Background In recent years, due to scarcity of evidence at the time of registration, approved indications/patient characteristics for anticancer drugs closely resemble those of the pivotal RCTs. At the same time, many authors complain it is hard to transfer the results of clinical trials to clinical practice, due to the high selectivity of patient eligibility criteria. In Italy, at the time of marketing, the majority of new anticancer drugs are subject to a compulsory electronic outcome registry called ‘oncoAIFA’. In order to prescribe these drugs and have them dispensed, clinicians need to enter the patient's clinical profile, to prove they correspond to approved indications, and each prescription. Subsequently, hospital pharmacists record every prescription they dispense. At the end of the treatment, physicians need to report the patient's outcome.

Purpose To compare baseline characteristics and outcomes of clinical trial patients with the one of a cohort of patients treated with new drugs for renal cell carcinoma, sorafenib and sunitinib, in the Veneto Region (North East of Italy, 4.9 million inhabitants).

Materials and methods Pivotal clinical trials for sorafenib and sunitinib for the indication ‘renal cell carcinoma’ were selected. Data of the Veneto Region patients treated with sorafenib and sunitinib were extracted from the oncoAIFA register for the period January 2007-March 2011. Baseline characteristics were compared between clinical trials and clinical practice: gender, age, ECOG performance status, number of metastatic organs. The outcome compared was the proportion of patients with disease progression or death.

Results In the Veneto Region, 209 patients were treated with sorafenib and 570 with sunitinib. For sorafenib, baseline characteristics were similar for gender (% male: 70% RCT, 70% register), ECOG performance status (% ECOG zero: 49% RCT, 49% register). Relevant differences were found for age (median 58 years RCT, 67 years register), number of metastatic sites (% > 2: 57% RCT, 27% register), and previous cytokines use (% yes: 83% RCT, 57% register). For sunitinib, gender (% male: 71% RCT, 69% register), median age (62 years RCT, 66 years register), and ECOG performance status (% ECOG zero: 62% RCT, 56% register) were similar, while the two populations differed greatly for number of metastatic sites (% >= 3: 57% for RCT, 18% for register). Regarding outcome, 38.1% of patients experienced disease progression or death in the sorafenib trial versus 58% in real life; this proportion was 21% in the sunitinib trial versus 46% in the register.

Conclusions Although approved indications for new drugs often resemble RCT patient characteristics, patients treated in clinical practice differ from the study populations. This difference is also described in patient outcomes.