854P - Sunitinib in Advanced RCC: Cost-Effectiveness Evaluation Based on Clinical Practice

Abstract

ABSTRACT The approval trials have established sunitinib, multi-targeted TKI, a standard treatment in patients with metastatic RCC. RCTs may fail to show relevant clinical benefit in wider population and routine use, as certain patient subtypes are under-represented, notably those with poorer prognostic factors and pretreated. The data were collected through national registry Onco-AIFA as part of the mandatory surveillance. RCC patients receiving sunitinib once daily, on schedule 4/2, between 2007 and 2011, had been checked prospectively for toxicity, clinical outcomes and length of treatment. Based on the difference in effectiveness in PFS between approval RCT and clinical practice, a new price adjusted for effectiveness has been calculated. A total of 106 patients (64 years, M 70/F 36) were reviewed, 77% had prior nephrectomy and 74% were treatment-naive. At first evaluation we found 28% partial responses, 25% stable diseases, 45% progressions and one discontinuation due to toxicity. The median PFS and OS were 7 and 11.3 months, respectively. Among 79 RCC patients (pts) with metastases (mets) at first diagnosis, 63% had lung mets, 21% pts had liver mets, 21% had bone mets and 9% had brain mets. Cox regression model showed in subgroups analysis significantly worse survival prognosis in males, brain and liver mets, ECOG PS > 1, non-clear cell histology and non prior nephrectomy. Sorafenib treatment after progression on sunitinib (33% pts) did not improved OS. Grade 3 thrombocytopenia, neutropenia, mucositis and HFS caused dosage reductions. Effectiveness adjusted price was 3,87 euro/mg as opposed to the ex-factory price of 2,46 euro/mg proportionally to the difference of PFS form RCTs (11 months) and that form clinical practice oncology (7 months). The results show that sunitinib clinical benefit in RCC patients was gained in selected responders, but in overall the effectiveness was nearly half of that reported in the approval RCTs. Evaluating cost-effectiveness ratio, price should be at least 36% lower than actual ex-factory price based on net clinical benefit achieved in real life practice. Post-marketing studies in real life practice are required in order to verify both effectiveness and safety in general population, testing for external validity of randomized trials. Disclosure All authors have declared no conflicts of interest.