Table 2 Characteristics of orphan drugs approved by the FDA from 1999 to 2018
Characteristics | Number (%) |
|---|---|
Novel orphan drugs | 214 |
Follow-up years since approval [median (IQR)] | 6.7 (3.0–12.6) |
Therapeutic area | |
Antineoplastic and immunomodulating agents | 99 (46.3) |
Alimentary tract and metabolism | 26 (12.1) |
Nervous system | 16 (7.5) |
Blood and blood forming organs | 13 (6.1) |
Various | 13 (6.1) |
Cardiovascular system | 9 (4.2) |
Systemic hormonal preparations, excl. sex hormones | 9 (4.2) |
Anti-infective for systemic use | 8 (3.7) |
Musculo-skeletal system | 8 (3.7) |
Antiparasitic products, insecticides and repellents | 6 (2.8) |
Respiratory system | 5 (2.3) |
Sensory organs | 1 (0.5) |
Therapeutic radiopharmaceuticals | 1 (0.5) |
Approval status | Â |
Priority review | 169 (79.0) |
Accelerated approvals | 55 (25.7) |
Breakthrough therapy | 53 (24.8) |
Approved with boxed warning | 63 (29.4) |
For long-term use | 73 (34.1) |
All postmarketing safety events up to 31 December 2019 | |
Number of safety events | 641 |
Withdrawal | 0 (0) |
Suspended marketing | 1 (0.16) |
Boxed warning | 48 (7.49) |
Contraindications | 50 (7.80) |
Drug interactions | 68 (10.61) |
Warnings and precautions | 453 (70.67) |
Adverse reactions | 443 (69.11) |