ISCT Reaffirms Position on Addressing Ethical Implications of US Expanded Access Pathway

International Society for Cell & Gene Therapy Publishes Report Exploring Areas for Potential Misuse and Outlines Plans for Future Guidelines to Keep Patients Safe

VANCOUVER, British Columbia–(BUSINESS WIRE)–The International Society for Cell & Gene Therapy (ISCT), the translational focused global community of cell and gene experts today reaffirms its position on ethical concerns relating to the US Expanded Access Pathway with the publication of the ISCT Expanded Access Working Group Short Report ‘The Future of Expanded Access to Unapproved Cell and Gene Therapies’.

The short report was created by the ISCT Expanded Access Working Group (EAWG), a subset of the Committee on the Ethics of Cell and Gene Therapy, comprising scientific, regulatory, and ethical experts. The EAWG report acknowledges that non-trial preapproval access can serve as an appropriate route for seriously and terminally ill patients to obtain investigational cell- and gene-based interventions; however, the committee identified three major concerns.

Firstly, expanded access can delay or halt clinical trials so that data on the safety and efficacy of interventions is never generated. This could negatively impact the progress of the sector. Secondly, expanded access might be used as a route for commercialization of unproven interventions, opening doors to unethical vendors. Finally, expanded access might be provided in ways that are inequitable or that increase existing disparities resulting in unfair access to treatments based on patients’ socioeconomic status.

Speaking on the significance of the report, Patricia J. Zettler, JD, (Chair, ISCT Expanded Access Working Group) JD said, “We have seen a substantial increase in patient interest in non-trial access, which has spurred controversy and contention around the ethics behind expanded access. To address these sector-wide concerns, ISCT formed the Expanded Access Working Group to identify practical, ethical, and regulatory issues emerging from the use – and possible abuse – of the expanded access pathway.

This initial short report examines the history of expanded access, identifying three examples of potential issues we see arising because of this access. Building on this report the working group will publish a final expanded report and develop a set of ethical guidelines to minimize the risks and help shape the future of non-trial access.”

In the coming year, the ISCT Expanded Access Working Group (EAWG) will publish a final expanded report developing a set of guidelines based on accessibility and equity for cell and gene therapy stakeholders to help ensure that uses of expanded access serve the interests of patients and benefit the field. To learn more about the ISCT Expanded Access Working Group visit our website here.

About the ISCT Expanded Access Working Group

The ISCT Expanded Access Working Group aims to identify and address practical, ethical, and regulatory issues that arise from the use, and potential misuse, of the expanded access pathway by various CGT stakeholders.

The Working Group aims to develop informed perspectives on expanded access that will help ISCT as it generates educational resources that promote innovation in translational research while upholding the highest ethical standards.

About the International Society for Cell & Gene Therapy

Established in 1992, the International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists, and industry partners with a shared vision to translate cell and gene therapy into safe and effective therapies to improve patients’ lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory, and Commercialization. Through strong relationships with global regulatory agencies, academic institutions, and industry partners, ISCT drives the advancement of research into a standard of care.

Comprised of over 2,700 cell and gene therapy experts across five geographic regions and representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders, and organizations invested in cell and gene therapy translation. For more information about the society, key initiatives, and upcoming meetings, please visit https://isctglobal.org, @ISCTglobal.

About Patricia (Patti) J. Zettler

Patti Zettler is the co-chair of the International Society of Cell & Gene Therapy’s (ISCT) Committee on the Ethics of Cell and Gene Therapy, also chairing its subcommittee on expanded access.

Zettler is an associate professor at The Ohio State University Moritz College of Law and a faculty member of Ohio State’s Drug Enforcement and Policy Center and its Comprehensive Cancer Center. Her research and teaching focus on FDA law and policy, torts, and legislation and regulation. Her scholarship has appeared in leading legal and health sciences journals such as the New England Journal of Medicine, JAMA, and Science, and has covered various topics including expanded access, biohacking, stem cell interventions, opioids, cannabis products, tobacco and nicotine products, and COVID-19 countermeasures.

Zettler also is a co-author of the 5th edition of Food and Drug Law: Cases and Materials (with Peter Barton Hutt, the late Richard A. Merrill, Lewis A. Grossman, Nathan Cortez, and Erika Lietzan). She currently serves on the Food and Drug Law Institute’s (FDLI) Board of Directors. Previously she served on the National Academies of Sciences, Engineering, and Medicine’s (NASEM) Committee on Reviewing the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE) and as a consultant to the NASEM Committee on Pain Management and Regulatory Strategies to Address Prescription Opioid Abuse, among other things.

Before entering academics, Zettler served as an attorney in the Office of the Chief Counsel at FDA. She received her undergraduate and law degrees from Stanford University, both with distinction.

Contacts

ISCT Contact
Lauren Reville, MSc

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