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European UrologyVolume 62, issue 6, pages e95-e106, December 2012
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
Published online 19 August 2012, page 1022
Refers to article:
How to Identify Active Novel Agents in Rare Cancers and then Make Them Available: A Need for a Paradigm Shift
December 2012 (Vol. 62, Issue 6, pages 1020 - 1021)
Refers to article:
A Phase 2 Trial of Sunitinib in Patients with Advanced Nonclear Cell Renal Cell Carcinoma
Accepted 20 June 2012
December 2012 (Vol. 62, Issue 6, pages 1013 - 1019)
We thank Professor Porta for his thoughtful comments  about the implicit issues raised by our report  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 , , and  and see ample justification for extension to rare cancers, as described by Porta .
One interesting extension of these ideas would be to establish registries of rare cancers. Even though this “uncontrolled” experience has well-known pitfalls, it is probably our best hope for obtaining “prior distributions” to support the design of targeted trials. We need a way to pool experience and “mine” that experience for hypotheses that can be investigated more formally.
By whatever specific mechanisms, there is a moral obligation to learn something from every patient we treat, particularly regarding rare cancers, for which there will probably never be data from large phase 3 trials. The comments of Porta  are extremely important and most welcome.
Conflicts of interest
The authors have nothing to disclose.
-  C. Porta. How to identify active novel agents in rare cancers and then make them available: a need for a paradigm shift. Eur Urol. 2012;62:1020-1021 Abstract, Full-text, PDF, Crossref.
-  N.M. Tannir, E. Plimack, C. Ng, et al. A phase 2 trial of sunitinib in patients with advanced non–clear cell renal cell carcinoma. Eur Urol. 2012;62:1013-1019 Abstract, Full-text, PDF, Crossref.
-  P.F. Thall, L.H. Wooten, C.J. Logothetis, R.E. Millikan, N.M. Tannir. Bayesian and frequentist two-stage treatment strategies based on sequential failure times subject to interval censoring. Stat Med. 2007;26:4687-4702 Crossref.
-  P.F. Thall, C. Logothetis, L.C. Pagliaro, et al. Adaptive therapy for androgen-independent prostate cancer: a randomized selection trial of four regimens. J Natl Cancer Inst. 2007;99:1613-1622 Crossref.
-  A. Siefker-Radtke, A. Kamat, D. Williams, et al. A phase II randomized four-regimen selection trial incorporating response for sequential chemotherapy in metastatic, unresectable urothelial cancer: final results from the M. D. Anderson Cancer Center. J Clin Oncol. 2009;27:251s
a Department of Genitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
b Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Corresponding author. The University of Texas MD Anderson Cancer Center, Department of GU Medical Oncology, Unit 1374, 1155 Pressler Street, Unit 1374, Houston, TX 77030, USA. Tel. +1 713 563 7265; Fax: +1 713 745 0422.
© 2012 Published by Elsevier B.V.
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