The Use of Patient-reported Outcomes (PRO) Within Comparative Effectiveness Research

TLDR

Patient‑reported outcomes are essential for capturing results that matter to patients and for informing decision making, and CER uses such data to provide clinicians, patients, and families with evidence to guide care choices. This paper aims to evaluate the role of patient‑reported outcomes in comparative effectiveness research, outlining suitable PRO measures, selection strategies, challenges, and recommendations for researchers, clinicians, and policymakers. The authors review how to select appropriate PRO instruments amid a growing array of questionnaires, address key challenges, and highlight emerging facilitators such as electronic health records, registries, and national monitoring initiatives. The study finds that linking PRO data to CER enhances decision making and offers recommendations to help researchers, clinicians, and policymakers apply PROs effectively in CER contexts.

Abstract

The goal of comparative effectiveness research (CER) is to explain the differential benefits and harms of alternate methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. To inform decision making, information from the patient's perspective that reflects outcomes that patients care about are needed and can be collected rigorously using appropriate patient-reported outcomes (PRO). It can be challenging to select the most appropriate PRO measure given the proliferation of such questionnaires over the past 20 years.In this paper, we discuss the value of PROs within CER, types of measures that are likely to be useful in the CER context, PRO instrument selection, and key challenges associated with using PROs in CER.We delineate important considerations for defining the CER context, selecting the appropriate measures, and for the analysis and interpretation of PRO data. Emerging changes that may facilitate CER using PROs as an outcome are also reviewed including implementation of electronic and personal health records, hospital and population-based registries, and the use of PROs in national monitoring initiatives. The potential benefits of linking the information derived from PRO endpoints in CER to decision making is also reviewed.The recommendations presented for incorporating PROs in CER are intended to provide a guide to researchers, clinicians, and policy makers to ensure that information derived from PROs is applicable and interpretable for a given CER context. In turn, CER will provide information that is necessary for clinicians, patients, and families to make informed care decisions.