Table 2 Possible composite hypotheses of testing for effectiveness and safety
From: Innovative thinking of clinical investigation for rare disease drug development
| Â | Safety | ||
|---|---|---|---|
Na | Sb | Ec | |
Effectiveness | Â | Â | Â |
N | NN | NS | NE |
S | SN | SS | SE |
E | EN | ES | EE |
From: Innovative thinking of clinical investigation for rare disease drug development
| Â | Safety | ||
|---|---|---|---|
Na | Sb | Ec | |
Effectiveness | Â | Â | Â |
N | NN | NS | NE |
S | SN | SS | SE |
E | EN | ES | EE |
Orphanet Journal of Rare Diseases