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.
Who should attend?
- 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
Programme Day One
Introduction to ATMPS
The regulatory landscape
- Definition of ATMPs and associated terms
- Classification of ATMPs
Understanding clinical trials
- 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
Insight into global regulations and requirements
- 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
Overcoming quality challenges
- 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
Risk-based approach for ATMPs
- 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
Programme Day Two
GMP for ATMPs
Delivery and shipping
- 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
Preparation for clinical trial
- Considering stability issues
- Challenges in transporting cell therapies/cryopreservation
- Preparation on site
Practical considerations for the Investigational Medicinal Product Dossier (IMPD)
- 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
Alison is an independent regulatory affairs consultant. Previously regulatory affairs manager for Smith & Nephew Wound Management, she has over 25 years’ experience of regulatory affairs in medicinal products, human tissue products and medical devices. She is a nominated UK expert for ISO (International Standards Organisation) TC150/WG 11 – Tissue Engineered Medical Products and a member of BSI Technical Panel RGM/1 – Regenerative Medicine.
Alison is a Module Advisor for the TOPRA* MSc in Regulatory Affairs (Pharma) (Module 9 – Biotechnology) and was Module Advisor for the MSc in Medical Technology Regulatory Affairs (Module 6 – Combination Products and Other Technologies) from 2006-2012. She is a member of the Topic Selection Panel for the MHRA Medical Device Technology Forum. She was a member of the MHRA (then MDA) working group responsible for production of the Code of Practice for Production of Human-Derived Therapeutic Products, 2002, and is the technical author for the BSI publication PAS 93: Characterisation of human cells for clinical application, published August 2011.
CellData Services provides strategic regulatory and development advice for a range of UK, EU and US clients seeking to develop and commercialise advanced therapy medicinal products (ATMPs), regenerative medicine therapies and drug-device combination products in the EU. Alison has extensive expertise in guiding smaller companies and academic groups through the regulatory complexities of development of novel regenerative medicine products, in particular scientific advice procedures and first-in-man clinical trials, and in recognition of this has received the first TOPRA Futures Award for contribution to regulatory science in new technology areas.
An independent consultant with 32 years of experience with regulatory affairs and pharmaceutical development. Experience at multiple levels within pharmaceutical companies and most recently as Vice President for Regulatory Affairs, gaining significant experience in co-ordinating, planning and running global development projects.
His experience is wide ranging, allowing fast, effective development of medicinal products, through early stages, through registration and continued Life Cycle Management.
Since acting as an independent consultant his roles have included:
- Interim Strategic advice to major pharmaceutical company for roll-out and compliance strategies for Russia / CIS, MENA, ASEAN, LATAM, South Africa and Switzerland, including ensuring appropriate staff development and strategies which ensure protection of intellectual property whilst maintain patient focus.
- Orphan drug registrations of vaccine products and synthetic molecules
- NCE approved in US and Europe for urology product (Simultaneous NDA and EU Centralised submission)
- Support for NBE during clinical phases and CMC support for a Biosimilar, Biosimilar / Biologic CMC reviewer for major pharma. Biosimilar experience includes working on first EU biosimilar onwards.
- Oversight of Regulatory strategy for ATMP (Cancer vaccine) and CMC lead for Stem Cell treatment
- Training to major pharmaceutical companies for Chemistry and Pharmacy section of the dossier and Life Cycle Management. Additional lead training / presentations on Life Cycle Management for TOPRA.
Furthermore, his responsibilities have provided senior management oversight and direction in the following key areas: -
- Strategic management of ATMP products for Biotech start-up company
- Strategic Biosimilar team, developing a global biotech and biosimilar strategy for key biosimilar products.
- Global Quality Leadership Team, providing oversight and direction for global quality processes.
- Innovation Team, providing oversight and direction for the innovation.
Additional experiences reflect raising funds for new start-up companies and associated due diligence activities. Also he has substantial awareness of management of P & L performance, growth initiatives related to service expansion, geographical expansion and innovative business development approaches.