FDA等机构药物有效性研究要求沿革及进展

Evolution and progress of requirements for drug efficacy investigation of FDA and other regulatory agencies

本文回顾了FDA等发达国家药品监管机构药物临床有效性评价的相关法规和技术要求的逐渐完善和成熟的发展历史,概括总结了经过半个多世纪的药物有效性研究的科学探索,逐步形成的以前瞻性、多中心、随机、盲法、对照试验设计为主的现行较为公认的药物临床有效性评价方法的基本技术要求,包括研究目的、研究计划与对照、纳入人群、随机和盲法等控制偏移的因素,主要疗效指标的选择与分析等;文章还对近几年新颁布的旨在提高药物有效性评价的可靠性、准确性、效率和降低成本等,FDA陆续出台的药物临床有效性评价的试验设计方法、临床疗效终点的设计与制定、药物疗效的数据使用、统计学分析和总结等新指导原则进行了较为全面的梳理和总结。

The paper reviews the history of the formation and improvement of the relevant regulations and guidance for drug efficacy investigation by the drug regulatory agencies in developed countries such as FDA,and summarizes the basic technical requirements of the current widely accepted method,i. e.,prospective,multicenter, random and blind clinical trial,which has been gradually formed after more than half a century of scientific exploration,it including study objective,plans and controls,factors controlling bias,such as randomization and blindness,subject inclusion criteria, selection and analysis of primary endpoints. In order to improve the reliability, accuracy,cost and time efficiency of drug efficacy evaluation,new guidelines have been issued by FDA and other regulatory agencies,including clinical trials design methods,design and development of clinical efficacy endpoints, statistical analysis and summary of drug efficacy data. The paper also outlines the progress of such new guidelines for drug clinical efficacy investigation.