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.
*School of Physical and Occupational Therapy, McGill University, Clinical Epidemiology, McGill University Health Center, Centre de recherche interdisciplinaire en réadaptation, Montreal, QC
†National Institute on Minority Health and Health Disparities, National Institutes of Health, Bethesda, MD
‡Health Outcomes Research, United BioSource Corporation, Bethesda, MD
§Center for Health Outcomes Research, United BioSource Corporation, Lexington, MA
∥Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA
¶Memorial Sloan-Kettering Cancer Center, New York, NY
#Lineberger Comprehensive Cancer Center & Department of Health Policy and Management, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC
**John Hopkins Bloomberg School of Public Health, Baltimore, MD
The tools and mechanisms described in this paper are not being endorsed by the ISOQOL. The instruments and projects presented are examples and are not an exhaustive list.
S. Ahmed is supported by a Fonds de la Recherche en Santé du Quebec (FRSQ) research career award.
The authors declare no conflict of interest.
Reprints: Sara Ahmed, PhD, Faculty of Medicine, McGill University, 3654 Prom Sir-William-Osler, Montreal, QC, Canada H3G 1Y5. E-mail: firstname.lastname@example.org.