Table 3.
Designs of 97 dose investigation studies planned by 79 paediatric development programmes. Listed are the median and interquartile range (IQR) of the median ages to be recruited in the different studies (or the median ages study conclusions apply to in the case of extrapolation exercises)
| 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 | ||