Patient and Societal Preferences for On-Demand Treatment in Hereditary Angioedema: Estimation of Utility Using a Discrete Choice Experiment
Author(s)
Patrick Yong, MBChB, PhD1, Timothy Craig, DO2, Sandra Christiansen, MD3, Paula Busse, MD4, Tomaz Garcez, MBChB5, Sinisa Savic, MBChB6, Rebekah Hall, PhD7, Siu Hing Lo, MA, MSc, PhD7, Caleb Dixon, MSc8, Paul Audhya, MD, MBA9, Alice Wang, MA9, Aleena Banerji, MD10, Sorena Kiani, MB, PhD11.
1Frimley Health NHS Foundation Trust, Frimley, United Kingdom, 2Penn State University, Hershey, PA, USA, 3University of California, San Diego, La Jolla, CA, USA, 4Mount Sinai School of Medicine, New York, NY, USA, 5Manchester University NHS Foundation Trust, Manchester, United Kingdom, 6The Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, United Kingdom, 7Acaster Lloyd, London, United Kingdom, 8Acaster Lloyd, Whitstable, United Kingdom, 9KalVista Pharmaceuticals, Cambridge, MA, USA, 10Harvard Medical School / Massachusetts General Hospital, Boston, MA, USA, 11Royal Free London NHS Foundation Trust, London, United Kingdom.
1Frimley Health NHS Foundation Trust, Frimley, United Kingdom, 2Penn State University, Hershey, PA, USA, 3University of California, San Diego, La Jolla, CA, USA, 4Mount Sinai School of Medicine, New York, NY, USA, 5Manchester University NHS Foundation Trust, Manchester, United Kingdom, 6The Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, United Kingdom, 7Acaster Lloyd, London, United Kingdom, 8Acaster Lloyd, Whitstable, United Kingdom, 9KalVista Pharmaceuticals, Cambridge, MA, USA, 10Harvard Medical School / Massachusetts General Hospital, Boston, MA, USA, 11Royal Free London NHS Foundation Trust, London, United Kingdom.
OBJECTIVES: Injectable on-demand therapies for hereditary angioedema (HAE) are associated with administration burden which may have a negative impact on patients’ health-related quality of life. We aimed to assess patient and societal preferences associated with on-demand treatments and estimate utility associated with administration route and administration-related side effects.
METHODS: An online survey using a discrete choice experiment (DCE) was conducted between December 2024 and January 2025 in adult HAE patients and general population in the US and UK. Participants chose between two hypothetical treatments described by four differentiating on-demand treatment attributes: (i) time to symptom relief onset, (ii) time to almost complete recovery, (iii) side effects and (iv) treatment type (ie, administration route, preparation, storage). Two approaches to anchor DCE estimates to a 0 (dead) to 1 (full health) scale were used: DCE-Visual Analog Scale and DCE-time trade-off.
RESULTS: Participants included 285 patients (N=76 UK, 209 US; mean age 45.0y) and 599 general population members (N=300 UK; 299 US; mean age 46.2y). Treatment type was ranked the most important attribute, accounting for 46.1% and 36.2% of patient and general population total preference share, followed by side effects (20.9%, 24.3%). For patients, intravenous infusion had the largest utility decrements (-0.157 to -0.063, p<0.001) compared to oral administration, based on both rescaling approaches; utilities for subcutaneous injection ranged -0.019 (p=0.012) to 0.009 (p=0.183). Injection-related side effects including skin reactions and painful burning/stinging were associated with significant utility decrements. Patients who were dissatisfied with subcutaneous administration had a significant utility decrement compared to those with no dissatisfaction (-0.014 vs. 0.032, p<0.001).
CONCLUSIONS: From patient and societal perspectives there was a preference for oral on-demand administration over injections. Injectable administration and related side effects were estimated to have a negative impact on quality of life resulting in a disutility compared to an oral alternative.
METHODS: An online survey using a discrete choice experiment (DCE) was conducted between December 2024 and January 2025 in adult HAE patients and general population in the US and UK. Participants chose between two hypothetical treatments described by four differentiating on-demand treatment attributes: (i) time to symptom relief onset, (ii) time to almost complete recovery, (iii) side effects and (iv) treatment type (ie, administration route, preparation, storage). Two approaches to anchor DCE estimates to a 0 (dead) to 1 (full health) scale were used: DCE-Visual Analog Scale and DCE-time trade-off.
RESULTS: Participants included 285 patients (N=76 UK, 209 US; mean age 45.0y) and 599 general population members (N=300 UK; 299 US; mean age 46.2y). Treatment type was ranked the most important attribute, accounting for 46.1% and 36.2% of patient and general population total preference share, followed by side effects (20.9%, 24.3%). For patients, intravenous infusion had the largest utility decrements (-0.157 to -0.063, p<0.001) compared to oral administration, based on both rescaling approaches; utilities for subcutaneous injection ranged -0.019 (p=0.012) to 0.009 (p=0.183). Injection-related side effects including skin reactions and painful burning/stinging were associated with significant utility decrements. Patients who were dissatisfied with subcutaneous administration had a significant utility decrement compared to those with no dissatisfaction (-0.014 vs. 0.032, p<0.001).
CONCLUSIONS: From patient and societal perspectives there was a preference for oral on-demand administration over injections. Injectable administration and related side effects were estimated to have a negative impact on quality of life resulting in a disutility compared to an oral alternative.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR170
Topic
Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases