Adaeze Ekwe, MSc, PhD
Junior Associate Editor
Queensland University of Technology (QUT)
Brisbane, Australia
Joaquim Vives, PhD
Contributing Editor
Banc de Sang i Teixits (BST)
Barcelona, Spain
Declarations of interest: none.
Topic Overview
As Cell & Gene Therapy (CGT) transition from experimental to commercial-stage interventions, regulatory systems, manufacturing paradigms and reimbursement models are being stress-tested. On June 5th, the Food and Drug Administration’s Center for Biologics Evaluation (FDA CBER) convened a multi-stakeholder CGT Roundtable with distinguished researchers, regulators and patient advocates to discuss how breakthrough one-time treatments can reach patients efficiently, addressing critical barriers and enablers for sustainable growth of the CGT ecosystem. This edition of the ISCT Telegraft’s Scientific Spotlight distills key insights from that meeting.
Insights Across the Ecosystem
The session had a strong focus on regulatory reforms, particularly on streamlining and reducing regulatory processes.
For Patients: access remains uneven due to geographic, socioeconomic, and insurance-related barriers. Stakeholders emphasized the need for speedy access to therapies especially in rare genetic diseases where timelines can mean the difference between life or death. The recent success story of the treatment of baby KJ Muldoon with the first-ever bespoke CRISPR therapy was highlighted as an example of what can be achieved with expedited regulatory pathways.
For Academics, Researchers and Clinicians: clinical pipeline crises and commercialization burden. Researchers highlighted the need to expedite the bench to bedside pathway, so that several transformative therapies can reach patients. Constraints to commercialization include complex and exorbitant manufacturing in addition strict requirements for early phase trials. Suggestions ranged from “Plug-and-Play approaches” rather than a one-size fits all. This displays a shift to deploying 'GMP-like' rather than full GMP (in some instances) to accelerated pathways for Biological License Approval (BLA) and streamlining regulatory pathways for N-of-few trials. There was also a mention of the need to “protect young researchers.”
For Developers and Industry: loss of innovation and market lead. Participants warned that the United States was at risk of losing their dominance in the cell and gene space to other countries “eager to fill the void,” such as China. Biotec startups are plagued by delayed approvals and frozen IPOs because of the current regulatory model.
For Regulators, Payers and Policy Makers: government representatives emphasized the need for transparency and to simplify the regulatory pathway. The Secretary of the U.S. Department of Health and Human Services (HHS) Robert F. Kennedy, Jr., invited the assembled stakeholders to identify regulations they considered unnecessary and wanted removed. The FDA commissioner Martin Makary, M.D., emphasized that they are committed to implementing changes based on the recommendation by the stakeholders.
Expert Perspectives
Speakers on the roundtable panel included pioneers in the field of cell and gene therapy such as Dr. Carl June and Dr. Catherine Bollard (former ISCT president). In his statement, Dr. June noted that due to the “slow, costly and inflexible” process of the US, “innovation is happening offshore” and proposed the FDA adopt a “two-tier model like China." This involves first-in-human exploratory trials requiring only local institutional review board (IRB) approval, while FDA involvement is reserved for larger, multisite trials.
Dr. Bollard spoke about the lack of progress especially in pediatric cases noting that “our children are not benefitting.”
Another speaker pointed out that lowered FDA regulations could undermine credibility and trust citing instances of unproven stem-cell clinics.
Global Viewpoint
Altogether participants agreed that in addition to streamlining regulations, speedy access to cell and gene therapies by patients can be achieved through the collaborative efforts of academia, government, industry and patient advocates. However, some questions remain unanswered.
What are the risk factors to reducing regulatory oversight of cell and gene therapies? How can we balance the need for speedy access to therapies to the rigorous scientific process which takes time?
What to Watch
References
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