利益相关者视角下罕见病药品供应困境及原因分析

Analysis on the Supply Dilemma and Causes of Rare Diseases Drugs from the Perspective of Stakeholders

本研究选取部分典型的罕见病药品,梳理当前罕见病药品存在的供应不稳定、“最后一公里”不通畅等困境,基于利益相关者视角,分析导致罕见病出现供应困境的具体原因,提出明确罕见病界定标准、及时更新罕见病目录、定期召开药事管理会、将罕见病药品配备使用情况纳入公立医院绩效考核、建立医院罕见病用药管理制度、协同利用罕见病患者组织力量、加强企业社会责任建设等解决对策,以期改善罕见病药品供应困境。

In this study,some typical rare disease drugs were selected to sort out the current supply difficulties of rare disease drugs,such as unstable supply and the"last mile"obstruction.Based on the stakeholders'perspective,the specific reasons for the supply difficulties of rare diseases were analyzed,and the definition standards of rare diseases were put forward,the catalogue of rare diseases was updated in time,pharmaceutical affairs management meetings were held regularly,the equipment and use of rare disease drugs were included in the performance evaluation of public hospitals,the management system of rare disease drugs in hospitals was established,the organizational strength of patients with rare diseases was utilized in coordination,and corporate social responsibility was strengthened.