提升罕见病药品可及性的机制设计与模式选择——对设立“中华罕见病种子基金”的探讨

Mechanism Design and Mode Selection for Enhancing Rare Disease Drug Accessibility:Exploration of the Establishment of‘Chinese Rare Disease Seed Fund’

在罕见病群体所面临的诸多困境中,用药支付困境始终是最难以解决且无法回避的难题。本文通过系统梳理国内罕见病用药保障政策及实践,认为当前中国多层次多主体的罕见病用药保障机制雏形已开始显现。然而,在职工医疗保险制度的财务压力和待遇清单制度的双重约束下,多项地方罕见病超待遇保障项目面临清理,罕见病用药保障受到冲击,急需在国家层面重组资源和重建替代性机制。基于此,笔者提出设立“中华罕见病种子基金”(以下简称“种子基金”)的构想,详细阐释其筹资原理,指出种子基金初始资本的本质是“制度套利”的红利,可被视为中国医疗保障制度第一层次的延伸,是多方共付的杠杆和平台。根据当前职工医疗保险基金运行和罕见病药品保障现状,测算出种子基金规模。结果表明,种子基金不仅能覆盖当前登记患者的全部年用药费用67亿元,而且仍有结余。笔者认为,种子基金提供的罕见病用药保障能力可完全满足罕见病患者的未来需要,且随着资金规模的扩大,种子基金的社会功能可逐渐扩展,成为践行共同富裕道路上的标杆性机制。

Among the many dilemmas faced by patients with rare diseases,the drug payment dilemma is always the most difficult and unavoidable.By reviewing domestic policies and practices on drug guarantee for rare diseases,this paper concludes that a multi⁃level mechanism involving multiple subjects is beginning to emerge in China.However,under the medical insurance system for employees and with the constraints of the treatment list,the medical system of safeguarding basic diseases will not be able to respond to the needs of groups with rare diseases.Many local⁃level super⁃treatment protection programs for rare diseases are on the brink of collapse.An innovation approach,or alternative mechanism,must be developed to ensure that the drug guarantee for rare diseases is not reduced,and social harmony and stability maintained.Efforts should be made to stick to the institutional bottom line of the medical guarantee treatment,bring no additional financial burdens at all levels,and fully exploit the potential of social resources and reorganize them nationwide.This paper proposes‘the conception of a seed fund’and elaborates on the funding rationale,nature of funds,and institutional functions.How does the seed fund work then?Taking advantage of the window period for the reform of the investment system of the basic medical insurance fund for urban employees,a portion of the fund is‘borrowed’and entrusted to the National Council for Social Security Fund for investment each year before the market investment.After‘returning’the‘principal’and interest to the provincial⁃level regions,the investment income is regarded as the initial capital of the seed fund,and a portion of the investment income is used annually for drug guarantee for rare diseases.The seed fund,essentially the result of the institutional arbitrage dividend and market investment operation,can be considered as a first⁃tier extension of China's medical insurance system in terms of positioning,and as a lever and platform for multi⁃payments for rare diseases in terms of function.Based on the current operation of the medical insurance fund for employees and the status of rare disease drug coverage,the scale of the seed fund and the cost of rare disease drugs eligible for inclusion in the seed fund are measured.Results show that after a five⁃year funding during the reform window,the seed fund can obtain about CNY 234.7 billion of initial capital,yielding CNY 14.1 billion of annual income;a balance remains after covering the full CNY 6.7 billion cost of drugs for patients currently enrolled in rare disease registries.Seed fund is an effective approach to ensure the sustainability of the medical insurance fund during the policy transition period and represents the CPC's solemn promise to‘common prosperity for all’.