{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T04:12:44Z","timestamp":1792642364247,"version":"4.5.0"},"reference-count":11,"publisher":"BMJ","issue":"12","license":[{"start":{"date-parts":[[2025,12,3]],"date-time":"2025-12-03T00:00:00Z","timestamp":1764720000000},"content-version":"unspecified","delay-in-days":2,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["J Immunother Cancer"],"accepted":{"date-parts":[[2025,11,17]]},"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.1136\/jitc-2025-013518","type":"journal-article","created":{"date-parts":[[2025,12,3]],"date-time":"2025-12-03T01:40:28Z","timestamp":1764726028000},"page":"e013518","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":8,"title":["Enabling access to genetically modified cell therapies through flexible approaches to manufacturing and cost recovery"],"prefix":"10.1136","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4847-0736","authenticated-orcid":false,"given":"Mark D","family":"Stewart","sequence":"first","affiliation":[{"name":"Friends of Cancer Research, Washington, District of Columbia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-9938-1331","authenticated-orcid":false,"given":"Christopher R","family":"Cabanski","sequence":"additional","affiliation":[{"name":"Parker Institute for Cancer Immunotherapy, San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeff D","family":"Allen","sequence":"additional","affiliation":[{"name":"Friends of Cancer Research, Washington, District of Columbia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John E","family":"Connolly","sequence":"additional","affiliation":[{"name":"Parker Institute for Cancer Immunotherapy, San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ben M","family":"Beneski","sequence":"additional","affiliation":[{"name":"Allogene Therapeutics, South San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Boro","family":"Dropuli\u0107","sequence":"additional","affiliation":[{"name":"Caring Cross, Gaithersburg, Maryland, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven A","family":"Feldman","sequence":"additional","affiliation":[{"name":"Laboratory for Cell and Gene Medicine, Stanford University School of Medicine, Stanford, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lee A","family":"Fleisher","sequence":"additional","affiliation":[{"name":"Rubrum Advising, Philadelphia, Pennsylvania, USA"},{"name":"Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick J","family":"Hanley","sequence":"additional","affiliation":[{"name":"Center for Cancer and Immunology Research, Children\u2019s National Hospital, Washington, District of Columbia, USA"},{"name":"Department of Pediatrics, The George Washington University, Washington, District of Columbia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristen","family":"Hege","sequence":"additional","affiliation":[{"name":"Independent Advisor, Burlingame, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8394-0855","authenticated-orcid":false,"given":"Natasha","family":"Kekre","sequence":"additional","affiliation":[{"name":"Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holly","family":"Fernandez Lynch","sequence":"additional","affiliation":[{"name":"Department of Medical Ethics and Health Policy, University of Pennsylvania, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0359-9023","authenticated-orcid":false,"given":"Crystal L","family":"Mackall","sequence":"additional","affiliation":[{"name":"Center for Cancer Cell Therapy, Stanford University School of Medicine, Stanford, California, USA"},{"name":"Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA"},{"name":"Department of Medicine, Stanford University School of Medicine, Stanford, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2025,12,2]]},"reference":[{"key":"2025120217402075000_13.12.e013518.1","doi-asserted-by":"crossref","first-page":"1836","DOI":"10.1038\/s41591-024-03035-1","article-title":"Enhancing pediatric access to cell and gene therapies","volume":"30","author":"Mackall","year":"2024","journal-title":"Nat Med"},{"key":"2025120217402075000_13.12.e013518.2","unstructured":"Friends of Cancer Research and Parker Institute for Cancer Immunotherapy . 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