Patient and Caregiver Health State Utilities in Tuberous Sclerosis Complex

Siu Hing Lo (), Jade Marshall, Hanna Skrobanski and Andrew Lloyd
Additional contact information
Siu Hing Lo: Acaster Lloyd Consulting Ltd
Jade Marshall: GW Pharma Ltd
Hanna Skrobanski: Acaster Lloyd Consulting Ltd
Andrew Lloyd: Acaster Lloyd Consulting Ltd

PharmacoEconomics - Open, 2022, vol. 6, issue 1, No 11, 105-121

Abstract: Abstract Background Tuberous sclerosis complex (TSC) is a rare multisystem disorder often associated with treatment-resistant epilepsy. Cost-effectiveness analysis for new antiseizure medications typically requires health state utilities (HSUs) that reflect the burden of a given condition. Objective This study aimed to estimate HSUs, with a focus on valuing the impact of seizure type and seizure frequency on health-related quality of life (HRQL) for patients with TSC and their caregivers. Methods A targeted literature review and qualitative research with healthcare professionals and caregivers informed the development of health state vignettes describing the experience of living with TSC or caring for a child with TSC. Vignettes were evaluated in interviews with the UK general population using the time trade-off (TTO) method. Results Sixteen vignettes were developed describing patient HRQL (n = 8) and caregiver HRQL (n = 8). Two hundred interviews were conducted via online video calls due to COVID-19 pandemic restrictions. Two hundred participants evaluated the patient (n = 100) and caregiver (n = 100) health state vignettes. Estimated utility scores varied consistently according to seizure type and seizure frequency. Patient TTO utility scores ranged between −0.234 (highest seizure frequency and multiple seizure types) and 0.725 (seizure-free state). Caregiver TTO utility scores ranged from 0.221 to 0.905. Conclusions Findings highlight the substantial burden of living with TSC and caring for a child with TSC. Patient and caregiver burden was greater for generalised versus focal seizures. The burden was greatest for a combination of both seizure types and worsened with increasing seizure frequency.

Date: 2022
References: View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s41669-021-00296-1 Abstract (text/html)

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:pharmo:v:6:y:2022:i:1:d:10.1007_s41669-021-00296-1

Ordering information: This journal article can be ordered from
http://www.springer.com/adis/journal/41669

DOI: 10.1007/s41669-021-00296-1

Access Statistics for this article

PharmacoEconomics - Open is currently edited by Timothy Wrightson and Christopher Carswell

More articles in PharmacoEconomics - Open from Springer
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().