In-Conference Workshop
In-Conference Workshop I
Innovative Clinical Trial Designs for Personalized Medicine
1:30-5:00pm | August 27, 2019 | ROOM #509, 5F
Organizer(s): Toshimitsu Hamasaki, National Cerebral and Cardiovascular Center
Moderator(s): H.M. James Hung, US Food and Drug Administration
Instructor(s): James Wason, Cambridge University and Newcastle University; Florian Klinglmueller, Austrian Medicines & Medical Devices Agency
The workshop will cover statistical methods for designing and implementing innovative trial designs for personalized medicine.
We will demonstrate adaptive multiple testing procedures defined by directed, weighted graphs that provide an intuitive visual tool for constructing multiple testing strategies that reflect the, often complex, contextual relations between hypotheses in clinical trials. These designs permit mid-trial design modifications based on unblinded interim data, while providing strong family wise error rate control in a wide range of scenarios including trials with multiple treatment comparisons, endpoints, subgroups, or combinations thereof. Examples of permitted adaptations are dropping of treatment arms, selection of subpopulations, and sample size reassessment. We will also discuss innovative approaches to the design and analysis of trials incorporating patient subgroups, including basket trials, umbrella trials and adaptive signature designs.
The practical implementation, operating characteristics, as well as regulatory considerations of such designs will be illustrated using real case studies.
About the instructors
Dr James Wason is a Professor of Biostatistics at Newcastle University and a group leader at the MRC Biostatistics Unit. He leads a research group focused on development of statistical methods for more efficient trials. His research interests are adaptive designs, using biomarkers prospectively during trials and analysis of composite endpoints. As well as working on methodology, James is actively working on applying novel methods in real clinical trials and is currently involved in several ongoing trials using novel designs.
Dr. Florian Klinglmueller is a statistician at the Austrian Medicines and Medical Devices Agency. He is works as an assessor of Scientific Advice and Centralized Marketing Authorization Procedures of the European Medicine Agency’s Procedures. He is an observer of EMA’s Biostatistics Working Party where he is involved in the review and drafting of new regulatory guidance documents. Previously he spent several years as a researcher at the Medical University of Vienna working on adaptive design and multiple testing procedures. He received his PhD in Mathematics from the Vienna University of Technology.
The in-conference workshop is FREE for the ISBS2019 main conference attendees and the registration is NOT required. Up to 50 seats are available and the seating is on a first-come, first-served basis.
In-Conference Workshop II
Adaptive Multi-Arm Multi-Stage (MAMS) designs in confirmatory clinical trials: a practical introduction to the statistical methodology and its application
1:30-5:00pm | August 28, 2019 | ROOM #509, 5F
Organizer(s): Yannis Jemiai, Cytel
Instructor(s): Lingyun Liu, Cytel; Yannis Jemiai, Cytel; Hrishikesh Kulkarni, Cytel
The development of new therapies has been challenging due to high cost and failure rate. More efficient trial design can save the time to market with less patients. Two arm group sequential design have been widely used for over 40 years which is more efficient compared to the traditional fixed sample design. Such design allows early stopping for overwhelming efficacy or futility, and therefore save sample size and development time. The natural generalization of two-arm group sequential designs is to compare multiple treatment arms to a common control in a multiple stages (MAMS designs). This course will discuss adaptive multi-arm multi-stage design in the confirmatory setting. We will start with the theory and design for two-arm group sequential designs. We will then extend the theoretical framework to MAMS design. We will discuss the various methods for controlling familywise error rate in face of adaptive treatment selection and sample size adaptation and the pros and cons for each approach. We will demonstrate how to design such studies with real clinical study and powerful software package. Practical considerations will also be discussed for successful implementation of such designed trials.
About the instructors
Dr. Lingyun Liu got her Ph.D. in statistics from Northwestern University. She is now a director in the strategic consulting team at Cytel. She has provided consultation over 100 studies on clinical development plan, innovative trial designs, multiple comparisons, and regulatory interactions across different therapeutical areas including oncology, immuno-oncology, diabetes, CNS, infectious diseases, cardiovascular and rare disease and orphan drugs. She also supports DMC meetings with multiple studies and provided instrumental guidance for interim decision making. Her research interest includes multiple comparison, innovative design, platform trials, and rare disease. She also provides technical inputs to the module development in the commercial software EAST, StatXact.
Dr. Yannis Jemiai is Senior Vice President at Cytel, where he leads the software and strategic consulting business units. He and his team of his team of highly-skilled statistical consultants advise biopharmaceutical and medical device companies, drawing on their research in adaptive designs, data monitoring, multiple comparison procedures, pharmacometrics, and data science. He has broad experience in a number of therapeutic areas, including oncology and rare diseases. Dr. Jemiai also oversees the development of Cytel’s software product lines, including trial design packages East® and Compass®, and exact statistics applications StatXact® and LogXact®. Dr. Jemiai earned his Ph.D. from Harvard University, an M.P.H. from Columbia University, and a B.A. in Molecular and Cellular Biology also from Harvard.
Mr. Hrishikesh Kulkarni is Customer Experience Manager at Cytel. He joined Cytel in 2007 and has been involved in the software quality, documentation, demos & training, and product marketing for East, EnForeSys and other Cytel products. Hrishikesh has M.Sc. in Statistics from University of Pune, India. He is based in Cambridge, MA.
The in-conference workshop is FREE for the ISBS2019 main conference attendees and the registration is NOT required. Up to 50 seats are available and the seating is on a first-come, first-served basis.