Table 3.
Characteristics of clinical postmarketing requirements and commitments for new therapeutics receiving original Food and Drug Administration approval, 2009–2018.
| PMR or PMC authority, No. (%) | Total clinical PMRs or PMCsb | PMR/PMC study indication | Study design | Study objective | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Original indication | Modified indication | New indication | Uncleard | Trial | Observational | Safety | Efficacy | Safety and Efficacy | ||||
| General population | Subgroupc | |||||||||||
| Total | 750 | 150 (20.0) | 305 (40.7) | 123 (16.4) | 61 (8.1) | 111 (14.8) | 576 (76.8) | 174 (23.2) | 330 (44.0) | 106 (14.1) | 314 (41.9) | |
| PMR | FDAAA | 331 | 94 (28.4) | 61 (18.4) | 48 (14.5) | 23 (6.9) | 105 (31.7) | 173 (52.3) | 158 (47.7) | 258 (77.9) | 8 (2.4) | 65 (19.6) |
| PREA | 207 | 0 (0) | 166 (80.2) | 19 (9.2) | 18 (8.7) | 4 (1.9) | 207 (100) | 0 (0) | 64 (30.9) | 3 (1.4) | 140 (67.6) | |
| AA | 59 | 19 (32.2) | 10 (16.9) | 24 (40.7) | 6 (10.2) | 0 (0) | 58 (98.3) | 1 (1.7) | 1 (1.7) | 34 (57.6) | 24 (40.7) | |
| AER | 3 | 3 (100.0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 3 (100.0) | 0 (0) | 0 (0) | 3 (100.0) | |
| PMC | 506Ba | 141 | 34 (24.1) | 66 (46.8) | 29 (20.6) | 10 (7.1) | 2 (1.4) | 129 (91.5) | 12 (8.5) | 7 (5.0) | 58 (41.1) | 76 (53.9) |
| Non-506Ba | 9 | 0 (0) | 2 (22.2) | 3 (33.3) | 4 (44.4) | 0 (0) | 9 (100.0) | 0 (0) | 0 (0) | 3 (33.3) | 6 (66.7) | |
PMR: postmarketing requirement; PMC: postmarketing commitment; FDAAA: Food and Drug Administration Amendments Act; PREA: Pediatric Research Equity Act; AA: Accelerated Approval; AER: Animal Efficacy Rule.
“506B” refers to postmarketing commitments subject to reporting requirements under section 506B of the Federal Food, Drug, and Cosmetic Act. Postmarketing commitments not subject to this rule are denoted as “Non-506B.”
“Clinical PMRs or PMCs” include those outlining new prospective cohort studies, registries, or clinical trials; completion or submission of results from ongoing prospective clinical studies; new retrospective observational studies; or new analysis, follow-up, or flexible analyses of clinical studies.
Postmarketing requirements and commitments generating evidence on subgroups of the original indication may evaluate demographic subgroups, clinical subgroups, or both.
“Unclear” indications were those for which a defined disease population could not be determined from postmarketing requirement or commitment description and, if identified, corresponding study registration on ClinicalTrials.gov.