Beitz J, Gnecco C, Justice R
Division of Oncology and Pulmonary Drug Products, U.S. Food and Drug Administration, Rockville, MD 20857, USA.
J Natl Cancer Inst Monogr. 1996(20):7-9.
Increasingly, quality-of-life (QOL) end points are being incorporated into randomized, controlled clinical trials in oncology. The Oncologic Drugs Advisory Committee (U.S. Food and Drug Administration) has recommended that beneficial effects on QOL and/or survival be the basis for approval of new anticancer drugs. Therefore, from a regulatory standpoint, for drugs that do not have an impact on survival, demonstration of a favorable effect on QOL is more important than most other traditional measures used to assess efficacy, such as objective tumor response. Trials incorporating QOL questions will be evaluated on the basis of how well they address the stated objectives. The clinical protocol should delineate investigators' hypotheses and choice of validated instruments and should specify a detailed statistical analysis plan describing strategies for handling missing data. The U.S. Food and Drug Administration welcomes the opportunity to explore with investigators the use of QOL instruments in the design of cancer clinical trials.
生活质量（QOL）终点越来越多地被纳入肿瘤学的随机对照临床试验中。肿瘤药物咨询委员会（美国食品药品监督管理局）建议，对生活质量和/或生存的有益影响应作为批准新抗癌药物的依据。因此，从监管角度来看，对于那些对生存没有影响的药物，证明其对生活质量有积极影响比用于评估疗效的大多数其他传统指标（如客观肿瘤反应）更为重要。纳入生活质量问题的试验将根据其实现既定目标的程度进行评估。临床方案应阐明研究者的假设和对经过验证的工具的选择，并应指定详细的统计分析计划，描述处理缺失数据的策略。美国食品药品监督管理局欢迎有机会与研究者探讨在癌症临床试验设计中使用生活质量工具的问题。
