代表委员履职故事丨全国政协委员杜丽群：探索建立系统性保障 推动“罕见病”不再“罕见”

Core Viewpoint - The article highlights the challenges faced by families of patients with rare diseases in China, particularly focusing on the high costs of medication and the need for systemic support to improve treatment accessibility and affordability [1][2]. Group 1: Challenges in Rare Disease Treatment - The average annual medication cost for patients with Duchenne Muscular Dystrophy (DMD) exceeds 100,000 yuan, creating a significant financial burden for ordinary families [1]. - The difficulties in diagnosis, medication access, and healthcare support are described as "three mountains" that weigh heavily on rare disease patients [1]. - The overall burden of rare disease treatment, including regular check-ups and standardized medication, is reported to be substantial [2]. Group 2: Initiatives and Progress - A rare disease reporting system covering 1.15 billion people has registered 1.64 million cases, indicating progress in tracking and managing rare diseases [2]. - The updated national medical insurance drug list published at the end of 2025 includes over 100 rare disease medications, covering more than 50 types of diseases, providing hope for more families [2]. - Efforts are being made to explore the establishment of a cost compensation mechanism for rare disease medications and to build a supply guarantee network for these drugs [2]. Group 3: Advocacy and Community Engagement - The article emphasizes the importance of gathering diverse opinions to formulate effective solutions for rare disease challenges [2]. - The commitment of healthcare professionals, such as Du Lijun, to advocate for patients and address their needs is highlighted, showcasing a dedication to improving healthcare access [3][4]. - The role of community support and the need for a robust social safety net for special children, including those with rare diseases, is underscored [2].