Integrating Medical Aid and Philanthropic Support for Rare Diseases: A Global Policy Analysis Based on the Case of Hemophilia

Abstract

In the global pursuit of health equity, the medical security of the rare disease population is an extremely urgent issue. There are over 7,000 known rare diseases globally, affecting approximately 350 million people. The high treatment costs of these diseases seriously threaten the lives, health, and quality of life of patients, imposing a heavy burden on individuals, families, and society. Against this backdrop, this study comprehensively employs methods such as comparative case studies and policy analysis. Taking hemophilia as an example, it deeply explores the relationship between medical assistance and charity support for rare diseases.By comparing the relevant policies of countries and regions such as China, the United States, the European Union, Japan, and Germany, the study finds that there is a lag in incorporating new gene therapies into medical insurance in China. In contrast, the European Union has reduced the prices of rare - disease drugs by an average of 20% - 30% through its joint procurement mechanism. In City W, China, the charity subsidy coverage rate for rare - disease patients in suburban areas is only 65%, which is much lower than the 90% in urban centers, while Australia has significantly narrowed the regional gap through its national charity resource allocation mechanism. In City W, China, the out - of - pocket expenses of hemophilia patients account for 30% of the total medical costs, while in Japan, the out - of - pocket expense ratio for rare - disease patients has been reduced to less than 10% through multi - party collaboration. In addition, there are problems such as policy fragmentation, low administrative efficiency, and insufficient stakeholder synergy.To address these issues, this study proposes a series of specific solutions. For example, a dynamic adjustment mechanism for the medical insurance catalog should be established to ensure that eligible new therapies are included in medical insurance in a timely manner. A cross - regional equity fund should be set up, including the construction of a special transfer payment fund pool, a regional medical insurance fund mutual assistance mechanism, and a policy coordination mechanism. Digital transformation should be promoted by building a national rare - disease information platform and using blockchain technology to ensure data security, and implementing the "One - Window Acceptance" approval model. The legislative framework should be improved to clarify the rights and responsibilities of all parties, and a tripartite cooperation mechanism among the government, charitable organizations, and pharmaceutical companies should be established. These strategies aim to improve the global rare - disease support system, promote medical fairness, and provide references for the formulation of global rare - disease policies.