Reply from authors re: Camillo Porta. How to identify active novel agents in rare cancers and then make them available: A need for a paradigm shift. Eur Urol 2012;62:1020-1: Research in rare tumors: A need for a paradigm shift

摘要

We thank Professor Porta for his thoughtful comments [1] about the implicit issues raised by our report [2] of sunitinib therapy for a heterogeneous mixture of rare subtypes of kidney cancer. We could not agree more that the academic oncology community must find a more efficient paradigm for the treatment of rare cancers. Moreover, we believe that the most productive path toward this goal is to embrace Bayesian notions in the context of adaptive trials that incorporate candidate biomarkers. We have previously championed these ideas in the context of the phase 2 selection problem [3-5] and see ample justification for extension to rare cancers, as described by Porta [1].