Patient-reported outcome measurement in drug discovery: a tool to improve accuracy and completeness of efficacy and safety data.

INTRODUCTION: Patient-reported outcomes (PROs) reflect how patients feel and function as conveyed directly by patients themselves, for example symptoms and physical functioning. PRO measures can be included in any phase of product development as primary, secondary, or exploratory endpoints to understand the impact of treatment on the patient experience. AREAS COVERED: In this review, the authors describe approaches commonly used to assess PROs in drug development programs, including how to select or develop outcomes and measures, implement these in trials, and analyze data. Barriers and facilitators for effectively including PROs in clinical trials are discussed, and strategies for making labeling claims based on PRO data are noted. EXPERT OPINION: Early planning with PRO experts is recommended to assure a successful PRO strategy in a product development program. Outcomes that are meaningful to patients should be rationally identified early in a product development program based on qualitative work, literature search and/or assumed mechanism of action. Measures corresponding to those outcomes should be selected or developed, with demonstration of robust psychometric properties in a related patient population. Ideally, these measures will be tested prior to a pivotal trial to support the design and analysis in the pivotal trial. Selection of timing and mode of assessments should be specified a priori and justified. If a labeling claim is sought, a statistical plan should be pre-specified. Strategies to minimize and handle missing data in analyses should be planned up front.