3.1
The main evidence for the clinical effectiveness of vinflunine was from 1 open-label, phase 3, randomised controlled trial (study 302, the registration trial) that compared vinflunine plus best supportive care with best supportive care alone in patients with advanced or metastatic transitional cell carcinoma of the urothelial tract whose disease had progressed after platinum-based chemotherapy. Results from 2 open-label, single-arm, phase 2 studies (study 202 and CA001) were also provided. The manufacturer's submission highlighted issues around using best supportive care alone as the control arm in study 302. In particular, patients had to be fit enough to receive chemotherapy but willing to accept randomisation to best supportive care. The manufacturer stated that patients in the trial had a poorer prognosis, as indicated by the high percentage (approximately 74%) of patients with visceral involvement in both groups in the trial. The manufacturer also highlighted that there is currently no standard chemotherapy regimen for patients with advanced transitional cell carcinoma of the urothelial tract whose disease has progressed after a prior platinum-containing chemotherapy, and there is a lack of trial evidence of survival advantage from chemotherapy in this clinical situation. Therefore, no standard active treatments were available to use as a control, and best supportive care was considered the most appropriate comparator for vinflunine.