Federal Office for Safety in Health Care www.basg.gv.at

Update on investigational ATMP guideline

Ilona Reischl, PhD BASG/AGES - Federal Office for Safety in Health Care Austrian Agency for Health and Food Safety Traisengasse 5, 1200 Vienna, Austria

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§  The practical application of the Clinical Trials Regulation has been delayed to Q2 2019

§  We have been working on the Guideline for investigational ATMPs since the beginning of 2016

§  National clinical trials assessors had been invited via the CTFG and have been participating in the drafting activities

Update Where are we now

Development of a guideline on Investigational ATMPs §  Initiate development of guideline – external consultation in Q4 2016 §  Finalise guideline after external consultation - completion Q2 2017

§  We are clearly delayed §  What are the reasons?

CAT Workplan 2016 Timelines

The guideline has not been sent out for comments yet Why? §  Efforts spent on GMP for ATMPs §  Finalisation of the Guideline on quality, non-clinical and clinical

aspects of gene therapy medicinal products à Adoption Q1 2018

§  Alignment issues – which content goes into which guidance document

§  Limitation of resources

Update Where are we now

What kept us busy? Now published

EudraLex The Rules Governing Medicinal Products

in the European Union Volume 4 Good Manufacturing Practice

Guidelines on Good Manufacturing Practice specific to Advanced

Therapy Medicinal Products

http://ec.europa.eu/newsroom/sante/newsletter-specific-archive-issue.cfm?newsletter_service_id=327&newsletter_issue_id=6070&page=1&fullDate=Wed%2022%20Nov%202017&lang=default https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-4/2017_11_22_guidelines_gmp_for_atmps.pdf

§  Revision of the Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.

§  Publication of the draft of the revised Guideline for external

consultation by 1Q 2018 §  Finalise the Revision of the Guideline by 4Q 2018 §  CAT will collaborate with the BWP for the revision of the quality

part of this guideline.

CAT Workplan 2017 Revision of ATMP specific guidelines

§  The initial plan of developing a Guideline on comparability of cell-based medicinal products has been changed, instead the format of a Q&A document is considered as better suitable

Why? §  There are already general texts in all documents (for cell- and

gene therapies), including in the guideline for investigational ATMPs

§  The general principles in ICH Q5E apply §  For case-by-case explanations a Q&A is better suited

Comparability Format of guidance

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§  Alignment with current EMA guidance and taking into consideration guidance from other agencies

§  Intended applicability for all ATMPs, coverage of quality, non-clinical and clinical aspects

à specific drafting groups

§  Main focus is on minimal requirements for early clinical trials, but guidance for later development will also be included

§  Differentiation between exploratory and pivotal clinical trials rather than phases

iATMP Content Unchanged

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§  Text for cell-based and gene therapy products is drafted separately and will be brought together at a later stage

§  Existing GL on non-clinical requirements for gene therapy products will be incorporated

§  Considerations for combination products will be included

Content Unchanged

Next drafting group meeting 6.12.2017 – joint meeting §  Cell based products - quality – close to first draft §  Gene therapy - quality – wait for finalisation of GT GL §  Preclinical – first draft ready §  Clinical – first draft ready The individual first drafts will be completed and compiled. There will be a need for alignment.

iATMP Status quo

Development of a guideline on Investigational ATMPs §  Discuss with developers (interested parties) the key aspects on

which they would like to have regulatory clarification on. •  Deadline: 2Q 2018

§  Publication of the draft of the Guideline for external consultation in Q4 2018

§  Finalise guideline after external consultation - completion Q3 2019

CAT Workplan 2018 Timelines

Federal Office for Safety in Health Care www.basg.gv.at

Thank you for your attention!

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