新华鲜报|约100种罕见病用药进医保！生命与希望在集结

Core Insights - The 2025 China Rare Disease Conference highlighted significant advancements in the diagnosis and treatment of rare diseases, showcasing a growing network of hospitals and an expanding list of covered diseases and medications [1][3][5] Group 1: Healthcare System Developments - The national medical insurance drug list now includes medications for 42 types of rare diseases, with 8.6 billion yuan allocated for rare disease drug payments in 2024, accounting for 7.7% of total drug payments during the agreement period [3][5] - A multi-layered insurance system is being developed, combining basic medical insurance, critical illness insurance, and medical assistance, while exploring commercial health insurance and social charity as additional support [3][5] Group 2: Research and Innovation - The conference emphasized the importance of research in transforming previously untreatable conditions into manageable diseases, with notable examples of successful drug development now included in the insurance system [5][6] - The establishment of the China Rare Disease Comprehensive Cloud Service Platform aims to enhance diagnostic applications and create a comprehensive map for rare disease treatment and support [5][6] Group 3: Global Contributions and Collaboration - China is positioning itself as a key player in global rare disease governance, having established a reporting system covering 1.15 billion people and registering 1.64 million cases [8] - The government is actively promoting policies to expedite the review and approval of rare disease medications and medical devices, while also setting up specialized funding and laboratories for rare diseases [8][9] Group 4: Challenges and Future Directions - Despite progress, challenges remain, including limited awareness of rare diseases in grassroots areas, high costs of innovative drug development, and the need for faster policy implementation to facilitate market access [9][10] - The commitment to prioritizing patient needs and enhancing the visibility of rare diseases is essential for improving the quality of life for affected individuals [9][10]