The hidden coordination burden behind cell and gene therapy access | HSES 2026

A new diagnosis — whether it’s cancer or a chronic condition newly identified through genetic testing — asks a lot of the people treating it, not just the people living with it. For health systems, the burden extends well past the exam room. Despite the real clinical strides in cell and gene therapy — treatments that work by modifying a patient’s cells at the genetic level to manage, treat or potentially cure disease — getting one of these therapies from approval to a patient’s bedside runs into roadblocks that have nothing to do with whether the science works.

Getting a cell or gene therapy through FDA approval is only the beginning. Health systems are finding that the harder problem is operational: scheduling apheresis, managing REMS requirements, coordinating multidisciplinary care teams, and tracking patients through years of required follow-up.

At the first Health System Executive Summit, Candy Tsourounis, PharmD, UCSF School of Pharmacy/UCSF Medication Outcomes Center, and Elise Wozniak, PharmD, Formulary & Drug Use Coordinator, Medication Outcomes Center, UCSF Health, got candid about the issues surrounding this groundbreaking therapy.

Where cell and gene therapies currently are

After decades of research, cell and gene therapies are finally entering clinics and making a measurable difference in patients’ lives. By targeting a patient’s genetic makeup and modifying it, these therapies can put diseases into remission or cure them outright. According to the FDA, gene therapy can work in one of several ways:

• Replacing a gene that is missing or causing a problem
• Adding a gene to help treat disease
• “Turning off” a gene that isn’t working properly
• Repairing a gene mutation through genome-editing technology

To do this, scientists use “vectors” — often a virus modified to remove its ability to cause infection — to deliver genetic material into cells. In other cases, doctors modify blood, bone marrow or other tissue taken from the patient, then reintroduce the changed cells so the new gene can take effect.

Cell and gene therapies (CAR-T, and gene therapies for conditions like spinal muscular atrophy (SMA) and sickle cell) have grown sharply in both clinical complexity and cost. The clinical bar to get one approved keeps falling, but the operational bar to deliver one safely — apheresis slot scheduling, manufacturing turnaround, REMS compliance, long-term (sometimes 15-year) follow-up obligations — keeps rising. A coordination failure isn’t just a clinical problem; it’s a dropped patient and a reimbursement denial.

“This has really changed how many health systems work together now, because you have to come out of the silos in order to understand each person’s role,” said Tsourounis. “Elise and I have weekly or biweekly meetings with our teams to go over where this patient is in their journey — where are we with contracting, where are we with clinical care, where are we with storage.”

Which failure points are causing the biggest barriers

Storage and handling trip up health systems before a patient is even scheduled.

“Each gene therapy seems to have its own set of rules and specific things that you need to consider,” Wozniak said.

Some require ultra-cold storage, some require a separate facility where cells can be manipulated under controlled conditions, and all of them require tracking chain of identity or chain of custody so the system always knows exactly where a patient’s product is in the process.

Site-of-care decisions compound that. Describing one therapy given as “really an easy injection, not an infusion,” Wozniak noted that drug labels often don’t settle the question:

“They don’t really tell you where you should get it, just kind of generally the timeline when it should be given. But you, as an institution, have to decide what works best.”

Billing is also another tricky barrier. Tsourounis said some of the costliest mistakes happen in this area. One therapy’s inner and outer packaging carried two different national drug code (NDC) numbers; billing under the wrong one meant the claim went unpaid.

“If you bill using the wrong NDC, you’re not going to be paid,” she said. “We had to learn that the hard way.”

Reimbursement is also inconsistent even when everything is billed correctly:

“You might say, ‘I’m expecting $1.7 million,’ but you might get this in $600,000 increments over a period of time,” Tsourounis said. “You need to track that to ensure you’ve gotten fully reimbursed.”

There’s also a financial tension unique to gene therapy’s slower-than-expected uptake. For hemophilia specifically, Tsourounis said, “we haven’t seen the uptake as much” as projected — so much so that “manufacturers have removed some gene therapies from the market because of that low interest, that low uptake,” driven partly by durable clotting-factor alternatives already meeting clinical need and partly by unresolved questions about long-term durability and safety, including cases of fatal acute liver failure now reflected in some products’ prescribing information.

That slow uptake creates a secondary bind.

“If you have a hemophilia treatment center at your organization, it’s likely operating on the 340B revenue that it’s generating from administering factors,” Tsourounis said, “and as factor use will go down, so too would those revenues.”

How to fix it

Both speakers’ core recommendation was to get full alignment across departments before committing to offer a therapy, not during rollout. Wozniak described assigning “the quarterback — the main person to kind of shepherd the entire process through” — coordinating pharmacy, contracting, revenue, and procedural teams from the start.

Her takeaway: “You need to be aligned before doing this.”

On the reimbursement side, Tsourounis said the fix is largely about timing and specificity. “Recognizing which payer requires a single case agreement — or a letter of agreement — is key to minimizing financial risk,” she said, “and not assuming that your organization will be paid under your contracted coverage.”

That means negotiating those agreements before a patient needs the drug, not after. Prior authorization requires similar discipline:

“It’s critical to work with your organization to get pre-approvals or prior authorizations, and to be very aware of the prior auth duration,” she said, since it’s possible to “run out of time” before a patient is actually ready to receive the therapy the authorization was approved for.

Finally, both speakers pointed to having the right expertise embedded in these decisions.

“It’s really important that you have a pharmacist at the table,” Tsourounis said, “so they can help you understand the process — how these therapies have to be stored, the chain of custody, and all the issues that come with it.”

She closed by urging systems to track outcomes once a therapy is live:

“I hope that if you are using gene therapies, you can assess whether they are working in your patient populations, and get some real-world evidence out there — that’s how we can help inform people on what the reality of what these therapies are offering patients.”