Skip to main content

Table 1 FDA orphan drug designations and approvals by therapeutic area, 1983–2019

From: Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Therapeutic area # of designations % of total designations # with at least one approval % of designations with at least one approval
Oncology 1910 37 267 14
Neurology 674 13 69 10
Infectious diseases 436 9 79 18
Metabolism 280 5 45 16
Hematology 254 5 65 26
Transplant 207 4 14 7
Pulmonary 206 4 15 7
Gastroenterology 195 4 17 9
Ophthalmology 153 3 16 10
Endocrinology 140 3 42 30
Vascular 135 3 20 15
Rheumatology 119 2 19 16
Dermatology 94 2 5 5
Pharmacology and toxicology & poisoning and chelators 87 2 18 21
Immunology 54 1 12 22
Nephrology and urology 54 1 9 17
Cardiology 41 1 7 17
Orthopedics 31 1 3 10
Obstetrics and gynecology 17  < 1 1 6
Otolaryngology 6  < 1 0 0
Nutrition 6  < 1 1 17