Table 1 Orphan drug designations and initial orphan drug approvals, by therapeutic area, 1983–2022

Oncology

38% (2405)

38% (333)

Neurology

14% (892)

10% (84)

Infectious Diseases

7% (461)

10% (90)

Metabolism

6% (370)

7% (61)

Hematology

5% (306)

8% (69)

Pulmonary

4% (280)

2% (19)

Gastroenterology

4% (243)

3% (25)

Transplant

4% (239)

2% (18)

Ophthalmology

3% (200)

2% (19)

Vascular

2% (155)

2% (21)

Rheumatology

2% (150)

3% (26)

Endocrinology

2% (147)

5% (43)

Dermatology

2% (103)

1% (8)

Pharmacology/Toxicology/Poisoning/Chelators

2% (99)

2% (22)

Nephrology/Uurology

1% (86)

2% (15)

Immunology

1% (76)

2% (14)

Cardiology

1% (50)

1% (8)

Orthopedics

1% (43)

 < 1% (4)

Obstetrics and Gynecology

 < 1% (19)

 < 1% (2)

Otorhinolaryngology

 < 1% (10)

 < 1% (0)

Nutrition

 < 1% (6)

 < 1% (1)