Consensus of recommendations guiding comparative effectiveness research methods.

Jacob B Morton,Robert Mcconeghy,Nicolle M Gatto,Kirstin Heinrich,Aisling R Caffrey

doi:10.1002/pds.4051

Abstract

Because of an increasing demand for quality comparative effectiveness research (CER), methods guidance documents have been published, such as those from the Agency for Healthcare Research and Quality (AHRQ) and the Patient-Centered Outcomes Research Institute (PCORI). Our objective was to identify CER methods guidance documents and compare them to produce a summary of important recommendations which could serve as a consensus of CER method recommendations. We conducted a systematic literature review to identify CER methods guidance documents published through 2014. Identified documents were analyzed for methods guidance recommendations. Individual recommendations were categorized to determine the degree of overlap. We identified nine methods guidance documents, which contained a total of 312 recommendations, 97% of which were present in two or more documents. All nine documents recommended transparency and adaptation for relevant stakeholders in the interpretation and dissemination of results. Other frequently shared CER methods recommendations included: study design and operational definitions should be developed a priori and allow for replication (n = 8 documents); focus on areas with gaps in current clinical knowledge that are relevant to decision-makers (n = 7); validity of measures, instruments, and data should be assessed and discussed (n = 7); outcomes, including benefits and harms, should be clinically meaningful, and objectively measured (n = 7). Assessment for and strategies to minimize bias (n = 6 documents), confounding (n = 6), and heterogeneity (n = 4) were also commonly shared recommendations between documents. We offer a field-consensus guide based on nine CER methods guidance documents that will aid researchers in designing CER studies and applying CER methods. Copyright © 2016 John Wiley & Sons, Ltd.

Highlights

As a result of an ever-increasing number of treatment options, real-world evidence is needed to inform clinical decision-making
While comparative effectiveness research (CER) is a newer term to describe an existing discipline that has carried various names, the focus of our search for methods guidelines was specific to this term that was popularized by the American Reinvestment and Recovery Act
The Patient-Centered Outcomes Research Institute (PCORI), Agency for Healthcare Research and Quality (AHRQ), and European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) present detailed methodological guidance, organized by topic area, which can serve as a how-to guide for researchers attempting to design and conduct CER.[4, 5, 10]

Summary

Introduction

As a result of an ever-increasing number of treatment options, real-world evidence is needed to inform clinical decision-making. In response to a number of recent CER funding initiatives based in the United States, under the 2009 American Recovery and Reinvestment Act and the 2010 Patient Protection and Affordable Care Act, which established the Patient-Centered Outcomes Research Institute (PCORI), several methods guidance documents have been developed recently.[2,3,4,5] Despite the utility of such guides, it remains unclear which documents should be followed and under which circumstances, as consensus between the guidance documents has not been determined To this end, this review sought to identify CER methods guidance documents, and identify areas of agreement among CER methods recommendations to create a consensus document that may assist in the design and conduct of high-quality CER, including observational studies and randomized clinical trials (RCTs)

Objectives

Methods

Results

Discussion

Conclusion