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Bayesian Statistics in Medical Device Clinical Trials

This guidance provides direction on the use of Bayesian statistical methods in medical device clinical trials. It covers key statistical aspects of trial design and analysis using Bayesian approaches, including prior information incorporation, sample size determination, interim analyses, and decision-making criteria.

  1. Schedule early meetings with FDA to discuss:
    • Prior information selection and justification
    • Trial design and analysis plans
    • Operating characteristics evaluation
  2. Develop comprehensive protocol including:
    • Prior information details
    • Success criteria
    • Sample size justification
    • Operating characteristics
    • Simulation results
  3. Implement proper trial conduct measures:
    • Randomization
    • Masking where applicable
    • Data quality control
    • Interim analysis procedures if planned
  4. Prepare detailed documentation of:
    • Statistical models and assumptions
    • Software code and calculations
    • Sensitivity analyses
    • Model checking results
  5. Plan for clear presentation of results:
    • Understandable labeling language
    • Appropriate summaries of posterior distributions
    • Credible intervals reporting
    • Comprehensive final report
  6. Consider post-market surveillance planning using Bayesian methods to update safety and effectiveness information

Key Considerations

Clinical testing

  • Clinical trial planning and rigorous conduct are important regardless of statistical approach
  • Basic tenets of good trial design remain the same for both Bayesian and frequentist trials
  • Randomization and masking principles should still be followed
  • Pre-specification of analysis method (Bayesian vs frequentist) is required

Software

  • Software programs capable of Bayesian calculations should be used (e.g., WinBUGS)
  • Program code and data used for simulations should be submitted
  • Electronic submission of calculations is recommended for FDA review

Labelling

  • Results from Bayesian trials need to be expressed clearly in device labeling
  • Bayesian terminology should be understandable
  • Credible intervals should be reported

Safety

  • Minimum sample size should be determined based on safety endpoints
  • Safety endpoints may require larger sample sizes than effectiveness endpoints
  • Post-market surveillance can use Bayesian approaches to update safety information

Other considerations

  • Prior information selection and incorporation must be carefully justified
  • Operating characteristics (type I/II errors) must be evaluated
  • Interim analyses and adaptive designs need pre-specification
  • Model assumptions should be checked and sensitivity analyses performed
  • Hierarchical models can be used to combine data across studies
  • Missing data handling requires appropriate methods and sensitivity analyses

    Relevant Guidances

  • Bayesian Statistics in Medical Device Clinical Trials: Design, Analysis and Implementation
  • MCMC: Markov Chain Monte Carlo techniques for Bayesian computation
  • DIC: Deviance Information Criterion for model selection
  • CODA: Convergence Diagnosis and Output Analysis for MCMC

Original guidance

  • Bayesian Statistics in Medical Device Clinical Trials
  • HTML / PDF
  • Issue date: 2010-02-05
  • Last changed date: 2020-02-28
  • Status: FINAL
  • Official FDA topics: Medical Devices, Radiation-Emitting Products, Premarket
  • ReguVirta summary file ID: 92673262c724b66c15067383a41580e9
This post is licensed under CC BY 4.0 by the author.