Drug | Initial FDA indication | Trajectories launched | Trajectories recommended by NCCN | Trajectories advanced to RCT | Trials (k) | Patients (n) | Grade 3–4 SAE, n (%) (95% CI) | Grade 5 SAE, n (%) (95% CI) |
Dasatinib | Chronic myeloid leukaemia | 12 | 0 | 0 | 18 | 763 | 96 (14.1%) (9.7% to 20%) | 0 |
Decitabine | Myelodysplastic syndromes | 2 | 0 | 0 | 2 | 59 | 18 (33.9%) (8.7% to 73%) | 1 (3.1%) (0.63% to 14%) |
Ixabepilone | Breast cancer | 1 | 0 | 0 | 1 | 85 | 13 (15.3%) (9.1 to 25%) | 5 (5.9%) (2.5% to 13%) |
Lapatinib | Breast cancer | 1 | 0 | 0 | 1 | 18 | 1 (5.6%) (0.78% to 31%) | – |
Lenalidomide | Myelodysplastic syndromes | 6 | 0 | 2 | 7 | 241 | 73 (30.1%) (16% to 49%) | 1 (1.8%) (0.66% to 4.6%) |
Nelarabine | T-cell acute lymphoblastic leukaemia/T-cell lymphoblastic lymphoma | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Nilotinib | Chronic myeloid leukaemia | 1 | 0 | 0 | 3 | 73 | 6 (9.4%) (4.2% to 19%) | 0 |
Panitumumab | Colorectal carcinoma | 2 | 0 | 1 | 2 | 68 | 8 (14.0%) (4% to 39%) | 1 (2.2%) (0.45% to 10%) |
Sorafenib | Renal cell carcinoma | 10 | 3 | 0 | 12 | 394 | 62 (16.7%) (11% to 25%) | 0 |
Sunitinib | Renal cell carcinoma/gastrointestinal stromal tumour | 25 | 2 | 4 | 47 | 2652 | 524 (21.7%) (18% to 26%) | 44 (2.9%) (2.2% to 3.7%) |
Temsirolimus | Renal cell carcinoma | 5 | 0 | 3 | 7 | 214 | 37 (18.7%) (11% to 29%) | 7 (5.4%) (2.5% to 11%) |
Vorinostat | Cutaneous T-cell lymphoma | 4 | 0 | 1 | 4 | 132 | 37 (21.8%) (7.6% to 49%) | 0 |
All drugs | 69 | 5 | 11 | 104 | 4699 | 875 (19.6%) (17% to 22%) | 59 (2.8%) (2.3% to 3.4%) |
NCCN, National Comprehensive Cancer Network; RCT, randomised controlled trial; SAE, severe adverse event.