Global Regulatory Perspectives Workshop

Pre-Conference Workshop
Wednesday, May 2
08:00 - 17:45

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The ISCT Global Regulatory Perspectives (GRP) workshop represents the diverse activities and partnerships between international regulatory bodies, industry, clinicians and academia.

The GRP Workshop is an annual one-day program.  Unique to this workshop is the attendance of regulators from North America, Europe, Asia, South & Central America and Australia & New Zealand.  It is a highly interactive program including various hypothetical case studies throughout.

Supporters

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Program Information

Moderator: Dominic Wall, PhD, FFSc(RCPA), Operations Director, Peter MacCallum Cancer Centre, Australia

Speakers:
John Ng, Chief Operating Officer, Tessa Therapeutics, Singapore

Patient specific cell-based immunotherapies have evolved into potent therapeutic products that are being considered for multiple markets on the basis of small-scale clinical studies. What does this mean for global supply where the clinical data will predominantly be developed in one market? How is this affected by complex overlays of local donor management and centralized single site manufacturing elsewhere, and what does this mean from the perspective of the inspectorates?

Case study to follow

Moderator: Christopher Bravery, PhD, Consulting on Advanced Biologicals Ltd

Speakers:
Nadine K. Kolas, PhD, Senior Policy Analyst, Health Canada, Canada
Victor Lietao Li, MD, Founder and CEO, Lion TCR Pte Ltd, Singapore

Unlike other biological medicinal products, autologous cell-based products in particular, pose unique challenges because the donor starting material needs to be procured at the hospital and shipped to the manufacturing site within a short period of time (typically less than 48h).  Consequently, when expanding operations into other markets, local rules for donor collection need to be addressed.

Some new markets will be too far from the initial manufacturing site to allow timely shipment, in which case manufacturing needs to be located closer to or in the new market.  Even simply replicating an existing process at another site can lead to small changes in procedures, equipment and raw materials, but also local regulatory expectations may differ.

Case Study to Follow

Moderator: Karen Nichols, Esq, Vice President Regulatory and Quality, Magenta Therapeutics, United States

Speakers:

Marc Turner, MBChB, PhD, MBA, Medical Director, Scottish National Blood Transfusion Service, Scotland
Shari Targum, MD, MPH, Branch Chief, Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States
Michael Pepper, MBChB, PhD, MD, Director, Institute for Cellular and Molecular Medicine and SAMRC Extramural Unit for Stem Cell Research and Therapy, University of Pretoria, South Africa

Sponsor wants to adapt their clinical study design to a pediatric population in Europe.  They want to use as much of the same design as possible although clinical assessments from the adult study suggest that apheresis yields patient-specific cell populations at collection which may contribute to more variability in manufacturing products.  How should the sponsor manage this variability?  What, if any, additional product characterization should be applied to the starting T-cell material as well as the final product? Collections will occur at various locations, even if processing is centralized, introducing another element of variability.  To what extent should the harvest be considered part of the trial design, and to what extent are failed cell harvests and insufficient recovered cell doses relevant to the regulatory strategy?  What is the role of a pediatric investigation plan when administering a personalized medicine?

Given that this is a small (in numbers) patient population, patients will present and perhaps not meet the inclusion criteria – will the sponsor opt for expanded access for these patients?  What is the regulatory view of doing such for a CAR-T product?

Moderator: Janet Macpherson, PhD, Development Manager, Royal Prince Alfred Hospital, Australia

Speakers:
Christiane Niederlaender, PhD, Senior Quality Assessor, MHRA, United Kingdom
Ramjay S. Vatsan, PhD, Team Lead, Division of Cellular & Gene Therapies, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States

Inherent variability between products is a given for autologous cellular immunotherapy products due to their patient-derived nature.  What strategies can be employed to help predict the likelihood of clinical success, and do regulators consider this important? What should be done to determine the host factors that predict biological response to a product? What technology is available to assist in determining treatment effectiveness? How much data is required? At what stage of product development should these evaluations be undertaken?

Speaker: Scott Burger, MD, Advanced Cell & Gene Therapy, United States

To view the full meeting program, please refer to the Program Schedule.

Confirmed Speakers and Moderators

Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd., United Kingdom
Scott Burger, MD, Principal, Advanced Cell & Gene Therapy, United States
Victor Lietao Li, MD, Founder and CEO, Lion TCR Pte Ltd, Singapore
Janet Macpherson, PhD,
Development Manager, Royal Prince Alfred Hospital, Australia
John Ng, Chief Operating Officer, Tessa Therapeutics, Singapore
Karen Nichols, Esq, VP Regulatory and Quality, Magenta Therapeutics, United States
Christiane Niederlaender, PhD, Senior Quality Assessor, MHRA, United Kingdom
Michael Pepper, MBChB, PhD, MD, Director, Institute for Cellular and Molecular Medicine and SAMRC Extramural Unit for Stem Cell Research and Therapy, University of Pretoria, South Africa
Shari Targum, MD, MPH, Branch Chief, Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States
Marc Turner, MBChB, PhD, MBA, Medical Director, Scottish National Blood Transfusion Service, Scotland
Ramjay S. Vatsan, PhD, Team Lead, Division of Cellular & Gene Therapies, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States
Dominic Wall, PhD, FFSc(RCPA), Operations Director, Peter MacCallum Cancer Centre, Australia

Organizing Committee

Karen Nichols, Esq. – Co-Chair
VP, Regulatory and Quality, Magenta Therapeutics, United States
Dominic Wall, PhD, FFSc(RCPA) – Co-Chair
Operations Director, Peter MacCallum Cancer Centre, Australia
Patrick Bedford, Commercialization Manager, Centre for Commercialization of Regenerative Medicine (CCRM), Canada
Christopher Bravery, PhD, Director, Consulting on Advanced Biologicals, United Kingdom
Scott Burger, MD, Principal, Advanced Cell & Gene Therapy, United States
Srinivasan Kellathur, PhD, Head, Advanced Therapy Products Unit, Health Sciences Authority, Singapore
Janet Macpherson, PhD, Development Manager, Dept of Cell & Molecular Therapies, Royal Prince Alfred Hospital, Australia
Jean-Hughes Trouvin, PhD, Professor, University Paris Descartes, France