Session: 904. Outcomes Research—Non-Malignant Conditions: Poster II
Hematology Disease Topics & Pathways:
Hemophilia, Diseases, Bleeding and Clotting
In economic evaluations, health-state-utility-valuations (HSUV) for quality-of-life (QoL) estimates inform the cost-effectiveness of new interventions. HSUVs are estimated by eliciting the preferences of people for different health states. However, people with inherited and long-term health conditions, such as hemophilia, may adapt to their given health state. This “disability paradox” could mean that QoL estimates for the same health state derived from people with hemophilia (PWH) may be valued higher compared to the general population (GP).
This study aimed to explore differences in preferences for the same health states elicited from PWH and GP and whether there were differences in the preferences elicited across hemophilia severity.
A discrete choice experiment (DCETTO) was completed by 283 PWH and 1,900 GP to derive HSUVs between July-to-September 2019 in the USA. Participants indicated their preferences for hypothetical EQ-5D-5L health states with a duration attribute. A total of 120 DCETTO choice tasks were administered online, with participants completing 15. Dominated and repeated scenarios were included to test for inconsistencies in responses. Conditional-logit regressions were used with random sampling of GP to match PWH for a balanced comparison. Model estimates were compared and QoL-weights developed to adjust for differences in preferences.
Responses were collected from 1,327 people (1,150 GP, 177 PWH including 104 Severe, 40 Moderate, 33 Mild) after removal of respondents failing consistency-checks. Sampling for age and gender (male-only), mean HSUV difference between PWH and GP was 0.17 (Figure 1). By disease severity, mean HSUV differences between severe PWH and moderate PWH with GP were 0.13 and 0.17, respectively. Data analysis for mild PWH was precluded due to limited sample size.
These findings demonstrate the presence of disability paradox in hemophilia. QoL estimates for the same health states derived from PWH may be valued higher compared to GP, indicating under-estimation of the impact of hemophilia.
Disclosures: O'Hara: F. Hoffmann-La Roche Ltd: Consultancy; HCD Economics: Current Employment, Current equity holder in private company. Martin: HCD Economics: Current Employment; uniQure: Consultancy. Morgan: HCD Economics: Current Employment; uniQure: Consultancy. Buckner: American Thrombosis and Hemostasis Network: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Consultancy; CSL Behring: Consultancy; Bayer: Consultancy; Biomarin: Consultancy; Takeda: Consultancy; Spark: Consultancy; Genentech: Consultancy; Kedrion: Consultancy; uniQure: Consultancy; Tremeau Pharmaceuticals: Consultancy. Skinner: Freeline Therapeutics: Research Funding; CSL Behring: Research Funding; Bayer: Consultancy, Research Funding; Novo Nordisk: Honoraria, Research Funding; Roche: Honoraria, Research Funding; Sanofi: Honoraria, Research Funding, Speakers Bureau; Sobi: Research Funding; Takeda: Honoraria, Research Funding; uniQure: Research Funding; Biomarin: Consultancy, Research Funding; Pfizer: Other, Speakers Bureau; Spark Therapeutics: Other, Speakers Bureau; Genentech: Consultancy, Honoraria. O'Mahony: UniQure: Honoraria; Freeline: Honoraria; Biomarin: Honoraria, Membership on an entity's Board of Directors or advisory committees. Mulhern: uniQure: Honoraria. Sawyer: uniQure: Current Employment, Current equity holder in publicly-traded company. Li: uniQure: Current Employment, Current equity holder in publicly-traded company.
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