药物临床价值评估的主要考虑因素及问题

Key considerations and issues of assessment for clinical value of drugs

药物研发和评价中的临床价值越来越受到重视。为了提升对药物研发和临床试验中药物临床价值的全面认识,本文从多角度,特别是从药物研发临床评价方面对影响药物临床价值评估的主要因素及问题进行了梳理、总结和分析。包括药物适用的疾病特点、疾病的治疗现状、药物的研发目的和临床定位,药物的临床试验目的和主要疗效指标,影响药物临床价值或临床价值发挥的其他因素等;同时,本文对药物临床价值评估需要关注的几个问题也进行了分析。包括不能仅看单一因素,需要多个因素综合考虑,药物临床价值的有无和临床价值的大小需要充分的证据支持,而不是根据其声称的药物研究目的和临床定位,药物临床价值与其临床试验中主要疗效指标的关系等。这些都是药物研发和上市注册临床试验和评价中需要充分考虑的问题。

The clinical value of drugs,R D and evaluation has been paid more and more attention. This paper summarizes and analyzes the main factors and problems which may influence the evaluation of drugs,clinical values in order to enhance the understanding in R D and evaluation from the clinical reviewers,view point. It mainly includes the characteristics of the target diseases of drugs,treatment status and current medical development of the target disease,therapeutic aim of drug,objective of the clinical trial and the primary endpoints in the pivotal study and other factors which may influence the clinical values. This paper also discusses the main points in the clinical value evaluation,the following points should be fully considered during the RD and evaluating of the clinical values for NDA： multiple factors should be considered,not only focus on single factor; clinical value should be supported by data and evidence,not just by announcing the therapeutic aim of drug or the objective of the clinical trial by the registered applicants,and also the relationship between clinical value and the primary endpoints in the pivotal study should be fully concerned.