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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

American Journal of Clinical Oncology: April 2016 - Volume 39 - Issue 2 - p 210–213
doi: 10.1097/COC.0000000000000251
Original Articles: Cancer Management Controversy

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.

Departments of *Surgery

Surgery and Oncology

Obstetrics and Gynecology, Johns Hopkins University School of Medicine

Department of Surgery and Health Policy & Management, Johns Hopkins University School of Medicine, Johns Hopkins University Bloomberg School of Public Health, Baltimore, MD

§Department of Surgery, Fox Chase Cancer Center Temple Health, Philadelphia, PA

The authors declare no conflicts of interest.

Reprints: Martin A. Makary, MD, MPH, Department of Surgery, The Johns Hopkins School of Medicine, Halsted 610, 600 North Wolfe Street, Baltimore, MD 21287. E-mail:

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