Posted April 06, 2022 | By Joanne S. Eglovitch
The International Council for Harmonization (ICH) released a draft ICH E11A guideline on Tuesday that establishes a framework for extrapolating drug development data from adult studies to the pediatric population.
“This new ICH guideline aims to promote the international harmonization of methodologies and strategies to incorporate pediatric extrapolation into overall drug development plans and to improve the speed of access to new drugs for pediatric patients, while reducing the number of children who need is being restricted for participation in clinical trials,” ICH said in an announcement.
ICH issued a draft document in June 2017 endorsing the need for a guideline. The document was subsequently issued for consultation by the European Medicines Agency. (RELATED: EMA revises discussion paper on pediatric extrapolation, focus on regulation October 13, 2017)
“Historically, extrapolating security was generally considered unacceptable. However, our understanding of similarities and differences between reference and target populations with regard to safety has evolved,” the guideline states. As described in the ICH E11(R1) guideline, the principle of using data generated in a reference population to determine the scope and size of data to be collected in a target population may also apply to the generating security data.”
The guideline “provides a roadmap to assist drug developers and regulators with the extent to which pediatric extrapolation can be applied, and the information to be collected to address knowledge gaps that support the safe and effective use of drugs in the pediatric population.” .”
The extrapolation framework consists of a pediatric extrapolation concept and then the preparation and implementation of an extrapolation plan. The concept should examine disease similarity and response to treatments between the reference and target pediatric populations, according to a guideline presentation.
An important factor in developing the concept is assessing the similarities and differences of the disease between a reference and a target population. The guideline states that “the evaluation of disease similarity is not intended to determine whether the disease in the reference and target populations is ‘exactly the same’, but rather whether the disease differs in such a way that pediatric extrapolation would be excluded.”
Once a concept has been developed, the extrapolation plans should cover the relevant studies to be performed, such as dose selection and dose range data to be collected. The plan should also address whether model-informed approaches are appropriate and whether biomarkers should be used.
The plan should address potential research designs, such as the use of bridging biomarkers, Bayesian strategies, and single-arm pharmacokinetic/pharmacodynamic (PK/PD) studies.
ICH expects guidance to be finalized in the second quarter of 2024.
I E11A guidelines
I E11A presentation
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