Precision medicine for pediatric cancers lags behind that for adult cancers: Citing hurdles in clinical trial recruitment and drug development, researchers are exploring ways to close gaps

Precision Medicine, pediatrics, clinical trials

Pediatric precision oncology faces challenges due to the rarity of pediatric cancers, which limits research and funding compared to adult cancers. Dr. Katherine Janeway highlights the need for more validated pre-clinical models and pharmaceutical interest in pediatric cancers. The RACE Act, effe…

Jan 9^th • 2 mins read

Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective

cancer, pediatrics, product labeling, united states food and drug administration, childhood cancer, denosumab, surrogate endpoints, adverse event, patient self-report, pediatric oncology, selumetinib, benefit-risk assessment, tisagenlecleucel, statistica

PROs have been feasible to collect from patients as young as 7 years old and were included in trials from 2013 onward. PROs were used as exploratory endpoints in four product applications but not included in product labeling. To include PRO data in FDA labeling, early interaction with the FDA, c…

Apr 30^th • 12 mins read

Pediatric Trials for Cancer Therapies With Targets Potentially Relevant to Pediatric Cancers

antineoplastic agents, adult, child, pediatrics, continental population groups, united states food and drug administration, statutes and laws, cancer therapy, childhood cancer, molecular target

The Research to Accelerate Cures and Equity (RACE) for Children Act was enacted in 2017. The Act authorizes the US FDA to require pediatric studies for new cancer drugs with molecular targets relevant to pediatric cancers. An examination of 78 adult cancer drugs approved by the FDA from 2007 to …

Oct 29^th • 10 mins read