Table 4 Multivariate relative risks of an application for an orphan designation at EMA or FDA
From: Drug development for exceptionally rare metabolic diseases: challenging but not impossible
Variable | Univariate RR (95% CI) | Multivariate RR (95% CI) |
|---|---|---|
Prevalence | ||
1-9/1.000.000 | 5.0 (2.7–9.2) | 2.8 (1.4–5.4) |
<1/1.000.000 | Ref | Ref |
Preclinical proof of concept? | ||
Yes | 6.0 (3.0–12.0) | 3.9 (1.9–8.3) |
No | Ref | Ref |