SESSION J

TITLE: Designing and Integrating the RCT/RWE in Safety Decision Making

SPEAKERS: Dr. Rima Izem, FDA, (On Behalf of the ASA Safety Working Group)
Dr. Richard C. Zink, TARGET PharmaSolutions Inc. (On Behalf of the ASA Safety Working Group)
Dr. William Wang, Merck (On Behalf of the ASA Safety Working Group)

 

Abstract

Traditional evaluation of the strength of evidence for establishing the efficacy and safety of health interventions is two-tiered. In the top tier is the gold standard of randomized clinical trials (RCTs), and in the lower tier are observational studies and other sources of real world evidence (RWE). However, this two-tiered view of evidence from clinical investigations is not nuanced enough for today’s needs and methodologies.

There is growing demand for fast, timely and relevant public health data on patient safety. This has resulted in increasing expectations for well-designed, well-executed and well-reported observational studies. In addition, there is a rise in demand for using RCTs to understand treatment effects in a more real world setting. To face these challenges and opportunities, the ICH and various regulatory authorities are coming up with guidance to incorporate RWE and RCTs into relevant decision making. These ideas are reflected in the recent update of ICH E2C for periodic safety update report, the E6/E8 renovation paper, the ICH E9 R1 estimand discussion, as well as the recent FDA/PMDA/EMA guidance on the use of real world evidence.

These opportunities are particularly relevant and potentially rewarding in safety monitoring and evaluation, and serve as the motivation for the ASA Safety Working Group to form a new work stream on “Integrating and Bridging RCT and RWE for Safety Decision Making”. This tutorial session will be based on the research of this work stream. It may include the following topics:

  1. Statistical and design considerations for real world evidence in health decision making
  2. Statistical and design considerations for randomized pragmatic trials
  3. Selected topics on advanced analytics the multi-source safety data

Speaker’s Bio:

Richard C. Zink is Principal Research Statistician Developer in the JMP Life Sciences division at SAS Institute. Prior to SAS, he spent eight years in the pharmaceutical industry, where he designed and analyzed clinical trials in diverse therapeutic areas including infectious disease, oncology, and ophthalmology. He is the 2018 Chair-Elect and Host of the Statistics Podcast for the Biopharmaceutical Section of the American Statistical Association, and an Associate Editor for Therapeutic Innovation & Regulatory Science, the scientific journal of DIA. Richard holds a Ph.D. in Biostatistics from the University of North Carolina at Chapel Hill, where he serves as an Adjunct Assistant Professor of Biostatistics. He is author of Risk-Based Monitoring and Fraud Detection in Clinical Trials Using JMP and SAS, co-editor of Modern Approaches to Clinical Trials Using SAS: Classical, Adaptive, and Bayesian Methods, and contributor to six other books on statistical topics in clinical trials and clinical research.

 

Dr. William (Bill) Wang is an executive director, clinical safety statistics, in the department of Biostatistics and Research Decision Sciences (BARDS), Merck Research Laboratories. He has over 24 years of experience in the pharmaceutical industry, with expertise and research publications in statistical design, analysis, clinical data management and their technology enablement. During his 17-year tenure at Merck, he supported regulatory filings in multiple therapeutic areas and established the BARDS Asia Pacific operation. Since 2010, he has served on the DIA China Regional Advisory Board and the DIA’s Global Community Leadership Council (CLC), including the chairmanship of the DIA China Statistics Community. He was a recipient of the DIA global inspire award in 2017. He is currently co-chairing an ASA safety working group, and a deputy topics-leader in the ICH E17 working group on multi-regional clinical trials.

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