Module 1 2021

01/07/2021

Extrapolation

• Extrapolation is often necessary for the whole paediatric or subsets of the paediatric population • Reducing unnecessary trials in children • For decrease the (efficacy) uncertainties with regard to the use of medicine in children • Extrapolation explicit in > 50 agreed PIPs • Consider the reflection paper on extrapolation; • Similarity in disease, treatment, prognosis between subgroups of patients • Similarity in mode of action of the medicine • PK/PD relation • Similarity in exposure https://www.ema.europa.eu/en/documents/scientific-guideline/adopted- reflection-paper-use-extrapolation-development-medicines-paediatrics- revision-1_en.pdf

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• The condition which is covered • The population for which a waiver is approved • The studies (measures) that need to be conducted PIP opinion contains

• Key binding elements • Study objective • Study design • Patient population; number of patient • Endpoints • .. • Timelines

The Organisation for Professionals in Regulatory Affairs

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