Article Abstract

Challenges of interpreting patient reported outcomes from clinical trials

Authors: Diane L. Fairclough


As Bell et al. (1) indicated in their paper entitled “Statistical Controversies in Cancer Research: Using standardized effect size graphs to enhance interpretability of cancer-related clinical trials with patient reported outcomes,” Patient Reported Outcomes (PROs) are frequently being gathered in clinical trials. The primary challenge is that these outcomes are multidimensional; rarely is there a single outcome but rather a number of symptoms measures or components of quality of life or health status. While there are formal statistical procedures to insure that the conclusions from a trial about any single outcome is valid (not a false positive), it does not address the clinically relevant questions as to whether the intervention is generally beneficial across the multiple outcomes.