Real-World Data Drives CAR T Treatment Decisions, Reveals Benefits of Earlier Administration | AMCP Nexus 2025

In 2017, the first two CAR T-cell therapies (chimeric antigen receptor T-cell) — Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) — were approved and have helped to transform treatment for blood cancer. Since then, the two therapies have been approved for additional indications and five new CAR T-cell therapies have come to market.

As the CAR T pipeline expands into solid tumors and autoimmune diseases, the current infrastructure may struggle to meet increased demand. Success will require sustained collaboration among payers, providers, manufacturers, and patients to ensure equitable access while managing costs effectively, speakers said today during a session at the Academy of Managed Care Pharmacy (AMCP) Nexus, which is being held at the Gaylord National Resort and Convention Center in National Harbor, Maryland, outside of Washington.

Patients face challenges related to access while payers and providers determine how to manage the high costs of these therapies and provide reimbursement for the centers.

Real-world evidence is beginning to drive decision-making for who, when, and where patients might receive CAR T-cell treatments. Andy Berg, Pharm.D., CEO of Audaire Health, outlined during the session how data can identify high-performing treatment centers, reduce administrative bottlenecks, and support value-based contracting arrangements. “Better data leads to better decisions and better outcomes,” he stated.

For example, emerging evidence suggests that earlier administration of CAR T therapies — before patients are heavily pretreated — significantly improves outcomes. Studies show that reducing wait times by two months can increase the number of eligible patients receiving treatment by 10%, and a lower tumor burden at infusion correlates with better response rates and reduced toxicity. One study Berg mentioned at the session has shown that treating with CAR T therapies when patients have a lower tumor burden led to higher rates of complete response, better overall survival, and better event-free survival.

Chester B. Good, M.D., MPH, senior medical director for value-based pharmacy at UPMC Health Plan, shared a personal story about his brother-in-law who died from complications before receiving the therapy. His account underscored the urgency of expanding access and streamlining referral processes.

Berg said real-world evidence can bridge the gap between clinical trials and what is happening with patients in the centers. “There are some ongoing trials on sequencing and understanding earlier positioning of the CAR T therapy versus standard of care, but for payers, this reinforces the importance of timeliness and reducing burdens and any sort of administrative burden and logistical delays to ensure patients are able to be qualified and eligible for therapy,” he said.

Real-world data about how physicians were addressing the risk of cytokine release syndrome and neurotoxicity informed the FDA’s decision in June 2025 to eliminate the Risk Evaluation and Mitigation Strategy (REMS) program for CAR T-cell therapies. This regulatory change opens possibilities for expanding treatment to community oncology settings, which could help to ease access for patients in rural communities or areas without an accredited center of excellence. A geographic concentration of accredited centers has created a significant barrier for patients in rural areas.

“Data can help us understand tech variation between sites as well as between individual providers within a site within the same center,” Berg said. “We can identify the performers by treatment times and total cost of care, as well as toxicity and efficacy. This could mean directing our contracting teams to work directly with that provider, that facility, possibly on a shared savings program.”

The Health Plan Perspective

Health plans are exploring various strategies to expand access, including hub-and-spoke models connecting academic centers with community oncologists, value-based agreements with manufacturers, and enhanced care coordination services covering housing and transportation. Cross-functional teams including pharmacy, care management, network contracting, and social services are essential for addressing the multifaceted challenges.

As the CAR T pipeline expands into solid tumors and autoimmune diseases, the high costs are of concern to payers, said Ryan Steadman, Pharm.D., senior vice president of pharmacy at CareSource, a nonprofit managed care organization serving primarily the Medicaid markets with more than 2.2 million members in 15 states.

“Over two-thirds of health plans have some form of restriction on these [CAR T] agents, whether it’s step therapy, prior authorization or some form of exclusionary criteria. I think that's going to continue to increase as we continue to look at the cost, along with the long-term durability of these agents.”

He said cross-functional teams including pharmacy, care management, network contracting, and social services are essential for addressing the multifaceted challenges. “We have to partner to create a hub-and-spoke model with the community oncologist to increase and expand the access to these therapies,” Steadman said.

Value-based agreements play a role and will continue to be a lever, as will working with manufacturers for assistance programs that might help with housing and transportation. But, he said, the right partnerships with the right centers and providers will be key to expanding access and addressing costs.

“Servicing the majority of lives in the Medicaid book of business, we know they are the vulnerable patients out there,” he said, noting that social determinants of health, education barriers, and complex insurance navigation create additional access hurdles beyond geography.