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Estimation of Health Utility Scores for Glycogen Storage Disease Type Ia

Author

Listed:
  • Eliza Kruger

    (Ultragenyx Pharmaceutical Inc.)

  • Daniel Aggio

    (Acaster Lloyd Consulting Ltd)

  • Hayley Freitas

    (Acaster Lloyd Consulting Ltd)

  • Andrew Lloyd

    (Acaster Lloyd Consulting Ltd)

Abstract

Background Glycogen Storage Disease Type Ia (GSDIa) is a rare genetic metabolic disorder characterized by hypoglycemia, hepatomegaly, growth failure, and possible seizures/death. Patients frequently consume cornstarch to maintain blood glucose. Evidence demonstrating the impact of GSDIa on health-related quality of life (HRQoL) is lacking. This study aims to understand the burden of GSDIa by obtaining utility values for economic appraisals. Methods A targeted literature review and interviews with experts (n = 4) and caregivers (n = 4) informed the development of health state vignettes describing different GSDIa severities by age and level of hypothetical treatment (i.e., gene therapy) response. Health states reflecting caregivers’ experiences were also developed. A convenience sample of the UK general public completed a time trade-off (TTO) exercise. Scores conceptually varied from 0 (dead) to 1 (full health). States were also rated using a visual analog scale (VAS) and the EQ-5D-5L. Data were descriptively summarized. Results One hundred participants completed the exercise (male: 48%; mean age: 42 years). Scores were lowest for the adolescent pre-treatment state (TTO = 0.38). Large increments in HRQoL were observed for the response (adult: TTO = 0.86; child: TTO = 0.84) and complete response (adult and child: TTO = 0.94) hypothetical treatment response states. Caregiver values were lowest for the pre-treatment state (TTO = 0.57) and highest for the complete response state (TTO = 0.95). VAS and EQ-5D-5L scores followed a similar pattern. Conclusion This study found an HRQoL burden on GSDIa patients and caregivers, with potential large improvement from a hypothetical treatment. These findings may be useful for families, clinicians, regulatory agencies, and in therapy economic evaluations.

Suggested Citation

  • Eliza Kruger & Daniel Aggio & Hayley Freitas & Andrew Lloyd, 2023. "Estimation of Health Utility Scores for Glycogen Storage Disease Type Ia," PharmacoEconomics - Open, Springer, vol. 7(4), pages 627-638, July.
    • Handle: RePEc:spr:pharmo:v:7:y:2023:i:4:d:10.1007_s41669-023-00397-z
    DOI: 10.1007/s41669-023-00397-z
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    References listed on IDEAS

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    1. Brendan Mulhern & Yan Feng & Koonal Shah & Mathieu F. Janssen & Michael Herdman & Ben Hout & Nancy Devlin, 2018. "Comparing the UK EQ-5D-3L and English EQ-5D-5L Value Sets," PharmacoEconomics, Springer, vol. 36(6), pages 699-713, June.
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