Quality of Life: Patient Reported Outcomes: Purpose, Types, Development, and Evaluation Part 1 (May 2, 2022 via “NIH VideoCast”): 30-min presentation.

• Summary: Dr. Kevin Weinfurt emphasizes that for endpoints and outcomes to meaningfully inform decisions, they must matter to patients, clinicians, and health systems. Patient-reported outcomes (PROs), which are becoming more commonly measured in pragmatic trials, are defined as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else” (FDA Guidance for Industry, 2009). The differing types of PROs are briefly overviewed, and the development and evaluation of PROs are discussed as well.

Rockhold FW, et al. Design and analytic considerations for using patient-reported health data in pragmatic clinical trials: report from an NIH Collaboratory roundtable. J Am Med Inform Assoc. 2020 Apr 1;27(4):634-638. (5-page paper)

• Summary: Pragmatic trials may leverage routinely collected health data to introduce pragmatism into the design, implementation, or management of the study, but quality issues associated with these data can limit their usefulness for research, particularly when defining study outcomes/endpoints. Patient-reported health data, including PROs, can be used to confirm and/or supplement routinely collected data, but patient-reported data can bring their own challenges. Therefore, to provide guidance for the appropriate use of patient-reported data in pragmatic trials, the strengths and limitations of these data are itemized, and criteria for determining when such data are appropriate (to fill gaps in the evidence collected from routinely collected data) are described.

NIH Pragmatic Trials Collaboratory (Living Textbook): Section 5 - Choosing PRO Measures (Chapter: Data, Tools, & Conduct - Patient-Reported Outcomes): 1-page website.

• Summary: Highlights questions trialists should ask themselves when considering PROs in pragmatic trials: (1) Will the measures from core outcome sets or other widely available measures be acceptable for my research question? (2) Are the PRO data already being collected? If so, how and where are the data collected? Are the data available (already linked) to the routinely collected health data (e.g., electronic health records, disease registries, administrative health data) or through other mechanisms? (3) Will the collection of the PRO be acceptable and not burdensome to my patient population?

Patient-Reported Outcomes in Clinical Trials (October 10, 2023 via “JAMA Network”): 14-min interview.

• Summary: A conversation between Drs. Ethan Basch and Melanie Calvert defines PROs, overviews the increasing use of PROs in clinical trials, discusses what can be done to optimize their use, and summarizes the strengths and limitations of these measures. A focus on the ethical considerations related to the collection of PROs is the focus of an associated JAMA publication (Rivera SC, et al. 2022).

Zigler CK, et al. Collecting patient-reported outcome measures in the electronic health record: Lessons from the NIH pragmatic trials Collaboratory. Contemp Clin Trials. 2024 Feb;137:107426. (7-page paper)

• Summary: The NIH Pragmatic Trials Collaboratory supports the design and conduct of several pragmatic trials, and many include PROs as primary/secondary outcomes. This article overviews challenges trial teams have encountered related to PROs, e.g., challenges related to competing health system priorities, clinician’s buy-in for adoption (especially in low resource settings), and lack of standardization of PROs in the electronic health record.

NIH Pragmatic Trials Collaboratory (Living Textbook): Section 2 - How Are PRO Measures Used? (Chapter: Data, Tools, & Conduct - Patient-Reported Outcomes): 1-page website.

• Summary: Describes the different roles of PROs in clinical research (including pragmatic trials), clinical care, and quality assurance. i.e., Within pragmatic trials, PROs may be used: (1) As measures of study endpoints/outcomes, (2) As monitoring tools for adverse events and symptoms, and/or (3) To define the intervention (e.g., more aptly described as patient-reported health data). A table is provided describing the use of PROs across 8 pragmatic trials within the NIH Collaboratory.

Calvert M, et al. The CONSORT Patient-Reported Outcome (PRO) extension: implications for clinical trials and practice. Health Qual Life Outcomes. 2013 Oct 29;11:184. (7-page paper)

• Summary: The CONSORT statement aims to encourage transparent and complete reporting of clinical trials and is associated with improved reporting of trials. Further, the number of trials that assess PROs has substantially increased over time. Therefore, the CONSORT PRO extension aims to improve the reporting of PROs in trials to bolster the impact of trial results to inform practice and policy.

NIH Pragmatic Trials Collaboratory - Patient-Centered Outcomes Core Toolkit (September 12, 2023): 5-page toolkit.

• Summary: A toolkit to enhance the validity of patient-reported data and to improve inclusion, diversity, equity, and accessibility (IDEA) in pragmatic trials research. Includes links to a variety of existing translated and/or culturally adapted PROs, relevant peer-reviewed literature focused on PRO collection in pragmatic trials, and other toolkits to support equity in the collection of PROs.

NIH Pragmatic Trials Collaboratory - Patient-Reported Outcomes (January 2014): 17-page document.

• Summary: Introduces the definition, measurement, and use of PROs in pragmatic trials. It is argued that the successful incorporation of patient-reported data collection in the clinical process requires clinician (/provider) engagement. Further, it is emphasized that the measurement process for patient-reported outcomes must not create undue burdens on patients, clinicians, or the health system.