Enabling access to genetically modified cell therapies through flexible approaches to manufacturing and cost recovery

Authors

Mark D. Stewart, Friends of Cancer Research, Washington, District of Columbia, USA mstewart@focr.org.
Christopher R. Cabanski, Parker Institute for Cancer Immunotherapy, San Francisco, California, USA.
Jeff D. Allen, Friends of Cancer Research, Washington, District of Columbia, USA.
John E. Connolly, Parker Institute for Cancer Immunotherapy, San Francisco, California, USA.
Ben M. Beneski, Allogene Therapeutics, South San Francisco, California, USA.
Boro Dropulić, Caring Cross, Gaithersburg, Maryland, USA.
Steven A. Feldman, Laboratory for Cell and Gene Medicine, Stanford University School of Medicine, Stanford, California, USA.
Lee A. Fleisher, Rubrum Advising, Philadelphia, Pennsylvania, USA.
Patrick J. Hanley, Center for Cancer and Immunology Research, Children's National Hospital, Washington, District of Columbia, USA.
Kristen Hege, Independent Advisor, Burlingame, California, USA.
Natasha Kekre, Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
Holly Fernandez Lynch, Department of Medical Ethics and Health Policy, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Crystal L. Mackall, Center for Cancer Cell Therapy, Stanford University School of Medicine, Stanford, California, USA.

Document Type

Journal Article

Publication Date

12-2-2025

Journal

Journal for immunotherapy of cancer

Volume

13

Issue

12

DOI

10.1136/jitc-2025-013518

Keywords

Chimeric antigen receptor - CAR; Gene therapy; Immunotherapy

Abstract

Genetically modified cell-based therapies hold transformative potential, particularly for patients with rare cancers and ultra-rare diseases. However, progress toward regulatory approval, reimbursement, and broad patient access is often constrained by misaligned regulatory, manufacturing, and financial frameworks that do not reflect the realities of treating small populations and low-throughput production models. Drawing on a collaborative white paper and public meeting convened by Friends of Cancer Research and the Parker Institute for Cancer Immunotherapy in May 2025, this commentary outlines three strategies to streamline regulatory pathways and enable timely, sustainable access: (1) flexible approaches to Chemistry, Manufacturing, and Controls requirements in small populations, (2) adaptable regulatory frameworks to support diverse manufacturing models, and (3) limited cost recovery mechanisms to bridge early access and development gaps. Recent regulatory and policy discussions have echoed these priorities, signaling an opportunity to align oversight with operational realities to advance innovation and access for patients in high-need settings.

APA Citation

Stewart, Mark D.; Cabanski, Christopher R.; Allen, Jeff D.; Connolly, John E.; Beneski, Ben M.; Dropulić, Boro; Feldman, Steven A.; Fleisher, Lee A.; Hanley, Patrick J.; Hege, Kristen; Kekre, Natasha; Fernandez Lynch, Holly; and Mackall, Crystal L., "Enabling access to genetically modified cell therapies through flexible approaches to manufacturing and cost recovery" (2025). GW Authored Works. Paper 8455.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/8455

Department

Pediatrics