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Platform Trials, Can they Benefit Animal Studies?

Master protocols and platform clinical trials have become an innovative and efficient approach to testing multiple compounds in a single and consistent framework. But how can they be applied to animal studies? At the BAYES2022: Bayesian Biostatistics conference (October 12–14, 2022, in Bethesda, Maryland), Cytel’s VP of Scientific Strategy and Innovation Kyle Wathen and Senior Research Principal Krishna Padmanabhan will be presenting. Dr. Wathen’s abstract on such platform trials is below along with his commentary on this important and timely upcoming talk. (See our previous post for more information on Dr. Padmanabhan’s talk.)


Platform Trials, Can they Benefit Animal Studies?

Cytel author: Kyle Wathen

In recent years, master protocols and in particular platform clinical trials have gained substantial support and provide an efficient way of testing multiple compounds in a single and consistent framework. In the traditional setting, each new compound requires independent proof-of-concept, phase 2A and/or 2B trials to be designed and conducted. In this setting, patients are often randomized between the new treatment and control, with the same control utilized in many studies. In contrast, platform clinical trials utilize a master protocol with each new compound added through an intervention specific appendix (ISA). This innovative approach leverages the common control, patient outcomes, and recruitment centers to improve efficiency in terms of operations, analysis, and decision-making.

In this presentation, I will begin with the key aspects of platform trials including terminology and discuss some of the key advantages. Next, I will discuss some of the intricate details and complications involved in simulating platform trials with multiple compounds. In particular, I will discuss the application to an animal study where we explore the incorporation of data on control from a previous study, how and when to borrow information in new ISAs, and potential benefits/risk of various Bayesian models utilized for decision-making. The last part of the presentation I will introduce an R package that was utilized for simulating this complex design.


What motivated you to explore this topic and why is this moment significant? What are the stakes of the questions being explored?

Platform trials + Bayesian models is an interesting topic as this approach potentially reduces patient resources and uses them more efficiently.  

What can Cytel offer our clients in regards to this topic?

We can offer simulation-guided design of platform studies using either Bayesian or Frequentist approaches. In addition, we can help with a complete exploration of options including platform studies.

What's an example of a finding that those attending your talk can look forward to learning more about?

In this application, we are able to use fewer animals in the later ISAs (treatments) while maintaining the same power.

Would sponsors need to simulate their own platform trials? Wouldn't they simply be joining an existing one? What is the benefit of a sponsor doing their own simulations?

They can always join an existing one, but if an existing one does not meet the need of the trial they want to run, then they could set up their own. You must simulate a platform study to understand how it will perform in practice as they are too complicated and there are too many intricacies to skip the simulation step. Cytel can help with this.

How can the conversation continue from here?

If someone thinks they may be interested in a platform study, or more generally master protocols, we can help with the design and evaluation of the options. Contact us to learn more.


We look forward to seeing you at BAYES2022!



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*Photo credit (blog thumbnail and accompanying social post): Elvert Barnes (CC BY-SA 2.0)

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