News Hub

Alejandro Barquero, MPharm, MSc 
CMC Manager
ISCT Legal and Regulatory Affairs Committee: Europe
DanausGT Biotechnology Ltd.
Madrid, Spain

Laura Mendieta, PhD
Senior Scientific and RA Officer Project Manager
ISCT Legal and Regulatory Affairs Committee: Europe 
Asphalion
Barcelona, Spain

Committee of Advance Therapy (CAT)

Minutes of the meetings

• Minutes for the meeting on 17-19 January 2024

https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-17-19-january-2024_en.pdf

CAT quarterly highlights and approved ATMPs

• November 2023 - January 2024

https://www.ema.europa.eu/en/documents/committee-report/cat-quarterly-highlights-approved-atmps-november-2023-january-2024_en.pdf

Updates from Regulatory Agencies

Updates from the EMA

• Progress update on pilot scheme for academic and non-profit developers of advanced therapy medicines – Feb 8th

https://www.ema.europa.eu/en/news/progress-update-pilot-academic-non-profit-developers-advanced-therapy-medicines

• EMA and the Heads of Medicine Agency (HMA) have launched two new public electronic catalogues of real-world data (RWD) sources and one for working data (WD) studies – Feb 15th

https://www.ema.europa.eu/en/news/launch-new-hma-ema-catalogues-real-world-data-sources-studies

• European Medicines Agency pre-authorisation procedural advice for users of the centralised procedure – March 27th

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-pre-authorisation-procedural-advice-users-centralised-procedure_en.pdf

• Clinical trials' transition to new EU system - All ongoing clinical trials in the EU must be transitioned to the Clinical Trials Information System (CTIS) by 31 January 2025.

https://www.ema.europa.eu/en/news/clinical-trials-transition-new-eu-system-one-year-left

Updates from the HMA

• EUCTR/CTIS

A new CTCG Best Practice Guide for sponsors – first substantial modification (SM) Part I after CTR transition and its 2 annexes (cover letter template, SM template for first SM post transition (version 1, dated 19 Mar 2024) providing further guidance on which documents to update in relation to the post transition SM, and supportive templates for compiling that SM – March 19th

https://www.hma.eu/fileadmin/dateien/HMA_joint/00-_About_HMA/03-Working_Groups/CTCG/CTCG-Best-Practice-Guide-to-sponsors-updating-the-application-dossier-PartI-after-CTR-transition_vs_1.0.pdf

A new recommendation paper on principles of Good Laboratory Practices for clinical trial applications under CTR, issued by CTCG together with relevant EMA and EC working groups and parties (version 1, March 2024) and an accompanying template table. This document is also mentioned in the latest version of the CTR Transition Q&A, updated earlier this month by the EU Commission, and provides considerations on the requirements regarding OECD GLP compliance (and regulatory acceptability) of pivotal non-clinical data submitted to support a clinical trial application submitted in the EU for sponsors, test facilities, and other interested parties – March 2024

https://www.hma.eu/fileadmin/dateien/HMA_joint/00-_About_HMA/03-Working_Groups/CTCG/GLP-recommendation-paper.pdf

An updated CTCG Best Practice Guide for sponsors of multinational clinical trials with different protocol versions approved in different Member States under the Directive 2001/20/EC that will transition to the Regulation (EU) No. 536/2014 (now version 4, dated March 2024) and its corresponding cover letter template, with further clarifications on low intervention clinical trials, auxilliary medicinal products and managing archiving rules when transition trials include countries ended under CTD and ongoing / transitioned to CTR.

https://www.hma.eu/fileadmin/dateien/HMA_joint/00-_About_HMA/03-Working_Groups/CTCG/2024_03_CTCG_Best_Practice_Guide_for_sponsors.pdf

ICH

• ICH Q12 IWG “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management" - publication of the ICH Q12 “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management Introductory Video” – March 4th

https://ich.org/page/quality-guidelines#12-2

• The ICH E2D(R1) draft Guideline presentation available now on the ICH website

Post-approval safety data: definitions and standards for management and reporting of individual case safety reports

https://database.ich.org/sites/default/files/ICH_E2D%28R1%29_Step3_Draft%20Guideline_2024_0205.pdf

MHRA

• MHRA announces two new UK Approved Bodies to certify medical devices – Jan 31st

https://www.gov.uk/government/news/mhra-announces-two-new-uk-approved-bodies-to-certify-medical-devices

EDQM

• EDQM has published a document a summary of the top ten deficiencies identified after the initial evaluation of new applications for Certificates of Suitability (CEP) for chemical purity – Feb 8th

https://www.edqm.eu/en/-/read-the-top-10-deficiencies-observed-in-new-cep-applications-for-chemical-purity-assessed-in-2023-to-improve-the-quality-of-your-applications-

Public Consultations

• Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials – Draft 2nd version - Consultation ends on 31 May 2024

https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-quality-non-clinical-clinical-requirements-investigational-advanced-therapy-medicinal-products-clinical-trials-second-version_en.pdf

• Non-inferiority and equivalence comparisons in clinical trials - Scientific guideline – Consultation ends on 31 May 2024

https://www.ema.europa.eu/en/documents/scientific-guideline/concept-paper-development-guideline-non-inferiority-equivalence-comparisons-clinical-trials_en.pdf

Other updates

• The new JOIN4ATMP project aims to accelerate European ATMP development and ensure wide-spread ATMP access while fostering the expansion of strong Good Manufacturing Practices (GMP) on a European level – Jan 25th

https://join4atmp.eu/