Clinical Trial Design, Endpoints and Regulatory Considerations in Heart Failure

Abstract

After the withdrawal of flosequinan in the early 1990’s, because of an increased risk of mortality and fatal arrhythmias, the bar for the approval of new drugs in heart failure has been raised and regulatory agencies have requested evidence for the efficacy of new treatments on mortality and morbidity end-points. However, more recently, regulatory agencies have become more open to include the assessment of functional capacity as an efficacy endpoint, at least in selected subgroups of patients. Therefore, a new therapy for the treatment of heart failure can be approved if it improves survival and/or reduces hospitalisations or if it safely improves functional capacity. This article reviews clinical trial design, trial endpoints and regulatory issues in Heart Failure trials.