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Eleanor M. Perfetto, PhD, MS

VACA20 2nd place – Eleanor Perfetto
Research Grant in Value Assessment and Health Outcomes Research, 2019 Challenge Award in Value Assessment and Health Outcomes Research, 2020 National Health Council

Methods Principles for Using Patient-Provided Information to Improve Real-World Evidence for Patient-Centered Value Assessment


Despite growing availability of real-world data (RWD) sources, concerns over bias, confounding, and lack of universally accepted methodological standards inhibits uptake of real-world evidence (RWE) by value assessment bodies (VAs). To improve the quality and patient-centricity of RWE, the Joint International Society for Pharmacoeconomics and Outcomes Research (ISPOR)-International Society for Pharmacoepidemiology (ISPE) Special Task Force on RWE in Health Care Decision Making included patient/stakeholder engagement as one recommended good-procedural practice when designing, conducting, and disseminating RWE. The objective of this project was to develop research-methods, good-practice recommendations on how and at which RWD study-design stage, patient-provided information (PPI) (e.g., experiences, preferences, perspectives, desired outcomes, etc.) should be used by researchers. Specifically, the project sought to identify how to use PPI to inform methods decisions (e.g., covariate identification, meaningful outcomes, etc.) to improve the rigor of RWE for use in patient-centered VA. A multi-disciplinary Advisory Board was established to guide the development and dissemination of recommendations. RWE-researchers (n=15) were presented with case studies (hypothetical research question and PPI) and asked to identify how they could apply PPI to study-design decisions using an RWE research-design framework. Interview responses were analyzed and translated into draft recommendations. A multistakeholder e-Delphi panel was assembled and draft recommendations were adapted into an e-Delphi survey to gain consensus on good-practice recommendations. Seventeen preliminary recommendations were identified and are currently being refined through an e-Delphi survey. Recommendations fall into key themes, including “Developing a Refined Research Question,” “Developing a Research Protocol,” and “Translation Phase.” Final recommendations will be described in an upcoming report. Applying PPI when designing studies will improve the rigor of RWE because study decisions will be based on patients’ lived experiences instead of researcher or clinician assumptions. PPI-informed RWE will contribute to a patient-centered evidence base to inform patient-centered VA.

It’s important to note that researchers who work on RWD studies are not expected to be the ones collecting the patient-provided information. That information, collected by others, can be leveraged by RWD researchers who are skilled at working with large data sets, but might not be experienced with patient engagement.

Eleanor M. Perfetto

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