Use and Interpretation of Pros in Early Phase Cancer Clinical Trials

• Activity Number: 147
• Type: Invited
• Date/Time: Monday, August 4, 2014 : 10:30 AM to 12:20 PM
• Sponsor: Health Policy Statistics Section
• Author(s): Amylou Dueck*+ and Pamela Atherton and Rui Qin and Jeff Sloan
• Companies: Mayo Clinic and Mayo Clinic and Mayo Clinic and Mayo Clinic
• Keywords: Patient-reported outcome ; Phase I ; Phase II ; Clinical trial ; Clinical significance ; Endpoint

Abstract

Use of patient-reported outcomes (PROs) in randomized phase III cancer clinical trials has become common practice with much published work offering solutions to the analytical challenges of multiple testing, missing data, and clinical significance. Is there a place for PROs in early phase, often non-randomized and/or uncontrolled cancer clinical trials? PROs in early phase clinical trials can provide preliminary estimates of patient benefit or identify areas for concern either to maximize drug delivery and/or compliance in later phase clinical trials or to spawn subsequent symptom intervention studies. Early phase clinical trials can also be a testing ground for endpoint refinement or establishing feasibility, acceptability, reliability, or validity of a PRO in the population of interest prior to launching a phase III clinical trial. In this session, I will present various approaches for analytically and graphically presenting and interpreting results of PROs in early phase clinical trials with examples including the use of PRO-CTCAE in a phase I and a randomized phase II selection clinical trial.