Implementation of Patient Experience Data in Clinical Trial Design: A Literature Review and Semi-Structured Interviews
Author(s)
Alice Vanneste, MSc1, Peter Sinnaeve, Prof.2, Isabelle Huys, PharmD, PhD1, Tom Adriaenssens, Prof.2.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Cardiovascular Diseases, Department of Cardiovascular Medicine, UZ Leuven, KU Leuven, Leuven, Belgium.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Cardiovascular Diseases, Department of Cardiovascular Medicine, UZ Leuven, KU Leuven, Leuven, Belgium.
OBJECTIVES: Patient-centred healthcare is essential for aligning research and development with patients’ needs and preferences. Integrating Patient Experience Data (PED) into clinical trial design can improve endpoint selection, recruitment, retention, and ensure trials are relevant to what matters to patients. However, evidence on how PED is currently used in clinical trial design is limited. This study aimed to investigate current trial design practices and how patients’ perspectives are integrated into these processes.
METHODS: A literature review was conducted in PubMed and Embase, identifying 231 articles related to PED use in clinical trials. To enrich these findings, 28 semi-structured interviews were conducted with diverse stakeholders, including patient organizations, physicians, researchers, developers, regulators, pricing and reimbursement agencies, and policymakers. Interviews were transcribed and analyzed using thematic framework analysis.
RESULTS: Literature and interviews indicate increasing interest in PED, particularly in using tools like patient-reported outcome measures and patient-reported experience measures. However, their application remains inconsistent across disease areas and trial phases, often due to a lack of standardized methods and validated tools. More research on clearly defining patient-relevant endpoints and core outcome sets were highlighted as opportunities in clinical trial design. Stakeholders also emphasized the need for earlier patient involvement in clinical trial design, regulatory support for effective PED integration, and stronger cross-sector collaborations to define guidelines for implementing PED in clinical trials.
CONCLUSIONS: Despite a clear growing trend, PED integration in clinical trial design remains fragmented. Advancing toward patient-centered research requires early and meaningful patient involvement, standardized outcome measures, and coordinated multi-stakeholder efforts. Strengthening partnerships across healthcare decision-making can ensure clinical trial designs reflect patients’ real needs and preferences, thereby powering value, access, and impact through patient-centered collaboration.
METHODS: A literature review was conducted in PubMed and Embase, identifying 231 articles related to PED use in clinical trials. To enrich these findings, 28 semi-structured interviews were conducted with diverse stakeholders, including patient organizations, physicians, researchers, developers, regulators, pricing and reimbursement agencies, and policymakers. Interviews were transcribed and analyzed using thematic framework analysis.
RESULTS: Literature and interviews indicate increasing interest in PED, particularly in using tools like patient-reported outcome measures and patient-reported experience measures. However, their application remains inconsistent across disease areas and trial phases, often due to a lack of standardized methods and validated tools. More research on clearly defining patient-relevant endpoints and core outcome sets were highlighted as opportunities in clinical trial design. Stakeholders also emphasized the need for earlier patient involvement in clinical trial design, regulatory support for effective PED integration, and stronger cross-sector collaborations to define guidelines for implementing PED in clinical trials.
CONCLUSIONS: Despite a clear growing trend, PED integration in clinical trial design remains fragmented. Advancing toward patient-centered research requires early and meaningful patient involvement, standardized outcome measures, and coordinated multi-stakeholder efforts. Strengthening partnerships across healthcare decision-making can ensure clinical trial designs reflect patients’ real needs and preferences, thereby powering value, access, and impact through patient-centered collaboration.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
P17
Topic
Clinical Outcomes, Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas