Providing access to transformative cell and gene therapies is one of the most urgent challenges in modern healthcare. These treatments offer the possibility of curing conditions once thought untreatable—yet in the United States, only a fraction of eligible patients are receiving them. The barriers are complex: financial risk for payers, short-term budgeting in a long-term outcomes space, fragmented delivery systems, and gaps in patient support. This month’s Bridging the Gap webinar tackled these issues head-on, exploring how innovative reimbursement models could break through access bottlenecks.
The August session, “Post-Bio 2025: Accessible Cell and Gene Therapies – The World Can’t Wait,” featured Dr. William Shrank, Chief Executive Officer of Aradigm, alongside moderator Olga Bukatova of Azenta Life Sciences. Dr. Shrank—a physician, former policymaker, and healthcare executive—brings deep experience from CMS, CVS Health, UPMC, and Humana. He joined permanent panelist George Eastwood, Executive Director of the Emily Whitehead Foundation, and co-founder Tom Whitehead, whose daughter Emily was the first pediatric patient to receive CAR T-cell therapy.
The Economic Disconnect: One-Time Costs vs. Long-Term Value
Dr. Shrank opened by noting a structural mismatch: the U.S. payment system for these kinds of conditions was designed primarily for ongoing chronic care, not for one-time curative therapies that require a substantial upfront investment.
“Payers have to incur that large cost now, even though the patient may not be on their books in a year or two,” he explained. “That distorts the calculus around value.”
This dynamic can make organizations cautious about committing resources when they face uncertainty about long-term return. For smaller employers or insurers, it may be especially challenging to balance near-term budget constraints with the long-term benefits these therapies can deliver. In many cases, access decisions focus on short-term financial exposure instead of lifetime benefit for patients and the system.
So what are the alternatives?
Building Smarter Value-Based Models
Value-based agreements (VBAs) between manufacturers and payers are emerging as one of the most practical tools to bridge the gap between high upfront costs and long-term patient outcomes. Unlike traditional fee-for-service payments, where manufacturers are compensated regardless of how well a therapy performs, VBAs link reimbursement to measurable clinical results. This means that if a therapy delivers on its promise—such as eliminating sickle cell crises or halting the progression of spinal muscular atrophy—manufacturers are rewarded. If not, the financial risk is shared.
Dr. Shrank emphasized that this type of accountability is essential for creating sustainable access. But he also pointed out a core difficulty: tracking patients over many years, particularly in the U.S. where individuals frequently change jobs, insurers, or even states. Without a reliable way to follow outcomes across those transitions, the risk-sharing model breaks down.
“We should reward the heck out of the manufacturer that cures the disease,” he said, “but also hold them accountable for performance.”
Achieving that balance requires infrastructure—systems that can monitor patient progress long after the therapy is administered. That includes not only clinical benchmarks but also agreements on how data is collected, who is responsible for reporting it, and how it is verified for payment purposes. These details are often overlooked but are critical for VBAs to succeed at scale.
George Eastwood echoed this concern, highlighting that outcome tracking cannot simply be layered onto the responsibilities of treatment centers already struggling with staff shortages and resource constraints. “It’s not connected. Clinical follow-up is one thing; payment verification is another. We need to segment and support both.”
These insights reveal that while VBAs are a promising mechanism, their success depends on building robust, coordinated infrastructure—something that requires cooperation across payers, providers, and manufacturers.
The Patient’s Perspective on Cost and Follow-Up
While financial models provide one lens on the problem, Tom Whitehead grounded the conversation in lived experience. He shared his family’s journey navigating the healthcare system as his daughter Emily battled leukemia.
By 2012, the costs of Emily’s failed standard treatments—including chemotherapy and hospitalizations—had reached nearly $3 million. In contrast, the CAR T-cell therapy that ultimately saved her life was billed at $475,000. “That sounded reasonable to me,” Tom reflected, noting the irony that the therapy which worked cost a fraction of the treatments that didn’t.
But, as he emphasized, the story doesn’t end once a therapy is delivered. Patients and families continue to carry the burden of long-term follow-up. For many, this means repeated travel to specialized centers, frequent lab tests, and ongoing monitoring that disrupts daily life.
Even with supportive insurance coverage, the indirect costs—time off work, transportation, and lodging—add up quickly. Families may also encounter turnover at manufacturer support programs, creating inconsistencies in navigating the system.
