Background

Advanced therapy medicinal products (ATMP) are an emerging class of innovative biopharmaceutical medicines, summarizing gene therapy, somatic cell therapy and tissue-engineered products. With the adoption of the ATMP regulation EC 1394/2007, ATMPs are regarded as medicinal products and must consequently comply with current EU drug legislation including GMP. Although the pharma industry recently increased their activities in this new area the development of these complex products is still focused at universities, hospitals and spin off companies derived thereof (small medium enterprises, SME).  This brings special challenges for compliance, for these SME's, with the regulatory requirements on marketing authorization and GMP.  With the publication of the new stand-alone guidance document on the GMP requirements in November 2017, EMA tried to define the expected standards for this special kind of medicinal products.

Target Group

This course is advisable to people who

• Are involved in basic or translational research on  cell-based therapy concepts with the perspective of clinical application
• Are responsible for quality aspects on ATMP
• Implement GMP in ATMP manufacturing
• Are involved in regulatory inspections of  ATMP
• Are responsible for GMP requirements during pre-approval phases

Programme

Regulatory Expectations on Tissues, Tissue Preparations and ATMPs – an Introduction

• Overview on Products and Therapies: Reality and Future
• Legal Framework in EU and Germany
• CTA, Hospital Exemption and Marketing Authorisation: Steps to Consider in the Development of ATMPs

Quality Aspects of ATMP – Practical Experiences from a CMO’s point of view

• Complexity of ATMPs and good Technology Transfer
• Quality Assessment on Cell Products after Non-Conformances
• The role of the QP

Requirements on Manufacturing of Cell-based products and Inspection Experiences

• Important Aspects for Characterisation and Control
• Quality of Reagents and Materials
• Relevant guidance documents
• Inspection Experiences and Findings
• Common Deficiencies in Clinical Trial Applications

Quality and manufacturing aspects of CAR-T-Cells

Microbiological Safety of Advanced Therapy Medicinal Products

• Aseptic manufacturing of ATMPs according new Guidelines on Good Manufacturing Practice specific to Advanced Therapy Medicinal Products.
• How to apply the Ph. Eur. 2.6.27 Microbial examination of cell-based preparations using Automated Growth-Based Methods, Method suitability studies and results.
• Microbiological security of ATMPs. A review of the main sources of microbial contamination: from starting and raw materials to finished products.
• The big challenge of the future: Viral security of allogeneic ATMPs. The immunosuppressed hosts factor and viral reactivation.

Sterility Testing of ATMP

• Challenges of sterility testing of ATMPs
• Matrix-specific selection of a rapid method for sterility testing
• Time to result / negative-to-date results
• Analytical characterization of ATMPs according to EMA expectations
• Requirements and categorization for ATMPs
• Examples and analytical designs for selected drugs
• Developing and control under GL(C)P & GMP
• Products on the market – description and characterization

The New EU-GMP Guideline for ATMP (Part I)

• Guideline Overview
• ATMPs & Quality Risk Management
• Zone Concept for ATMP-Facility
• Focus: Qualification & Monitoring

The New EU-GMP Guideline for ATMP (Part II)

• Focus: Process-Validation & Media Fill
• Focus: Documentation
• How to certify/release an ATMP Batch
• Specific Products/Processes

GMP for ATMP – Considerations to European and US Requirements from industrial point of view

• Essential Effects of the New Guideline
• Challenges in Practice
• US Requirements

Development and implementation of a large scale GMP production plant for EMA – approved ATMP cartilage substitute

• Scaling-up ATMP production does not involve only dimensional considerations
• A correct management of production complexity reflects on the overall process quality
• Hardware and Software innovative approaches are fundamental for a successful and cGMP compliant outcome

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