Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective.

ABSTRACT:

Introduction

The European Medicines Agency (EMA) offers scientific advice to support the qualification procedure of novel methodologies, such as preclinical and in vitro models, biomarkers, and pharmacometric methods, thereby endorsing their acceptability in medicine research and development (R&D). This aspect is particularly relevant to overcome the scarcity of data and the lack of validated endpoints and biomarkers in research fields characterized by small samples, such as pediatrics.

Aim

This study aimed to analyze the potential pediatric interest in methodologies qualified as "novel methodologies for medicine development" by the EMA.

Methods

The positive qualification opinions of novel methodologies for medicine development published on the EMA website betw

SUBMITTER: Giannuzzi V 

PROVIDER: S-EPMC11007141 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature