罕见病专项立法的逻辑依据与构建路径

Logical basis and construction path of special legislation on rare diseases

罕见病确诊难、就医难、药物可及性低等问题,已成为我国乃至全球范围内的公共卫生治理难题。我国罕见病防治与保障措施碎片化现象明显,相关政策急需以法律形式确定,罕见病药品研发缺乏有效的法律与政策激励。专项立法是破解罕见病防治与保障困局的有效路径,当下我国推进罕见病专项立法是实现社会正义和保障患者健康权的内在要求,有广泛的社会共识与域外成熟经验可供借鉴。应明确罕见病专项立法防治兼保障的功能定位,确立合理的差别待遇的价值导向,坚持中央统筹推进与地方先行先试并行的思路,推进罕见病专项立法构建。

The difficulties in diagnosing rare diseases,seeking medical treatment,and low drug accessibility have become a public health governance challenge in China and even globally.The fragmentation of China’s rare disease prevention and protection system is evident,and relevant policies urgently require legal authorization and solidification.Research and development of rare disease drugs lack effective legal and policy incentives.Special legislation is an effective way to solve the dilemma of rare disease prevention and protection.Promoting special legislation for rare diseases in China at present is an inherent requirement for achieving social justice and protecting the right to health of patients.There is a broad social consensus and mature experience outside the region for reference.The functional positioning of special legislation for the prevention and protection of rare diseases should be clarified,a reasonable value orientation for differential treatment should be established,and the construction of special legislation for rare diseases should be promoted through adhering to the idea of central coordination and local pilot parallel promotion.