A new patient-focused approach to the treatment of metastatic renal cell carcinoma: establishing customized treatment options

center dot Six targeted agents - sorafenib, sunitinib, pazopanib, bevacizumab, temsirolimus and everolimus - have been approved for the treatment of advanced renal cell carcinoma (RCC) based on evidence from large randomized controlled trials (RCTs). However, no head-to-head trials have been conducted to evaluate the relative efficacy of these agents in this setting. center dot Patient populations included in clinical trials do not accurately reflect the wider population of patients with RCC, as certain subgroups, such as the elderly or those with co-morbidities, are typically under-represented. center dot The optimum choice of therapy should be based on patient characteristics, nature of disease, and history and aims of therapy; however, there is currently no clear guidance for physicians in this decision-making process. center dot A patient-focussed schema has been developed that acknowledges nine different patient-, disease-, and treatment-related factors relevant to clinical decision-making, and provides a visual indication of the strength of evidence with which a particular agent can be recommended for use in specific subgroups. center dot To demonstrate the applicability of this tool, a review of all available evidence (published articles, congress presentations and personal communications) for sorafenib in RCC was conducted by a panel of experts, findings from which showed that sorafenib can be recommended for use in various subgroups of differing age, prognosis, performance status, tumour burden and distribution, treatment history and co-morbidity. center dot This patient-focussed approach has broad application and can be used to assess other agents and tumour types. Randomized controlled trials (RCTs) show that six targeted agents - sorafenib, sunitinib, temsirolimus, everolimus, bevacizumab and pazopanib - improve outcome in advanced renal cell carcinoma (RCC). The populations enrolled in the pivotal phase III studies differed, and, to date, no head-to-head comparisons allow us to judge relative efficacy and tolerability. Populations recruited to RCTs under-represent certain patient subtypes, notably the elderly and those with comorbidities. Choosing the agent most appropriate in a specific case requires that we take into account the characteristics of the patient, the nature of their disease, and the history and aims of therapy. Data from expanded access programmes and clinical experience may be as relevant as the results of RCTs when making this difficult decision. To show how different sources of data can be integrated, we propose a schema that acknowledges nine patient-, disease-, and treatment-related factors relevant to clinical decision-making and provides an easily understood visual indication of the strength with which a particular agent can be recommended for use in specific subgroups of patients. As an example, we show how this tool shows the suitability of sorafenib in RCC subpopulations of differing age, prognosis, performance status, tumour burden and distribution, treatment history, and comorbidity. This patient-focused approach has broad application to other agents and tumour types.