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Designing and Implementing Real-World Patient-Reported Outcomes-Emerging Recommendations: A Good Practices Report of

Angela J Rylands1, Konrad Maruszczyk2, Olalekan Lee Aiyegbusi2

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Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
|March 20, 2026
PubMed
Summary
This summary is machine-generated.

High-quality real-world patient-reported outcomes (RW-PROs) are crucial for decision-making. This report outlines nine methodological considerations for collecting robust RW-PRO data in routine care, balancing rigor with feasibility for patient-centered healthcare.

Keywords:
health technology assessmentmethodological guidancepatient experience datapatient-reported outcomes (PRO)prospective real-world studiesreal-world PROs (RW-PROs)real-world data (RWD)real-world evidence (RWE)regulatory decision making

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Area of Science:

  • Health Services Research
  • Clinical Informatics
  • Patient-Reported Outcomes

Background:

  • Real-world evidence (RWE) is increasingly used in regulatory, reimbursement, and clinical decisions.
  • Collecting high-quality patient-reported outcomes (PROs) outside traditional trials presents unique challenges.
  • Existing guidance inadequately addresses the methodological and operational complexities of prospective real-world PRO (RW-PRO) data collection.

Purpose of the Study:

  • To review methodological considerations for prospective PRO collection in real-world settings.
  • To examine how RW-PROs can complement clinical outcomes for capturing patient experience and treatment effectiveness.
  • To address stakeholder uncertainty regarding feasible and robust RW-PRO data generation for decision-making.

Main Methods:

  • A Task Force reviewed emerging methodological considerations for prospective RW-PRO collection.
  • Identified nine interrelated considerations for planning and implementing RW-PRO studies.
  • Examined the balance between scientific rigor, feasibility, patient burden, and operational constraints.

Main Results:

  • Nine key methodological considerations were identified: clear objectives, patient engagement, representativeness, instrument selection, data quality, feasible assessments, rigorous analysis, responsible technology use, and early engagement with regulatory bodies.
  • Best practices for RW-PRO research are context-dependent, varying by study, evidentiary use, and population.
  • The report outlines options, tradeoffs, strengths, and limitations rather than a single prescriptive model.

Conclusions:

  • RW-PRO evidence can inform regulatory and HTA decisions, enhance treatment outcome understanding, and support patient-centered care.
  • Transparent reporting of methodological choices and their implications is crucial for RW-PRO data interpretability.
  • Adopting emerging practices ensures RW-PRO data are meaningful, interpretable, and reflect patient-important outcomes.