“When you’re a parent with a sick kid, you’ll give up every material thing you have,” Tom said. “But we need to make follow-up easier and less costly”.
A New Insurance Model: Risk Pooling for Sustainability
Recognizing these challenges, Dr. Shrank outlined Aridigm’s model for spreading financial risk more effectively. By pooling costs across large groups of employers and insurers, the approach creates stability through scale. Instead of a single employer absorbing the full impact of a million-dollar therapy, expenses would be distributed across the pool—making coverage more predictable and sustainable.
This “trusted intermediary” would negotiate collaboratively with providers and manufacturers, pay providers upfront, and tie manufacturer payments to long-term outcomes. Just as importantly, the model builds in patient-centered supports such as concierge care, travel assistance, and lodging, helping families overcome practical barriers to treatment.
“It’s really been a guiding principle for us—to maintain sort of maniacal patient-centeredness in this process,” Dr. Shrank said.
Targeted to launch in 2026, the design is being shaped in partnership with employers, payers, and advocacy groups to ensure solutions reflect the needs of all stakeholders.
The Role of Advocacy in Changing the Conversation
Questions from the audience highlighted the power of patient voices—whether in advocating for at-home blood monitoring to ease follow-up or in engaging insurers directly. Dr. Shrank emphasized that having patients “in the middle of the conversation” helps shift focus from competing financial interests to shared goals of equitable access.
George Eastwood underscored this point: “This is not a category you can explain in 10 seconds. It’s nuanced. Bringing payers, technologists, and families together builds understanding and trust.”
Audience Questions: Extending the Conversation
The session closed with insightful questions from the audience that underscored both the practical and human challenges of access.
One audience member asked whether at-home blood testing could help ease the burden of long-term follow-up, especially for families navigating pediatric care. George Eastwood noted that while technology exists, the real hurdles lie in reimbursement and state-by-state policies.
Dr. Shrank added that providers need new payment models that reward doing what is best for patients—whether in-person or virtually. “We need a thoughtful payment model that allows the provider to do both what’s best for the patient and what’s best for their relationship—rewarding that, rather than just rewarding in-person visits,” he said.
Another question came in asking how families can help advocate with insurers, inspired by her grandson’s survival through CAR T therapy. George emphasized the importance of including patient and family voices in payer conversations, while Dr. Shrank stressed that advocacy builds trust among stakeholders. “When the patient or the patient’s family is in the middle of that conversation, you can bring the pieces together better,” he said.
These exchanges reinforced a central theme: patients and families are essential partners in shaping the future of access to cell and gene therapies.
Key Takeaways from the Session
- Fragmented payment systems make it difficult to reconcile high upfront costs with long-term value, creating access barriers.
- Value-based agreements are promising, but require robust systems to track patient outcomes over years—even across insurance changes.
- Patient experience matters: travel, lodging, and long-term follow-up care can be major obstacles without targeted support.
- Risk pooling and pre-negotiated contracts could make coverage more predictable and sustainable for payers and providers.
- Advocacy and transparency are essential to building trust among payers, providers, manufacturers, and patients.
By the end of the conversation, one message was clear: cell and gene therapies break the existing model—and fixing it will take collaboration, innovation, and a relentless focus on patient-centered solutions.
As Dr. Shrank put it, “We all agree curing is better than treating forever. Now we just have to build the system to make it possible.”
Learn More
Stay tuned for the next Bridging the Gap webinar, where science, compassion, and innovation continue to meet at the frontier of life-changing medicine.
About the Guest Speaker
William H. Shrank, M.D.
CEO, Aradigm
Dr. William Shrank is CEO of Aradigm, an insurance company launched in November 2024 with funding from Andreessen Horowitz, focused on improving access and affordability for cell and gene therapies. Previously, he was Venture Partner in Bio and Health at Andreessen Horowitz and served as Chief Medical Officer at Humana, where he led care delivery, clinical operations, health equity, and population health initiatives.
Before Humana, Dr. Shrank was Chief Medical Officer for UPMC’s Insurance Services Division, overseeing clinical operations and policy for 3.5 million members. He also served as Chief Scientific Officer at CVS Health and as Director of the Research and Rapid-Cycle Evaluation Group at CMS’s Center for Medicare and Medicaid Innovation.
Earlier in his career, he practiced at Brigham and Women’s Hospital and taught at Harvard Medical School. Dr. Shrank earned his M.D. from Cornell, completed residency at Georgetown, and holds degrees from UCLA and Brown University.