Preclinical rare cancer research to inform clinical trial design

Abstract

With improved biobanking of rare cancer tissue enabling more in-depth tumour profiling, as well as generation of clinically relevant rare cancer preclinical models, treatment strategies for patients with rare cancer will rapidly advance. The future successes of rare cancer research, with alternative approaches for diagnosing, researching and conducting clinical trials, will ultimately benefit patients with all types of cancer. This Comment article argues that the future successes of rare cancer research will require alternative approaches for diagnosing, researching and conducting clinical trials, which will ultimately benefit patients with all types of cancer.