Why Cell and Gene Therapy's Biggest Barrier Isn't Scientific

Cell and gene therapies have a huge curative potential but come with a large price tag that can result in access barriers. At Asembia’s AXS26 Summit, a panel of health care leaders gathered to discuss how the US health system can consistently and equitably get cell and gene therapies to the patients who need them.

Moderated by Joe DePinto, head of cell, gene, and advanced therapies at McKesson, the session brought together clinical, payer, and health system perspectives on this widespread and timely problem.

When a Million-Dollar Cure Meets an Annual Budget

DePinto set the stage by defining the problem with advanced therapies as one with enormous clinical promise hamstrung by structural financial misalignment.

“I like to think of it as a cost density issue, where you have a large outlay of cost at one point, but the value is seen over an extended period,” DePinto said. “Typically, our US health care system, the payer environment, has year-to-year budgets.”

This mismatch of a million-dollar-plus upfront expenditure weighed against a payer's annual budget cycle sits at the heart of why patients are falling through the cracks. Cell therapies currently range from $500,000 to $700,000 per treatment, while gene therapies can run $1 million to $3 million. That cost density problem is compounded by lingering questions about durability of response, misalignment between FDA-label coverage criteria and pivotal trial data, and uncertainty about which delivery channel best fits a given patient population and payer mix. A 2023 analysis noted that payers face high budgetary impact from large upfront payments, which has prompted growing interest in alternative payment structures.¹

Access Gap: Why Patients Never Get the Treatment They Qualify For

Navneet Majhail, MD, physician-in-chief of blood cancers at the HCA Sarah Cannon Cancer Network, drew on his experience overseeing one of the nation's largest cell and gene therapy networks to describe the scope of the access gap.

“If you look at the data that's out there, basically 1 in 5 patients who can potentially benefit from a CAR [chimeric antigen receptor] T therapy gets one today,” he said. “That's the gap we've got to fulfill.”

He presented additional data from across the Sarah Cannon network, finding that among patients with lymphoma who have already cleared many of the initial geographic and referral barriers and made it all the way to a CAR T-cell capable treatment center, 40% still did not receive an infusion. The leading reason was disease progression during the authorization and manufacturing window. This finding is consistent with broader published literature showing that the gap between eligibility and actually receiving CAR T-cell therapy is driven by delays in prior authorization, limited treatment center availability, manufacturing timelines of 2 to 4 weeks, and health disparities related to geography, race, and insurance status.²

“From when someone gets referred to us, it might be a good 8 to 12 weeks before they get an infusion with these autologous products," Majhail noted. "Time is of the essence, often, as we try to get these patients through.”

The Integrated System Perspective on Cell and Gene Therapies

Michael Evans, PharmD, chief pharmacy officer at Geisinger, offered a window into what it looks like to manage cell and gene therapy as both a payer and a provider.

“The floor is moving under our feet,” Evans said. “We know what's approved today. We know what step it is in therapy, but that will change tomorrow. We need to be very flexible.”

Evans noted that with all of these changes, you need to have a robust team dedicated to revenue management, prior authorization, and formulary navigation. He described a growing pattern in which payers quietly add eligibility criteria to their approval processes without notifying providers, creating a situation where a therapy may be administered in good faith and denied after the fact. His team now obtains "a signed and notarized letter" confirming payment before any therapy is administered. This presents a significant operational burden and can sometimes even put patients outside of their treatment windows.

Evans also underscored the challenge of premium setting in a fast-moving landscape: "When we place our Medicare bid for 2027, we have to predict the population of patients we think we're going to administer—and that will be very complex, because the floor is moving."

In Advanced Therapies, Nobody's Winning. That Could Change.

Will Shrank, MD, cofounder and CEO of Aradigm Health, introduced Aradigm as a carve-out insurance product focused exclusively on cell and gene therapy, pooling risk across self-insured employers and fully insured payers, capping volatility at the premium level, and operating on a cost-plus model with year-end dividends returned to customers. The platform currently covers approximately 1 million members, with plans to scale to 7 to 8 million by mid-year which Shrank said would make Aradigm the nation's largest cell and gene therapy–focused platform.

"Nobody's winning today," Shrank said on the topic of the advanced therapy landscape. "Everybody's struggling. If you're a purchaser, the volatility, unpredictability, and the truly existential threat if you get disproportionately hit by a handful of these claims, is a huge challenge. If you're a provider, it's hard to build a sustainable business model around a category of therapies that you have to invest millions of dollars to be able to deliver, and you don't know what the volume is going to look like."

He argued that the fragmentation of the US health care system in which purchasers, providers, payers, and manufacturers each optimize independently is a large cause of the access gap, not any single bad actor. His proposed a framework of structured collaboration in which each stakeholder gives something to gain a great deal. Providers who receive guaranteed volume and upfront payment at sustainable rates are more willing to meet quality indicators. Purchasers who pool together gain protection in numbers. Manufacturers, freed from the transactional complexity of pharmacy benefit manager relationships, can engage in more meaningful outcomes-based contracting.

"There is a way for everybody to give a little, and we're going to get a lot," Shrank said. He specifically cited value-based agreements as more viable in this category than in traditional pharmaceuticals due to the binary nature of the therapies. "If you've got a $3 million therapy that purports to cure something, and the outcome is binary: either the patient's cured or they're not. That is where we can have a real conversation about value."

References

1. Horrow C, Kesselheim AS. Confronting high costs and clinical uncertainty: innovative payment models for gene therapies. Health Aff (Millwood). 2023;42(11):1532-1540. doi:10.1377/hlthaff.2023.00527
2. Perales MA. Promoting CAR T access focuses on awareness, capacity, and economics. Targeted Oncology^®. Published April 2026. Accessed April 29, 2026. https://www.targetedonc.com/view/promoting-car-t-access-focuses-on-awareness-capacity-and-economics