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February 15, 2019

The Orphan Drug Act Revisited

Author Affiliations
  • 1New York University School of Medicine, New York
  • 2Harvard Law School, Cambridge, Massachusetts
  • 3Division of Medical Ethics, NYU Langone Medical Center, New York, New York
JAMA. 2019;321(9):833-834. doi:10.1001/jama.2019.0290

The Orphan Drug Act (ODA) was first passed in 1983 to address the concern that pharmaceutical manufacturers were not pursuing drug development for diseases that affect limited patient populations. The concern was in part that companies viewed pursuing these therapies as undesirable because the markets for them are small in comparison with the markets for more widespread chronic diseases. To promote the development of orphan drug therapies, the ODA provided companies that engaged in research for drugs with populations of fewer than 200 000 patients with tax incentives, research subsidies, and extended market exclusivity. With the new incentives in place, drug companies began developing more of these drugs. As of August 2018, a total of 503 new orphan therapies had been approved following the passage of the act.1

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