Table 2.
Guidance on preclinical efficacy in EMA and FDA TAGs: distribution and specification
| Therapeutic area guidelines (TAGs) | EMA (N = 114) | FDA (N = 120) |
|---|---|---|
| Any guidance on preclinical efficacy | 29 (25%) | 50 (42%) |
| Types of preclinical efficacy study design elements | No. of TAGs | Rating of guidance | No. of TAGs | Rating of guidance | ||
|---|---|---|---|---|---|---|
| Generala | Specifica | Generala | Specifica | |||
| Efficacy models | 10 (9%) | 3 (30%) | 7 (70%) | 30 (25%) | 12 (40%) | 18 (60%) |
| Effect characteristicsb | 14 (12%) | 9 (64%) | 5 (36%) | 19 (16%) | 13 (68%) | 6 (32%) |
| Dosing characteristics AND/OR administrationc | 12 (11%) | 5 (42%) | 7 (58%) | 29 (24%) | 15 (52%) | 14 (48%) |
| Receptor binding and specificity | 0 (0%) | 0 (0%) | 0 (0%) | 5 (4%) | 2 (40%) | 3 (60%) |
| Otherd | 6 (5%) | 2 (33%) | 4 (67%) | 15 (13%) | 5 (33%) | 10 (67%) |
a
Supporting Information Table S2 gives examples for how guidelines' guidance on preclinical efficacy was rated as general or specific.
b
For example, nature, frequency, intensity of pharmacological effects, time to onset or duration of effects.
c
For example, dose duration, dose interval, administration route, dose response or comparison of nontoxic dose findings.
d
Other preclinical efficacy studies, for example, special studies to assess pharmacological actions other than the intended therapeutic effects.