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Reforming the Orphan Drug Act for the 21st Century

List of authors.
  • Ameet Sarpatwari, J.D., Ph.D.,
  • and Aaron S. Kesselheim, M.D., J.D., M.P.H.

The pharmaceutical market has undergone radical changes, including markedly increased prices for rare-disease drugs. The revenue generated by such products raises questions about whether the Orphan Drug Act should be reformed to better align with the needs of patients.

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Funding and Disclosures

Disclosure forms provided by the authors are available at NEJM.org.

Author Affiliations

From the Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Boston.

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