多位专家呼吁：成立国家级罕见病基金

Core Viewpoint - The establishment of a rare disease special fund is crucial for improving the treatment and support for over 20 million rare disease patients in China, addressing high-cost medication issues and ensuring sustainable funding sources for treatment [1][6][8] Group 1: Current Situation and Challenges - Over 7,400 rare diseases affect more than 420 million people globally, with over 20 million patients in China [1] - Rare diseases often require lifelong treatment with high costs, leading to irreversible damage if patients cannot afford medication [2] - The current healthcare system lacks a dedicated funding mechanism for rare diseases, making it difficult for patients to access necessary treatments [3][6] Group 2: Local Initiatives and Models - Shanghai has pioneered a multi-tiered funding mechanism for rare diseases since 2013, establishing a special fund for Gaucher disease that significantly reduced out-of-pocket expenses for patients [2][3] - Zhejiang Province has set up a rare disease medication guarantee fund, serving as a model for other regions [4][5] - Jiangsu Province has also proposed a structured funding mechanism for rare diseases, emphasizing government-led, multi-channel financing [5] Group 3: Expert Opinions and Recommendations - Experts advocate for the establishment of a national rare disease special fund, drawing on successful local models to address the high costs of rare disease medications [6][7] - The fund should target patients not covered by insurance and those facing high personal payment ratios, integrating various existing support resources [7][8] - Funding sources could include government allocations, healthcare reform surpluses, and public welfare funds, with a focus on effective utilization and transparent management [8]