Health state utilities for beta-thalassemia: a time trade-off study

Antony P. Martin, Enrico Ferri Grazzi (), Claudia Mighiu, Manoj Chevli, Farrukh Shah, Louise Maher, Anum Shaikh, Aliah Sagar, Hayley Hubberstey, Bethany Franks, Juan M. Ramos-Goñi, Mark Oppe and Derek Tang
Additional contact information
Antony P. Martin: Formerly HCD Economics
Enrico Ferri Grazzi: HCD Economics
Claudia Mighiu: HCD Economics
Manoj Chevli: Celgene Ltd, a Bristol-Myers Squibb Company
Farrukh Shah: University College London Hospital
Louise Maher: Celgene Ltd, a Bristol-Myers Squibb Company
Anum Shaikh: Formerly HCD Economics
Aliah Sagar: Formerly HCD Economics
Hayley Hubberstey: Formerly HCD Economics
Bethany Franks: HCD Economics
Juan M. Ramos-Goñi: Formerly Axentiva Solutions
Mark Oppe: Formerly Axentiva Solutions
Derek Tang: Bristol Myers Squibb

The European Journal of Health Economics, 2023, vol. 24, issue 1, No 3, 27-38

Abstract: Abstract Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies.

Keywords: Beta-thalassemia; Hemoglobin; Anemia; Time trade-off; Transfusion burden (search for similar items in EconPapers)
JEL-codes: I12 (search for similar items in EconPapers)
Date: 2023
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s10198-022-01449-7 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:eujhec:v:24:y:2023:i:1:d:10.1007_s10198-022-01449-7

Ordering information: This journal article can be ordered from
http://www.springer. ... cs/journal/10198/PS2

DOI: 10.1007/s10198-022-01449-7

Access Statistics for this article

The European Journal of Health Economics is currently edited by J.-M.G.v.d. Schulenburg

More articles in The European Journal of Health Economics from Springer, Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ) Contact information at EDIRC.
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().