Event Details

Overview

In this webinar we will review the range of statistical methodologies used to harness the potential of Real-World Data (RWD) in clinical development, particularly in the context of rare diseases and small populations like paediatrics. The session will include theoretical understanding and practical case studies, with a special focus on Bayesian methods and causal inference.


Tim Friede will present how randomized controlled trials can benefit from the inclusion of real world data, especially in rare diseases. There are various promising ways of linking data from RCTs and RWD. Therefore, a more routine joint consideration of RCT and RWD data appears desirable, in particular in rare diseases.


Brad Carlin will provide a brief review of the Bayesian adaptive approach to clinical trial design and analysis, and then will discuss a variety of areas in which Bayesian methods offer a better (and perhaps the only) path to regulatory approval. Topics to be covered are expected to include:

  • Leveraging historical controls and other auxiliary data
  • Methods for rare and pediatric disease
  • Causal inference tools to incorporate RWD/RWE


Following the talks there will be discussion and Q&A.


Who is this event intended for? Biostatisticians and drug developers in pharmaceutical industry, as well as students, people working in academia and regulators who are involved in/interested in learning about the challenges and benefits of using RWD in clinical trials in small populations and rare diseases.

What is the benefit of attending? Attendees will gain insights into different uses of RWD in small populations and rare diseases.


Timings

14:00-15:30 BST | 15:00-16:30 CEST

Speakers

  • Tim Friede (Professor of Biostatistics at University Medical Center Goettingen)

    Tim Friede

    Professor of Biostatistics at University Medical Center Goettingen

    Since January 2010 Tim Friede is Professor of Biostatistics at the University Medical Center Göttingen where he heads up the Department of Medical Statistics. He graduated in Mathematics (Dipl.-Math.) from the University of Karlsruhe and obtained a PhD (Dr.sc.hum.) from the Faculty of Medicine at the University of Heidelberg. In 2001 he joined the Department of Mathematics and Statistics at Lancaster University as NHS Training Fellow in Medical Statistics and was later promoted to Lecturer in Biostatistics. From 2004 on he worked for Novartis Pharma AG, Basel before joining Warwick Medical School as Associate Professor of Medical Statistics in October 2006. Tim Friede's methodological research interests are in clinical biostatistics including designs for clinical trials (in particular flexible adaptive designs) and generalized evidence synthesis (including systematic reviews and meta-analyses) as well as applications in rare diseases and cardiovascular medicine.

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  • Brad Carlin (Senior Advisor, Data Science and Statistics at Cencora-PharmaLex)

    Brad Carlin

    Senior Advisor, Data Science and Statistics at Cencora-PharmaLex

    Brad Carlin is a statistical researcher, methodologist, consultant, and instructor. He currently serves as Senior Advisor for Data Science and Statistics at Cencora-PharmaLex, an international pharmaceutical consulting firm. Prior to this, he spent 27 years on the faculty of the Division of Biostatistics at the University of Minnesota School of Public Health, serving as division head for 7 of those years. He has also held visiting positions at Carnegie Mellon University, Medical Research Council Biostatistics Unit, Cambridge University (UK), Medtronic Corporation, HealthPartners Research Foundation, the M.D Anderson Cancer Center, and AbbVie Pharmaceuticals. He has published more than 190 papers in refereed books and journals, and has co-authored three popular textbooks: “Bayesian Methods for Data Analysis” with Tom Louis, “Hierarchical Modeling and Analysis for Spatial Data” with Sudipto Banerjee and Alan Gelfand, and "Bayesian Adaptive Methods for Clinical Trials" with Scott Berry, J. Jack Lee, and Peter Muller. From 2006-2009 he served as editor-in-chief of Bayesian Analysis, the official journal of the International Society for Bayesian Analysis (ISBA). During his academic career, he served as primary dissertation adviser for 20 PhD students. Dr. Carlin has extensive experience teaching short courses and tutorials, and won both teaching and mentoring awards from the University of Minnesota. During his spare time, Brad is a health musician and bandleader, providing keyboards, guitar, and vocals in a variety of venues.

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Tickets

PSI Member
Member Price Complimentary
PSI Non-Member
Standard Price Complimentary

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