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The Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugs

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  • Aaron S Kesselheim
  • Jessica A Myers
  • Daniel H Solomon
  • Wolfgang C Winkelmayer
  • Raisa Levin
  • Jerry Avorn

Abstract

Introduction: The Orphan Drug Act encourages drug development for rare conditions. However, some orphan drugs become top sellers for unclear reasons. We sought to evaluate the extent and cost of approved and unapproved uses of orphan drugs with the highest unit sales. Methods: We assessed prescription patterns for four top-selling orphan drugs: lidocaine patch (Lidoderm) approved for post-herpetic neuralgia, modafinil (Provigil) approved for narcolepsy, cinacalcet (Sensipar) approved for hypercalcemia of parathyroid carcinoma, and imatinib (Gleevec) approved for chronic myelogenous leukemia and gastrointestinal stromal tumor. We pooled patient-specific diagnosis and prescription data from two large US state pharmaceutical benefit programs for the elderly. We analyzed the number of new and total patients using each drug and patterns of reimbursement for approved and unapproved uses. For lidocaine patch, we subcategorized approved prescriptions into two subtypes of unapproved uses: neuropathic pain, for which some evidence of efficacy exists, and non-neuropathic pain. Results: We found that prescriptions for lidocaine patch, modafinil, and cinacalcet associated with non-orphan diagnoses rose at substantially higher rates (average monthly increases in number of patients of 14.6, 1.45, and 1.58) than prescriptions associated with their orphan diagnoses (3.12, 0.24, and 0.03, respectively (p 75%). Increases in lidocaine patch use for non-neuropathic pain far exceeded neuropathic pain (10.2 vs. 3.6 patients, p

Suggested Citation

  • Aaron S Kesselheim & Jessica A Myers & Daniel H Solomon & Wolfgang C Winkelmayer & Raisa Levin & Jerry Avorn, 2012. "The Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugs," PLOS ONE, Public Library of Science, vol. 7(2), pages 1-7, February.
    • Handle: RePEc:plo:pone00:0031894
    DOI: 10.1371/journal.pone.0031894
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    References listed on IDEAS

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    1. Wellman-Labadie, Olivier & Zhou, Youwen, 2010. "The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?," Health Policy, Elsevier, vol. 95(2-3), pages 216-228, May.
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    1. Caridad Pontes & Corinne Zara & Josep Torrent-Farnell & Merce Obach & Cristina Nadal & Patricia Vella-Bonanno & Michael Ermisch & Steven Simoens & Renata Curi Hauegen & Jolanta Gulbinovic & Angela Tim, 2020. "Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab," Applied Health Economics and Health Policy, Springer, vol. 18(1), pages 5-16, February.
    2. Amelia Smith & Stephen Doran & Maria Daly & Cormac Kennedy & Michael Barry, 2021. "Effect of an Online Reimbursement Application System on Prescribing of Lidocaine 5% Medicated Plaster in the Republic of Ireland," Applied Health Economics and Health Policy, Springer, vol. 19(1), pages 133-140, January.
    3. Dyfrig A Hughes & Jannine Poletti-Hughes, 2016. "Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study," PLOS ONE, Public Library of Science, vol. 11(10), pages 1-12, October.
    4. Daniel Tobias Michaeli & Thomas Michaeli & Sebastian Albers & Tobias Boch & Julia Caroline Michaeli, 2024. "Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 25(6), pages 979-997, August.

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