Table 1.
Total Number and Distribution of Clinical Studies and Study Participants for Each Agent According to 3 Levels of Review
| Identified studies | Level 1: Availability of randomized controlled trials | Level 2: Availability of randomized controlled trial in FDA‐approved indication | Level 3: Availability of randomized controlled trial evaluating agent's effectiveness | |||||
|---|---|---|---|---|---|---|---|---|
| Accelerated approval agents | Total number of clinical studies identified | Number of participants included | Number of randomized trials (%) | Number of participants (%) | Number of trials (%) | Number of participants (%) | Number of “evaluation” trials (%) | Number of participants (%) |
| Agalsidase | 36 | 11,437 | 7 (19.4) | 10,541 (92.2) | 4 (100.0) | 193 (100.0) | 4 (100.0) | 193 (100.0) |
| Alemtuzumab | 326 | 26,962 | 59 (18.1) | 16,136 (59.8) | 8 (20.0) | 969 (11.9) | 7 (87.5) | 923 (95.3) |
| Bedaquiline | 28 | 6,594 | 18 (64.3) | 2,661 (40.4) | 6 (50.0) | 781 (77.0) | 4 (66.7) | 436 (55.8) |
| Bortezomib | 804 | 79,503 | 168 (20.9) | 35,936 (45.2) | 67 (59.3) | 22,388 (78.8) | 28 (41.8) | 7,462 (33.3) |
| Brentuximab | 107 | 9,256 | 16 (14.9) | 4,096 (44.3) | 6 (100.0) | 685 (100.0) | 3 (50.0) | 491 (71.7) |
| Carfilzomib | 121 | 11,882 | 17 (14.0) | 6,076 (51.1) | 6 (87.5) | 2,996 (98.9) | 4 (66.7) | 2,918 (97.4) |
| Cetuximab | 750 | 102,336 | 257 (34.3) | 31,333 (30.6) | 75 (42.4) | 29,870 (57.3) | 41 (54.7) | 24,700 (82.7) |
| Clofarabine | 156 | 18,312 | 21 (13.5) | 5,728 (31.3) | 1 (10.0) | 405 (11.5) | 0 (0.0) | 0 (0.0) |
| Crizotinib | 102 | 23,216 | 24 (23.5) | 17,116 (73.7) | 9 (64.3) | 1,997 (94.7) | 5 (55.6) | 1,227 (61.4) |
| Darunavir | 206 | 65,040 | 112 (54.4) | 40,580 (62.4) | 64 (73.6) | 17,350 (91.3) | 24 (37.5) | 6,931 (39.9) |
| Deferasirox | 90 | 11,339 | 25 (27.8) | 8,391 (74.0) | 10 (35.7) | 1,000 (48.9) | 8 (80.0) | 1,746 (95.6) |
| Deferiprone | 57 | 7,345 | 35 (61.4) | 2,082 (28.3) | 7 (29.2) | 526 (18.9) | 4 (57.1) | 233 (44.3) |
| Eltrombopag | 102 | 8,614 | 33 (32.4) | 3,922 (45.5) | 8 (30.8) | 736 (20.5) | 8 (100.0) | 736 (100.0) |
| Enfuvirtide | 67 | 7,294 | 30 (44.8) | 3,718 (50.9) | 21 (77.8) | 2,956 (91.6) | 11 (52.4) | 863 (30.0) |
| Etravirine | 79 | 18,824 | 32 (40.5) | 14,142 (75.1) | 19 (67.9) | 3,680 (92.4) | 14 (73.7) | 2,777 (75.5) |
