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dc.contributor.authorKesselheim, Aaron Seth
dc.contributor.authorMyers, Jessica Amelia
dc.contributor.authorSolomon, Daniel Hal
dc.contributor.authorWinkelmayer, Wolfgang C.
dc.contributor.authorLevin, Raisa
dc.contributor.authorAvorn, Jerry
dc.date.accessioned2012-04-26T17:10:52Z
dc.date.issued2012
dc.identifier.citationKesselheim, Aaron S., Jessica A. Myers, Daniel H. Solomon, Wolfgang C. Winkelmayer, Raisa Levin, and Jerry Avorn. 2012. The prevalence and cost of unapproved uses of top-selling orphan drugs. PLoS ONE 7(2): e31894.en_US
dc.identifier.issn1932-6203en_US
dc.identifier.urihttp://nrs.harvard.edu/urn-3:HUL.InstRepos:8623550
dc.description.abstractIntroduction: 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<0.001 for all)). By contrast, for imatinib, approved uses increased significantly over off-label (0.97 vs. 0.47 patients, p<0.001). Spending on off-label uses was highest for lidocaine patch and modafinil (>75%). Increases in lidocaine patch use for non-neuropathic pain far exceeded neuropathic pain (10.2 vs. 3.6 patients, p<0.001). Discussion In our sample, three of four top-selling orphan drugs were used more commonly for non-orphan indications. These orphan drugs treated common clinical symptoms (pain and fatigue) or laboratory abnormalities. We should continue to monitor orphan drug use after approval to identify products that come to be widely used for non-FDA approved indications, particularly those without adequate evidence of efficacy.en_US
dc.language.isoen_USen_US
dc.publisherPublic Library of Scienceen_US
dc.relation.isversionofdoi:10.1371/journal.pone.0031894en_US
dc.relation.hasversionhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC3283698/pdf/en_US
dash.licenseLAA
dc.subjectmedicineen_US
dc.subjectdrugs and devicesen_US
dc.subjectdrug research and developmenten_US
dc.subjectepidemiologyen_US
dc.subjecthealth care policyen_US
dc.subjectscience policyen_US
dc.subjecttechnology developmenten_US
dc.titleThe Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugsen_US
dc.typeJournal Articleen_US
dc.description.versionVersion of Recorden_US
dc.relation.journalPLoS ONEen_US
dash.depositing.authorKesselheim, Aaron Seth
dc.date.available2012-04-26T17:10:52Z
dc.identifier.doi10.1371/journal.pone.0031894*
dash.contributor.affiliatedKesselheim, Aaron
dash.contributor.affiliatedFranklin, Jessica
dash.contributor.affiliatedSolomon, Daniel


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