Background In adults with advanced-stage Hodgkin's lymphoma, the CD30-directed
antibody–drug conjugate brentuximab vedotin combined with multiagent chemotherapy has …

Childhood cancer is considered rare, corresponding to~ 3% of all malignant neoplasms in
the human population. The World Health Organization (WHO) reports a universal …

Simple Summary Clinical trials in pediatric oncology and personalized medicine are
challenging due to the rarity of the disease, the low prevalence, and the ever-improving …

As precision medicine becomes more precise, the sizes of the molecularly targeted
subpopulations become increasingly smaller. This can make it challenging to conduct …

Simple Summary Solid tumors account for~ 60% of pediatric, as well as adolescent and
young adult (AYA), cancers, and outcomes for patients with these progressive diseases …

Introduction The unique evidentiary, economic and ethical challenges associated with
health technology assessment (HTA) of precision therapies limit access to novel drugs and …

Simple Summary The detection of genomic aberrations in cancers has yielded a wealth of
information to discover oncogenic drivers or pathogenic variants that are relevant for the …

This article provides a summary of discussions from the American Statistical Association
(ASA) Biopharmaceutical (BIOP) Section Open Forum organized by the ASA BIOP Statistical …

Phosphatase of regenerating liver 3 (PRL3) is a specific tumor antigen overexpressed in a
broad range of adult cancer types. However, its physiological expression in pediatric …

Despite high-level endorsement, the number of adaptive Phase II/III trials in rare cancers
needs to be improved, with better understanding of their value for clinical decisions in daily …