构建罕见病数据信托的中国方案——以制定《上海市促进浦东新区罕见病医药发展若干规定》为视角

Developing Chinese Approach to Rare Disease Data Trust——From the Perspective of Formulating the"Stipulation of Shanghai Municipality on Promoting the Development of Drugs for Rare Diseases in Pudong New Area"

罕见病数据共享是解决诊疗难题、提高药品可及性的关键,必须以主体协同与统一立法来克服数据孤岛和高成本研发的挑战。在《关于构建数据基础制度更好发挥数据要素作用的意见》出台的背景下,数据要素价值的开发利用不断推进,发展出与医疗数据共享相一致的罕见病数据共享模式。然而,现有模式设计无法适应罕见病的诊疗需求,在实践中仍面临诸多挑战。数据信托作为一种创新模式,因其在平衡数据权益、保障数据安全以及促进数据高效利用方面的优势,显示出与罕见病领域的高度契合性。基于此,罕见病数据共享应立足于现实需求,通过发挥浦东新区法规先行先试的优势,以患者需求为出发点,确立共享、开放和公益的基本原则。同时,须注重个人隐私与数据安全的保护,通过优化收益分配规则,构建起罕见病数据信托的中国方案。最后,应通过对内推广最佳实践,对外建立互认机制,实现数据信托效力的外溢,促进各主体共同应对罕见病挑战。

Rare disease data sharing is key to solving difficulties in diagnosis and treatment and improving drug accessibility,thus it is imperative to overcome challenges of data silos and the high costs of research and development through collaboration among entities and unified legislation.In the context of the promulgating"Opinions on Establishing Basic System on Data to Better Play the Role of Data as A Productive Factor",the exploitation and utilization of the value of data as a productive factor are constantly promoted to form rare disease data sharing mode consistent with medical data sharing,however,the existing model design cannot meet the needs of development in the rare disease field and still faces many practical challenges.As an innovative model,data trust,due to its advantages in balancing data rights,ensuring data security,and promoting efficient data utilization,shows a high degree of compatibility with the rare disease sector.Based on this,rare disease data sharing should be grounded in practical needs,by leveraging the advantages of Pudong New Area regulations in pioneering trial and taking patients’needs as the starting point,to establish basic principles of sharing,openness,and public welfare.At the same time,attention is paid to the protection of personal privacy and data security to develop Chinese approach to rare disease data trust by optimizing rules on income distribution.Finally,it is advisable to realize the spillover of the effect of data trust and promote all entities to jointly address the challenges of rare diseases.