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