Statistics Applied to Clinical Trials - Mars 2016

 

 

   

Friday, March 11, 2016

UCL, Auditoire MORE 53 - Place Montesquieu, 2 - Louvain-la-Neuve

Seminar organized jointly by

 

 

Programme

 

14:00 - 14:10   Welcome and introduction - Prof. Catherine Legrand (UCL)
14:10 - 14:30   Dr. Everardo Saad (IDDI) - Response assessment: why do we need it?
14:30 - 15:15   Saskia Litière (EORTC) - RECIST and beyond
15:15 - 15:45   Coffee break
15:45 - 16:30   Dr. Patrick Therasse (Servier) - How to implement RECIST in practice
16:30 - 17:00   Discussion time - All

Abstract

Response Assessment in Cancer Trials: "Response assessment: why do we need it?"
Everardo D. Saad, M.D. | Senior Medical Expert |IDDI, Louvain-la-Neuve, Belgium

Abstract
Several types of endpoints may be used to assess the efficacy of anticancer therapy. Historically, tumor shrinkage has been considered an important indicator of chemotherapy activity, and in many diseases there is an association between objective responses and treatment benefit at the patient level. As a result, objective responses have been used as primary or secondary endpoints in cancer clinical trials, and different criteria have been proposed over the years in order to standardize response assessment. Although response rates have not been confirmed as good surrogates for overall survival in various disease settings, response assessment is an essential component in the development of cytotoxic agents as well as a very useful indicator of treatment efficacy in the clinic. On the other hand, problems arise when response rates are used to assess the benefit of some targeted agents and immune checkpoint inhibitors. This talk will provide an overview of the goals, methods and limitations of response assessment in oncology.

Biography
Everardo Saad is a medical oncologist with a special interest in clinical-trial methodology. He graduated in Medicine and trained in Internal Medicine in Sao Paulo, Brazil, and later pursued a fellowship in Medical Oncology at the University of Texas M.D. Anderson Cancer Center, in Houston, USA. After several years of clinical practice, he shifted his career toward consultancy in clinical research. He has over 15 years of experience in design and analysis of clinical trials for pharmaceutical/biotech companies and academic groups. He is a member of IDDI’s team of consultants, providing support to internal teams and clients. He currently serves as associate editor of the journal Trials, and has published more extensively in the area of efficacy endpoints in oncology.
   
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Response Assessment in Cancer Trials: RECIST and beyond.
Saskia Litière, Ph.D | Biostatistician | EORTC, Brussels, Belgium

Abstract
Assessment of tumour burden is an integral part of clinical trial methodology in oncology. The RECIST criteria, implemented in 2000 and updated in 2009, provide a tool for uniform assessment of tumour burden in multicentre clinical trials across multiple solid tumour types. This talk will start with a brief history of RECIST and an overview of the criteria. I will review some alternative response assessment criteria together with the level of evidence on which they are based. The talk will finish with an overview of the questions which the RECIST Working Group is currently trying to address, and some thoughts on the challenges the RECIST Working Group faces in the rapidly changing world of onco-therapeutics.
For more information on RECIST, please visit http://www.eortc.org/recist/

Biography
Saskia Litière obtained her degree of Applied Mathematics at the University of Ghent in 2001. In that same year she started her career as a statistician in the Center for Statistics (Censtat) at the Hasselt University. Over a period of nine years she obtained the degree of MSc in Biostatistics, she worked as statistical consultant, she was involved in the teaching of several Bachelor and Master level courses in statistics and she successfully defended her PhD thesis on the topic of misspecification in generalized linear mixed models. In September 2010 she joined the EORTC statistics department. She has worked with the EORTC Breast Cancer Group and is currently the statistician of the EORTC Soft Tissue and Bone Sarcoma Group. She is also a member of the RECIST Working Group and is involved in the ongoing update of RECIST.
  
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Response Assessment in Cancer Trials: "How to Implement RECIST in practice"
Patrick Therasse, M.D. PhD | Head of Oncology Development | Servier, Paris, France

Abstract
Since their first publication in 2000, RECIST criteria have been rapidly and broadly used by the scientific community mostly because these criteria provided an updated and more comprehensive framework than the former WHO criteria to facilitate the assessment of tumor response in clinical trials. Surprisingly, the new criteria are also used to help decision making in clinical practice situations while they were actually not designed for that so somehow reflecting the interest of physicians to refer to some sort of guidance even in uncontrolled settings. The RECIST working group continued its efforts to improve the criteria wherever possible by collecting (and answering) questions from the scientific community in real time, expanding the reference datasets with new data (targeted therapies) and testing new hypotheses on the datasets. These continuous efforts led to a revised RECIST version (referred to as 1.1) published in 2009 which further reduced the quantity of information needed to perform an adequate response assessment in clinical trials. This revised version also added precisions (based on accumulated experience with formal Q&A with the scientific community) and integrated the most relevant/validated references for radiological assessments. The last 7 years the RECIST working group has continued to search for improvements looking at both the methodological backbone of the criteria (mathematical model behind response calculation and dynamic imaging performance) as well as specific disease and treatment settings. So far, this research as well as the surveillance of the work published by others on these topics has however not yet justified a revision of the 1.1Version. Lately an additional common effort with active contributors to the development of immunotherapy has been initiated to validate concepts proposed in the immune Response Criteria (iRC) guidelines with the RECIST datasets (also expanded with immunotherapy clinical trial data) and consider the integration of iRC into the next version of RECIST.

Biography
Patrick Therasse is an MD, PhD, who focused his career on applied research and drug development in Oncology. Since 2014, he is the head of oncology development for Servier (France). Before working for Servier, he worked eight years for GlaxoSmithKline leading the clinical development program for Cancer Immunotherapeutics. He started his career at the European Organization for Research and Treatment of Cancer (EORTC) as a research fellow and then served as Director of the EORTC Data Center for eleven years. He worked in all fields of oncology with a specific interest in breast cancer. At the EORTC he coordinated the research efforts leading to the publication of the Response Criteria In Solid Tumors (RECIST) guideline in 2000 and continued with the 1.1 Version. He is still active on the RECIST committee. His areas of expertise encompass phase I to phase III drug development with cytotoxics, small molecules or active/passive immunotherapy and translational research.