Session Details

Workshop 2 - PTF Session – Regulatory aspects of Unproven Cell Therapies in North America
Friday, September 13, 2019 03:30 PM - 05:00 PM
Plenary Hall
The ISCT Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell & Gene Therapy (UCT) is a group of prominent academic, industry, bioethics and regulatory experts in the cell and gene therapy field. This session is focused on current regulatory and scientific efforts to curb the growth of a global industry offering unproven cell-based interventions for a wide variety of health conditions. In the scientific literature, there is an increasing number of documented cases of harm caused by unregulated administration of cell-based interventions. There is also a global regulatory trend towards pathways of accelerated clinical translation for cell and gene therapies in parallel with intensified regulatory actions against businesses that flout widely accepted safety and efficacy standards. In the U.S., this trend has been exemplified by the recent introduction and implementation of the Regenerative Medicine Advanced Therapy (RMAT) designation and by sustained Food and Drug Administration (FDA) and Federal Trade Commission (FTC) actions against businesses offering unproven cell-based interventions. In contrast to these efforts by federal regulators, there exists a growing push for cell product deregulation at state and federal level with stated goal the weakening of FDA oversight.

Chair: Laertis Ikonomou, PhD, Boston University, USA

FDA's Approach to Investigation Cell Therapies
Peter Marks, MD, PhD, Center for Biologics Evaluation and Research, FDA, USA
Peter Marks, MD, PhD, U.S. Food and Drug Administration, USA
Learning objectives:
•      Review the U.S regulatory framework for cell therapies
•      Describe FDA's actions to facilitate the development of cell therapies using appropriate regulatory mechanisms
•      Review the agency's efforts to address the inappropriate marketing cell therapy products

FDA Regulation of Animal Cells, and Cell- or Tissue-Based Products
Lynne Boxer, DVM, Division of Therapeutic Drugs for Non-Food Animals, Office of New Animal Drug Evaluation, Center for Veterinary Medicine, FDA, USA
Learning objectives:
•      Animal cells, tissues, and cell- or tissue-based products (ACTPs), meeting the definition of a drug, are regulated as new animal drugs and require FDA approval prior to marketing. Attendees will gain an understanding of how the new animal drug regulations are applied to ACTPs.
•      Attendees will learn about the pre-approval considerations for ACTPs, adverse events reported to FDA, and the risks associated with use of unapproved ACTPs.
•      Attendees will learn about FDA’s efforts to promote development of safe and effective ACTPs.

The Pitfalls of Unregulated Cell THerapy in Ophthalmology
Ajay Kuriyan, MD, MS, Department of Ophthalmology, University of Rochester Medical Cente, USA
Learning objectives:
•      Understand the prevalence of “cell therapy” clinics offering ophthalmic treatments
•      Learn about the different ophthalmic conditions targeted by “cell therapy” clinics
•      Understand the ocular complications associated with treatments at “cell therapy” clinics

Right to Try Laws and Expanding Access to Experimental Stem Cell Interventions
Kristin Matthews, PhD, Rice University's Baker Institute for Public Policy, USA
The presentation will:
•      Review Right to Try laws passed in states and the federal government;
•      Review recent efforts in Texas to include expanded access to stem cell interventions;
•      Analyze impact of state efforts on stem cell clinical trials, FDA oversight and marketing of unproven stem cell based interventions