a) | |||
Characteristic | FDA approval (N = 14) | No FDA approval (N = 73) | P-value* |
Neurological endpoint | 0 (0%) | 34 (46.6%) | 0.00059 |
No neurological endpoint | 14 (100%) | 39 (53.4%) | |
Regulatory precedent | 9 (64.3%) | 31 (42.5%) | 0.15411 |
No regulatory precedent | 5 (35.7%) | 42 (57.5%) | |
Prevalence < 5/1,000,000 | 2 (14.3%) | 40 (54.8%) | 0.00742 |
Prevalence ≥ 5/1,000,000 | 12 (85.7%) | 33 (45.2%) | |
Orphan status designation | 14 (100%) | 56 (76.7%) | 0.06229 |
No orphan status designation | 0 (0%) | 17 (23.3%) | |
b) | |||
Characteristic | Orphan status (N = 70) | No orphan status (N = 17) | P-value* |
Neurological endpoint | 19 (27.1%) | 15 (88.2%) | < 0.0001 |
No neurological endpoint | 51 (72.9%) | 2 (11.8%) | |
Regulatory precedent | 39 (55.7%) | 1 (5.9%) | 0.00022 |
No regulatory precedent | 31 (44.3%) | 16 (94.1%) | |
Prevalence < 5/1,000,000 | 25 (35.7%) | 17 (100%) | < 0.0001 |
Prevalence ≥ 5/1,000,000 | 45 (64.3%) | 0 (0%) |