Drugs Against Rare Diseases: Are The Regulatory Standards Higher?
The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as "Rare Diseases Guidance"). In our opinion, the FDA should consider: (a) explicitly acknowledging the standards are higher for rare diseases for the reasons presented in this article; and (b) illustrating innovative development pathways that may be acceptable for rare diseases, including case studies.
University of Maryland, School of Pharmacy, 20 N. Pine Street, Baltimore, Maryland, USA. jgobburu@rx.umaryland.edu.
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Gobburu
University of Maryland, School of Pharmacy, 20 N. Pine Street, Baltimore, Maryland, USA.
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Pastoor
D017277:Drug Approval; D017408:Guidelines as Topic; D006801:Humans; D009965:Orphan Drug Production / Q000592:standards*; D035583:Rare Diseases / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards*