Fordham Law Review Volume 86 Issue 4 Article 15 2018 Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs Julien B. Bannister Fordham University School of Law Follow this and additional works at: https://ir.lawnet.fordham.edu/flr Recommended Citation Julien B. Bannister, Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs, 86 Fordham L. Rev. 1889 (2018). Available at: https://ir.lawnet.fordham.edu/flr/vol86/iss4/15 This Note is brought to you for free and open access by FLASH: The Fordham Law Archive of Scholarship and History. It has been accepted for inclusion in Fordham Law Review by an authorized editor of FLASH: The Fordham Law Archive of Scholarship and History. For more information, please contact
[email protected]. Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs Erratum Law; Administrative Law; Agency; Legislation; Food and Drug Law; Medical Jurisprudence; Law and Society This note is available in Fordham Law Review: https://ir.lawnet.fordham.edu/flr/vol86/iss4/15 NOTES REGULATING RARE DISEASE: SAFELY FACILITATING ACCESS TO ORPHAN DRUGS Julien B. Bannister* While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment. Congressional and regulatory efforts to stimulate the development of rare-disease treatments, while laudable, have not resolved the fundamental issues surrounding rare-disease treatment development. Indeed, small patient populations, incomplete scientific understanding of rare diseases, and high development costs continually limit the availability of rare-disease treatments. To illustrate the struggle of developing and approving safe rare-disease treatments, this Note begins by discussing the approval of Eteplirsen, the first drug approved for treating a rare disease called Duchenne muscular dystrophy.