STATISTICS COLLOQUIUM
Shing M. Lee
Associate Professor
Department of Biostatistics
Columbia University
Incorporating patient-reported outcomes in dose-finding clinical trials
Abstract
Oncology dose-finding clinical trials have traditionally determined the maximum tolerated dose based on toxicity outcomes captured by clinicians. With the availability of more rigorous instruments for measuring toxicity directly from patients, there is a growing interest to incorporate patient-reported outcomes (PRO) in clinical trials to inform patient tolerability. In this talk, I will discuss the methodological aspects of dose-finding clinical trials and the key issues regarding the use of PROs in the setting of dose-finding clinical trials. I will present our proposed three extensions of the continual reassessment method (CRM), termed PRO-CRMs, that incorporate both clinician and patient outcomes. The first method is a marginal modeling approach whereby clinician and patient toxicity outcomes are modeled separately. The other two methods impose a constraint using a joint outcome defined based on both clinician and patient toxicities and model them either jointly or marginally.
For more information, contact: Tracy Burke at tracy.burke@uconn.edu