This course has been designed to provide a comprehensive overview of the regulatory, quality and good manufacturing practice (GMP) requirements to consider when developing advanced therapy medicinal products (ATMPs).
Industry experts 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. 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 addressed.
This programme will be of benefit to all those involved with or considering developing an ATMP.
Benefits of attending:
- Understand the GMP requirements for clinical trials in the 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.
Programme Day One
Introduction to ATMPs
- Definition of ATMPs and associated terms
- Classification of ATMPs
The regulatory landscape
- 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 Device Directive (93/42/EEC) and forthcoming regulation
Understanding clinical trials
- 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 the EU and US
- Understanding breakthrough status/PRIME
Insight into global regulations and requirements
- 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 the US and Europe - ramifications for your product
Overcoming quality challenges
- 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
Risk-based approach for ATMPs
- Examining 3.2.A Adventitious Agents
- Level of data required EU/US
Programme Day Two
GMP for ATMPs
- What is required: examine the major differences between GMP for ATMPs and conventional therapies
- Overcoming potential pitfalls when manufacturing cells
- Current GMP interpretations
- Multiple manufacturing sites for autologous products
- Point-of-care preparation devices
Delivery and shipping
- Considering stability issues
- Challenges in transporting cell therapies/cryopreservation
- Preparation on site
Preparation for clinical trial
- 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
Practical considerations for the IMPD
- Terms and definitions
- Guidance on IMPD content for ATMPs
- Data requirements for first-in-human vs later clinical trials
Ms Alison Wilson,
CellData Service ,
Alison Wilson 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.
She is now Principal Consultant at CellData Service who provide 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. She 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.
Mr Andrew Willis,
Andrew Willis is an independent consultant providing expert advice and training on global regulatory solutions and pharmaceutical development. Previously, he worked for Catalent Pharma Solutions as VP Regulatory Affairs & Consulting Services. Catalent is the world’s leading contract manufacturer and distributor of pharmaceuticals, and he was head of a team of internal and external regulatory affairs consultants. He qualified as a Chemist from the University of Glamorgan, after which he furthered his understanding of pharmaceutical development, working as a research chemist with Parke Davis. He has 10 years manufacturing and analytical experience prior to entering regulatory affairs as a Senior Executive Officer with responsibility for submission of European MAAs and project management of development programs. He currently has a total of 28 years pharmaceutical experience with extensive knowledge in the development and manufacture of sterile, solid oral, inhalation, topical and biotech pharmaceutical products. These experiences have allowed knowledge of many Biotech products requirements with experiences of growth hormones and multiple cancer treatments, including development and clinical registration of the first genetically modified live bacterium for such treatment. He has extensive experience of major European and US regulatory projects, in the clinical and marketing authorisation stages, and has significant experience in coordinating and managing meetings with European and US Health Authorities. Specific experience includes the project management of a large MAA requiring full clinical data, followed by Mutual Recognition of the application in all of the European Concerned Member States. The project recorded successful outcomes in all major markets (26 countries) and was viewed as highly successful by the client, meeting very stringent project timings.
- 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
11 Thurloe Pl,
The Rembrandt Hotel is located at 11 Thurloe Place, London, SW7 2RS. The hotel’s location in central London couldn’t be better, whether you’re travelling for business or leisure. You’ll be right between two of London’s most fashionable areas – South Kensington and Knightsbridge – within walking distance of museums, theatres, Harrods and Hyde Park.
You can hop on the Tube at South Kensington and take the District, Circle or Piccadilly lines direct to the City of London, Heathrow Airport and all other areas of the city. Look out of your window and you may well see the Victoria and Albert Museum – it’s right across the street.