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The impact on quality of life of diet restrictions and disease symptoms associated with phenylketonuria: a time trade-off and discrete choice experiment study

Author

Listed:
  • Sara Olofsson

    (The Swedish Institute for Health Economics, IHE)

  • Katarina Gralén

    (The Swedish Institute for Health Economics, IHE)

  • Christina Hoxer

    (BioMarin Pharmaceutical Inc)

  • Paul Okhuoya

    (BioMarin Pharmaceutical Inc)

  • Ulf Persson

    (The Swedish Institute for Health Economics, IHE)

Abstract

Phenylketonuria (PKU) is a metabolic disorder leading to a deficiency in the metabolism of phenylalanine (Phe). Elevated Phe levels in the blood, tissue and brain may lead to emotional, cognitive, and physical symptoms in adults. To control blood Phe levels, most individuals with PKU need to follow a strict, life-long Phe-restricted diet. The main objective of this study was to estimate the impact of PKU-related disease symptoms and dietary restrictions on health-related quality of life (HRQoL). This study was designed as a web-based survey using the methods of Time Trade-Off (TTO) and Discrete Choice Experiment with duration (DCETTO) in a survey of the Swedish general population and population with PKU. Data were collected using questionnaires for TTO diet (n = 509), TTO symptoms (n = 507), and DCETTO (n = 1117). The disutility of diet restrictions ranged from 0.050 for a partially restricted diet without medical food to 0.193 for a fully restricted diet with medical food in the TTO (DCE: 0.043–0.108). The disutility of experiencing symptoms (emotional, cognitive and physical) ranged from 0.148 for mild symptoms to 0.593 for severe symptoms in the TTO (DCE: 0.122–1.522). The study shows that both diet and symptoms have a significant negative impact on HRQoL in PKU and that utility estimates are largely consistent across methods and samples.

Suggested Citation

  • Sara Olofsson & Katarina Gralén & Christina Hoxer & Paul Okhuoya & Ulf Persson, 2022. "The impact on quality of life of diet restrictions and disease symptoms associated with phenylketonuria: a time trade-off and discrete choice experiment study," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 23(6), pages 993-1005, August.
    • Handle: RePEc:spr:eujhec:v:23:y:2022:i:6:d:10.1007_s10198-021-01415-9
    DOI: 10.1007/s10198-021-01415-9
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    References listed on IDEAS

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    1. Brazier, John & Ratcliffe, Julie & Salomon, Joshua & Tsuchiya, Aki, 2016. "Measuring and Valuing Health Benefits for Economic Evaluation," OUP Catalogue, Oxford University Press, edition 2, number 9780198725923.
    2. Henrik Andersson & James K. Hammitt & Kristian Sundström, 2015. "Willingness to Pay and QALYs: What Can We Learn about Valuing Foodborne Risk?," Journal of Agricultural Economics, Wiley Blackwell, vol. 66(3), pages 727-752, September.
    3. Albrecht, Gary L. & Devlieger, Patrick J., 1999. "The disability paradox: high quality of life against all odds," Social Science & Medicine, Elsevier, vol. 48(8), pages 977-988, April.
    4. Menzel, Paul & Dolan, Paul & Richardson, Jeff & Olsen, Jan Abel, 2002. "The role of adaptation to disability and disease in health state valuation: a preliminary normative analysis," Social Science & Medicine, Elsevier, vol. 55(12), pages 2149-2158, December.
    5. Mark Oppe & Kim Rand-Hendriksen & Koonal Shah & Juan M. Ramos‐Goñi & Nan Luo, 2016. "EuroQol Protocols for Time Trade-Off Valuation of Health Outcomes," PharmacoEconomics, Springer, vol. 34(10), pages 993-1004, October.
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    More about this item

    Keywords

    Phenylketonuria; Phenylalanine hydroxylase deficiency; Quality of life; Burden of illness; Time trade-off; Discrete choice;
    All these keywords.

    JEL classification:

    • I1 - Health, Education, and Welfare - - Health
    • I31 - Health, Education, and Welfare - - Welfare, Well-Being, and Poverty - - - General Welfare, Well-Being

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