国外罕见病治疗保障体系对我国的伦理启示

Ethical Enlightenment of Foreign Rare Disease Treatment Guarantee System to China

通过总结国外在罕见病研究和药物研发方面的政策法规,参考国外罕见病发展中的治疗保障体系相关政策支持,结合中国国情,分析中国可借鉴的可行之处,进而对当下罕见病研究中存在的伦理问题提出一些建议:基于公平原则调整地域间差异,建立全国的罕见病信息收集中心;提高药企科研伦理素养,加强伦理监督;政府和社会履行义务以保障罕见病患者权益。以期为中国罕见病治疗保障的相关政策制定提供参考。

By summarizing the policies and regulations of foreign countries on rare disease research and drug research and development,referring to the relevant policy support of treatment guarantee system in the development of rare diseases abroad,and combining with China s national conditions,this paper analyzed the feasible points that China can learn from,and then put forward some suggestions on the ethical problems existing in the current rare disease research,so as to provide reference suggestions for the relevant policy formulation of rare disease treatment guarantee in China:adjusting regional differences based on the principle of fairness,and establishing a national rare disease information collection center;improving the ethical quality of scientific research in pharmaceutical companies,and strengthening ethical supervision;and the government and society fulfill their obligations to protect the rights and interests of patients with rare diseases.