Clinical value of biological therapies in oncology: measurement of outcomes for benefits evaluation

Abstract

Introduction: Oncology represents a major challenge in setting criteria for inclusion of new treatments. The high costs associated with cancer explain the great concern of the healthcare payers with the added value of new cancer treatments. Following the precepts of evidence-based medicine allows the use of best available evidence in decision-making process. The evaluation of new health technologies should consider its added value, which includes among others, efficacy of the treatment. In this way is necessary know whether the outcomes used for the analysis of this new technology are correct or appropriate to capture the real risks and benefits. Objective: The objective of this study was to evaluate outcome measures commonly used in oncology and to understand their differences. Discussion: The evaluation of metastatic melanoma treatment with the immunobiological agent ipilimumab indicated a higher late survival when compared to treatment previously available. However, results interpretation only through the median survival did not allow the capture and proper expression of long-term treatment benefits, as prolonged survival. Such benefits could be captured by the use of mean survival analysis, since it reflects the entire extension of the curve, considering all follow up period and patients who effectively have benefit. Conclusion: Thus, all outcomes of interest must be evaluated and with different methods, in order to obtain as much information as possible about benefits of each treatment.