1. European Medicines Agency. Orphan designation: overview. https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview. Accessed 10 June 2021.
2. Kesselheim AS. B, Innovation and the Orphan Drug Act, 1983–2009: regulatory and clinical characteristics of approved orphan drugs. In: Field MJ, Boat TF, editors. Rare diseases and orphan products: accelerating research and development. Washington, DC: National Academies Press (US); 2010. p. 291–308.
3. Swann J. The story behind the Orphan Drug Act. 2018. https://www.fda.gov/industry/orphan-products-development-events/story-behind-orphan-drug-act. Accessed 15 Feb 2022.
4. European Medicines Agency. Legal framework: orphan designation. https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation/legal-framework-orphan-designation. Accessed 15 Feb 2022.
5. Brown DG, Wobst HJ. A decade of FDA-approved drugs (2010–2019): trends and future directions. J Med Chem. 2021;64(5):2312–38.