Edward Bein
Title: The psychometrics of clinical trial endpoints derived from聽patient-reported outcome measures: Causal inference validity.
AbstractThe 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聽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. NOTE: Social gathering (Open area outside Rm1140) from 3-3:30pm |