MAPPING PATIENT-REPORTED OUTCOMES EVIDENCE REQUIREMENTS ACROSS GLOBAL MARKETS
Author(s)
Francesco Cottone, PhD1, Stephanie Fairhurst, BSc2, Sarah Knight, MSc2;
1Daiichi Sankyo, Global Oncology HEOR and RWE, Rome, Italy, 2Clarivate, London, United Kingdom
1Daiichi Sankyo, Global Oncology HEOR and RWE, Rome, Italy, 2Clarivate, London, United Kingdom
OBJECTIVES: Regulatory and health technology assessment (HTA) bodies increasingly recognize the importance of incorporating patient perspectives in clinical trials, via capturing patient-reported outcomes (PRO) data. However, requirements for PRO results evaluation in regulatory or reimbursement decisions vary across markets. This project aimed to identify Guidance documents regarding the use of PRO data from 10 regulatory agencies and 16 HTAs around the globe.
METHODS: A targeted literature review was conducted in stages: 1) HTA and regulatory websites hand searches, 2) OVID database search, 3) Google Scholar and Google hand searches (up to five pages). Information was extracted into an Excel data table, organized by topics: PRO measure content validity, psychometric measurement properties, meaningful change thresholds, requirements for PRO data inclusion in product labelling, clinical trial data statistical analysis considerations, clinical trial design and conduct considerations, preferences for communication or submission of PRO data, and recommendations for using specific PRO measures.
RESULTS: The number and detail of Guidance documents varied significantly across considered bodies. The Guidance and requirements reflected each organisations priorities for using the data, e.g. several HTA bodies reported preferences for deriving QALYs from EQ-5D or SF-6D. Some PRO requirements differed between bodies, e.g. IQWiGs universal response threshold of ≥ 15% of the scale range for an additional benefit rating versus FDAs preference for deriving meaningful score differences within each specific context of use. New initiatives may promote alignment in requirements, e.g. Joint Clinical Assessment (JCA) in Europe.
CONCLUSIONS: The heterogeneity in the amount and detail of PRO Guidance from stakeholders makes it challenging for sponsors to plan effective global PRO strategies. Greater alignment on PRO data requirements could improve transparency for sponsors wishing to submit PRO results for evaluation. It will be interesting to follow whether initiatives like JCA will lead to clearer and more aligned PRO evidence requirements in the future.
METHODS: A targeted literature review was conducted in stages: 1) HTA and regulatory websites hand searches, 2) OVID database search, 3) Google Scholar and Google hand searches (up to five pages). Information was extracted into an Excel data table, organized by topics: PRO measure content validity, psychometric measurement properties, meaningful change thresholds, requirements for PRO data inclusion in product labelling, clinical trial data statistical analysis considerations, clinical trial design and conduct considerations, preferences for communication or submission of PRO data, and recommendations for using specific PRO measures.
RESULTS: The number and detail of Guidance documents varied significantly across considered bodies. The Guidance and requirements reflected each organisations priorities for using the data, e.g. several HTA bodies reported preferences for deriving QALYs from EQ-5D or SF-6D. Some PRO requirements differed between bodies, e.g. IQWiGs universal response threshold of ≥ 15% of the scale range for an additional benefit rating versus FDAs preference for deriving meaningful score differences within each specific context of use. New initiatives may promote alignment in requirements, e.g. Joint Clinical Assessment (JCA) in Europe.
CONCLUSIONS: The heterogeneity in the amount and detail of PRO Guidance from stakeholders makes it challenging for sponsors to plan effective global PRO strategies. Greater alignment on PRO data requirements could improve transparency for sponsors wishing to submit PRO results for evaluation. It will be interesting to follow whether initiatives like JCA will lead to clearer and more aligned PRO evidence requirements in the future.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR93
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas