The Orphan Drug Act: Restoring the Mission to Rare Diseases : American Journal of Clinical Oncology

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Original Articles: Cancer Management Controversy

The Orphan Drug Act

Restoring the Mission to Rare Diseases

Daniel, Michael G.; Pawlik, Timothy M. MD, MPH, PhD; Fader, Amanda N. MD; Esnaola, Nestor F. MD, MPH, MBA; Makary, Martin A. MD, MPH

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American Journal of Clinical Oncology 39(2):p 210-213, April 2016. | DOI: 10.1097/COC.0000000000000251


The Orphan Drug Act has fostered drug development for patients with rare cancers and other diseases; however, current data suggest that companies are gaming the system to use the law for mainstream drugs. We identify a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits. Since the law was passed, the proportion of new FDA-approved drugs that were submitted as orphan drugs has increased with a peak last year of 41% of all FDA-approved drugs approved as orphan drugs. On the basis of the current data, we suggest that patients with rare cancers and other diseases may suffer due to dilution of the incentives and benefits. We propose reform to increase submission scrutiny, decrease benefits based on off-label use, and increase price transparency.

Copyright © 2016 Wolters Kluwer Health, Inc. All rights reserved.

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