Table 2.
Distribution of ATC-1 groups based on the biotechnological and small-molecule status of drugs in rare disease applications.
| ATC-1 code | Biotechnological drugs | Small-molecule drugs | ||||
|---|---|---|---|---|---|---|
| n, (%) | Mean age ± SD | The most frequent drug, (%) | n, (%) | Mean age ± SD | The most frequent drug, (%) | |
| A | 103, (6.0) | 9.1 ± 3.7# | Elosulfase alfa, (89.3) | 454, (16.2) | 5.0±4.5 | Sapropterin, (42.5) |
| B | 92, (5.4) | 8.8 ± 5.2 | Coagulation factor VIIa, (32.6) | 242, (8.7) | 9.2±4.9 | Iloprost, (59.1) |
| C | - | - | - | 219, (7.8) | 8.2±5.3 | Bosentan, (33.8) |
| D | - | - | - | 73, (2.6) | 4.5±3.3 | Isotretinoin, (95.9) |
| G | - | - | - | 161, (5.8) | 8.6±5.4 | Sildenafil, (54.0) |
| H | 172, (10.1) | 9.3 ± 4.3 | Somatropin, (99.4) | 100, (3.6) | 9.2±5.2 | Hydrocortisone, (28.0) |
| J | 60, (3.5) | 8.6 ± 5.7# | Intravenous immunoglobulin, (86.7) | 216, (7.7) | 6.4±5.2 | Valganciclovir, (37.5) |
| L | 1248, (73.2) | 9.8 ± 5.0 | Canakinumab, (20.8) | 923, (33.0) | 10.0±4.9 | Mycophenolate, (29.6) |
| M | 12, (0.7) | 12.6 ± 5.0# | Denosumab, (100,0) | 128, (4.6) | 8.8±3.8 | Ataluren, (35.2) |
| N | - | - | - | 68, (2.4) | 9.2±4.9 | Trihexyphenidyl, (22.1) |
| P | - | - | - | 16, (0.6) | 1.9±3.6 | Pyrimethamine, (100.0) |
| R | 13, (0.8) | 9.3 ± 5.9 | Omalizumab, (53.8) | 52, (1.9) | 7.9±5.1 | Mannitol, (51.9) |
| S | - | - | - | 3, (0.1) | 11.3±9.1 | Dexamethasone, (66.7) |
| V | - | - | - | 126, (4.5) | 6.6±5.5 | Calcium folinate, (52.4) |
| Others* | 4, (0.2) | 10.5 ± 4.8 | - | 16, (0.6) | 7.5±6.0 | - |
| Total | 1704, (100.0) | 9.7 ± 4.9# | Canakinumab, (15.3) | 2797, (100.0) | 8.1±5.3 | Mycophenolate, (9.8) |
A, Alimentary tract and metabolism; B, Blood and blood-forming organs; C, Cardiovascular system; D, Dermatological; G, Genito urinary system and sex hormones; H, Systemic hormonal preparations, excl. sex hormones and insulins; J, Antiinfectives for systemic use; L, Antineoplastic and immunomodulating agents; M, Musculoskeletal system; N, Nervous system; P, Antiparasitic products, insecticides, and repellents; R, Respiratory system; S, Sensory organs; V, Various; *, Applications that have no defined ATC code. # p < 0.05 biotechnological vs. conventional drugs group.