Table 2.
Characteristics of FDA approvals (n = 42) and clinical trial publications leading to these approvals (n = 44)a
| Trial characteristic | No. (%) |
|---|---|
| Approved immunotherapy type (n = 42) | |
| Anti–PD-1 | |
| Pembrolizumab | 11 (26.2) |
| Nivolumab | 11 (26.2) |
| Cemiplimab | 1 (2.4) |
| Anti–PD-L1 | |
| Atezolizumab | 3 (7.1) |
| Avelumab | 2 (4.8) |
| Durvalumab | 2 (4.8) |
| Anti–PD-1 + Anti–CTLA-4 | |
| Nivolumab + ipilimumab | 3 (7.1) |
| Anti–PD-1 + chemotherapy | |
| Pembrolizumab + carboplatin + paclitaxel/nab-paclitaxel | 1 (2.4) |
| Pembrolizumab + carboplatin + pemetrexed | 1 (2.4) |
| Pembrolizumab + pemetrexed + platinum | 1 (2.4) |
| CAR-T cell therapy | |
| Tisagenlecleucel | 2 (4.8) |
| Axicabtagene ciloleucel | 1 (2.4) |
| Anti–CTLA-4 | |
| Ipilimumab | 2 (4.8) |
| PegInterferon | 1 (2.4) |
| Approval type (n = 42) | |
| Regular | 22 (52.4) |
| Accelerated | 20 (47.6) |
| Approval indication (n = 42) | |
| First line | 8 (19) |
| Second line and beyond | 30 (71.4) |
| Adjuvant | 3 (7.1) |
| Maintenance | 1 (2.4) |
| Immunotherapy drug approved alone (n = 42) | 35 (83.3) |
| Immunotherapy drug approved in combination (n = 42) | 7 (16.7) |
| Year of FDA drug approval (n = 42) | |
| 2018 | 13 (31.0) |
| 2017 | 13 (31.0) |
| 2016 | 6 (14.3) |
| 2015 | 6 (14.3) |
| 2014 | 2 (4.8) |
| 2011 | 2 (4.8) |
| Tumor type (n = 42) | |
| Non-small cell lung cancer | 12 (28.6) |
| Melanoma | 7 (16.7) |
| Urothelial cancer | 5 (11.9) |
| Large B-cell lymphoma | 3 (7.1) |
| Renal cell carcinoma | 2 (4.8) |
| Colorectal cancer | 2 (4.8) |
| Hepatocellular carcinoma | 2 (4.8) |
| Squamous cell carcinoma of the head and neck | 2 (4.8) |
| Hodgkin lymphoma | 2 (4.8) |
| Merkel cell carcinoma | 2 (4.8) |
| Cervical cancer | 1 (2.4) |
| Cutaneous squamous cell carcinoma | 1 (2.4) |
| Gastric or gastroesophageal junction adenocarcinoma | 1 (2.4) |
| Small cell lung cancer | 1 (2.4) |
| Acute lymphoblastic leukemia | 1 (2.4) |
| Phase of published trial supporting the approval (n = 44) | |
| Phase 1 | 4 (9.1) |
| Phase 1/2 | 2 (4.5) |
| Phase 2 | 21 (47.7) |
| Phase 3 | 17 (38.6) |
| Randomization status of published trial supporting the approval (n = 44) | |
| Randomized clinical trial | 23 (52.3) |
| Single-arm clinical trial | 21 (47.7) |
| Supporting trial published data on PROs (n = 44) | 21 (47.7) |
aCAR = chimeric antigen receptor; CTLA-4 = cytotoxic T lymphocyte-associated protein 4; FDA = Food and Drug Administration; PD-1 = programmed cell death protein 1; PD-L1 = programmed cell death ligand 1; PRO = patient reported outcome.