Pfize

Abstract

The Outline of the 15th Five-Year Plan for National Economic and Social Development of the People’s Republic of China call for implementing a health-first development strategy during the 15th Five-Year Plan period. Guided by the two major strategic priorities of accelerating the “Healthy China” initiative and advancing high-quality population development, China’s healthcare service and security system will continue to undergo a shift toward a people-centered approach over the next five years. This transformation aims to provide equitable, accessible, systematic, continuous, high-quality, and efficient health services and security for all citizens. 

In China’s ongoing efforts to advance systemic reforms and enhance nationwide health outcomes, individuals with rare diseasesrepresent one of the most vulnerable groups that cannot be overlooked. The prevention and treatment of rare diseases has become a public health challenge. China places great significance on the prevention and treatment of rare diseases, having incorporated the improvement of rare disease drug security policiesinto the “Healthy China 2030” Blueprint as early as 2016. In 2025, the World Health Organization adopted a resolution titled “Rare Diseases: a Global Health Priority for Equity and Inclusion,” urging member states to “accelerate efforts toward achieving and extending universal health coverage by 2030, ensuring healthy lives and well-being for all individuals, including persons living with a rare disease, throughout their life course.” China’s active participation in advancing this resolution demonstrates its confidence and commitment to providing healthcare services and security for all people, including those with rare diseases.

Globally, drug development in the rare disease field has accelerated in recent years. With the emergence and application of innovative therapies, treatment approaches for certain rare diseases have undergone fundamentalshifts. Treatment models have also evolved into comprehensive, lifelong health management approaches resembling those for chronic diseases. Patients now have the opportunity to achieve the same quality of life as healthy individuals through long-term, standardized treatment. China has made significant progress in the field of chronic disease prevention and control and is advancing the entire chain of services spanning diagnosis, treatment, rehabilitation, and health management for chronic conditions. The characteristics of certain rare diseases, such as hemophilia, parallel those of chronic diseases in several respects. The necessary conditions are also in place for the development of an integrated service model for hemophilia covering the entire chain of diagnosis, treatment, rehabilitation, and health management.

China has consistently prioritized the diagnosis, treatment, and healthcare security of hemophilia patients, establishing a national hemophilia registry system and developed a preliminary three-tiered national hemophilia treatment system. Ondemand (episodic) and regular replacement therapy (prophylaxis) medications for children, as well as on-demand replacement therapy medications for adults, have been included in the Basic Medical Insurance (BMI) scheme. However, the current clinical treatment model for hemophilia in China remains predominantly ondemand replacement therapy. Adult patients experience an average of 52 bleeding episodes per year,  with nearly 90% of those over 30 years old developing disabilities. Further, 35% of adult patients over 18 years of age are employed,  while only 16% are married. 

Reducing the disability rate among hemophilia patients across all age groups has positive implications for enhancing population health and the quality of the labor force, alleviating long-term medical and social burdens, and achieving universal health coverage while promoting high-quality population development. China can leverage its successful experience in chronic disease management and use hemophilia as a focal point to establish a full-chain service model for the diagnosis, treatment, rehabilitation, and health management of rare diseases. This approach will also contribute Chinese solutions to global efforts in preventing and treating rare diseases.

During the 15th Five-Year Plan period, Pfizer stands ready to collaborate with healthcare institutions, charities, and industry partners to jointly advance the “Hemophilia Healthy 2030” initiative and actively support China in exploring a full-chain service model for the diagnosis, treatment, and health management of hemophilia tailored to its own circumstances. This model aims to provide hemophilia patients with disease management and insurance coverage throughout their entire lives. In this report, we offer three recommendations for policymakers to consider:

First, establish clear medium- and long-term prevention and treatment goals for hemophilia. Based on analysis of existing data from the National Hemophilia Registry, by 2030, the proportion of adult patients receiving regular prophylaxis can be increased, while disability rates among adult patients can be reduced. By 2035, the disability rate of patients of all ages can be significantly reduced.

Second, improve access to innovative therapies and enhance medical insurance coverage of prophylaxis for adults. Innovative drugs with annual treatment costs within the affordable range of BMI funds may be included in the National Reimbursement Drug List (NRDL) through the national negotiation process. This would facilitate a shift in treatment models for adult patients and thus reduce disability rates

Finally, strengthen the capacity of hemophilia centers and develop a quality control indicator system for hemophilia. This would standardize diagnosis, treatment, rehabilitation, and patient management services, gradually shifting the focus of China’s clinical treatment model for hemophilia from emergency care and disability management to lifelong health management.

Download the full report：Reducing the Disability Rate of Hemophilia Patients and Advancing High-quality Population Development by Establishing a Full-lifecycle Medical Security System for Rare Diseases