Outcome research: Rationale and objectives

Outcome research measures the clinical, economic and humanistic outcomes of pharmacological therapies on patients suffering from different diseases. Outcome research can be applied either to phase II and III clinical trials to assess new drugs for registration or to already marketed drugs to assess their real value when used in clinical practice in a large population of patients. Oncology can be a major field of application because there is a large amount of new treatments put on the market, often without demonstration of significant improvement in survival or Quality of Life but with higher prices than the old molecules. Patients and disease specific outcomes have been identified for cancer by a Working Group set up by the American Society of Clinical Oncology, with the former being defined as more critical to establish the value of new treatments. Survival, drug toxicity end impact on Quality of Life are the most important patient's outcomes to consider before recommending anti-cancer therapies for use in clinical practice. The present paper will describe the outcomes in oncology and will deal with the transferability to clinical practice of the results of clinical trials.