Pharmaceutical Development of ATMPs

The programme will cover the regulatory landscape and the definition and classification of ATMPs and provide practical guidance on how to overcome quality and specific GMP challenges. Comparison of the differences between GMP for ATMPs and conventional therapies will be discussed, as will the requirements for clinical trials and the preparation of the IMPD. Advice on the specific transportation requirements of these products will also be included.

This programme will be of benefit to all those involved with or considering developing an AMTP.

Benefits of attending:

• Understand the GMP requirements for clinical trials in US and Europe
• Gain a practical insight into other markets
• Determine critical quality attributes
• Develop a successful QC strategy
• Examine the major differences between GMP for ATMPs and conventional therapies
• Overcome potential pitfalls when manufacturing cells
• Discuss multi-manufacturing sites for autologous products
• Consider stability issues
• Examine risk analysis for biological materials and understand Annex 16
• Gain an introduction to GMO approval requirements

This seminar will be of benefit to all those involved with or considering developing an Advance Medicinal Therapeutic Product.

• R&D personnel involved in research on cell or gene-based therapies
• Managers involved in the development and manufacture of ATMPs
• Quality assurance and quality control personnel responsible for quality aspects of ATMPs
• GMP managers responsible for implementing GMP in ATMP manufacture
• Regulatory personnel involved in inspections of ATMPs

• Definition of ATMPs and associated terms
• Classification of ATMPs

• Examining the European regulatory landscape for ATMPs – detailed analysis of existing regulation 1394/2007
• Considering factors lying outside of the regulation’s scope, e.g. combination products
• Links to related directives, eg Tissues and Cells Directives (2002/98/EC and 2004/23/EC); Medical Devices Directive (93/42/EEC) and forthcoming regulation

• EU clinical trial requirements
• The US IND

• Opportunities to meet with regulators to maximise approval chances
• ATMP-specific options in the EU: certification procedure, risk-benefit approach
• Accelerated access opportunities in EU and US
• Understanding breakthrough status/PRIME

• Examining key markets including Europe, US and Japan
• Evaluate the regulatory differences between regions to help you build a strategic approval route
• GMP requirements at Phase I in US and Europe – ramifications for your product

• Overview of frequent quality concerns
• Potency assay development
• Determining critical quality attributes
• Developing a successful QC strategy for short shelf-life product
• QP release: timing and logistical challenges for ATMPs

• Examining 3.2.A Adventitious Agents
• Level of data required EU/US

• What is required: examine the major differences between GMP for ATMPs and conventional therapeutics
• Overcoming potential pitfalls when manufacturing cells
• Current GMP interpretations
• Multiple manufacturing sites for autologous products
• Point-of-care preparation devices

• Considering stability issues
• Challenges in transporting cell therapies/cryopreservation
• Preparation on site

• Optimising materials for regulatory compliance – vendor qualification
• Risk analysis for biological materials
• The comparability concept and its importance in preparing for clinical trials
• Introduction to GMO approval requirements

• Terms and definitions
• Guidance on IMPD content for ATMPs
• Data requirements for first-in-human vs later clinical trials