An article published by Seminars in Oncology describes the work of the National Clinical Trials Network (NCTN) who are “specifically charged with improving the care of patients with rare cancers through clinical trials research by conducting definitive, randomised, late phase clinical treatment trials.” In this context the authors discuss the “understanding of rare and molecularly-defined cancers, and propose a pathway for study of these diseases.”
Based on the observation that rare cancers present a high response rate to targeted therapies, probably due to identification of oncogenic drivers with little inter-patient variability, the authors have suggested that “bucket or basket” trials to be efficient. The authors describe these trials, which involves including patients with a wide variety of histological assessments “as they all harbour (sic) the cognate anomaly”, as a methodology pursued by NTCN. According the authors NTCN possesses an unprecedented international reach to perform widespread molecular screening and histological analysis of tumours and enrol these patients.
The authors also describe the Molecular Analysis for Therapy Choice (NCI-MATCH) project, an extension of the basket trial, under development in the NCTN. In the NCI-MATCH project multiple drugs/histological subtypes will be simultaneously studied, enrolling patients who have progressed on at least one line of standard therapy for a further molecular tumour analysis that will test for “response rate and progression free survival primary endpoints.”