1. US Food and Drug Administration. Rare Diseases at FDA. 2022. https://www.fda.gov/patients/rare-diseases-fda.
2. The Orphan Drug Act
3. Swan J. The Story Behind the Orphan Drug Act. 2018. https://www.fda.gov/industry/fdas-rare-disease-day/story-behind-orphan-drug-act.
4. US Food and Drug Administration. Total number of orphan drug designation decisions that resulted in orphan designation in the month: Fiscal Year 2020. 2020. https://www.accessdata.fda.gov/scripts/fdatrack/view/track.cfm?program=osmp&id=OSMP-OOPD-Number-orphan-drug-designations-resulted-in-orphan-designation.
5. Regulators adopt more orphan drugs