| Gefitinib | 366 | 55,600 | 118 (32.2) | 28,174 (50.7) | 47 (58.8) | 16,934 (80.3) | 35 (74.5) | 12,793 (77.9) |
| Gemtuzumab | 73 | 15,908 | 19 (26.0) | 5,757 (36.2) | 13 (100.0) | 6,759 (100.0) | 11 (84.6) | 6,490 (96.0) |
| Ibritumomab | 105 | 10,615 | 17 (16.2) | 7,526 (70.9) | 10 (100.0) | 1,446 (100.0) | 8 (80.0) | 1,432 (99.0) |
| Ibrutinib | 165 | 25,195 | 27 (16.4) | 18,019 (71.5) | 2 (14.3) | 804 (17.9) | 2 (100.0) | 804 (100.0) |
| Imatinib | 631 | 82,090 | 132 (20.9) | 50,049 (60.9) | 36 (37.9) | 9,776 (47.5) | 24 (66.7) | 7,433 (76.0) |
| Lenalidomide | 696 | 82,986 | 161 (23.1) | 35,899 (43.3) | 7 (6.9) | 1,163 (3.5) | 5 (71.4) | 872 (75.0) |
| Lopinavir | 365 | 109,939 | 210 (57.5) | 52,313 (47.6) | 133 (75.1) | 43,016 (84.0) | 64 (48.1) | 16,615 (40.2) |
| Maraviroc | 140 | 20,631 | 69 (49.3) | 9,708 (47.1) | 42 (73.7) | 7,584 (86.2) | 28 (66.7) | 5,657 (74.6) |
| Natalizumab | 95 | 67,003 | 27 (28.4) | 61,079 (91.2) | 14 (73.7) | 4,230 (81.1) | 13 (92.9) | 4,088 (96.6) |
| Nelarabine | 23 | 4,154 | 2 (8.7) | 1,534 (36.9) | 1 (100.0) | 1,900 (100.0) | 1 (100.0) | 1,900 (100.0) |
| Nilotinib | 167 | 26,758 | 33 (19.8) | 19,316 (72.2) | 10 (47.6) | 2,258 (64.1) | 6 (60.0) | 2,136 (94.6) |
| Ofatumumab | 114 | 12,450 | 28 (24.6) | 4,145 (33.3) | 8 (44.4) | 2,641 (61.2) | 7 (87.5) | 2,371 (89.8) |
| Omacetaxine | 18 | 903 | 1 (5.6) | 898 (99.4) | 1 (100.0) | 5 (100.0) | 0 (0.0) | 0 (0.0) |
| Oxaliplatin | 1,417 | 257,595 | 536 (37.8) | 56,832 (22.1) | 183 (69.3) | 99,762 (87.0) | 36 (19.7) | 23,378 (23.4) |
| Panitumumab | 198 | 28,500 | 64 (32.3) | 11,010 (38.6) | 21 (50.0) | 7,695 (68.2) | 12 (57.1) | 6,994 (90.9) |
| Pomalidomide | 110 | 10,977 | 27 (24.5) | 6,373 (58.1) | 8 (53.3) | 1,414 (65.5) | 5 (62.5) | 920 (65.1) |
| Ponatinib | 30 | 3,177 | 4 (13.3) | 1,720 (54.1) | 1 (100.0) | 307 (100.0) | 1 (100.0) | 307 (100.0) |
| Pralatrexate | 29 | 1,388 | 5 (17.2) | 708 (51.0) | 4 (80.0) | 786 (79.6) | 2 (50.0) | 137 (28.6) |
| Raltegravir | 297 | 49,113 | 153 (51.5) | 24,253 (49.4) | 89 (78.1) | 18,701 (94.0) | 55 (61.8) | 15,439 (82.6) |
| Tenofovir | 716 | 234,714 | 436 (60.9) | 82,261 (35.0) | 212 (66.0) | 71,525 (67.5) | 38 (17.9) | 12,098 (17.2) |
| Tipranavir | 82 | 13,849 | 32 (39.0) | 8,921 (64.4) | 17 (56.7) | 3,455 (83.5) | 7 (41.2) | 1,185 (34.3) |
| Treprostinil | 83 | 13,997 | 40 (48.2) | 10,402 (74.3) | 13 (56.5) | 1,476 (77.0) | 9 (69.2) | 1,448 (98.1) |