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Table 2 Characteristics of orphan drugs approved by the FDA from 1999 to 2018

From: Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 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  
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)