ASSESSING THE INCREMENTAL VALUE OF PATIENT EXPERIENCE EVIDENCE IN HTA AND REIMBURSEMENT DECISION-MAKING
Author(s)
Geof Gray1, Lorna M. Richards, PhD2, Puneet Kumar, M.Pharm3, Dana Michelle Saavedra Roman, MPH4, Jacquelyne Brauneis, MPH5.
1Research Strategist, Syneos Health, Methven, New Zealand, 2Syneos Health, Bridegwater, NJ, USA, 3Syneos Health, London, United Kingdom, 4Syneos Health, San Diego, CA, USA, 5Syneos Health, Bridgewater, NJ, USA.
1Research Strategist, Syneos Health, Methven, New Zealand, 2Syneos Health, Bridegwater, NJ, USA, 3Syneos Health, London, United Kingdom, 4Syneos Health, San Diego, CA, USA, 5Syneos Health, Bridgewater, NJ, USA.
OBJECTIVES: Patient experience evidence (PEE), (patient-reported outcomes, treatment satisfaction, symptom burden, functional impacts, and treatment burden), is increasingly recognized as relevant to healthcare value assessment. Its contribution to health technology assessment (HTA) and reimbursement decision-making is not consistently defined, evaluated, or communicated. This study developed a methodological framework to assess the contribution of PEE alongside conventional clinical, safety, and economic evidence.
METHODS: Targeted review of HTA guidance, assessment reports from HTA bodies (NICE, IQWiG/G-BA, HAS, CADTH, and ICER), and methodological literature from the past 5 years. Sources were reviewed to identify how PEE is considered in clinical benefit assessment, comparative value assessment, and deliberative decision-making. Findings were synthesized in a framework describing how PEE can complement, contextualize, or differentiate conventional clinical and economic evidence. Key considerations included measurement validity, disease relevance, clinical meaningfulness, uncertainty, and alignment with HTA decision criteria.
RESULTS: The framework identified four ways PEE adds value: (1) capturing benefits, burdens, and trade-offs not fully reflected by conventional endpoints; (2) demonstrating changes in quality of life, daily functioning, symptom burden, and treatment convenience; (3) differentiating interventions with similar efficacy and safety profiles with outcomes and attributes meaningful to patients and caregivers; (4) contextualizing clinical and economic evidence by clarifying unmet need, treatment burden, and the meaningfulness of observed effects. PEE contribution was influenced by fit-for-purpose validated measures, alignment with disease and treatment context, magnitude and relevance of observed changes, data completeness, and applicability to local decision criteria.
CONCLUSIONS: The framework highlights opportunities to incorporate PEE in value dossiers, economic model assumptions, and HTA submission strategies, particularly when traditional measures do not fully characterize outcomes, burdens, and trade-offs experienced by patients and caregivers. This framework provides a structured approach for assessing and communicating PEE contribution in HTA and reimbursement submissions and may support more transparent, patient-centered, and decision-relevant assessments of healthcare value.
METHODS: Targeted review of HTA guidance, assessment reports from HTA bodies (NICE, IQWiG/G-BA, HAS, CADTH, and ICER), and methodological literature from the past 5 years. Sources were reviewed to identify how PEE is considered in clinical benefit assessment, comparative value assessment, and deliberative decision-making. Findings were synthesized in a framework describing how PEE can complement, contextualize, or differentiate conventional clinical and economic evidence. Key considerations included measurement validity, disease relevance, clinical meaningfulness, uncertainty, and alignment with HTA decision criteria.
RESULTS: The framework identified four ways PEE adds value: (1) capturing benefits, burdens, and trade-offs not fully reflected by conventional endpoints; (2) demonstrating changes in quality of life, daily functioning, symptom burden, and treatment convenience; (3) differentiating interventions with similar efficacy and safety profiles with outcomes and attributes meaningful to patients and caregivers; (4) contextualizing clinical and economic evidence by clarifying unmet need, treatment burden, and the meaningfulness of observed effects. PEE contribution was influenced by fit-for-purpose validated measures, alignment with disease and treatment context, magnitude and relevance of observed changes, data completeness, and applicability to local decision criteria.
CONCLUSIONS: The framework highlights opportunities to incorporate PEE in value dossiers, economic model assumptions, and HTA submission strategies, particularly when traditional measures do not fully characterize outcomes, burdens, and trade-offs experienced by patients and caregivers. This framework provides a structured approach for assessing and communicating PEE contribution in HTA and reimbursement submissions and may support more transparent, patient-centered, and decision-relevant assessments of healthcare value.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA310
Topic
Health Technology Assessment, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas