ACCEPTABILITY OF PROMS IN EU-4 AND THE UK TO ASSESS CLINICAL BENEFIT AND WILLINGNESS TO PAY UTILIZING THE SIAT FRAMEWORK AND THE SMART CHECKLIST - A MODIFIED DELPHI STUDY
Author(s)
Gordon Guyatt, MD, MSc, FRCPC1, Peter Ashley, MSc2, Francisco Javier Badia, MPH, PhD3, Yvonne-Beatrice Böhler, MBA, MD4, Melanie Calvert, PhD, FMedSci5, Finn Børlum Kristensen, PhD, MD6, Andrea Marcellusi, PhD7, Roy Bentley, MSc, JD, PhD8, Peter O'Donovan, BSc, MSc9, Charlotte Oswald, BSc9, Ashley Enstone, MRes, BSc9, Robin Wyn, BSc, MSc9, Olivier Chassany, PhD, MD10.
1Department of Health Research Methods, McMaster University, Hamilton, ON, Canada, 2EP Independent Consultants, Lincolnshire, United Kingdom, 3Omakase Consulting, Barcelona, Spain, 4TH Koeln - University of Applied Sciences, Leverkusen, Germany, 5University of Birmingham, Birmingham, United Kingdom, 6Danish Centre for Health Economics - University of Southern Denmark, Hilleroed, Denmark, 7University of Milan, Milano, Italy, 8SHIONOGI B.V., Florham Park, NJ, USA, 9Adelphi Values PROVE, Bollington, United Kingdom, 10Assistance Publique–Hôpitaux de Paris (AP-HP), Paris, France.
1Department of Health Research Methods, McMaster University, Hamilton, ON, Canada, 2EP Independent Consultants, Lincolnshire, United Kingdom, 3Omakase Consulting, Barcelona, Spain, 4TH Koeln - University of Applied Sciences, Leverkusen, Germany, 5University of Birmingham, Birmingham, United Kingdom, 6Danish Centre for Health Economics - University of Southern Denmark, Hilleroed, Denmark, 7University of Milan, Milano, Italy, 8SHIONOGI B.V., Florham Park, NJ, USA, 9Adelphi Values PROVE, Bollington, United Kingdom, 10Assistance Publique–Hôpitaux de Paris (AP-HP), Paris, France.
OBJECTIVES: Patient‑reported outcome measures (PROMs) are routinely included in clinical trials, yet their perceived value to inform clinical review by European Health Technology Assessment (HTA) agencies and payer’s willingness to pay remain unclear. This study aimed to understand national HTA and payer perspectives on the use of PROMs as secondary endpoints in the EU-4 and the UK in different diseases (except oncology and rare diseases).
METHODS: A targeted literature and analogue review were conducted to inform the development of materials for a modified-Delphi panel consisting of two rounds of 1:1 interviews and online surveys, followed by a virtual roundtable meeting. The study gathered insights from a panel comprised of 10 HTA / payer experts from the EU-4 and the UK. Eight opinion leaders comprised a Steering Committee to provide oversight.
RESULTS: The SIAT (selection, implementation, analysis, treatment effect) framework was developed which outlines considerations for market access-related decision-makers to utilize evidence based on PROM selection, implementation, analysis and interpretation of treatment effect. Panelists identified several issues in sponsor dossiers, including poor measurement justification, inadequate evidence of validity, reliability, responsiveness, and meaningful change, as well as missing data. The SMART Set checklist, which guides sponsor’s selection and implementation of fit for purpose PROMs, was introduced to and positively received by panelists. Panelists highlighted that use of the SMART set to substantiate the incorporation of specific PROMs in a given development program would increase evidence credibility and facilitate its use in access-related decision-making. Experts noted the EU JCA (Joint Clinical Assessment), through PICOs (population, intervention, comparator, outcomes), will impact the perceived relevance of PROMs included in clinical trials.
CONCLUSIONS: Findings suggest that PRO evidence can inform market access-related decision-making. Sponsors can improve acceptability of PROMs by providing clear rationale for their selection, providing robust evidence of measurement properties, defining meaningful change thresholds, and reducing data missingness.
METHODS: A targeted literature and analogue review were conducted to inform the development of materials for a modified-Delphi panel consisting of two rounds of 1:1 interviews and online surveys, followed by a virtual roundtable meeting. The study gathered insights from a panel comprised of 10 HTA / payer experts from the EU-4 and the UK. Eight opinion leaders comprised a Steering Committee to provide oversight.
RESULTS: The SIAT (selection, implementation, analysis, treatment effect) framework was developed which outlines considerations for market access-related decision-makers to utilize evidence based on PROM selection, implementation, analysis and interpretation of treatment effect. Panelists identified several issues in sponsor dossiers, including poor measurement justification, inadequate evidence of validity, reliability, responsiveness, and meaningful change, as well as missing data. The SMART Set checklist, which guides sponsor’s selection and implementation of fit for purpose PROMs, was introduced to and positively received by panelists. Panelists highlighted that use of the SMART set to substantiate the incorporation of specific PROMs in a given development program would increase evidence credibility and facilitate its use in access-related decision-making. Experts noted the EU JCA (Joint Clinical Assessment), through PICOs (population, intervention, comparator, outcomes), will impact the perceived relevance of PROMs included in clinical trials.
CONCLUSIONS: Findings suggest that PRO evidence can inform market access-related decision-making. Sponsors can improve acceptability of PROMs by providing clear rationale for their selection, providing robust evidence of measurement properties, defining meaningful change thresholds, and reducing data missingness.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR47
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas