ROUNDTABLE Bypassing the Trial: Where do we Draw the Line for US Expanded Access?
Wednesday, May 31, 2023 01:00 PM - 02:00 PM
Room 252A
GRP
Roundtable
Assess the use of Expanded Access in terms of ethics, regulation, risk, and reimbursement.
Moderator
Beth Roxland, JD, MBioethics, Senior Advisor on Law, Policy and Bioethics, Roxland Consultants Ltd., USA
Panelists
Lynn O'Donnell, PhD, Director, Cell Therapy Manufacturing & Engineering, The Ohio State University, USA
Leigh Turner, PhD, Professor, University of California, Irvine, USA
Kevin Bosse, PhD, RAC-US, CABP(H), Director of Regulatory Affairs, Nationwide Children's Hospital, USA
Session Description
Members of ISCT ECGT’s Working Group on Expanded Access and other field leaders will share perspectives and engage audience members in a discussion of the benefits and challenges posed by non-trial access to investigational cell and gene therapies. Institutions have had success in both delivering as well as themselves manufacturing CGTs under these programs. This roundtable would discuss those successes to provide “best practices” information for other institutions, as well as discuss the challenges and pitfalls discovered during the process. Panel members will discuss how a code of ethics could potentially help nudge the field toward ethical and away from problematic uses of non-trial access pathways, with potential elements of such a code being discussed and debated among the panelists and audience. Regulatory implications, as well as cost reimbursement and point-of-care manufacturing will also be covered to ensure a full spectrum of discussion. Audience members will leave with an appreciation of the benefits and challenges associated with non-trial access to investigational cell and gene therapies and have an opportunity to contribute to the discussion over how ISCT should promote ethical use of these important pathways.
Learning Objective
Moderator
Beth Roxland, JD, MBioethics, Senior Advisor on Law, Policy and Bioethics, Roxland Consultants Ltd., USA
Panelists
Lynn O'Donnell, PhD, Director, Cell Therapy Manufacturing & Engineering, The Ohio State University, USA
Leigh Turner, PhD, Professor, University of California, Irvine, USA
Kevin Bosse, PhD, RAC-US, CABP(H), Director of Regulatory Affairs, Nationwide Children's Hospital, USA
Session Description
Members of ISCT ECGT’s Working Group on Expanded Access and other field leaders will share perspectives and engage audience members in a discussion of the benefits and challenges posed by non-trial access to investigational cell and gene therapies. Institutions have had success in both delivering as well as themselves manufacturing CGTs under these programs. This roundtable would discuss those successes to provide “best practices” information for other institutions, as well as discuss the challenges and pitfalls discovered during the process. Panel members will discuss how a code of ethics could potentially help nudge the field toward ethical and away from problematic uses of non-trial access pathways, with potential elements of such a code being discussed and debated among the panelists and audience. Regulatory implications, as well as cost reimbursement and point-of-care manufacturing will also be covered to ensure a full spectrum of discussion. Audience members will leave with an appreciation of the benefits and challenges associated with non-trial access to investigational cell and gene therapies and have an opportunity to contribute to the discussion over how ISCT should promote ethical use of these important pathways.
Learning Objective
- Understand rationale for, existing approaches and best practices to non-trial access
- Raise awareness of potential benefits and misuses of non-trial access pathways
- Identify strategies (e.g. a code of ethics) to support ethical use of non-trial access to investigational cell and gene therapies.
Beth Roxland JD, MBioethics
Senior Advisor on Law, Policy and Bioethics
Roxland Consultants Ltd
Moderator
Senior Advisor on Law, Policy and Bioethics
Roxland Consultants Ltd
Moderator
Kevin Bosse PhD, RAC-US, CABP(H),
Director of Regulatory Affairs
Nationwide Children’s Hospital
Panelist
Director of Regulatory Affairs
Nationwide Children’s Hospital
Panelist
Lynn O'Donnell PhD
Director, Cell Therapy Manufacturing & Engineering
The Ohio State University
Panelist
Director, Cell Therapy Manufacturing & Engineering
The Ohio State University
Panelist