Enabling access to genetically modified cell therapies through flexible approaches to manufacturing and cost recovery
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.
Authors
- Lee A. Fleisher (ORCID: https://orcid.org/0000-0003-2899-2294)
- Boro Dropulić
- Holly Fernandez Lynch (ORCID: https://orcid.org/0000-0001-7813-9879)
- Natasha Kekre (ORCID: https://orcid.org/0000-0001-8394-0855)
- Jeff Allen (ORCID: https://orcid.org/0000-0002-0586-3054)
- John E. Connolly (ORCID: https://orcid.org/0000-0002-9680-3216)
- Steven A. Feldman (ORCID: https://orcid.org/0000-0003-2098-3860)
- Kristen Hege (ORCID: https://orcid.org/0000-0002-9609-5029)
- Mark Stewart (ORCID: https://orcid.org/0000-0002-4847-0736)
- Christopher R. Cabanski (ORCID: https://orcid.org/0009-0007-9938-1331)
- Patrick J. Hanley (ORCID: https://orcid.org/0000-0002-0102-7892)
- Crystal L. Mackall (ORCID: https://orcid.org/0000-0003-0359-9023)
- Ben M Beneski
Institutions
- Children's National (US)
- University of Ottawa (CA)
- George Washington University (US)
- Independent Sector (US)
- Allogene Therapeutics (United States) (US)
- Parker Institute for Cancer Immunotherapy (US)
- Center for Cancer and Blood Disorders (US)
- School District of Philadelphia (US)
- Collaborative Drug Discovery (United States) (US)
- Green Cross International (US)
- University of Pennsylvania (US)
- Friends of Cancer Research (US)
- Stanford University (US)
Publication Details
- Journal
- Journal for ImmunoTherapy of Cancer
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1136/jitc-2025-013518
- Citations
- 2
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 2.09
Funders
- Bristol-Myers Squibb
- Gilead Sciences
- Children's Hospital of Philadelphia
- University of Pennsylvania
- Children's National Hospital
- Universitat de Barcelona
- Perelman School of Medicine, University of Pennsylvania