Chapter 13 - Incorporating Patient-Reported Outcomes Into Early-Phase Trials

Abstract

Early-phase cancer trials are designed to evaluate the safety, tolerability, and preliminary efficacy signals of a new therapeutic agent or combination. In a phase 1 trial the maximal tolerated dose is determined based on clinician assessment of toxicity during the first cycle of treatment. However, this may not always reflect the patient’s experience of tolerability over time. With more anticancer agents receiving accelerated approval based on early-phase trial data, and an expanding array of orally administered agents, the need to incorporate the patient’s perspective in early-phase trials has become more essential. The value of patient-reported outcomes (PROs) in later-phase trials can be extended and modified for the inclusion of PROs in the early drug development. This chapter traces the history of PRO inclusion in clinical trials, highlights selected PRO assessment tools, and outlines the important study design issues to consider when measuring, analyzing, and reporting PROs in early-phase clinical trials.