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Session Overview
Session
Short Course 3: Implementing the estimand framework in global drug development: Application of causal inference approaches
Time:
Sunday, 03/Sept/2023:
11:00am - 12:30pm

Location: Lecture Room U1.141 hybrid


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Presentations

Implementing the estimand framework in global drug development: Application of causal inference approaches

Mouna Akacha1, Björn Bornkamp1, Alex Ocampo1, Jiawei Wei2

1Novartis Pharma AG, Basel, Switzerland; 2Novartis Institutes for Biomedical Research Co., Shanghai, China

This half-day short course introduces how causal inference approaches are relevant and used in the implementation of estimands framework in drug development. It includes 4 lectures:

Lecture 1 - Introduction to Estimands and Causal Inference:

  1. Overview of the estimand framework and key points in ICH E9(R1)
  2. Introduction to causal inference, including potential outcomes, causal effects and common assumptions;

Lecture 2 - Estimation Methods of Causal Effects Targeting at Hypothetical Estimands:

  1. Introduction to common estimation methods, e.g., g-computation, IPW (Inverse probability weighting)
  2. RCT examples illustrated using R code;

Lecture 3 - Principal Stratum:

  1. Introduction to principal stratum estimand
  2. Estimation strategies
  3. Case studies in RCTs;

Lecture 4 - Conditional and Marginal Treatment Effects:

  1. Introduction to conditional and marginal treatment effects
  2. Appropriate estimators for conditional and marginal estimands
  3. RCT examples illustrated using R code.


 
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