罕见病用药难如何破？
罕见病，一个看似遥远而陌生的词汇，实则与我们每个人息息相关。据统计，全球大约有3亿人被罕见病所困扰，而在中国，这一数字接近2000万。这些疾病多因先天性遗传因素导致，与出生缺陷关系密切，对患者及其家庭带来巨大的生活挑战和经济负担。
近期，2024罕见病合作交流会在北京召开，国家卫生健康委妇幼司副司长沈海屏在会上强调了做好出生缺陷防治工作的重要性，指出这将对加强罕见病诊疗起到关键作用。自5年前，全国因出生缺陷导致的婴儿死亡率和5岁以下儿童死亡率已显著降低，神经管缺陷、唐氏综合征等严重致残出生缺陷的发生率也有所下降，这表明在防治出生缺陷方面取得了积极进展。
然而，罕见病患者依然面临两大困境：一是“无药可医”，二是“有药难负担”。北京病痛挑战公益基金会发起人王奕鸥指出，罕见病患者群体在医疗资源获取、药物可及性等方面存在巨大挑战。为破解这一难题，国家卫生健康委罕见病诊疗与保障专家委员会副主任委员兼办公室主任、全国罕见病诊疗协作网办公室副主任李林康提出，应通过研究、提升罕见病诊疗能力、优化诊疗网络等方式，为罕见病患者提供更精准、更有效的治疗方案。
对外经济贸易大学保险学院副院长孙洁强调，商业健康险在罕见病用药可及性方面的作用不容忽视。她建议，应通过政策引导、市场机制优化，让商业健康险与医保体系相配合，解决罕见病患者在“最后一公里”用药难的问题。清华大学药学院研究员杨悦则从政策制定和产业投入的角度出发，提出政府需制定有利政策，激发企业研发动力，形成政府、企业、市场多方协同的良性循环。
综上所述，破解罕见病用药难问题，需要政府、科研机构、医疗机构、商业保险等多方面共同努力，通过政策支持、技术创新、优化医疗资源分配等措施，为罕见病患者提供更全面、更有效的医疗保障。这一过程既是对医学科技的挑战，也是对社会人文关怀的考验，需要全社会的共同参与和关注。
英语如下：
Title: “Decoding the Challenge of Rare Disease Medication: Multi-Party Collaboration in Search of Solutions”
Keywords: rare disease medication, prevention of birth defects, collaborative conference
Content: How to Break the Challenge of Rare Disease Medication?
Rare diseases, a seemingly distant and unfamiliar term, are actually closely tied to each of us. Statistics show that around 300 million people worldwide are affected by rare diseases, with nearly 20 million in China. These diseases, often caused by先天 genetic factors, are closely related to birth defects and pose significant challenges to patients and their families in terms of both life and financial burden.
Recently, the 2024 Rare Disease Collaboration and Exchange Conference was held in Beijing, with Deputy Director Shen Haiping of the National Health Commission’s Women and Children Health Bureau emphasizing the importance of strengthening efforts to prevent birth defects. This, he pointed out, would be crucial in enhancing rare disease diagnosis and treatment. Over the past five years, there has been a notable decrease in infant mortality rates due to birth defects and the incidence of severe disabilities caused by conditions like neural tube defects and Down Syndrome, indicating progress in the prevention of birth defects.
However, rare disease patients still face two major challenges: “no cure for lack of medicine” and “difficulty in accessing affordable medicine”. Wang Yigu, founder of the Beijing Patient Challenge Charity Foundation, noted that rare disease patient groups face significant challenges in accessing medical resources and obtaining medicines. To address this issue, Vice Chairman and Secretary-General of the National Rare Disease Diagnosis and Assurance Expert Committee, as well as Deputy Secretary-General of the National Rare Disease Diagnosis and Treatment Network Office, Li Lankang, suggested that efforts should be made to improve rare disease diagnostic capabilities, optimize diagnostic networks, and provide more precise and effective treatment plans for rare disease patients.
Associate Dean of the Insurance School at the University of International Business and Economics, Sun Jie, stressed that the role of commercial health insurance in ensuring access to rare disease medication cannot be overlooked. She recommended that through policy guidance and market optimization, commercial health insurance should complement the national health insurance system to solve the “last mile” problem of accessing rare disease medication. Researcher Yang Yue from the School of Pharmaceutical Sciences at Tsinghua University proposed that the government should formulate favorable policies to stimulate the industry’s research and development efforts, fostering a cooperative cycle involving the government, enterprises, and the market.
In summary, to break the challenge of rare disease medication, cooperation is needed from various sectors including the government, research institutions, healthcare facilities, and commercial insurance. Through policy support, technological innovation, and optimized distribution of medical resources, a more comprehensive and effective healthcare system can be provided for rare disease patients. This process is a challenge to medical science and a test of social compassion, requiring the active participation and attention of the entire society.
【来源】http://www.chinanews.com/life/2024/07-22/10255254.shtml
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