December 4-8, 2017 at Atlantic City, New Jersey
The 73rd Deming Conference on Applied Statistics
Sponsored by Metropolitan Section, ASQ and Biopharmaceutical Section, ASA

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12/6/2016 (Tuesday) 1:30 PM - 4:30 PM

Session H: Improving our understanding of “less well-understood” adaptive trials: challenges, best practices and sharing of case studies

Dr. Eva Miller, Independent Statistical Consultant
Dr. Weili He, Merck

Moderator: Xiaoming Li
The draft adaptive design guidance released by FDA in 2010 included references to adaptive study designs that were described as “less well-understood”. There was relatively little regulatory experience with such designs and their properties were felt not to be adequately understood at the time of draft guidance release. In order to promote greater use of adaptive designs, especially those that were categorized as less well-understood, the Best Practice Subteam of the DIA Adaptive Designs Scientific Working Group has worked on describing and characterizing these designs, identifying challenges associated with them, and making suggestions on design or study conduct improvements that could make them more acceptable. In addition, the Best Practices Subteam conducted an extensive anecdotal search and reviewed trials from multiple sponsors who had employed these designs. We learned that these “less well understood” AD designs and associated statistical design and analysis methodologies can make difficult research situations more amenable to research, and, therefore are needed in our toolbox. This tutorial will cover the following two main parts: In the first part we will describe challenges of the “less well-understood” trials and suggest possible improvements to study design and/or study conduct; in the second part we will describe a few case studies in greater detail to give a more complete picture of lessons learned and best practices.

Dr. Eva Miller is Co-Chair of the DIA ADSWG BP subteam and is an independent biostatistical consultant. Eva has demonstrated biostatistical leadership with over 17 years of drug development experience across all phases of clinical trials (especially Phases II-IV) in the pharmaceutical industry and CROs. Before entering the pharmaceutical industry, Eva served New Jersey State government in health care research as a biostatistician and psychometrician. She has global experience managing people and projects across a broad range of therapeutic areas, including: Oncology, Cardiovascular diseases, Diabetes, other Endocrine/Metabolic diseases, Women’s Reproductive Health, Gastrointestinal diseases, Infectious Diseases and Vaccines, Mental Disorders, Musculoskeletal disorders, Arthritis, Neurology, Ophthalmology, Urology, Skin and Soft Tissue, and Pain Management. Eva has extensive submission experience and experience interacting with health authorities. She has developed global, harmonized SOPs and specific quality processes and procedures for Biostatistics, and has a proven record of innovation and problem-solving and excellent knowledge of a variety of study designs and statistical methodology. Eva is particularly strong in the design and implementation of adaptive trials. She has excellent consulting skills and a collaborative approach. Her experience is reflected in the number of publications and presentations. Eva has her Ph.D. from University of Pennsylvania and is an active member of both DIA and ASA.

Dr. Weili He is a Director of Clinical Biostatistics at Merck. Her research interests include survival and longitudinal data modeling, missing data imputation, cancer Phase I & II designs, repeated categorical data modeling, surrogate marker evaluations, adaptive design methodologies and implementations, and methods for benefit-risk assessment. Dr. He has published extensively in the areas of adaptive designs and benefit-risk evaluations, and is the author of more than 50 peer-reviewed publications in statistical and medical journals. She is also an editor of the book, “Practical Considerations for Adaptive Trial Design and Implementation”, published by Springer in 2014, and the book, “Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments”, published by CRC Press in 2016. She has also been involved in many professional activities and services, including serving as co-chair of the QSPI Benefit-Risk working, co-chair of the DIA ADSWG KOL lecture series, co-lead of the DIA ADSWG Best Practice Subteam until January 2016, Associate Editor for the journal of Statistics in Biopharmaceutical Research (SBR) and a referee for other statistical journals.

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