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Quality-Adjusted Life Years in Erythropoietic Protoporphyria and Other Rare Diseases: A Patient-Initiated EQ-5D Feasibility Study

Author

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  • Jasmin Barman-Aksözen

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland
    Institute of Laboratory Medicine, Municipal Hospital Zurich Triemli, 8063 Zurich, Switzerland
    Swiss Reference Centre for Porphyrias, Municipal Hospital Zurich Triemli, 8063 Zurich, Switzerland
    These authors contributed equally to this work.)

  • Anna-Elisabeth Minder

    (Swiss Reference Centre for Porphyrias, Municipal Hospital Zurich Triemli, 8063 Zurich, Switzerland
    Division for Endocrinology, Diabetology and Porphyria, Municipal Hospital Zurich Triemli, 8063 Zurich, Switzerland
    These authors contributed equally to this work.)

  • Francesca Granata

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland
    Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, S.C Medicine and Metabolic Disorders, 20122 Milano, Italy)

  • Mårten Pettersson

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland)

  • Cornelia Dechant

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland)

  • Mehmet Hakan Aksözen

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland)

  • Rocco Falchetto

    (International Porphyria Patient Network (IPPN), 8032 Zurich, Switzerland)

Abstract

Erythropoietic protoporphyria (EPP) is an ultra-rare inborn error of metabolism characterised by painful phototoxic burn injuries after short exposure times to visible light. Patients with EPP are highly adapted to their condition which makes the quantification of their health-related quality of life (QoL) challenging. In the presented patient-initiated feasibility study, we describe a new approach to assess treatment benefits in EPP by measuring QoL with the generic EQ-5D instrument in five patients under long-term (≥two years) treatment with afamelanotide, the first approved therapy for EPP. For the study, we selected patients with EPP who in addition were affected by an involuntary treatment interruption (caused by a temporary reimbursement suspension) because we hypothesized that individuals who had previously unlearned their adaptation are better able to assess their life without treatment than treatment-naïve patients. QoL under treatment was comparable to the age-matched population norm, and retrospective results for a treatment interruption and phototoxic reaction time point were comparable to the QoL of patients with chronic neuropathic pain and acute burn injuries, respectively. The results were accepted by the National Institute for Health and Care Excellence in England for their evaluation of the cost-effectiveness of afamelanotide, i.e., the calculation of quality-adjusted life years.

Suggested Citation

  • Jasmin Barman-Aksözen & Anna-Elisabeth Minder & Francesca Granata & Mårten Pettersson & Cornelia Dechant & Mehmet Hakan Aksözen & Rocco Falchetto, 2023. "Quality-Adjusted Life Years in Erythropoietic Protoporphyria and Other Rare Diseases: A Patient-Initiated EQ-5D Feasibility Study," IJERPH, MDPI, vol. 20(7), pages 1-15, March.
    • Handle: RePEc:gam:jijerp:v:20:y:2023:i:7:p:5296-:d:1109856
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    References listed on IDEAS

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    1. Jasmin Barman-Aksözen & Nicole Hentschel & Mårten Pettersson & Eva Schupp & Francesca Granata & Cornelia Dechant & Mehmet Hakan Aksözen & Rocco Falchetto, 2024. "Fair Funding Decisions: Consistency of the Time Horizons Used in the Calculation of Quality-Adjusted Life Years for Therapies for Very Rare Diseases by the National Institute for Health and Care Excel," IJERPH, MDPI, vol. 21(5), pages 1-14, May.

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