We spend a lot of time talking about the major pharmacotherapeutic targets: hypertension, diabetes, hyperlipidemia, etc. But what about those disease states that aren’t quite so common? How are therapies for those conditions developed, and what kind of regulatory support exists for manufacturers? Let tl;dr shed some light on rare diseases and their orphan drug treatments!

Pharmacy School, Practice, ProfessionalStephanie KujawskiNovember 14, 2023orphan drugs, rare diseases, FDA approval of orphan drugs, CDER, ARC rare diseases, GARD rare diseases, why do drug companies develop orphan drugs