Table 2.
Timing Between FDA Milestones for PMR Studies of New Drugs and Biologics Approved Between 2009 and 2012
| PMR | Studies (no.) | Timing between FDA milestones Months, median (IQR) [range] |
Timing of primary outcome ascertainment Months, median (IQR) [range]* |
|||
|---|---|---|---|---|---|---|
| Approval to protocol submission | Protocol submission to study completion | Study completion to final report submission | Approval to final report submission | Primary outcome | ||
| FDAAA | ||||||
| Clinical trials | 35 | 4 (3–6) [− 59–41]† | 53 (42–67) [5–104] | 6 (5–9) [− 11–36]‡ |
64 (44–80) [9–145] |
12 (3–48) [0–99] |
| Prospective cohort studies | 5 | 4 (4–5) [0–10] | 53 (50–111) [28–163] | 6 (5–7) [2–8] |
62 (56–122) [40–176] |
36 (15–60) [12–120] |
| Registries | 19 | 5 (3–9) [− 1–13] † | 86 (66–136) [6–195]‡ | 8 (6–12) [6–120]‡ |
123 (87–146) [57–221]‡ |
21 (12–72) [1–180] |
| Pediatric Research Equity Act§ | ||||||
| Clinical trials | 53 | 15 (7–37) [− 2–73]†‡ | 38 (29–51) [6–102]‡ | 6 (6–8) [2–57]‡ |
66 (55–87) [1–170] |
3 (1–6) [0–24] |
| Accelerated approval§ | ||||||
| Clinical trials | 7 | 3 (2–9) [− 30–18]† | 72 (56–80) [47–98] | 6 (6–6) [5–7] |
93 (64–95) [22–110] |
46 (14–60) [12–84] |
FDA, Food and Drug Administration; FDAAA, Food and Drug Administration Amendments Act; IQR, interquartile range; no., number; PMR, postmarketing requirement; range, full range
*Primary endpoint duration based on PMR descriptions or corresponding ClinicalTrials.gov data. Number of PMR studies excluded due to unavailable primary outcome duration: 4 FDAAA clinical trials, 6 registries; 20 PREA, 1 AA
†Protocol submission date before drug approval date: 2 FDAAA, 1 PREA, 1 AA, leading to negative duration
‡Number of PMR studies excluded due to unavailable study completion dates (1 FDAAA, 3 PREA), final report submission dates (1 FDAAA), protocol submission dates (3 PREA)
§During this period, the eligible postmarket requirements under PREA and AA were only clinical trials