ArticleTherapeutic innovation & regulatory science2025
Identification and Description of Regulatory and Health Technology Assessment Agencies' Guidance Related to Patient Experience Data in North America, Europe, and Asia Pacific.
Article in Therapeutic innovation & regulatory science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Authors and funding
8 authors.
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Abstract
objectivesTo identify and describe patient experience data (PED)-related guidance issued by regulatory and health technology assessment (HTA) agencies in North America, Europe, and Asia Pacific.
methodsNational agency websites were manually searched (October 2021, updated October-December 2023) to identify standalone PED guidance, and general submission/clinical trial or disease-specific guidance in four therapy areas, including PED-related recommendations. Identified documents were reviewed for requirements/expectations on types of PED; concepts of interest and instruments/tools; validation, analysis, and interpretation; study design, and endpoint definitions.
resultsA total of 34 regulatory and 21 HTA documents were reviewed across 7 and 11 agencies, respectively. Most regulatory agencies expected data collected via clinical outcome assessments; concepts of interest were similar; specific tools were not usually recommended, although the use of validated instruments was expected; and meaningful within-patient change was generally considered key to interpretation. HTA guidance on PED varied primarily based on country requirement for utility estimates to inform economic evaluation (little/no guidance beyond the concept of interest) versus interest in PED on signs, symptoms, or disease impact on functioning (generally more comprehensive details on PED expectations).
conclusionThrough PED, patient voice is an increasingly important factor in regulatory, access, and reimbursement decision-making. Regulatory and HTA agencies in North America, Europe, and Asia Pacific have varying expectations regarding PED. This misalignment can necessitate the collection of multiple types of PED to meet all requirements (resulting in complex clinical trial protocols and significant patient burden), and lead to differences in data interpretation or gaps in the expected data. Therefore, PED measurement standards should be harmonized globally, and the collection of required PED should be ensured early during drug development to ensure decisions can be made based on valid and reliable data.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.