Cell and gene therapy continues to advance across a growing number of rare genetic conditions. As more of these therapies move closer to the clinic, a central tension has come into focus: scientific success does not automatically translate into patient access. Access is shaped not only by scientific progress, but also by regulatory frameworks, economic incentives, infrastructure readiness, and patient experience.
To explore these issues, Charlotte Guzzo, COO and co-founder of Sano Genetics, convened an expert panel representing bioethics, patient advocacy, regulatory policy, and health policy. Speakers included Dr. Alison Bateman-House (NYU), Annie Kennedy (EveryLife Foundation for Rare Diseases), Monica Veldman (Alliance for Regenerative Medicine), and Max Bronstein (Aviva Strategies). The discussion focused on how the current ecosystem supports or limits patient access to cell and gene therapies in the U.S. today.
Cell and gene therapies (CGTs) involve complex manufacturing processes, specialized clinical sites, and long-term patient monitoring. These requirements place significant demands on health systems that were largely designed for more standardized models of care, not highly individualized treatments.
As a result, access challenges increasingly arise after clinical success has been demonstrated. Even when therapies show strong efficacy, patients may face delays or barriers related to site availability, payer coverage, contracting complexity, or the logistics of treatment and follow up. These logistical barriers can prevent groundbreaking scientific breakthroughs from making a meaningful difference in the patient populations they were carefully developed for.
The panel discussed how regulatory continuity and clear expectations influence development planning and patient access. Ongoing changes in leadership at the Food and Drug Administration (FDA) and evolving review practices can create uncertainty for developers, patient communities, and investors. This uncertainty does not stop at approval; it can ripple downstream, affecting manufacturing scale-up, site readiness, and payer confidence. These challenges add an extra layer of complexity to precision medicine drug development and have undoubtedly contributed to recent drops in biotech funding.
Clear guidance, consistent reviewer expertise, and transparency in evidence expectations were highlighted as important factors for enabling predictable timelines and reducing risk across development programs.
Incentive programs remain a critical foundation for rare and ultra rare disease development. The panel discussed how mechanisms such as the Rare Pediatric Disease Priority Review Voucher influence whether early-stage programs are funded and sustained.
Uncertainty around the future of these incentives can directly affect pipeline viability. Without stable and durable incentives, investment decisions for small patient populations with limited commercial upside become significantly harder to justify, threatening long-term innovation in these areas.
Approval does not guarantee access. The panel highlighted several factors that influence whether patients can receive approved therapies, including treatment center capacity, reimbursement policies, and payer perceptions of novel regulatory pathways.
Families may also face substantial practical burdens, such as extended travel, temporary relocation near treatment centers, and the cost of long-term follow up care. These factors can limit uptake even when therapies are clinically available. While fit-for-purpose reimbursement models have been widely discussed, the panel emphasized that meaningful progress will be required as more advanced therapies reach approval and real-world delivery accelerates.
Patient advocacy organizations were described as critical contributors across the development and access lifecycle. Beyond supporting approvals, advocacy groups often lead efforts related to natural history studies, registries, newborn screening, and data stewardship.
Panelists emphasized the importance of engaging advocacy communities early and understanding their priorities, including concerns around data ownership, transparency, and long term access to care. These groups are often closest to the practical realities families face once a therapy exists, making them essential partners in shaping access strategies that work beyond the clinic.
Access to cell and gene therapy depends on infrastructure that spans manufacturing, clinical delivery, reimbursement systems, and long term monitoring. Panelists noted that advocacy and policy efforts increasingly focus on these foundational elements rather than approvals alone.
Ensuring that infrastructure evolves alongside scientific innovation was described as essential for enabling access at scale and supporting sustainable delivery models. Without this alignment, access risks becoming the limiting factor for the field’s future progress.
The discussion highlighted the need for coordination across regulators, policymakers, patient advocacy groups, payers, and industry stakeholders. Aligning incentives, evidence expectations, and infrastructure investments will be critical to translating innovation into real world patient impact. As cell and gene therapies mature, access must be treated as a systems-level challenge that requires early, integrated planning rather than a post-approval afterthought.
Watch the on-demand webinar to explore these themes in more depth and hear directly from the panelists.