The psychometrics of clinical trial endpoints derived from patient-reported outcome measures: Causal inference validity
Edward Bein, PhD
Senior Statistician
Where: In-person | 2001 McGill College Avenue, room 1140;
Note: Social gathering (Open area outside Rm1140) from 3-3:30pm
Abstract
The US Food and Drug Administration increasingly emphasizes the importance of assessing how patients 鈥渇eel, function, and survive鈥� when evaluating new treatments in clinical trials, often via the use of endpoints derived from patient-reported outcome measures (PROs). This presentation examines possible pitfalls in using PRO-derived endpoints to estimate and test hypotheses about treatment effects. It discusses the notion of whether endpoints possess 鈥渃ausal inference validity,鈥� and presents inferential remedies when such validity is not achieved. The pitfalls and remedies are exemplified in a simulation study using the Rasch item response theory measurement model to analyze a PRO.
Speaker Bio
Ed Bein is on sabbatical from the US Food and Drug Administration, where he is a senior statistician at the Center for Drug Evaluation and Research. He has reviewed randomized trials evaluating treatments for infectious diseases, including COVID-19 and observational studies of the long-term safety of recently approved drugs. He has a Ph.D. in biostatistics from UC Berkeley and was a clinical psychologist in his previous career.