Clinical cancer research is undergoing profound changes, mostly because of the advent of personalized cancer medicines. Some remarkably efficacious drugs have even been approved based on uncontrolled phase I clinical trials.

In this webinar, we will challenge the view that the expected benefits from new personalized cancer medicines are generally sufficient to forgo randomization to a standard-of-care arm. Apparently improved outcomes in a single-arm early trial may be due to the prognostic nature of the target and to selection bias, rather than result from a true effect of therapy. Moreover, the predictive role of biomarkers cannot be ascertained in a definitive way without randomization to a control arm.

We will start by reviewing the evolving nature of clinical research, especially with targeted agents, and the very promising examples of the approach to developing personalized cancer medicines. We will then claim that randomization is an essential feature of clinical trials throughout the continuum of clinical drug development and discuss the potential loss of a clear demarcation between traditional phases of this development. Implementing randomization throughout the continuum of development requires important statistical considerations, adaptive designs, and a change of focus from the drug alone to the drug/patient match.

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Speakers

Marc Buyse, ScD, Chief Scientific Officer, International Drug Development Institute (IDDI)

Marc Buyse holds a ScD in biostatistics from the Harvard School of Public Health (Boston, MA). He is the founder of the International Drug Development Institute (IDDI) and of CluePoints, two biostatistical service organizations based in the US and Europe. He is interested in clinical trial design, meta-analysis, validation of biomarkers and surrogate endpoints, statistical methods in oncology, statistical detection of errors and fraud, statistical monitoring of clinical trials, and medical data sharing (http://publicationslist.org/marc.buyse). He served on the first Independent Review Panel for clinicalstudydatarequest.com.

Message Presenter

Everardo Saad, Senior Medical Expert, IDDI

Dr. Everardo Saad

Everardo Saad, Senior Medical Expert, IDDI

Dr. Everardo Saad, IDDI’s Senior Medical Expert, has over 15 years of experience in Medical Oncology and clinical trial designs. He graduated in Medicine and trained in Internal Medicine in Sao Paulo, and did his fellowship in Medical Oncology at the University of Texas M.D. Anderson Cancer Center, in Houston. After practicing for several years, he shifted his professional career towards education and research in Medical Oncology, and has a special interest in clinical trial methodology, the assessment of endpoints, and the development of novel therapies for cancer patients.

Message Presenter

Who Should Attend?

Clinical trialists, statisticians, physicians, and other professionals involved in the design, conduct and analysis of clinical trials on personalized cancer medicine.

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IDDI

International Drug Development Institute (IDDI) is an expert center in biostatistical and integrated eClinical services for Pharmaceutical and biotechnology companies in several disease areas, including oncology and ophthalmology.

IDDI optimizes the clinical development of drugs, biologics and devices thanks to proven statistical expertise and operational excellence. Founded in 1991, IDDI has offices in Belgium, Boston (MA), Raleigh (NC) and San Francisco (CA).