Four Decades of Orphan Drugs and Priorities for the Future

N Engl J Med. 2024 Jul 11;391(2):100-102. doi: 10.1056/NEJMp2401487. Epub 2024 Jul 6.

Authors

Michael S Sinha¹, Ariel D Stern¹, Arti K Rai¹

Affiliation

¹ From the Center for Health Law Studies, Saint Louis University School of Law, St. Louis (M.S.S.); Harvard Business School and the Harvard-MIT Center for Regulatory Science - both in Boston (A.D.S.); Hasso Plattner Institute, University of Potsdam, Potsdam, Germany (A.D.S.); and Duke Law School and the Duke-Margolis Center for Health Policy, Durham, NC (A.K.R.).

PMID: 38973724
DOI: 10.1056/NEJMp2401487

No abstract available

Publication types

Historical Article

MeSH terms

Drug Approval / legislation & jurisprudence
Drug Industry / history
Drug Industry / legislation & jurisprudence
History, 20th Century
History, 21st Century
Humans
Orphan Drug Production* / legislation & jurisprudence
Rare Diseases* / drug therapy
United States
United States Food and Drug Administration / legislation & jurisprudence