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PharmacoEconomics - Open
Published in: PharmacoEconomics - Open 1/2022

Open Access 01-01-2022 | Generalized Seizure | Original Research Article

Patient and Caregiver Health State Utilities in Tuberous Sclerosis Complex

Authors: Siu Hing Lo, Jade Marshall, Hanna Skrobanski, Andrew Lloyd

Published in: PharmacoEconomics - Open | Issue 1/2022

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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.
Appendix
Available only for authorised users
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Metadata
Title
Patient and Caregiver Health State Utilities in Tuberous Sclerosis Complex
Authors
Siu Hing Lo
Jade Marshall
Hanna Skrobanski
Andrew Lloyd
Publication date
01-01-2022
Publisher
Springer International Publishing
Published in
PharmacoEconomics - Open / Issue 1/2022
Print ISSN: 2509-4262
Electronic ISSN: 2509-4254
DOI
https://doi.org/10.1007/s41669-021-00296-1

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