Cell and gene therapy program implementation: Lessons from leading health systems
The U.S. cell and gene therapy market is growing at a 33% compound annual growth rate, with multiple CAR-T therapies achieving or approaching blockbuster status.1 Yet only two out of ten eligible patients receive these potentially curative treatments.2 The gap between market expansion and patient access reflects the operational complexity health systems face in delivering cell and gene therapies (CGTs).
At the December 2025 ASHP Midyear Clinical Meeting, pharmacy leaders from Children's Hospital of Philadelphia, Froedtert ThedaCare, and Moffitt Cancer Center shared what they've learned building cell and gene therapy programs. Their insights cut across governance, finance, access, and infrastructure, offering a practical view of what it takes to close the gap between CGT's clinical potential and patient access.
The operational complexity is unlike anything pharmacy has faced
At Children's Hospital of Philadelphia (CHOP), the cell and gene therapy journey spans three decades on the research side. Commercial hospital operations, however, have compressed into roughly five to ten years of intensive development. Richard Dyke, Director of Pharmacy Business Operations, and his team are standardizing processes for therapies that keep evolving, often adapting workflows mid-implementation.
Three characteristics make CGT fundamentally different from traditional specialty pharmacy. First, these therapies are intensely patient-specific in handling. Second, they carry time sensitivity at every stage. Even therapies that aren't clinically time-critical develop urgency through the manufacturing process, insurance verification, and cell viability windows. Third, the stakeholder pressure is unrelenting. These are logistically complex, high-stakes, expensive therapies, often with curative intent. Families, providers, and CFOs all demand real-time visibility into every step.
The manufacturing timeline alone illustrates the complexity. Cell collection, manufacturing, and distribution can begin weeks to months in advance, depending on the therapy.3 Lead times for product delivery can be days to weeks to months, with strict quality assurance, temperature and environmental controls throughout. When manufacturing doesn’t yield cells meeting Food & Drug Administration (FDA) specifications (a situation that has become less frequent but still occurs) the conversation with payers and families about proceeding with suboptimal manufacturing specs requires clinical judgment and careful documentation.
Governance structures are evolving in real time
Traditional Pharmacy and Therapeutics committee structures weren't designed for CGT. Justin Konkol, Executive Director of Cancer Services at Froedtert ThedaCare, found that the standard Pharmacy and Therapeutics (P&T) committee approach broke down when confronted with CGT's unique financial, operational, and clinical dynamics. These therapies don't fit existing formulary review processes.
The response at many institutions has been to create dedicated governance. CHOP calls theirs a "Cell and Gene Therapy Onboarding Workgroup" while Froedtert uses an "Innovative Therapies Committee." These structures bring together stakeholders who rarely sat in the same room before: pharmacy, finance, revenue cycle, legal, supply chain, clinical administration, and strategy.
Konkol's team is now implementing a two-step committee process. When an agent is six to twelve months from FDA approval, they conduct clinical review and data mining to identify which patients within their health system's patient population could benefit. This early work surfaces hard questions: If only two or three patients might benefit, is the operational investment justified? Should they create referral pathways to regional partners instead?
CHOP’s governance model includes separate arms for research and commercial CGT operations, a distinction that matters because 30 years of CGT research experience doesn’t directly translate to commercial therapy management. Their CGT Business and Operations Executive Committee oversees commercial therapies while the Cell and Gene Therapy Collaborative oversees research. CGT Operations Leadership and the Cell and Gene Therapy Collaborative coordinate to ensure connection and collaboration between research and commercial activities with specialized workgroups for onboarding newly FDA-approved commercial therapies.
Financial sustainability remains the central challenge
The cost structure of CAR-T therapy is sobering. Drug acquisition alone often exceeds $400,000, representing roughly 75 percent of total cost. Add hospital stays for administration and monitoring (typically two to three weeks), apheresis and diagnostic procedures, cytokine release syndrome management, and follow-up care, and total cost per patient frequently exceeds $500,000.4
These costs must be weighed against the potential for durable clinical benefit. Five-year follow-up data show durable remissions in 30-40% of patients with large B-cell lymphoma, with those maintaining complete response at two years showing near-complete disease-specific survival at five years.5,6 Importantly, real-world evidence confirms these outcomes translate outside clinical trials: CIBMTR registry data show comparable response rates even among the 57% of patients who would not have met original trial eligibility criteria, including older patients whose outcomes matched younger cohorts.7 For comparison, standard-of-care treatments for relapsed/refractory hematologic malignancies can cost $125,000-$430,000 annually with ongoing therapy8—costs that accumulate year over year and can ultimately exceed CAR-T's one-time investment for patients who achieve durable remission.
Reimbursement timelines compound the challenge. At CHOP, reimbursement timelines can vary, but are typically three to six months for payment, a cash flow reality that’s becoming a prominent concern for health system finance leaders. Invoice terms with manufacturers create additional pressure, as product is invoiced on delivery while reimbursement lags months behind.
Single case agreements have become standard practice for CGT. At Moffitt Cancer Center, Donn Davis, Pharmacy Business, Contracting, and Revenue Integrity Manager, has developed template agreements to reduce turnaround time, and most major payers have now standardized their own CGT agreement processes. These agreements clearly delineate what's covered (typically diagnostic testing, evaluation, therapy administration, and specified follow-up care) but Davis stresses that institutions must map out total cost of care including potential complications, extended treatment needs, and post-therapy monitoring.
