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  1. CDER Center for Clinical Trial Innovation (C3TI)

Bayesian Supplemental Analysis (BSA) Demonstration Project

CDER Center for Clinical Trial Innovation (C3TI)

C3TI aims to increase experience in Bayesian statistical methods in simple trial settings across sponsors, CDER clinical reviewers, and CDER statisticians, including deepening an understanding of their applicability, opportunities, and challenges. Bayesian statistical approaches can offer different perspectives in the assumptions made or addressed for an analysis when compared to frequentist approaches. Areas with emerging use of Bayesian methods include early-phase trials, trials with complex adaptive designs, and trials that incorporate borrowing of prior information, such as pediatric trials. 

With the Bayesian Supplemental Analysis (BSA) demonstration project, C3TI will partner with sponsors to integrate Bayesian analysis in parallel to frequentist analysis during their trial, providing an opportunity for both CDER and sponsors to learn new methods without impacting review criteria. In contrast to the Complex Innovative Design (CID) Program (more information is available on the CID Program webpage), this demonstration project focuses on simple non-adaptive trial designs that do not incorporate data from previous trials. In addition, during the review of trials participating in this demonstration project, the Bayesian analyses would be supplementary descriptive analyses that would not change the primary analysis methods or decision criteria (e.g., frequentist null hypothesis testing at 1-sided 0.025) for the trial. Increasing the experience and understanding of Bayesian approaches when used as supplementary analyses in an experiment-friendly environment may facilitate their use as primary analyses in appropriate settings in the future.  

Advance understanding and use of Bayesian statistics

This demonstration project seeks to advance the understanding of Bayesian statistical methods and establish processes for integrating these approaches into clinical trial design in the future (e.g., processes for pre-aligning on specifications of prior distributions), which would be shared with parties across the drug development landscape to drive appropriate adoption of Bayesian statistical methods. For consideration and selection to participate within this demonstration project, the trial must be a phase 3 efficacy, safety, or non-inferiority standalone trial (i.e., not incorporating data from previous trials beyond informing the non-inferiority margin) with a simple non-adaptive design. The Bayesian analysis should supplement the primary analysis and may be used to evaluate the primary endpoint in the overall study population and/or in relevant subgroups (i.e., for subgroup analysis). Participating sponsors would support the conduct of the supplementary descriptive Bayesian analyses.

Benefits of participation

Please note that sponsors who are interested in a trial with a Bayesian primary analysis may reach out to C3TI through the program mailbox to be directed to the proper subject matter experts for support.

Sponsors and other industry parties who are willing to participate in this demonstration project will benefit from demonstrating the value of Bayesian methods in drug development, testing new analytical methods in an experiment-friendly environment without impacting the regulatory decision, and partnering with additional FDA/CDER subject matter experts on Bayesian statistics. Furthermore, by participating, sponsor(s) would receive additional CDER support to reach alignment on the statistical analysis plan for the supplemental Bayesian analysis.

See example statistical analysis plans for reference:

Eligibility Criteria for BSA Demonstration Project Proposals

  • The sponsor has an active pre-Investigational New Drug (IND) or IND for the product(s) included in the proposal.
  • The trial must be a phase 3 efficacy, safety, or non-inferiority standalone trial (i.e., not incorporating data from previous trials beyond informing the non-inferiority margin) with a simple non-adaptive design.
  • The Bayesian analysis should supplement the primary analysis and may be used as a supplemental analysis of the primary endpoint in the overall study population and/or in relevant subgroups (i.e., for subgroup analysis). Participating sponsors would support the conduct of the supplementary descriptive Bayesian analyses.
  • The sponsor and FDA can reach agreement on the trial design information to be publicly disclosed.
    • Sponsors participating in demonstration projects will be expected to share select details of their clinical trials and the implementation of clinical trial innovations as they progress, starting as early as the finalization of study design. This sharing may include updates, lessons learned, and relevant insights gathered during the trial. It is understood that these shared details will reflect general principles and innovative aspects, while maintaining the necessary confidentiality of proprietary or sensitive information.

Instructions on how to submit a proposal can be found on the C3TI Demonstration Program Proposal Submission webpage.

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