Premium
Drugs Against Rare Diseases: Are The Regulatory Standards Higher?
Author(s) -
Gobburu J,
Pastoor D
Publication year - 2016
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.415
Subject(s) - food and drug administration , drug development , medicine , regulatory science , drug approval , intensive care medicine , drug , risk analysis (engineering) , pharmacology , pathology
The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as “Rare Diseases Guidance”).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.