Journal of biopharmaceutical statistics
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Cancer causes premature death and significant, often devastating, symptoms. While prolongation of survival is an obvious end point for new cancer drug approval, the US Food and Drug Administration (FDA) has also utilized end points that evaluate patient symptoms. In this article we discuss the end points, evidence, and analyses supporting cancer drug approvals based on evaluations of tumor-related signs and symptoms. ⋯ Drug sponsors are encouraged to include symptom assessments in cancer clinical trials and to perform further research to improve symptom-assessment methods. The FDA routinely meets with sponsors at End of Phase 2 Meetings to discuss drug development plans and the design of phase 3 trials. We encourage sponsors to request special protocol assessments (SPA) after meeting with the FDA to get written confirmation of the adequacy of plans for assessing cancer morbidity and quality of life, including protocols, end points, statistical analysis plans, and draft case report forms.