International Society for Cell & Gene Therapy establishes working group to tackle regulatory and ethical issues with Expanded Access

International Society for Cell & Gene Therapy establishes working group to tackle regulatory and ethical issues with Expanded Access

Vancouver, Canada, February 28, 2022 - The International Society for Cell & Gene Therapy (ISCT), the global society of clinicians, researchers, regulators, technologists, and industry partners dedicated to the translation of cell and gene therapy (CGT) into safe and effective therapies to improve patients’ lives, today announces it has established a task force to identify and address practical, ethical, and regulatory issues that arise from the use, and potential misuse, of expanded access within the CGT sector.

Expanded access is a pathway available under FDA regulations that in limited circumstances can provide patients with preapproval access to investigational products outside clinical trials, when no comparable or satisfactory alternative therapy options are available. In the US, patient and developer interest in expanded access has increased in recent years. This trend is expected to continue or even accelerate, specifically for the CGT sector. One major reason for this is that the FDA is no longer exercising enforcement discretion for premarket approval requirements for regenerative medicine products.

ISCT has formed the ‘ISCT Expanded Access Working Group’ in response to growing concerns in the field that expanded access is being utilized in ways that fall outside its intended purpose. This Working Group will create resources with and for the CGT community on the regulatory framework for expanded access, as well as to guide ethical and appropriate uses of the pathway.

“Inappropriate use of expanded access could harm the entire CGT sector, undermine research efforts, and ultimately delay approval of products critical for many patients. As the CGT sector is continuing to grow, and the therapeutic potential of CGTs is generating increased attention from scientific and patient communities, there is also growing interest in expanded access to CGT products. Expanded access is an important mechanism for providing access to unproven, but promising, interventions for seriously and terminally ill patients who lack approved treatment options. However, at the same time, expanded access should not be understood as a mechanism for commercializing unproven, unlicensed interventions,” said Patricia J. Zettler, chair, ISCT Expanded Access Working Group. “Expanded access to CGT products can pose difficult ethical and regulatory questions. This is why ISCT is launching this Working Group, which we hope will help enable expanded access to CGT products while rigorous research continues, to serve the goal of developing safe and effective therapies for patients.”

While expanded access refers to a US pathway, the misuse of expanded access in the United States could have international repercussions. ISCT is a global interdisciplinary organization that brings together regulatory experiences from across the world and has a strong interest in assuring well-intentioned regulatory structures maintain their integrity. Drawing on the CGT experience and knowledge of its global leaders, ISCT will evaluate expanded access against similarly intentioned programs in other regions and other regulatory frameworks, and research and monitor global developments across the field.

As the leading organisation representing all stakeholders within the CGT field and with experience in science, regulatory, commercialization, manufacturing, and bioethics, ISCT is well-positioned to educate all stakeholders across the CGT community, including the wider public, on complex regulatory and ethical topics such as expanded access and to place these issues in an appropriate global context.

The Expanded Access Working Group will operate under the remit of the ISCT Committee on the Ethics of Cell and Gene Therapy (ECGT), formerly the ISCT Presidential Task Force on the Use of Unproven and/or Unethical Cell & Gene Therapies (PTF).

The ISCT Expanded Access Working Group has been tasked with specific objectives. These include:

• providing resources that help all CGT stakeholders and regulators to provide expanded access in ways that serve the interests of patients without undermining rigorous research
• identifying and addressing practical, ethical, and regulatory issues that arise from the use, and potential misuse, of the established expanded access pathway
• developing a code of ethics for CGT stakeholders using expanded access and seeking endorsement from societies and professional organizations in the field.

The group will be chaired by Patricia J. Zettler, The Ohio State University and its membership includes Laertis Ikonomou, The State University of New York at Buffalo, US, Aaron Levine, Georgia Institute of Technology, US, Bambi Grilley, Baylor College of Medicine, US, Karen Nichols, Esq., Vertex Pharmaceuticals, US and Leigh Turner, University of California Irvine, US. Wilson Bryan, Director of the Office of Tissues and Advanced Therapies (OTAT), will serve as the FDA liaison to provide regulatory expertise where relevant.   

ISCT is organizing a Roundtable Session at its Annual Meeting, in San Francisco, US, in May 2022 that will address the various purposes and scopes of investigational treatment pathways, their limitations, and the considerations at play in risk vs. benefit assessments. This session will bring together global experts to discuss, debate, and solve complex ethical and regulatory issues that arise from the use, and potential misuse, of these pathways. The ISCT Expanded Access Working Group will leverage the discussions in the Roundtable Session to develop impactful resources for CGT stakeholders.