An Introduction and Overview of Innovative Trial Design



“An introduction and overview of innovative trial design" will be presented by Kert Viele, Ph.D. of Berry Consultants, on Thursday, September 20, 2018 3-4 pm EDT/2-3 pm CDT/12-1 pm PDT.

The webinar can be accessed by following the instructions provided on the public Innovative Clinical Trial Resource (ICTR) webpage at


In our first Innovative Clinical Trial Resource (ICTR) webinar, Kert Viele, Ph.D. will review the main component of traditional (gold standard) randomized clinical trials and a variety of innovative trial designs. Innovative trial designs to be discussed include flexible sample sizes; enrichment; use of historical information; multiple arm and multiple domain trials; and novel endpoints. Dr. Viele will discuss the potential advantages and disadvantages of each innovative trial design relative to traditional clinical trial designs to illustrate the tradeoffs that should be considered when adopting innovative clinical trial designs.

The traditional clinical trial design dates back to the “Streptomycin in Tuberculosis” trial of 1946. Standard features of the traditional or gold standard randomized clinical trial, such as control arms, randomization, stratification, and blinding, are designed to minimize bias and maximize conclusiveness and interpretability of results. At the same time the traditional clinical trial design often requires large resource expenditures and usually focuses on narrow research questions which are sometimes at odds with our current clinical uncertainties. Innovative trial designs may help address some of these challenges. We will begin by reviewing the purpose of each component of a traditional clinical trial, and then introduce a variety of innovative trial designs. Each innovation represents a deviation from the traditional design, and as such results in benefits and risks. While smaller sample sizes are a standard goal of innovative clinical trials, we will also discuss how an equivalently or slightly larger sample size may help answer broader questions and minimize the risk of a failed trial. For some innovations, the cost is minimal, while in others potential risks must be carefully weighted. For example, the use of historical control information can significantly reduce the size of a trial, particularly in rare diseases, but this gain requires careful matching of the historical data to the current trial, else the trial presents significant inferential risks due to mismatches. At the conclusion of the webinar, attendees should be familiar with a wide variety of innovative clinical trial designs and the central tradeoffs to be considered in adopting them.

About the Presenter:

Kert Viele is a Director and Senior Statistical Scientist with Berry Consultants, where he leads Berry Consultants’ research enterprise. He is a leader in clinical trial implementation of Bayesian hierarchical modeling, with expertise in platform and basket trials as well as clinical trials incorporating the use of historical information. Prior to joining Berry Consultants in 2010, he was a faculty member at the University of Kentucky, where he received the Provost’s Award for Outstanding Teaching and was an investigator for NSF and NIH funded research. He has developed over 100 custom Bayesian adaptive clinical trials for clients in industry, government, and academia, and currently serves on several data safety monitoring boards for randomized clinical trials. A former editor of the journal Bayesian Analysis, Dr. Viele is also an author of FACTS (Fixed and Adaptive Clinical Trial Simulator), clinical trial simulation software currently licensed to multiple pharmaceutical, academic, and government organizations.

About the Innovative Clinical Trial Resource Program:

The goals of the NHLBI  Innovative Clinical Trials Resource (ICTR) are two-fold: 1) provide statistical consultative services to awardees of the “Catalyzing Innovation in Late Phase Clinical Trial Design and Statistical Analysis Plans Initiative” FOAs (U34/X01) and 2) develop and provide an educational program on the application of non-traditional clinical trial design and analysis for the NHLBI research community.