Pediatric gene therapy: From pipeline to balance sheet reality

Pediatric cell and gene therapy is no longer a pipeline story—it's a balance sheet reality. Multiple million-dollar-plus approvals, imminent FDA decisions, and more than 260 products advancing toward market are converging on health system financial planning. For leaders, the question is no longer whether these therapies reshape your organization. It's whether your strategy is built to lead when they do.

Why this matters

Recent pediatric-relevant approvals range from $2 million to $4 million [RH3.1]per treatment and reflect the continued arrival of extremely expensive, one-time therapies that may be clinically transformative—but create an upfront financial commitment for children’s hospitals and integrated delivery networks that could impact health organizations’ bottom lines. Examples include:

• Kresladi for leukocyte adhesion deficiency type I—a disease that prevents white blood cells from adhering to blood vessel walls, migrating to infection sites or forming pus—has comparable benchmark pricing ranging from $2 million to $4 million for similar therapies.
• Zevaskyn for recessive dystrophic epidermolysis bullosa—a rare genetic skin disorder that prevents the skin’s outer and inner layers from adhering properly, leading to severe, painful blistering and scarring from minor friction—with a reported cost of $3.1 million.
• Itvisma for spinal muscular atrophy type 2—a genetic neuromuscular disorder where people with this disease cannot stand or walk independently—with a reported cost of $2.59 million.
• Waskyra for Wiskott-Aldrich syndrome—a disorder that makes patients highly susceptible to severe infections, easy bleeding, autoimmune conditions, and certain cancers like lymphoma—has comparable benchmark pricing ranging from $2 million to $4 million for similar therapies.

For many health systems and children’s hospitals, the costs of these therapies may mean reevaluating how they budget for rare disease care, manage financial risk, and coordinate access to emerging advanced therapies.

Near-term 2026 planning considerations

Several pediatric or pediatric-adjacent products [NH6.1][MS6.2]are expected to undergo FDA review, receive approval, and enter the market in 2026, signaling continued expansion across rare metabolic, neurologic, immunologic, and oncologic conditions.

As the financial impact of individual cell and gene therapies continues to grow, health system executives should carefully consider the following strategic implications:

1. Reimbursement risk remains high. Coverage policies, coding, prior authorization requirements, payment timelines, and reimbursement methodologies continue to evolve and often lag behind clinical adoption. Delays or denials can create substantial financial risk for health systems. Leaders should ensure strong coordination across pharmacy, revenue cycle, contracting, finance, and payer relations to optimize reimbursement processes before patients begin treatment.
2. Operational readiness is a competitive requirement. Cell and gene therapies fundamentally increase operational complexity by blurring inpatient and outpatient care models, requiring months of patient-specific manufacturing. Organizations that get ahead are implementing cross-functional orchestration across pharmacy, finance, revenue cycle, contracting, and clinical operations—including chain of custody, specialized storage, prior authorization, and single-case agreement workflows.
3. Site-of-care and access models are evolving. As some therapies transition to outpatient or alternative care settings, organizations should evaluate where these therapies can be delivered safely and efficiently while maintaining the specialized infrastructure required for administration and follow-up. Leaders should assess whether existing outpatient infusion centers, specialty pharmacy services, care coordination teams, and reimbursement processes are equipped to support these therapies, while considering the financial and operational implications of shifting care outside the inpatient setting.

4. Technology evolution increases complexity. Multi-target CAR-T, off-the-shelf allogeneic approaches, in vivo therapies, alternative delivery routes, and point-of-care manufacturing may improve access and scalability over time, but they also will introduce new operational, safety, and contracting considerations.

   Operational: New storage and handling requirements, chain of identity/custody, manufacturing coordination, scheduling, staffing, and administration workflows.

   Safety: Enhanced patient selection, monitoring for delayed adverse events, management of toxicities (e.g., cytokine release syndrome), long-term follow-up, and regulatory reporting.

   Contracting: Single-case agreements, payer coverage negotiations, outcomes-based or value-based reimbursement arrangements, manufacturer contracting, and financial risk-sharing models.

Pediatric cell and gene therapy has crossed a threshold — and health systems that treat it as a future-state challenge are already behind. The financial stakes are real, the operational demands are immediate, and the window to build a strategy is narrowing with each new approval.

Leaders that align pharmacy, finance, clinical operations, and payer strategy before the next therapy arrives will be positioned to deliver transformative care without absorbing preventable losses. Those who wait will find themselves reacting to a landscape they could have helped shape.