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Table 2 Statistical analysis of clinical, regulatory, and epidemiological factors associated with a) FDA approval for compounds intended to treat lysosomal storage disorders and b) orphan drug designation

From: Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

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%)

  1. *P-values are from Fisher’s Exact Test.