Financial toxicity extends to patients themselves. The highest-risk patients financially aren’t the uninsured, but those with coverage gaps. Silver or bronze tier plans may leave patients responsible for 20 percent of a half-million-dollar therapy. Konkol’s team at Froedtert now requires both primary and secondary insurance coverage before proceeding with CAR-T.
Access is expanding, carefully
The regulatory landscape is shifting to potentially broaden CGT access. In June 2025, the FDA removed Risk Evaluation and Mitigation Strategies (REMS) requirements on existing BCMA- and CD-19-directed CAR-T therapies, though manufacturers still must certify treatment centers.9 Texas law H.B. 3057 now allows certified, in-network providers to administer CAR-T without requiring accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT).10 This represents a significant change from the Centers of Medicare and Medicaid Services (CMS) 2017 determination that cellular therapies must be administered at FACT-certified centers like bone marrow transplant programs.
The CMS CGT Access Model, which began rolling out in January 2025 across 34 Medicaid programs, represents a new framework for outcomes-based payment.11 It is important to note that this model currently covers only sickle cell disease gene therapies (Casgevy and Lyfgenia), not CAR-T products. However, the model establishes infrastructure for outcomes-based agreements where payment ties to treatment success—an approach that could eventually extend to other cell and gene therapies, and that health systems should monitor closely.
For health systems, the access question creates tension between expertise and geography. Patients currently drive three to six hours for treatment. Expanding access while maintaining safety requires new partnership models that don't yet exist at scale. Konkol has been exploring partnerships with rural and regional hospitals to provide CGT with remote case management support from Froedtert.
The infrastructure investment is substantial
Froedtert is developing a formalized business plan for CGT that includes a centralized Coordination Office housed within pharmacy to manage intake, routing, and cross-functional collaboration. The office will include a dedicated program manager, nurse support, physician partner, and pharmacy partner. This infrastructure didn't exist five years ago because the volume didn't justify it.
The challenge becomes more acute as CGT expands beyond oncology. Neurology and autoimmune disease pipelines are advancing, but these service lines often lack the financial counselors, social workers, and centralized case management that cancer programs have built. Konkol recently received an inquiry from Froedtert's neurology team asking how to manage CGT patients when they have no infrastructure for this complexity.
The question facing health systems isn't whether to invest in CGT infrastructure. The pipeline is expanding beyond rare diseases into indications that affect far larger patient populations, including autoimmune conditions and common cancers. This evolution is precisely why health systems must build scalable CGT capabilities now rather than reacting after volume forces their hand.
The reimbursement landscape is shifting
Current payment models create significant financial risk for health systems administering CAR-T. The Medicare reimbursement structure illustrates this challenge: average CAR-T product costs approach $500,000, while the Medicare base DRG payment for FY 2026 is approximately $314,000. Every organization should be considering and investing the resources into development of outpatient and/or home infrastructures to deliver CGT therapies to ensure program viability. High-cost outlier payments help offset this gap but typically do not fully close it, meaning health systems may absorb significant losses per patient depending on payer mix and negotiated rates. This financial exposure is a primary driver of why CGT programs require dedicated financial planning and governance.12
The direction points toward outcomes-based reimbursement where manufacturers share risk on therapy effectiveness. Several payers are launching designated Centers of Excellence networks for cellular, gene, and molecular therapies. While the CMS CGT Access Model currently applies only to sickle cell gene therapies, its outcomes-based framework could serve as a template for future CAR-T reimbursement models.
Health systems are watching how these outcomes-based models will affect their financial position. When outcomes-based agreements refund payers for therapies that don't produce results, questions remain about how health systems that have already provided care and incurred costs will be treated. Sustainable payment models will need to keep providers in the financial equation.
Cost reduction through scale and process optimization offers some hope. As more centers gain CGT experience and manufacturing processes mature, costs should decline. But "should" isn't a strategy, and health systems are building programs today with today's economics.
The path forward
Health systems managing CGT well share common characteristics: dedicated governance structures that bring finance and clinical operations together early, standardized processes for therapy onboarding and patient financial clearance, proactive engagement with payers on single case agreements, and realistic assessments of which therapies their patient population and infrastructure can support.
The U.S. CGT market's rapid growth trajectory is not slowing. Multiple CAR-T therapies have achieved blockbuster status, with more on track for 2026.13 For pharmacy leaders, the question isn’t whether cell and gene therapy will transform their operations. It's whether they'll build the systems to manage that transformation proactively or reactively.
For the 80 percent of eligible patients who don't currently receive CAR-T therapy, building these systems isn't an operational exercise. It's the difference between access and exclusion from potentially curative treatment.
This article is based on the Accelerate Pharmacy Solutions Midday Symposium "Transforming Tomorrow: Navigating Cell & Gene Therapy at Health Systems" presented at ASHP Midyear Clinical Meeting. Speakers included Kevin Chinn (Cencora), Richard Dyke, PharmD (Children's Hospital of Philadelphia), Justin Konkol, PharmD (Froedtert Health), and Donn Davis, PharmD (Moffitt Cancer Center).
Sources
1. Evaluate Pharma. Net U.S. CGT Sales Projections. 2024.
4. Association of Community Cancer Centers. "The Economics of CAR T-Cell Therapy." 2024.
6. https://ascopubs.org/doi/10.1200/JCO-25-00507
10. Texas Legislature. H.B. 3057, 89th Legislature. 2025.
11. Centers for Medicare & Medicaid Services. "Cell and Gene Therapy Access Model." 2025.
13. Cell Therapy Market Size & Share Analysis - Growth Trends and Forecast(2026-2031)." 2026.
