Journal
CANCER INVESTIGATION
Volume 41, Issue 5, Pages 491-494Publisher
TAYLOR & FRANCIS INC
DOI: 10.1080/07357907.2023.2206497
Keywords
Patient-reported outcomes; quality of life; clinical trials; single arm trials; methodology
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Recently, many cancer drugs have been approved based on single-arm studies without direct comparison with standard, leading to potential bias in the analysis of quality of life and patient-reported outcomes. Caution is needed to avoid overrating methodologically weak evidence and to involve PROs experts in planning and interpreting analyses.
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.
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