Module232025

5/7/2025

Examples of Sources and Types of Data to Evaluate for Similarity of Disease and Response to Treatment

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Moving forward from concept to plan

Integrate evidence

Comprehensive review of existing evidence + Evaluation of strength of evidence

Paediatric extrapolation plan Dose selection Model-informed approaches Efficacy studies

• Quantitative synthesis of existing data (frequentist approaches by comparison of point

Knowledge gap identification

estimates and associated CIs)

• mechanistic and/or empirical approaches

• inclusion of systems

biology/pharmacology population-level data

• meta-analytic techniques

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