An institutional audit of the use of novel drugs in pediatric oncology

Abstract

Background: Significant challenges persist in treating children with rare, relapsed, or refractory malignancies. Novel molecularly targeted drugs promise improved outcomes for these children with reduced toxicity. However, there is often limited evidence to substantiate their clinical efficacy and guide their use. This raises issues for clinical decision-making, ethical concerns surrounding equity of access to these often-expensive agents, and the management of families' expectations for cure. This audit evaluated the off-label use of novel drugs and associated clinical outcomes in order to guide the development of future clinical and ethical guidelines. Aim: To evaluate the patterns in the ..