Lawrenceville, NJ, USA—June 23, 2026—Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of an ISPOR Good Practices Report outlining 9 areas to strengthen the robustness, transparency, and feasibility of real-world patient-reported outcomes studies. The report, “Designing and Implementing Real World Patient-Reported Outcomes- Emerging Recommendations: A Good Practices Report of an ISPOR Task Force,” was published in the June 2026 issue of Value in Health.
"Patient-reported outcome assessments crucially capture the patients’ direct report of their health status or behavior without interpretation by a clinician or anyone else, providing a true measure of the patient voice that is essential for developing patient focused medicines” said author and ISPOR Task Force Co-Chair, Angela Rylands, CPsychol, PhD, Global Head of Patient-Centered Outcomes, Kyowa Kirin International, London, England, United Kingdom. “Real-world patient-reported outcomes can provide valuable insights from larger and more diverse patient populations that are more representative of patients encountered in routine clinical practice. However, obtaining adequate patient-reported outcome data from certain real-world data sources—such as medical claims databases, electronic health records, surveys, or digital health technologies—can be challenging," added fellow Task Force Co-Chair, Melanie Calvert, PhD, FMedSci, Centre for Patient-Reported Outcomes Research, and NIHR Senior Investigator, University of Birmingham, Birmingham, England, United Kingdom .
There are few recommendations for the use of patient-reported outcomes in the context of real-world studies. Indeed, existing guidelines are inadequate for real-world patient-reported outcome contexts, underscoring the need for comprehensive, tailored guidance. To fill that gap, this Good Practices Report provides recommendations for the prospective collection and use of patient-reported outcomes in real-world studies.
It is intended for sponsors, academic investigators, patient organizations, clinicians, and methodologists planning or conducting pragmatic trials, observational cohort studies, registries, or hybrid real-world designs. The focus is on how to operationalize patient-reported outcomes in real-world contexts to inform regulation, reimbursement, clinical care, and health policy. While comprehensive, the guidance is non‑prescriptive and should be adapted to disease area, study design, infrastructure, and intended decision use.
The Task Force recommends focusing on 9 key methodological aspects and considerations:
- Develop Clear and Feasible Study Objectives
- Work with Patients as Partners
- Define the Study Population
- Select Fit-For-Purpose Patient-Reported Outcome Instruments in Real-World Settings
- Ensure Data Quality
- Implement Real-World Patient-Reported Outcomes Pragmatically
- Analyze Real-World Patient-Reported Outcomes Data Rigorously
- Use Technology Responsibly
- Engage Regulators and Health Technology Assessment Bodies Early
Beyond highlighting the 9 aspects, the report makes 3 additional substantive contributions that give the framework meaningful context and real-world grounding:
- The Challenges Framework. The report explicitly catalogs the barriers that have limited real-world patient-reported outcome adoption: unclear instrument selection guidance, variability in stakeholder perceptions of PRO value, higher missing data risk, patient burden and compliance concerns, methodological gaps in bias reduction and missing data handling, and operational reliance on paper-based collection. The exploration of these barriers validates known friction points and frames the 9 recommendations as responses to real problems rather than abstract ideals.
- Value-in-Context Framing. The report distinguishes between pre- and post-marketing authorization contexts, helping researchers understand why they are collecting real-world patient-reported outcomes and what evidentiary standard applies across situations ranging from early access and rare disease natural history studies to pharmacovigilance, label expansion, and formulary decision support.
- Three Case Examples. The report includes 3 detailed case examples—an X-linked hypophosphatemia adolescent study, an erythropoietic protoporphyria post-authorization registry, and an acute pain pathways cohort—mapped against all 9 focus areas, acknowledging real-world limitations and trade-offs, making the guidance tangible rather than theoretical.
The 18-member Task Force spans pharma (Kyowa Kirin, GSK, ABPI), regulators (FDA, NICE), academia (University of Birmingham, Johns Hopkins, Mayo Clinic, University of Tasmania), patient advocates, and ISPOR staff. The Task Force's development process was rigorous, including presentations at ISPOR conferences in the United States, Europe, and Asia, plus 2 rounds of written review by external reviewers.
“The utilization of patient-reported outcomes in real-world studies can significantly enhance our understanding of burden of disease and patients’ experiences of living with their condition,” said Task Force member, Konrad Maruszczyk, PhD, Centre for Patient-Reported Outcomes Research, University of Birmingham, Birmingham, England, United Kingdom
. “Patient-reported outcomes also provide evidence of therapy effectiveness and tolerability in real-world settings. By offering direct patient insights, real-world patient-reported outcome data can inform regulatory and health technology assessment decisions, potentially increasing patient access to treatments.”
The need for methodologically robust integration of patient-reported outcomes in real-world studies has been emphasized by another Task Force member, Onyeka Illoh, OD, PhD, Division of Clinical Outcome Assessment, US Food and Drug Administration: “As the field continues to advance toward more patient-focused drug development, integrating patient-reported outcomes into real-world studies is not only beneficial but essential. Equally important is understanding how to apply patient-reported outcomes empirically in real-world contexts to support credible inferences. This Good Practices Report lays the groundwork and represents a meaningful step in the right direction.”
About ISPOR’s Task Force on Patient Reported Outcomes (PROs) in Prospective Real World Study Design
Objective: The task force’s goal is to develop emerging good practice recommendations for designing prospective real-world studies that collect patient-reported outcome (PRO) data.
Rationale: Global regulators, payers, and policy makers have indicated that PRO data can provide valuable information on the effectiveness, safety, and tolerability from the patient perspective. Currently, there are no regulatory, health technology appraisal guidance nor publications pertaining to the standardization of PRO data usage in real-world studies.
While several guidelines on the implementation of PROs exist, they mainly focus on randomized controlled trials or clinical practice. They provide little or no recommendations for the use of PROs in the context of real-world evidence generation, which would address the needs of regulators and policy makers. Practicalities of PRO data collection in routine care and methods of its analysis often pose a challenge for real-world researchers.
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ABOUT ISPOR
ISPOR—The Professional Society for Health Economics and Outcomes Research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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ABOUT VALUE IN HEALTH
Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help healthcare leaders make evidence-based decisions. The journal’s current impact factor score is 6.0 and its 5-year impact factor score is 5.7. Value in Health is ranked 5th of 124 journals in Health Policy and Services, 12th of 185 journals in Health Care Sciences & Services, and 37th of 617 journals in Economics. Value in Health is a monthly publication that circulates to more than 55,000 readers around the world.
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ABOUT ISPOR GOOD PRACTICES REPORTS
ISPOR has earned an international reputation for research excellence based, in part, on its Good Practices Reports. These highly cited reports are expert consensus recommendations on good practice standards for outcomes research (clinical, economic, and patient-reported outcomes) and on the use of this research in healthcare decision making. ISPOR Task Forces comprise subject matter experts representing different stakeholders from diverse work environments (ie, regulators, payers, manufacturers, technology assessors, etc from research, government, academic, and industry sectors around the world). All ISPOR Good Practices Reports are published in the Society’s scientific journal, Value in Health, and are made freely available as part of the Society’s mission. The Society’s Good Practices Reports have been recognized with an ASAE “Power of A” award that acknowledges innovative, effective, and broad-reaching programs that have a positive impact on the world.