Table 1

Sample demographics per drug

DrugInitial FDA indicationTrajectories launchedTrajectories recommended by NCCNTrajectories advanced to RCTTrials (k)Patients (n)Grade 3–4 SAE, n (%) (95% CI)Grade 5 SAE, n (%) (95% CI)
DasatinibChronic myeloid leukaemia12001876396 (14.1%) (9.7% to 20%)0
DecitabineMyelodysplastic syndromes20025918 (33.9%) (8.7% to 73%)1 (3.1%) (0.63% to 14%)
IxabepiloneBreast cancer10018513 (15.3%) (9.1 to 25%)5 (5.9%) (2.5% to 13%)
LapatinibBreast cancer1001181 (5.6%) (0.78% to 31%)
LenalidomideMyelodysplastic syndromes602724173 (30.1%) (16% to 49%)1 (1.8%) (0.66% to 4.6%)
NelarabineT-cell acute lymphoblastic leukaemia/T-cell lymphoblastic lymphoma0000000
NilotinibChronic myeloid leukaemia1003736 (9.4%) (4.2% to 19%)0
PanitumumabColorectal carcinoma2012688 (14.0%) (4% to 39%)1 (2.2%) (0.45% to 10%)
SorafenibRenal cell carcinoma10301239462 (16.7%) (11% to 25%)0
SunitinibRenal cell carcinoma/gastrointestinal stromal tumour2524472652524 (21.7%) (18% to 26%)44 (2.9%) (2.2% to 3.7%)
TemsirolimusRenal cell carcinoma503721437 (18.7%) (11% to 29%)7 (5.4%) (2.5% to 11%)
VorinostatCutaneous T-cell lymphoma401413237 (21.8%) (7.6% to 49%)0
All drugs695111044699875 (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.