On May 22, Friends of Cancer Research (Friends) hosted a public webinar exploring next steps for supporting sustainable development and manufacturing models that can expand patient access to genetically modified cell-based therapies. The webinar, “Unlocking Next-Generation Therapies Meeting Recap: Sharing Next Steps and Opportunities with Advocates,” revisited policy discussions from the May 9th public meeting, and proposals from the collaborative white paper developed with the Parker Institute for Cancer Immunotherapy (PICI).
- Insights were distilled into actionable guidance designed to equip patients, advocates, and industry professionals with the knowledge and context needed to expand access- working together to translate scientific innovation and supportive policy discussions into meaningful practice.
- Discussions reaffirmed the need for cross-sector collaboration to ensure that regulatory flexibility, innovative manufacturing models, and reimbursement strategies evolve in parallel, particularly for rare diseases and small patient populations. Existing Chemistry, Manufacturing, and Controls (CMC) frameworks often fall short in addressing the complexities of individualized therapies, highlighting the importance of developing regulatory infrastructure that is both adaptable and fit-for-purpose.
What policy opportunities exist to overcome current barriers and ensure that patients, including those with rare diseases, can access the potentially transformative benefits of engineered cell-based therapies?
Translating proposals into scalable solutions requires confronting foundational challenges- starting with economic viability.
Many genetically modified cell-based therapies, particularly those targeting rare diseases, are not designed with traditional commercial returns in mind. As Kristen Hege, Independent Board of Directors Member, emphasized– realizing the full potential of these therapies demands a shift in how we think about value– how can we ensure that therapies serving small patient populations are supported by development and manufacturing models that don’t rely exclusively on volume-based incentives, but instead reflect their transformative clinical value?
Closing the gap between scientific progress and systemic readiness is equally critical.
Patrick Hanley, Children’s National Hospital, raised concerns shared across sectors- scientific innovation is outpacing regulatory, manufacturing, and reimbursement infrastructure. Without proactive efforts to align timeliness, adapt evidentiary standards, and modernize coordinating mechanisms, promising therapies risk being stalled within systems not built to support them. These are exactly the dynamics the Friends and PICI’s white paper seeks to address.
And most importantly- speed alone is not the goal. Lasting progress requires systems that are not only efficient, but also transparent, ethically grounded, and trusted by the public.
Holly Fernandez Lynch, University of Pennsylvania, emphasized that in fast-moving fields such as cell-based gene therapies, stakeholder confidence must evolve alongside scientific progress. These therapies are already demonstrating remarkable success in early-phase development, offering new hope for patients with limited options. But translating that promise into broad, real-world impact requires more than scientific success. Strengthening public trust through clear regulatory governance, meaningful patient engagement, and transparency in decision-making is essential to building sustainable and equitable pathways for access. Innovation must be supported by systems that uphold legitimacy, reflect readiness, and respond to the needs of both patients and the broader public. Only then can the full promise of these therapies be realized timely, responsibly, and at scale.
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