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Innovations & Thérapeutiques en Oncologie

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Methodological challenges for precision medicine in oncology Volume 3, issue 1, January-February 2017

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Authors
1 IR4M-UMR8081
Université Paris Sud et Comité statistique de ARCAGY-GINECO
Rue Ampère
91405 Orsay Cedex
France
2 OncoStat, Institut Gustave Roussy
Service de biostatistique et d’epidémiologie & INSERM CESP
114, rue Edouard Vaillant
94805 Villejuif cedex
France
* Tirés à part

New methods have been required for evaluating targeted therapies, as the classic sequence – phase I: toxicity; phase II: efficacy; phase III: comparison with standard treatment – is no longer effective. As the parallelism between dose-toxicity and dose-efficacy, which was observed for cytotoxic chemotherapies, can no longer be supported, early trials are required to simultaneously assess toxicity and early signals of efficacy, based on biomarkers when available. Phase II primary endpoints have also been brought into question, as RECIST criteria are not well-suited to functional modifications in the tumour. New phase III trials, including more homogeneous targeted populations, will use more flexible designs, including interim analyses and adaptive design, allowing the sample size, and sometimes the trial design, to be modified during the course of the trial. This article discusses these new methodological challenges.