全国政协委员孙洁：探索建立国家级罕见病专项基金，保障用药支付

Core Viewpoint - The accessibility of innovative drugs for rare disease patients is a significant barrier, prompting calls for a national-level rare disease special fund to alleviate financial burdens on patients and their families [1][2]. Group 1: Policy Recommendations - Establishing a national-level rare disease special fund is essential, with clear principles and development goals to integrate various compensation policies [1]. - A multi-faceted funding and payment mechanism involving health insurance, critical illness insurance, commercial insurance, medical assistance, and charitable funds is necessary to address the payment issues for high-value rare disease drugs [2]. Group 2: Regional Initiatives - Provinces like Jiangsu, Zhejiang, and cities like Qingdao have made significant strides in rare disease drug coverage, serving as models for others [2]. - Zhejiang Province's establishment of a rare disease drug guarantee fund in December 2019, which includes a personal payment cap, has shown stable operational effects and provides a potential framework for national implementation [2]. Group 3: Fund Management and Legislation - The rare disease special fund is viewed as an ideal payment solution, enhancing public awareness and supporting local innovation in drug development [3]. - The fund should be managed by provincial social security accounts, with diverse funding sources including government allocations, donations, and lottery funds [3]. - Emphasizing the importance of legislative support for the establishment of a sustainable payment mechanism, the fund should balance patient rights with equitable resource distribution [3].