Access to life-saving cell and gene therapies is one of the most critical challenges facing healthcare today. While these therapies offer remarkable hope for patients battling life-threatening conditions like cancer and rare genetic diseases, the reality is that only a fraction of eligible patients are actually receiving them. The reasons are rarely scientific. More often, they stem from how care is delivered, reimbursed, and regulated.
This was the focus of the July 2025 Bridging the Gap webinar, hosted by Azenta Life Sciences and the Emily Whitehead Foundation. Titled “From BIO 2025 to Bedside: The World Can’t Wait—Accelerating the Path from Approval to Access,” the session featured Dr. Kamal Menghrajani, physician-scientist and former Assistant Director for Cancer Innovation and Public Health at the White House Office of Science and Technology Policy.
Dr. Menghrajani was joined by George Eastwood, Executive Director of the Emily Whitehead Foundation; Albert Ribickas, Assistant Director of Cell Therapy at Moffitt Cancer Center; and co-hosts Olga Bukatova of Azenta and Tom Whitehead of the Emily Whitehead Foundation.
From the White House to the Field
In January 2025, Dr. Menghrajani helped lead the first White House forum on cell and gene therapy, bringing together regulators, policymakers, biotech leaders, and patient advocates to identify specific, actionable solutions to improve access.
The effort resulted in the creation of three cross-sector working groups, each focused on a critical barrier to progress: access and affordability, manufacturing and commercialization, and capital investment. These groups were tasked with identifying actionable solutions to advance patient access to therapies, streamline production and regulatory pathways, and ensure sustainable financial models to support continued innovation—particularly in rare and underserved disease areas.
“We’ve figured out the science,” she said. “Now we need to figure out how to give patients access. This is a man-made problem.”
Policy Momentum, but Funding Fears Loom
The discussion came just months after several encouraging developments from The Biden Cancer Moonshot effort, including:
- New navigation services reimbursable under Medicare and Medicaid, helping patients enroll in clinical trials and avoid delays in care.
- ARPA-H’s expanded support for high-risk, high-reward cancer innovations, including gene and cell therapy platforms.
- A proposed FDA rule to reduce nicotine levels in combustible tobacco products—targeting one of cancer’s biggest drivers.
These moves reflect a genuine shift toward rethinking healthcare delivery and access. But despite progress, serious risks remain. In particular, potential cuts to NIH funding could undermine everything from early-stage rare disease research to translational science.
“We’ve heard people describe it as an extinction-level event,” Dr. Menghrajani noted. “If the NIH can’t fund foundational science, private industry isn’t going to fill the gap. And that means fewer therapies developed—especially for rare diseases.”
She emphasized that federal investment must be protected for these initiatives to succeed. “The work is starting,” she said. “But we need continuity, capital, and commitment.”
Financing Innovation: What We Heard at BIO 2025
One of the webinar’s highlights was a recap of BIO 2025, where conversations around reimbursement and equity took center stage. George Eastwood moderated a standout session titled “Reimbursement Innovation—Unlocking the Future of Advanced Therapy Access,” which called for new financial models that reflect the reality of how these therapies work.
“Commercialization isn’t the end. If anything, it’s where the real work begins,” Eastwood said.
Panelists discussed several approaches gaining traction:
- Installment payments for one-time, high-cost therapies
- Outcomes-based agreements, where payment depends on long-term results
- Risk-sharing models that align government and private payers
Dr. Menghrajani also highlighted the Medicaid Cell and Gene Therapy Access Model now being implemented by the Center for Medicare and Medicaid Innovation (CMMI). In this pilot, the federal government negotiates pricing with therapy developers and enables states to opt into a shared-outcomes system—starting with sickle cell disease.
With 35 states enrolled and over 84% of Medicaid patients covered, it’s a promising test case that could eventually scale to more diseases and therapies.
“We need models like this that address both payment and outcomes,” she said. “It’s the kind of infrastructure that brings access into reality.”
Technology Is Ready—But Infrastructure Lags
Beyond policy, much of the conversation focused on the need to rethink how we deliver therapies. The rise of platform approaches, such as CRISPR paired with lipid nanoparticles, offers new hope for scalable, faster-to-market therapies. The recent case of Baby KJ, treated for a rare liver disorder at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine, using a custom gene therapy in under a year, was cited as a powerful example.
Still, regulatory and manufacturing systems remain fragmented.
“We have the tools,” said Ribickas. “But the space, the people, and the processes aren’t where they need to be yet. We have to modernize how these therapies get to patients.”
Automation, digitization, and AI-supported manufacturing are all being explored to reduce cost and increase consistency. The goal: make these therapies not just available, but accessible and sustainable.
Advocacy Is the Engine
As always in Bridging the Gap sessions, the power of advocacy was front and center. From Tom Whitehead’s FDA testimony to patients founding biotech companies to cure their children’s diseases, personal stories are fueling policy shifts across the field.
“When patients speak, systems listen,” said Eastwood. “It’s up to all of us—manufacturers, providers, advocates—to carry those voices into every conversation.”
Bukatova echoed that sentiment, adding that even tech companies and logistics partners play a role. “It takes the entire value chain—cryo logistics, tracking, data integration—to make cell and gene therapy viable at scale,” she said. “And that means every part of the ecosystem must align around access.”
Key Takeaways
- The science of cell and gene therapy has outpaced access; regulatory, economic, and awareness barriers remain.
- The Biden Cancer Moonshot’s 2025 initiatives—including navigator reimbursement, ARPA-H funding, and FDA rulemaking—are active and continuing.
- Medicaid’s new outcomes-based gene therapy payment model is a first step toward sustainable financing—but requires continued oversight and support.
- “Platform therapy” approaches and regulatory innovation could dramatically cut costs and timelines, especially for rare diseases.
- Elevating all voices—patients, caregivers, manufacturers, policymakers—is essential to closing the awareness and access gap.
Stay Updated
Join us for the next Bridging the Gap webinar, where science, compassion, and innovation continue to meet at the frontier of life-changing medicine.
NOTE: After the July webinar, U.S. Senate Releases Proposed NIH Funding Levels for FY 2026: https://www.acr.org/News-and-Publications/senate-proposes-fy2026-funding
About the Guest
Kamal Menghrajani, M.D.
Former Assistant Director, Cancer Innovation & Public Health, The White House (Office of Science and Technology Policy)
Dr. Kamal Menghrajani received her MD from the University of North Carolina at Chapel Hill. She completed her Internal Medicine residency at the University of Michigan, and her fellowship and chief fellowship in hematology and medical oncology at Memorial Sloan Kettering Cancer Center. Kamal subsequently joined the faculty at MSK on the leukemia service, where she used computational biology, genomics, and population sciences to study clonal hematopoiesis and myeloid diseases. She was the recipient of an NIH K award, an ASCO YIA, and she earned a Masters degree in Biostatistics from the Mailman School of Public Health at Columbia University.
In 2023, Dr. Menghrajani was asked to join the White House to serve as the oncologist for the Cancer Moonshot and the physician for the Health Outcomes team. As a White House Fellow and then an Assistant Director at the White House Office of Science and Technology Policy, Dr. Menghrajani created and executed strategic initiatives around cell & gene therapy, global health, clinical trials, rare diseases, the decline in life expectancy, and more.