New drug development in childhood cancer

Purpose of review: Future advances in childhood cancer treatment will pivot on developing biology-driven new drug development pathways that build on current knowledge of oncogenic pathways; however, we need to address major barriers to accessing new drugs for clinical evaluation in childhood cancers. Recent findings: Through legislative change, substantial incentives to the pharmaceutical industry to invest in the ultra-rare diseases, such as childhood cancers, have encouraged greater engagement with paediatric oncology drug development consortia. Disappointingly, this has not translated into paediatric-focussed drug development. Adult disease-driven drug development will continue to dominate until biology/target-driven approaches prevail.There are specific challenges to undertaking early drug development trials in children with incurable disease. The balance between risk and benefit for a child participating in trials wherein the chance of clinical benefit is indeterminate has the potential for unrealistic optimism by both physicians and families. Importantly, innovative trial designs that assess safety and maximize information on potential efficacy from small patient numbers are needed. Summary: International collaboration in early phase trial consortia addresses these challenges. Academic networks concentrating early phase trials expertise and delivery of innovative trial designs will maximize appropriate selection of drugs that can translate into therapeutic advantage when incorporated into standard care. © 2014 Wolters Kluwer Health.