拓展性同情使用临床试验用药制度的相关问题研究

Issues Related to Expanded Access to Investigational Drugs for Compassionate Use

目的研究国外部分国家拓展性同情使用临床试验用药制度及申请批准情况,为我国拓展性临床试验用药制度设计提供参考。方法通过查阅美国、欧盟药品监管机构发布的法规政策、技术指南、年度报告及相关文献,深入分析欧美拓展性临床试验用药的相关概念、类别、申请要求、风险获益评估等内容。结果和结论拓展性同情使用临床试验用药是在不能通过已上市药物有效治疗严重疾病或危及生命疾病时,获得研究药物的潜在方式。美欧等地区对拓展性研究药物制定了较为完善的法规体系,因为临床试验用药并未上市,安全性和有效性证据收集不足,使用时更要保护使用者的安全,同时伦理委员会应当严格履行职责,保证患者知情同意,并对治疗方案严格审查。

Objective To study the laws and regulations and application approval by some foreign drug regulatory agencies concerning expanded access to investigational drugs for treatment use so as to provide reference for the design of related guidelines in China.Methods By referring to the laws and regulations,technical guidelines,annual reports and relevant literature issued by drug regulatory agencies of the United States and the European Union,the relevant concepts,categories,application requirements,risk and benefit assessment of drugs used in overseas extended clinical trials were analyzed in depth.Results and Conclusion Compassionate use of clinical trial drugs is a potential way in which patients obtain research drugs when they are suffering from serious diseases or life-threatening diseases that cannot be effectively treated with marketed drugs.As research drugs are not approved into the market,evidence about their safety and efficacy is lacking.In order to ensure the safety of subjects,ethical committees need to perform their duties by conducting strict examination of treatments.