Table 1 List and Main Characteristics of Included FDA Drug Approvals (n = 35).
Drug | Biomarker gene | Indication | Original (O) or supplemental (S) approval | Accelerated Approval or regular | No. of trials | No. of non-enriched trials* | No. of RCTs | No. of Trials with OS/PFS | No. of patients enrolled in trials |
|---|---|---|---|---|---|---|---|---|---|
Ado-Trastuzumab Emtansine | ERBB2 | Breast cancer | O | regular | 4 | 0 | 1 | 2 | 1237 |
Afatinib | EGFR | Lung cancer | O | regular | 6 | 0 | 2 | 6 | 2329 |
Anastrozole | ESR1, PGR | Breast cancer (early stage) | S | accelerated | 1 | 1 | 1 | 1 | 9366 |
Anastrozole | ESR1, PGR | Breast cancer (advanced stage) | S | regular | 1 | 0 | 1 | 0 | 668 |
Cetuximab | EGFR | Colorectal cancer | O | accelerated | 3 | 0 | 1 | 3 | 525 |
Cetuximab | KRAS | Colorectal cancer | S | NA | 5 | 5 | 5 | 5 | 4141 |
Crizotinib | ALK | Lung cancer | O | accelerated | 2 | 1 | 0 | 0 | 302 |
Dabrafenib | BRAF | Melanoma (single agent) | O | regular | 3 | 0 | 1 | 3 | 514 |
Dabrafenib | BRAF | Melanoma (with trametinib) | S | accelerated | 1 | 0 | 1 | 0 | 162 |
Dasatinib | BCR/ABL1 | Blood cancer | O | accelerated | 2 | 0 | 0 | 0 | 46 |
Denileukin Diftitox | IL2RA | Other | O | accelerated | 2 | 0 | 1 | 0 | 106 |
Erlotinib | EGFR | Lung cancer | S | regular | 1 | 0 | 1 | 1 | 173 |
Everolimus | ERBB2 | Breast cancer | S | regular | 1 | 0 | 1 | 1 | 724 |
Everolimus | ESR1 | Breast cancer | S | regular | 1 | 0 | 1 | 1 | 724 |
Exemestane | ESR1 | Breast cancer | S | regular | 1 | 1 | 1 | 1 | 4724 |
Fulvestrant | ESR1 | Breast cancer | O | regular | 2 | 2 | 2 | 2 | 924 |
Imatinib | KIT | Blood cancer | S | regular | 2 | 2 | 0 | 0 | 30 |
Imatinib | BCR/ABL1 | Blood cancer (Ph + ALL, adult patients) | S | regular | 2 | 0 | 0 | 0 | 55 |
Imatinib | BCR/ABL1 | Blood cancer (Ph + ALL, Pediatric patients in combination with chemotherapy) | S | accelerated | 3 | 0 | 0 | 3 | 64 |
Imatinib | PDGFRB | Blood cancer | S | regular | 2 | 2 | 0 | 0 | 31 |
Lapatinib (with capecitabine) | ERBB2 | Breast cancer | O | regular | 3 | 0 | 1 | 1 | 631 |
Lapatinib (with letrozole) | ERBB2 | Breast cancer | S | accelerated | 1 | 1 | 1 | 1 | 1286 |
Letrozole | ESR1, PGR | Breast Cancer (early stage) | S | accelerated | 1 | 0 | 1 | 1 | 8010 |
Letrozole | ESR1, PGR | Breast cancer (advanced stage) | O | regular | 3 | 0 | 3 | 1 | 2013 |
Panitumumab | EGFR | Colorectal cancer | O | accelerated | 1 | 0 | 1 | 1 | 463 |
Panitumumab | KRAS | Colorectal cancer | S | regular | 2 | 2 | 2 | 2 | 1292 |
Pertuzumab | ERBB2 | Breast cancer (Metastatic) | O | regular | 2 | 0 | 1 | 2 | 903 |
Pertuzumab | ERBB2 | Breast cancer (Neo-adjuvant) | S | accelerated | 2 | 0 | 2 | 0 | 642 |
Trametinib | BRAF | Melanoma | O | regular | 3 | 1 | 1 | 3 | 516 |
Trastuzumab | ERBB2 | Metastatic Breast cancer | O | regular | 7 | 0 | 1 | 0 | 1163 |
Trastuzumab | ERBB2 | Breast cancer, adjuvant | S | regular | 2 | 0 | 2 | 2 | 3987 |
Trastuzumab | ERBB2 | metastatic gastric adenocarcinoma | S | regular | 1 | 0 | 1 | 1 | 594 |
Vemurafenib | BRAF | Melanoma | O | regular | 3 | 0 | 1 | 1 | 856 |
Ceritinib | ALK | Lung cancer | O | accelerated | 2 | 0 | 0 | 0 | 266 |
Lenalidomide | del (5q) | Blood cancer | O | regular | 2 | 1 | 0 | 0 | 191 |
