70万一针救命药进医保 罕见病男孩上学了

Core Viewpoint - The story highlights the journey of a child with spinal muscular atrophy (SMA) and the impact of national healthcare policies on access to life-saving treatments, showcasing the importance of medical advancements and insurance negotiations in improving patient outcomes [1][14]. Group 1: Medical Condition and Treatment - The child, known as Little Stone, was diagnosed with SMA at eight months old, with initial medical advice suggesting a grim prognosis and no available treatments in China [3][5]. - A special drug, Nusinersen, was introduced in China in early 2019, but its high cost of approximately 700,000 yuan per injection posed a significant financial burden for the family [5][10]. - In 2020, the drug's price was reduced to 550,000 yuan per injection, and the company offered a buy-one-get-five-free deal, improving the child's condition through ongoing rehabilitation and treatment [8][12]. Group 2: Healthcare Policy and Insurance Negotiations - The first round of national healthcare negotiations in 2021 saw the drug's price drop dramatically from 550,000 yuan to around 30,000 yuan, making it accessible for more patients [10][12]. - The negotiations aimed to balance the interests of patients, pharmaceutical companies, and the healthcare fund, reflecting a significant shift in the availability of rare disease treatments in China [10][14]. - Following the successful negotiations, the number of patients using Nusinersen in 2022 increased significantly, with a reported 2.6 times growth in sales compared to the previous year [12]. Group 3: Social Integration and Support - Little Stone successfully enrolled in elementary school, with the institution making special accommodations to support his learning needs, demonstrating a positive societal response to children with disabilities [16][18]. - The family continues to face challenges, including ongoing rehabilitation sessions and the aging of family members who provide support [20]. - The narrative emphasizes the importance of community support, family involvement, and healthcare policies in enabling children with rare diseases to integrate into society and pursue education [21].