The PCORI Methodology Report* provides minimal requirements to follow in the conduct of scientifically valid patient-centered outcomes research. The report addresses two legislative mandates: the development of methodology standards and development of a translation framework to guide the choice of study designs for specific research questions. It details the context and rationale for the PCORI Methodology Standards, published in late 2012, and most recently updated in February 2019. It includes stories and examples that illustrate different ways that good methodology makes a difference to patients and their care.

The PCORI Methodology Report includes an Executive Summary, an Introduction, and four sections:

• Section I: Patient-Centered Outcomes Research reviews the need for methodological standards in patient-centered outcomes research. The standards guide researchers to adopt approaches most likely to produce the high-quality and relevant evidence needed to inform health decisions.

• Section II: Identifying and Addressing Evidence Gaps in PCOR discusses the importance of a systematic process for prioritizing topics and determining which research designs can provide information that is both useful and timely. These activities align a program of patient-centered outcomes research with questions about what works best, for whom, and under what circumstances.

• Section III: PCORI Methodology Standards explains how PCORI developed and updated its set of 54 methodology standards, discusses each group of standards, and summarizes the justifications for the standards. These standards can improve the way research questions are selected and formulated, how studies are designed and analyzed to address these questions, and how findings are reported.

• Section IV: Advancing Understanding and Appropriate Use of Methods for PCOR emphasizes the role of the Methodology Standards in promoting transparency, which is a critical component of good research practices. Transparency enables other researchers to assess the reproducibility and validity of findings. This section also outlines the next steps for PCORI’s continued development of standards to support the design, conduct, analysis, and reporting of high-quality patient-centered outcomes research.

View the PCORI Methodology Report

• View the 2017 version of the Methodology Report and Methodology Standards
• View the 2013 version of the Methodology Report and Methodology Standards
• Read what key leaders say about the report and standards

Draft report and background materials:

• Draft Methodology Report (Posted July 23, 2012)
• Public comments on the Draft Methodology Report (Received July 23-September 14, 2012)
• Overview of the Methodology Committee’s Response to Public Comment
• Proposed revisions to the Methodology Standards by the Methodology Committee (Published November 19, 2012)
• PCORI Methodology Committee Research Recommendations (As presented to the Board November 19, 2012, and published December 14, 2012)

PCORI’s First Methodology Report

Reports to Support the Development of the First Report

All statements in these reports, including their findings and conclusions, are solely those of the authors and do not necessarily represent the views of PCORI, its Board of Governors, or Methodology Committee. PCORI has not peer-reviewed or edited this content. The report is being made available free of charge for the information of the scientific community and general public as part of PCORI’s ongoing research programs. Questions or comments about this report may be sent to PCORI at [email protected].

Review of Guidance Documents for Selected Methods in Patient-Centered Outcomes Research

• Enhancing the Patient's Voice: Standards in the Design and Selection of Patient-Reported Outcomes Measures for Use in Patient-Centered Outcomes Research
  
• Minimal Standards in the Prevention and Handling of Missing Data in Observational and Experimental Patient-Centered Outcomes Research
• Standards for the Design, Conduct, and Evaluation of Adaptive Randomized Clinical Trials
• Standards for Causal Inference Methods in Analyses of Data from Observational and Experimental Studies in Patient-Centered Outcomes Research
• Standards in Addressing Heterogeneity of Treatment Effectiveness in Observational and Experimental Patient Centered Outcomes Research
• Standards in the Design, Conduct, and Evaluation of Diagnostic Testing for Use in Patient Centered Outcomes Research
• Standards in the Conduct of Registry Studies for Patient-Centered Outcomes Research
• Standards in the Use of Collaborative or Distributed Data Networks in Patient Centered Outcomes Research
• The Design and Selection of Patient-Reported Outcomes Measures for Use in Patient-Centered Outcomes Research

Review and Synthesis of Evidence for Eliciting the Patient's Perspective in Patient-Centered Outcomes Research

• Integrating Patients’ Voices in Study Design Elements with a Focus on Hard-to-Reach Populations

Expert Stakeholder Interviews to Identify Evidence for Eliciting the Patient’s Perspective in Patient-Centered Outcomes Research

• Eliciting Patient Perspective in Patient-Centered Outcomes Research - A Meta-Narrative Systematic Review
• Eliciting Patient Perspective in Patient-Centered Outcomes Research - Expert Interviews Part 1
• Eliciting Patient Perspective in Patient-Centered Outcomes Research - Expert Interviews Part 2
• Eliciting Patient Perspective in Patient-Centered Outcomes Research - Expert Interviews Part 3
• Eliciting Patient Perspective in Patient-Centered Outcomes Research - Expert Interviews Part 4

Methods for Setting Priorities in Research

• Methods for Involving Patients in Topic Generation for Patient-Centered Comparative Effectiveness Research – An International Perspective
• Value of Information and Research Prioritization
• Value-of-Information Analysis for Patient-Centered Outcomes Research Prioritization
• Peer Review - A Research Priority
• Prioritizing Future Research through Examination of Research Gaps in Systematic Reviews

*The PCORI Methodology Standards were updated February 26, 2019, and are available here.
An updated Methodology Report will be posted in later in 2019.

Posted: April 19, 2013; Updated: February 26, 2019