Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - Scientific guideline

This guideline provides guidance on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with advanced therapy investigational medicinal products.

Keywords: Investigational ATMPs, gene therapy medicinal product, cell therapy medicinal product, tissue engineered product, exploratory trial, first in human trial, confirmatory trial

Current version

Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials

Adopted Reference Number: EMA/CAT/22473/2025 Legal effective date: 01/07/2025

Overview of comments received on 'Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials’ (EMA/CAT/123573/2024) – second public consultation

Reference Number: EMA/264632/2024

Document history

Draft guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - Second version

Draft: consultation closed Consultation dates: 25/03/2024 to 31/05/2024 Reference Number: EMA/CAT/123573/2024 Summary:

This guideline provides guidance on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with advanced therapy investigational medicinal products.

Note: This is a short, second public consultation for the guideline. All comments received during the first public consultation have been reviewed and incorporated, where possible, in this guideline. Stakeholders can consult the ‘Overview of comments’ document: comments submitted on the first version of this guideline should not be resubmitted.
 

Overview of comments received on 'Draft guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - first version'

Reference Number: EMA/62329/2024

Draft guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - First version

Draft: consultation closed Consultation dates: 21/02/2019 to 01/08/2019 Reference Number: EMA/CAT/852602/2018