The Value of Patient-Reported Outcomes in Single-Arm Cancer Trials

被引：0
|
作者：
Di Maio, Massimo [1 ,2 ,3 ]
机构：
[1] Univ Turin, Ordine Mauriziano Hosp, Dept Oncol, Turin, Italy
[2] Univ Turin, Dept Oncol, Via Magellano 1, I-10128 Turin, Italy
[3] Ordine Mauriziano Hosp, Div Med Oncol, Via Magellano 1, I-10128 Turin, Italy
来源：
CANCER INVESTIGATION | 2023年 / 41卷 / 05期
关键词：
Patient-reported outcomes; quality of life; clinical trials; single arm trials; methodology;
D O I：
10.1080/07357907.2023.2206497
中图分类号：
R73 [肿瘤学];
学科分类号：
100214 ;
摘要：
Recently, many cancer drugs have been approved by regulatory agencies based on single-arm studies, where analysis of quality of life (QoL) and patient-reported outcomes (PROs) is not a direct comparison with the standard, but the simple description of changes versus baseline and trends over time. This analysis can be potentially biased by missing data: QoL level could be overestimated, and symptoms underestimated. Caution is needed to avoid that a methodologically weak evidence in PROs and QoL data is overrated, if not for the drug approval, for the interpretation of data by scientific community and the communication with patients. PROs experts should be involved in planning, conduction, and interpretation of analyses.
引用
收藏
页码：491 / 494
页数：4
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