Conference Coverage

‘Real-world’ data show CAR T therapies are cost effective


– Chimeric antigen receptor (CAR) T-cell therapy has been hailed as a major advance and a game changer, but their cost has redefined the meaning of “expensive.”

However, new “real-world” data now suggests that CAR T-cell therapy may actually be cost effective, as it may lower other expenses related to the illness.

When used in a population of older adults with non-Hodgkin lymphoma, these new data show that CAR T-cell therapy cut related expenditures, compared with health care costs prior to receiving this treatment.

“CAR T therapy was associated with fewer hospitalizations, shorter time in the hospital, fewer ED visits, and lower total health care costs,” said lead study author Karl M. Kilgore, PhD, of Avalere Health in Washington, D.C.

He presented the findings at the 2019 annual meeting of the American Society of Hematology (abstract 793).

CAR T-cell therapies were approved in the United States in 2017. First came tisagenlecleucel (Kymriah, Novartis), for the treatment of pediatric and young adult patients with acute lymphoblastic leukemia, with a price tag of $475,000. Closely following it was axicabtagene ciloleucel (Yescarta, Kite/Gliead), indicated for adult patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma who are ineligible for autologous stem cell transplant, with a price tag of $373,000.

The formidable price tags sent shock waves through the blood cancers community, which is struggling to incorporate this novel approach because of the remarkable responses that have been seen.

Real-world experience

In the current study, Dr. Kilgore and colleagues evaluated the demographic and clinical characteristics of Medicare patients who received CAR T therapy (axicabtagene ciloleucel or tisagenlecleucel) and then compared health care utilization, costs, and outcomes pre– and post–CAR T therapy.

“The goal of this study was to look at the real use of CAR T-cell therapy and real-world data on the use of these therapies,” said Dr. Kilgore. “And to look at health care utilization.”

Data was obtained from the Centers for Medicare & Medicaid Services 100% Medicare Fee-for-Service Part A and B claims data, and patients were included in the study if they had been diagnosed with lymphoma and received CAR T therapy between Oct. 1, 2017, and Sept. 30, 2018.

A total of 177 patients met all of the inclusion criteria and were included in the analysis.

The average age was 70 years, more than half (58.8%) were male, and they were primarily white (87.6%). Nearly all patients (91.5%) had a primary diagnosis of diffuse large B-cell lymphoma (DLBCL), as well as multiple comorbidities with 74.6% having a Charlson Comorbidity Index score of 3 or less. Fewer than 5% of patients had undergone a previous autologous stem cell transplant, and 51% had one or more comorbidities that would have disqualified them from participating in CAR T clinical trials (for example, renal failure, heart failure, recent history of deep vein thrombosis/pulmonary embolism).

Just over half of participants (52%) had been treated with intravenous chemotherapy in the 6 months prior to receiving CAR T-cell therapy, and 60% received outpatient lymphodepletion.

During their index episode of care for CAR T infusion, the patients spent a median of 16 days (interquartile range, 10) in the hospital during and 45.5% required ICU care after infusion. In the 6-month period prior to CAR T-cell therapy (preindex), 51.2% had been hospitalized at least once, and almost 20% had three or more periods of hospitalization. Of that group, 27.1% were readmitted during the postindex period.

Among patients who required hospitalization, the median length of stay preindex was 7 days and 5 days post index. The number of ED visits was also lower in the post versus preindex (15.8% vs. 29.9%).

“Patients spent 17% less time in the hospital 6 months after CAR T-cell therapy than before,” he said.

While there were no deaths during the postindex period, a small percentage (less than 5%) were admitted to hospice care. It is unclear if any patients received chemotherapy during the 100-day postindex period, which would suggest disease progression. However, the authors note that claims for the period might be lagging behind for some patients.

Dr. Kilgore pointed out that half of the patients had one or more chronic conditions that, in some cases, would have excluded them from clinical trials. “But 73% remain alive at 6 months,” he said. “We have data that goes out to 21 months, and over 50% are still alive at almost 2 years.”

As for cost, the median total health care costs during the preindex period were $51,999 (mean, 58,820; standard deviation, 45,701) and $14,014 post index (mean, 23,738; SD, 29,698). This extrapolates into $9,749 pre- versus $7,121 post index for each patient per month, which is a 27% decrease in expenditures.

Dr. Kilgore explained that the total paid amounts for CAR T from all sources (Medicare and patient) varied significantly, depending on whether patients were treated in a clinical trial and whether the hospital was reimbursed under standard Medicare prospective payment system for inpatient facilities or through the PPS-exempt payment system.


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