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ASCO Educational Book; 2008
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Clinical Decision-making in Rare Tumors: What Is Needed from Clinical Trials

Paolo G. Casali, MD

From the Istituto Nazionale Tumori

Author's disclosure of potential conflicts of interest is found at the end of this article.

Address reprint requests to Paolo Giovanni Casali, MD, Istituto Nazionale Tumori, Via G. Venezian 1, Milano, Italy I-20133; e-mail: paolo.casali{at}istitutotumori.mi.it

Overview: Sarcomas are a good model for rare tumors. They are not so rare as to prevent the feasibility of randomized clinical studies; therefore, these studies have been conducted. However, some trials involving adult soft tissue sarcomas have been unable to address clinical practice in a noncontroversial way. This may be a result of tension between "statistical precision" and "clinical precision" and may be more frequent in rare tumors, although it also exists in frequent tumors. Innovations in the methodology of clinical research are needed to meet the needs of clinical decision-making in rare tumors. Innovations in the methodology of clinical decision-making are needed as well. Improvements in both areas may help reduce the degree of uncertainty at the patient's bedside and may serve as a model for frequent tumors, because in the age of targeted therapies, all tumors tend to be split into rare subgroups.