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Clin Pharmacol Ther
; 100(4): 322-3, 2016 10.
Article
in English
| MEDLINE
| ID: mdl-27326701
ABSTRACT
The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as "Rare Diseases Guidance").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.