Orphan drug designations by risk factors
| Risk factors | ODDs with an MA (n) | R&D (n) | Failures (n) | Total | % Failures | |
| MA failures | Abandoned | |||||
| Age-related type of condition | ||||||
| Not affecting children | 24 | 102 | 8 | 45 | 179 | 29.6% |
| Affecting children | 108 | 335 | 26 | 140 | 609 | 27.3% |
| Therapeutic area | ||||||
| Cardiovascular and respiratory diseases | 10 | 37 | 3 | 25 | 75 | 37.3 |
| Dermatological diseases | 1 | 8 | 1 | 4 | 14 | 35.7 |
| Endocrine diseases | 6 | 16 | 1 | 4 | 27 | 18.5 |
| Gastrointestinal diseases | 2 | 11 | 0 | 6 | 19 | 31.6 |
| Haematologic diseases | 12 | 35 | 0 | 8 | 55 | 14.5 |
| Inborn errors of metabolism diseases | 32 | 37 | 6 | 16 | 91 | 24.2 |
| Infectious and immunitary system diseases | 8 | 58 | 1 | 19 | 86 | 23.3 |
| Neurological and psychotic diseases | 9 | 54 | 2 | 20 | 85 | 25.9 |
| Oncologic diseases | 49 | 147 | 17 | 74 | 287 | 31.7 |
| Ophthalmic diseases | 1 | 22 | 1 | 4 | 28 | 17.9 |
| Poisoning/overdose diseases | 0 | 5 | 1 | 0 | 6 | 16.7 |
| Renal, urinary and reproductive diseases | 0 | 3 | 1 | 1 | 5 | 40 |
| Others | 2 | 4 | 0 | 4 | 10 | 40 |
| Sponsor type | ||||||
| Commercial | 132 | 405 | 34 | 178 | 749 | 28.3 |
| Non-commercial | 0 | 32 | 0 | 7 | 39 | 17.9 |
| Sponsorship transferred | 40 | 117 | 16 | 53 | 226 | 30.5 |
MA, marketing authorisation; ODD, orphan drug designation; R&D, research and development.