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: 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.
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Authors | Aaron S Kesselheim, Jessica A Myers, Daniel H Solomon, Wolfgang C Winkelmayer, Raisa Levin, Jerry Avorn |
Journal | PloS one
(PLoS One)
Vol. 7
Issue 2
Pg. e31894
( 2012)
ISSN: 1932-6203 [Electronic] United States |
PMID | 22363762
(Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)
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Chemical References |
- Benzhydryl Compounds
- Naphthalenes
- Prescription Drugs
- Lidocaine
- Modafinil
- Cinacalcet
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Topics |
- Benzhydryl Compounds
(economics, pharmacology)
- Cinacalcet
- Costs and Cost Analysis
- Drug Approval
(statistics & numerical data)
- Humans
- Lidocaine
(economics, pharmacology, therapeutic use)
- Modafinil
- Naphthalenes
(economics, pharmacology)
- Neuralgia
(drug therapy)
- Off-Label Use
(economics, statistics & numerical data)
- Orphan Drug Production
(economics, statistics & numerical data)
- Prescription Drugs
(economics)
- Public Health
- United States
- United States Food and Drug Administration
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