The Orphan Drug Act: Restoring the Mission to Rare Diseases

@article{Daniel2016TheOD,
  title={The Orphan Drug Act: Restoring the Mission to Rare Diseases},
  author={Michael Daniel and Timothy M. Pawlik and Amanda Nickles Nickles Fader and Nestor F. Esnaola and Martin A. Makary},
  journal={American Journal of Clinical Oncology},
  year={2016},
  volume={39},
  pages={210–213}
}
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… Expand
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