"Extensive Proof-of-Concept Studies in TNF-Alpha Antagonists might be Responsible for A Delay of Patient Access in Pediatric Rheumatology"

Juergen Brunner

doi:10.26717/bjstr.2021.39.006244

Abstract

Highlights

In order to improve the route to market for approved pediatric therapeutics, the current Pediatric Regulation in the EU and the Food and Drug Administration (FDA) Amendments Act (FDAAA) were both adopted in 2007
It compares data obtained from studies using etanercept, infliximab and adalimumab in juvenile idiopathic arthritis (JIA) and adult rheumatoid arthritis (RA) and argues that efficacy and safety in children could be extrapolated from the data in a corresponding adult disease, for chimeric or pegylated biologics, PK/PD parameter analysis would need to be investigated before selecting the effective dose in children
The question arises – is it important to carry out confirmatory studies in children? Are these studies really necessary or can the data for biologics be extrapolated if the expression of the respective target is the same in adults and children? The data reviewed suggests that the results obtained in adult RA studies are likely to be useful in predicting the dose, efficacy and safety for children with JIA

Summary

Introduction

In order to improve the route to market for approved pediatric therapeutics, the current Pediatric Regulation in the EU and the Food and Drug Administration (FDA) Amendments Act (FDAAA) were both adopted in 2007. After almost a decade of experience of PIPs, it seemed important to evaluate the usability of data derived from clinical trials for new medicinal products in children for marketing authorization This is important in order to understand the need for, and extent of, clinical studies for new drugs in children in the future. The aim of this study is to evaluate whether proof-of-concept clinical trials need to be carried out at the existing rate and frequency, and whether data to support the use of new drugs in children can be extrapolated from adult trials of equivalent indications with focus on rheumatology. It compares data obtained from studies using etanercept, infliximab and adalimumab in juvenile idiopathic arthritis (JIA) and adult rheumatoid arthritis (RA) and argues that efficacy and safety in children could be extrapolated from the data in a corresponding adult disease, for chimeric or pegylated biologics, PK/PD parameter analysis would need to be investigated before selecting the effective dose in children

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Discussion

Conclusion