Asembia 2025: Tracking Long-Term Outcomes Key to Advanced Therapy Value

In an interview with Pharmaceutical Executive, Fran Gregory, VP, emerging therapies, Cardinal Health, shared key insights into the evolving landscape of cell and gene therapies. Speaking about the current state of the market, the anticipated growth of the pipeline, and the critical steps needed to ensure long-term sustainability, Gregory highlighted both the transformative potential of these therapies and the systemic challenges that must be addressed. Her perspective offered a comprehensive look at where the field stands today—and where it's heading next.

Pharmaceutical Executive: How are we seeing patient outcomes evolve with the rollout of these advanced therapies, and what still needs improvement?

Fran Gregory: That was a central theme of our session. We know these treatments are significantly improving patient outcomes—lives are being saved. But we now need to back that up with robust health economic modeling and clear, data-driven evidence. It's time to move into a space where we're showing, through statistical models, how these therapies provide long-term value to patients.

Anna Purdum, who was also on my panel, discussed health economic modeling and the role ICER plays in evaluating these therapies. ICER’s work helps demonstrate the value these treatments bring to patients. What really needs more focus now is long-term outcomes tracking—making sure patients are followed over time so we can truly understand the durability and sustained impact of these therapies. That data can then feed into health economic analyses, which we can take to payers to help shape sustainable payment models that support long-term access and financial viability.

Full Interview Summary: The session began with an overview of the cell and gene therapy (CGT) landscape—where the industry stands today and what the future may hold. The discussion included a pipeline review, highlighted key products, and identified both current successes and future areas of focus. Since the first CGT approval eight years ago, there has been significant progress. The conversation emphasized the need to continue advancing the field to ensure its momentum is sustained.

The CGT pipeline is rapidly evolving and holds tremendous promise, with projections estimating over 100,000 patients may benefit by 2030. However, this comes with a projected $75–80 billion cost to the healthcare system, prompting the need for sustainable payment models. Currently, around half of the pipeline targets oncology and hematology, but the field is expanding to include neurology, ophthalmology, rheumatology, and immunology, signaling broader therapeutic impact.

Patient outcomes from CGTs are transformative, yet there’s a growing need for long-term data to quantify their durability and cost-effectiveness. The discussion underscored the importance of robust health economic modeling and outcomes tracking to demonstrate value and guide reimbursement strategies. Insights from groups like ICER already point to the value of CGTs, but more evidence is required to solidify these claims.

Innovative payment models are crucial for long-term sustainability. The most promising models include outcomes-based contracts, where reimbursement aligns with patient response, and annuity payments, which spread costs over time. Models based on performance guarantees are less popular due to their all-or-nothing structure.

Looking ahead, the boldest move the industry can make is to collaboratively develop systems that ensure patient access while addressing affordability. Achieving this balance will require cooperation across stakeholders—payers, manufacturers, providers, and intermediaries—to ensure CGTs deliver on their full potential.