PDA EU00181 CMC Regulatory Compliance Strategy for Advanced Therapy Medicinal Products

11 Sep - 12 Sep 2023
Seville, Spain

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There has been a surge in clinical development of Advanced Therapy Medicinal Products (ATMPs), also referred to as Cellular & Gene Therapy Products (CGTPs), both in the EU and the USA.

Many start-up companies, as well as large biopharmaceutical companies, are planning or have already entered manufacturing these cellular therapies or gene therapies (e.g., genetically engineered viral vectors, mRNA-lipid nanoparticles). While off to a slow start in market approval, the FDA has projected that by 2025 there could be up to 10-12 new ATMPs approved for the market each year.

While many CMC teams are familiar with the CMC regulatory compliance strategy for other biological products (e.g., monoclonal antibodies), they may not understand the significant differences and extra challenges in the CMC regulatory compliance strategy for ATMPs. These extra CMC challenges are due to the increased size and complexity of the nucleic acid-based biologics; the increased challenge in manufacturing of living biologic medicines (substantially manipulated patient cells, genetically engineered viruses and cells); and the limited size of the manufacturing runs (frequently patient-specific batches). This course will provide insights and practical guidance for the CMC teams to develop a cost-effective, risk-based CMC regulatory compliant strategy for the manufacture and control of ATMPs from first-in-human (FIH) clinical studies through market approval. The course emphasis will include the limited FDA and EMA CMC regulatory guidance. Real-world case examples of CMC regulatory compliance problems and delays affecting ATMPs will be discussed.

  1. Understand how CMC Regulatory, cGMPS and Quality System interactively provide patient protection for the manufactured cell and gene therapy products
  2. Explain why a risk-based flexible approach is absolutely required for these advanced therapy medicines
  3. Discuss where it is appropriate and acceptable to apply a minimum CMC regulatory compliance continuum across the manufacturing of these products during clinical development
  4. Learn how to effectively manage product comparability for ATMP manufacturing process changes
Note PDA is committed to deliver safe and secure in-person events. In conjunction with the venue and vendors supporting our event, PDA strictly adheres to all national, provincial and local government regulations. PDA is closely monitoring the situation and will continue to update this information if required. 

Agenda

Discover What's Happening Each Day
Day 1 11 Sep Day 2 12 Sep
Monday, 11 September

Monday, 11 September 2023

9:00 Welcome and Introduction


9:10 CMC Regulatory Compliance is Challenging for ATMPs

  • Discussion of the increasing diversity of ATMPs and their associated regulatory terminology
  • The FDA/EMA regulatory systems in place for ATMPs (IND → BLA; IMPD → MAA)

10:30 Coffee Break


11:00 CMC Regulatory Compliance Differences

  • Discussion of ATMPs being different from the existing biologic medicines
  • CMC regulatory compliance consequences due to those differences

12:30 Lunch Break


13:30 Risk-Managed CMC Regulatory Compliance Strategy for ATMPs

  • Why a risk-managed approach to CMC regulatory compliance is absolutely necessary for ATMPs
  • How three interactive risk-based components protect patients – CMC, cGMPS, Quality System

15:00 Coffee Break


15:30 Recommended Risk-Based Approach for ATMPs

  • Introduction to the ‘Minimum CMC Regulatory Compliance Continuum'
  • Application to ATMPs: Quality by Design (QbD) – QTPP, CQA, CPP – and Quality Risk Management (QRM)

17:00 End of Training Course Day 1

Tuesday, 12 September

Tuesday, 12 September 2023

9:00 Applied Risk-Based, Minimum CMC Regulatory Compliance Continuum for ATMPs

  • ATMP raw materials
  • ATMP starting materials

10:30 Coffee Break


11:00 Applied Risk-Based Minimum CMC Regulatory Compliance Continuum for ATMPs

  • ATMP upstream and downsream manufacturing processes for the API
  • ATMP formulation and drug product manufacturing processes

12:30 Lunch Break


13:30 Specific Challenging CMC Regulatory Compliance Issues for ATMPs

  • Level of manufacturing process control needed – need to identify CQAs sooner than later
  • CMC challenges in patient administration of drug product

15:00 Coffee Break


15:30 Challenges in Demonstrating Product Comparability After Changes

  • Demonstrating ATMP comparability after manufacturing process changes
  • Three risk-based considerations for this comparison

17:00 End of Training Course

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Location and Travel

Venue Details and Accommodations

PLEASE READ  PDA is not affiliated or contracted with any outside hotel contracting company. If someone other than PDA or the PDA chosen hotel contacts you suggesting that they represent any PDA event, they do not. It is PDA's recommendation that you book your hotel directly through the official PDA chosen hotel that is listed on our web site.

PDA recommends the reservation at the following hotel

Melia Lebreros
C. Luis de Morales, 2
41018 Seville
Spain

Tel: +34 954 57 94 00

PDA Europe has reserved a limited number of rooms by 01 August 2023.

Book your room at the PDA Group Rate via the above-mentioned reservation button.


The hotel is located in the heart of Seville, next to the Royal Alcazar and the Plaza de Espana including a 270 qm swimming pool and a fully equipped fitness area.

How to Get Here
By Air The hotel is only 8 kilometers away from Seville International Airport.
By Car On site parking is available. Please contact the hotel for more information.

Registration

Pricing Options

Standard Registration

Member Price

€ 1.895

GovernmentMember Only

€ 1.895

Health AuthorityMember Only

€ 1.895

Early Career ProfessionalMember Only

€ 1.895

StudentMember Only

€ 1.895

AcademicMember Only

€ 1.895

Non-Member

€ 1.895

See Qualifying Criteria for Member Types.

ALL PRICES IN EURO (EUR), EXCLUDING VAT.

WAYS TO REGISTER: 1. Online Registration   2. registration-europe@pda.org

PDA MEMBERS: Please remember to log in before registering If you come from a Governmental or Health Authority Institution or from an Academic Organization, please contact registration-europe@pda.org before registering.

If you need to apply for a visa to enter the event country, PDA Europe must be informed of this fact at least 4 weeks before the start of the event.

Sponsor information will be available soon

We're currently finalizing our list of sponsors and will share the details shortly.

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