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Helle Lynggaard1, Oliver N Keene2, Tobias Mütze3

  • 1Novo Nordisk A/S, Bagsvaerd, Denmark.

Pharmaceutical Statistics
|August 8, 2024
PubMed
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This paper addresses challenges in applying the estimand framework to non-inferiority trials. It offers guidance on defining estimands for these trials, ensuring robust clinical trial design and interpretation.

Area of Science:

  • Clinical Trials Methodology
  • Biostatistics
  • Pharmaceutical Research

Context:

  • The estimand framework, crucial for defining the target of estimation in clinical trials, has primarily been applied to superiority trials.
  • Non-inferiority trials present unique challenges not fully addressed by existing estimand guidance, particularly from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).

Purpose:

  • To provide comprehensive guidance for defining estimands in clinical trials with a non-inferiority objective.
  • To explore how the estimand framework can incorporate pre-ICH addendum methods for non-inferiority, including the role of Per Protocol analysis sets.

Summary:

  • This paper discusses key considerations for defining estimands in non-inferiority trials, examining how the estimand framework can be adapted.
Keywords:
ICH E9(R1)clinical question of interestestimand frameworknon‐inferiorityregulatory guidancesuperiority

Related Experiment Videos

  • It addresses the formulation of relevant clinical questions, the appropriateness of 'conservative' estimands, and the impact on trial design elements like multiple primary estimands, non-inferiority margins, assay sensitivity, and switching objectives.

    • Impact:

    • Offers practical recommendations and considerations for trial teams designing non-inferiority trials.
    • Enhances the rigorous application of the estimand framework in non-inferiority settings, improving the clarity and validity of trial results.
    • Contributes to the standardization of statistical approaches in pharmaceutical development for non-inferiority studies.