The childhood cancer data initiative: enabling data sharing to drive research advances and transform pediatric cancer diagnosis and treatment.

Purpose Of Review: With growing emphasis on data-driven research in pediatric oncology, particularly in the context of advances in molecular characterization and precision medicine, there is an urgent need for comprehensive data-sharing initiatives. This review explores how the Childhood Cancer Data Initiative (CCDI) addresses this critical need.

Recent Findings: CCDI plays a key role in enhancing pediatric cancer research by improving data integration, sharing, and collaboration.

Stakeholder Perspectives on Randomized Clinical Trials for Children With Poor-Prognosis Cancers.

Importance: In poor-prognosis children's cancers, new therapies may carry fresh hope for patients and parents. However, there is an absolute requirement for any new therapy to be properly evaluated to fulfill scientific, regulatory, and reimbursement requirements. Randomized clinical trials (RCTs) are considered the gold standard, but no consensus exists on how and when they should be deployed to best meet the needs of all stakeholders.

Frequency of pathogenic germline variants in pediatric medulloblastoma survivors.

Background: Medulloblastoma is the most common malignant brain tumor in children. Most cases are sporadic, but well characterized germline alterations in , , , , , and predispose to medulloblastoma. However, knowledge about pathogenic/likely pathogenic (P/LP) variants that predispose to medulloblastoma vary based on genes evaluated, patient demographics, and pathogenicity definitions.

Considerations on Design and Analysis of External Control in Pediatric Oncology.

Pediatric cancer consists of a diverse group of rare diseases. Due to limited patient populations, standard randomized and controlled trials are often infeasible. As a result, single-arm trials are common in pediatric oncology and the use of external controls is often desirable or necessary to help generate actionable evidence and contextualize trial results.

Pediatric Cancer Drug Development: Leveraging Insights in Cancer Biology and the Evolving Regulatory Landscape to Address Challenges and Guide Further Progress.

The discovery and development of anticancer drugs for pediatric patients have historically languished when compared to both past and recent activity in drug development for adult patients, notably the dramatic spike of targeted and immune-oncology therapies. The reasons for this difference are multifactorial. Recent changes in the regulatory landscape surrounding pediatric cancer drug development and the understanding that some pediatric cancers are driven by genetic perturbations that also drive disparate adult cancers afford new opportunities.