EQ-5D-5L-DERIVED UTILITY DRIVERS OF HRQOL IN HAE AND COMPARISON WITH PATIENT REPORTED QUALITATIVE FINDINGS
Author(s)
Patrick Yong, MBChB, PhD1, Sinisa Savic, MBChB, PhD2, Luke Stainer, BSc, MSc3, You-Ren Chou, MSc3, Emma Louise Prentice, BSc, MSc3, Jasmina Medjedovic, MSc4, Joan Mendivil, MSc, MD4.
1Frimley Health NHS Foundation Trust, Frimley, United Kingdom, 2University of Leeds, Leeds, United Kingdom, 3Tolley Ltd, Buxton, United Kingdom, 4Pharvaris, Zurich, Switzerland.
1Frimley Health NHS Foundation Trust, Frimley, United Kingdom, 2University of Leeds, Leeds, United Kingdom, 3Tolley Ltd, Buxton, United Kingdom, 4Pharvaris, Zurich, Switzerland.
OBJECTIVES: Hereditary angioedema (HAE) is a rare genetic disorder characterised by unpredictable attacks of swelling affecting various body parts, and is associated with a substantial health-related quality of life (HRQoL) burden. The aim of this work was to identify key drivers of HRQoL burden in HAE, derived from EQ-5D-5L responses and qualitative interviews.
METHODS: EQ-5D-5L data collected in the Phase 3 RAPIDe-3 randomised controlled trial (NCT06343779) in people with HAE were analysed (n=118 attacks; n=79 patients). Domain disutilities were calculated using the Rowen 2026 updated value set, stratified by severity and primary attack location. To separate patient-reported burden from population preference weighting, EQ-5D-5L raw domain scores were examined alongside tariff-weighted disutilities, and compared with RAPIDe-3 qualitative interview data.
RESULTS: Based on raw domain scores, the pain/discomfort and usual activities domains were key drivers of HRQoL impact, supporting qualitative findings in which activities of daily living and work/school were prioritised as important aspects of daily life impacted by HAE attacks. Pain/discomfort contributed the greatest absolute domain disutility, reflecting patient burden and substantial population preference weights assigned to this domain. The anxiety/depression domain demonstrated a non-linear severity gradient, with disproportionately greater contribution to moderate versus mild as well as severe attacks, potentially demonstrating heightened anticipatory anxiety in addition to symptom burden. Domain profiles were similar across peripheral and abdominal locations. The most frequently reported negative impact of attacks in qualitative interviews was fatigue (55.8% of participants), however fatigue is absent from the EQ-5D framework, potentially underestimating total attack burden.
CONCLUSIONS: EQ-5D-5L domain analysis using the Rowen 2026 value set identifies the pain/discomfort and usual activities domains as primary drivers of HRQoL burden in HAE attacks. The non-linear anxiety pattern and absence of fatigue from the EQ-5D highlight important measurement gaps. An HAE specific utility study could quantify these impacts for future health technology assessment submissions.
METHODS: EQ-5D-5L data collected in the Phase 3 RAPIDe-3 randomised controlled trial (NCT06343779) in people with HAE were analysed (n=118 attacks; n=79 patients). Domain disutilities were calculated using the Rowen 2026 updated value set, stratified by severity and primary attack location. To separate patient-reported burden from population preference weighting, EQ-5D-5L raw domain scores were examined alongside tariff-weighted disutilities, and compared with RAPIDe-3 qualitative interview data.
RESULTS: Based on raw domain scores, the pain/discomfort and usual activities domains were key drivers of HRQoL impact, supporting qualitative findings in which activities of daily living and work/school were prioritised as important aspects of daily life impacted by HAE attacks. Pain/discomfort contributed the greatest absolute domain disutility, reflecting patient burden and substantial population preference weights assigned to this domain. The anxiety/depression domain demonstrated a non-linear severity gradient, with disproportionately greater contribution to moderate versus mild as well as severe attacks, potentially demonstrating heightened anticipatory anxiety in addition to symptom burden. Domain profiles were similar across peripheral and abdominal locations. The most frequently reported negative impact of attacks in qualitative interviews was fatigue (55.8% of participants), however fatigue is absent from the EQ-5D framework, potentially underestimating total attack burden.
CONCLUSIONS: EQ-5D-5L domain analysis using the Rowen 2026 value set identifies the pain/discomfort and usual activities domains as primary drivers of HRQoL burden in HAE attacks. The non-linear anxiety pattern and absence of fatigue from the EQ-5D highlight important measurement gaps. An HAE specific utility study could quantify these impacts for future health technology assessment submissions.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE409
Topic
Clinical Outcomes, Economic Evaluation, Patient-Centered Research
Disease
Rare & Orphan Diseases