare delivery system. Attention to research and development in China have resulted in increased therapeutic product manufacturing capacity, and China is progressively establishing a domestic supply system for therapeutics ranging from basic replacement therapies to advanced treatments including gene therapy for inherited bleeding disorders. Concurrently, the ongoing development of the tiered healthcare system is expected to optimize the allocation of medical resources and enhance the standardization of diagnosis and treatment nationwide. In the future, the successful development of domestic innovative drugs, combined with an efficient and cost-effective healthcare delivery system, will lay a solid foundation for improving treatment outcomes and the quality of life for patients in China.