Table 1 Characteristics of orphan drugs
Single-arm trial (n=82) | Randomized controlled trial (n=151) | Total (n=233) | ||
|---|---|---|---|---|
Approval year | ||||
2001–2005 | 5 (6.1%) | 17 (11.3%) | 22 | |
2006–2010 | 9 (11.0%) | 18 (11.9%) | 27 | |
2011–2015 | 28(34.1%) | 42 (27.8%) | 70 | |
2016–2021 | 40 (48.8%) | 74 (49.0%) | 114 | |
Prevalence | ||||
1–5 / 10,000 | 38 (46.3%) | 66 (43.7%) | 104 | |
1–9 / 100,000 | 31 (37.8%) | 59 (39.1%) | 90 | |
1–9 / 1,000,000 | 6 (7.3%) | 21 (13.9%) | 27 | |
< 1 / 1,000,000 | 7 (8.5%) | 5 (3.3%) | 12 | |
Modality | ||||
Chemical | 52 (63.4%) | 92 (60.9%) | 144 | |
Biologics | 30 (36.6%) | 59 (39.1%) | 89 | |
ATC code | ||||
A (Alimentary tract and metabolism) | 6 (7.3%) | 20 (13.2%) | 26 | |
B (Blood and blood forming organs) | 9 (11.0%) | 20 (13.2%) | 29 | |
C (Cardiovascular system) | 1 (1.2%) | 8 (5.3%) | 9 | |
D (Dermatologicals) | 0 | 2 (1.3%) | 2 | |
G (Genito urinary system and sex hormones) | 1 (1.2%) | 0 | 1 | |
H (Systematic hormonal preparations, excl. sex hormones and insulins) | 0 | 7 (4.6%) | 7 | |
J (Antiinfectives for systemic use) | 0 | 3 (1.3%) | 3 | |
L (Antineoplastic and immunomodulating agents) | 64 (78.0%) | 52 (34.4%) | 116 | |
M (Musculo-skeletal system) | 1 (1.2%) | 8 (5.3%) | 9 | |
N (Nervous system) | 0 | 17 (11.3%) | 17 | |
P (Antiparasitic products, insecticides and repellents) | 0 | 5 (3.3%) | 5 | |
R (Respiratory system) | 0 | 4 (2.6%) | 4 | |
S (Sensory organs) | 0 | 3 (2.0%) | 3 | |
V (Various) | 0 | 2 (1.3%) | 2 | |
Target age segment | ||||
Children with or without adult | 24 (29.3%) | 55 (36.4%) | 79 | |
Adult only | 58 (70.7%) | 96 (63.6%) | 154 | |
Primary efficacy endpoint | ||||
Pharmacodynamic/Response biomarker | 69 (84.1%) | 67 (44.4%) | 136 | |
Clinical outcome | 13 (15.9%) | 84 (55.6%) | 97 | |
Severity of the disease outcome | ||||
High mortality | 67 (81.7%) | 67 (44.4%) | 134 | |
Others | 15 (18.3%) | 84 (55.6%) | 99 | |
Prior approval outside of US | ||||
Yes | 9 (11.0%) | 34 (22.5%) | 43 | |
No | 73 (89.0%) | 117 (77.5%) | 190 | |
Alternative treatment in US | ||||
Not available | 25 (30.5%) | 51 (33.8%) | 76 | |
Exists | 57 (69.5%) | 100 (66.2%) | 157 | |
Drug usage | ||||
Mono therapy | 78 (95.1%) | 128 (84.8%) | 206 | |
Combination therapy | 4 (4.9%) | 23 (15.2%) | 27 | |
Designation | ||||
Fast track | ||||
Yes | 17 (20.7%) | 51 (33.8%) | 68 | |
No | 65 (79.3%) | 100 (66.2%) | 165 | |
Breakthrough therapy | ||||
Yes | 31 (37.8%) | 38 (25.2%) | 69 | |
No | 51 (62.2%) | 113 (74.8%) | 164 | |
Priority reivew | ||||
Yes | 52 (63.4%) | 87 (57.6%) | 139 | |
No | 30 (36.6%) | 64 (42.4%) | 94 | |
Accelerated approval | ||||
Yes | 43 (52.4%) | 31 (20.5%) | 74 | |
No | 39 (47.6%) | 120 (79.5%) | 159 | |