Module232025

12/05/2025

What is it not

• It is not following a decision tree with binary decisions

‘…whether the disease and expected response to treatment can be considered sufficiently similar between a target and reference population is not simply a “ yes or no ” question’

• Not a ‘tool’ used to retrofit available data to support a benefit/risk assessment

• Not a ‘tool’ used because one knows a development is ‘unfeasible or challenging to complete’ over time

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What it is

ICH E11A: ‘ an approach to providing evidence in support of effective and safe use of drugs in the pediatric population when it can be assumed that the course of the disease and the expected response to a medicinal product would be sufficiently similar in the pediatric [target] and reference (adult or other pediatric) population ’  also to addressing one of the most significant challenges that is to avoid the unnecessary enrolment of children in clinical trials

• Its use should be agreed prospectively and based on scientific considerations, approached as a continuum

• It starts with the clinical question and is about identifying (extrapolation concept) and addressing uncertainties (extrapolation plan)

 quantitative tools, like M&S, statistical approaches (eg Bayesian stats), RWD may be leveraged to fill gaps in knowledge and/ or reduce uncertainties

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https://www.ema.europa.eu/en/ich-guideline-e11a-pediatric-extrapolation-scientific-guideline

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