罕用药临床试验及受试者用药保障分析

Analysis of clinical trial of orphan drugs and participants medication guarantee

罕见病患者数量少、临床试验成本高、支持性资源不足等诸多因素导致罕用药临床试验开展面临阻碍,患者无法得到有效治疗。本文通过分析罕用药临床试验的现状,梳理罕用药临床试验后受试者药物治疗保障的相关国际渊源,在此基础上对罕用药临床试验及受试者用药保障问题进行伦理审视并提出对策建议,为国家相关部门的决策提供理论参考,以促进我国罕见病医疗保障制度的实施。

A small number of rare patients,high cost of clinical trials,lack of supportive resources and other causes make orphans clinical trials to face a lot of obstacles,thus patients cannot get effective treatment. This paper analyzes the present situation of orphan drug clinical trials and the relevant international origin of the drug therapy of the participants after orphan drug clinical trials. On this basis,this paper makes the ethical examination of the orphan drug clinical trials and the medication guarantee of participants,then puts forward the suggestions.This paper aims to provide theoretical reference for the relevant departments of the country to make decision and promote the implementation of our rare disease health care system.