Table 3.
Dose investigation study design | Number of studies (by rarity of disease) | Median age (years) (IQR) | Median number of patients per study (IQR) | |
---|---|---|---|---|
Orphan | Non-orphan | |||
Sequential cohort dose escalation trial (with intra-subject escalation) | 2 (5.0%) | 1 (1.8%) | 15 (2.8) | 30 (18) |
Sequential cohort dose escalation trial (without intra-subject escalation) | 6 (15.0%) | 5 (8.8%) | 9.3 (0.5) | 40 (24.5) |
Within subject dose escalation trial | 4 (10.0%) | 4 (7.0%) | 11 (2.9) | 22 (15) |
Non-randomized cross-over trial | 1 (2.5%) | 0 | 9 (0) | 15 (0) |
Fixed dose strategy trial (> 1 dosing rule of novel medicine) | 5 (12.5%) | 13 (22.8%) | 11.3 (5.1) | 61.5 (93) |
Fixed dose strategy trial (1 dosing rule of novel medicine) | 15 (37.5%) | 25 (43.9%) | 9.6 (2.6) | 30 (18.5) |
TDM trial | 1 (2.5%) | 1 (1.8%) | 8 (2) | 47.5 (2.5) |
Extrapolation exercise: | 6 (15.0%) | 8 (14.0%) | 9.3 (4.6) | 0 |
• M&S study | 2 | 5 | ||
• Prospective literature review | 2 | 1 | ||
• Citation in PIP of completed paediatric dose investigation trial in current medicine | 2 | 1 | ||
• Citation in PIP of completed paediatric dose investigation trial in related medicine | 0 | 1 |