Immuno-Oncology Resource Center

Immuno-Oncology Resource Center

April 26, 2018
7 min read

Analysis determines true cost for CAR T-cell therapy

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Inmaculada Hernandez

Patients treated with chimeric antigen receptor T-cell therapy will incur on average $30,000 to $36,000 in additional costs aside from drug expenses, according to a research letter published in JAMA Oncology.

Patients who present with severe cytokine release syndrome may pay up to $56,000 more.

“To our knowledge, this study provides the first estimates of the total costs of chimeric antigen receptor [CAR] T-cell immunotherapies in the United States,” Inmaculada Hernandez, PharmD, PhD, assistant professor of pharmacy and therapeutics at University of Pittsburgh, and colleagues wrote.

Last year, FDA approved the first two CAR T-cell therapies.

Tisagenlecleucel (Kymriah, Novartis) is approved for treatment of patients aged up to 25 years with refractory B-cell precursor acute lymphoblastic leukemia, as well as those whose disease is in second or later relapse.

Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adults with relapsed or refractory large B-cell lymphoma who underwent two or more lines of systemic therapy.

Treatment with tisagenlecleucel has been priced at $475,000 and axicabtagene ciloleucel has been priced at $373,000. However, these prices do not account for costs associated with treatment, including hospital stays and treatment for related toxicities.

“Patients undergoing CAR T-cell therapy require leukapheresis, administration of chemotherapy for depletion of lymphocytes and administration of CAR T-cell therapy, and a high proportion of patients receiving CAR T-cell therapy require ICU hospitalizations for cytokine release syndrome, a severe but common side effect of CAR T-cell therapy,” Hernandez told HemOnc Today. “All of this is related with facility costs — costs reimbursed to the hospital — and physician costs, which, again, are not accounted for by the price tag announced by the manufacturer.”

Hernandez and colleagues identified 11 scenarios that accounted for variability in receipt of treatment, development of cytokine release syndrome and response to therapy.

“Our analysis included the facility and provider costs associated with cytokine release syndrome,” Hernandez said. “Specifically, we accounted for both the probabilities and costs associated with the occurrence of mild to moderate cytokine release syndrome and of severe cytokine release syndrome.”

The researchers used data from the FDA and the ELIANA trial to determine the probability of each scenario. The researchers used the 2017 Medicare Physician Fee Schedule rates, Medicare Hospital Outpatient Prospective Payment System amounts, Medicare Part B payment limits, and Health Care and Utilization Project estimates to calculate physician costs for leukapheresis, administration of lymphodepletion therapy and CAR T-cell therapy, costs of other drugs, and facility costs.

“It is important to quantify the total costs of this therapy because these nondrug costs are certainly not negligible. It is important to account for them when doing pharmacoeconomic evaluations of these therapies and deciding on their coverage,” Hernandez said. “Moreover, the outcomes-based agreements announced for tisagenlecleucel would only refund pharmaceutical costs for nonresponders. So, it would be important, as well, to account for those nondrug costs when payers negotiate pricing agreements with manufacturers.”


Total cost of CAR T-cell therapy

The researchers used the sum of the expected costs in each scenario and the probability of each scenario to determine the mean expected cost per patient.

The mean total expected cost of tisagenlecleucel was $510,963. Costs ranged from $478,777 for patients who did not develop cytokine release syndrome to $531,813 for patient who did develop cytokine release syndrome.

The mean total expected cost decreased to $432,131 for tisagenlecleucel when researchers adjusted for the outcomes-based pricing arrangement announced by Novartis.

Every year about 600 U.S. patients are eligible for tisagenlecleucel. Using the mean total expected cost, the researchers estimated the annual expenditure would be $259 million.

The mean total expected cost of axicabtagene ciloleucel was $402,647.

With 7,500 U.S. patients eligible for axicabtagene ciloleucel, the estimated annual expenditure would be $3 billion.

Nondrug costs accounted for 7% of total costs.

“However, this doesn’t mean that these costs are negligible, because this 7% accounts for $30,000 for the average patient and up to $60,000 for those who present with cytokine release syndrome,” Hernandez said.

This study was not designed to assess benefits associated with CAR T-cell therapy or to provide a cost-effectiveness analysis. However, as HemOnc Today previously reported, the Institute for Clinical and Economic Review concluded that with the available information, CAR T-cell therapy costs align with its benefits. More analysis should be done as more long-term data on the therapy become available, according to Hernandez.


Reimbursement for CAR T-cell therapy

It is unclear how reimbursement models for CAR T-cell therapy will be designed, Hernandez said.

“Payers should factor in nondrug costs when making decisions on the coverage of these therapies and when negotiating prices with manufacturers. For the health care systems perspective, the key point is that health care resources are not unlimited,” Hernandez told HemOnc Today. “Money needed to cover new highly expensive therapies with a high budget impact, such as CAR T-cell therapy, needs to come from somewhere — either from a reallocation of funds from other therapies and health services that will no longer be covered or from increased premiums.”

Programs like Novartis’s outcomes-based pricing for tisagenlecleucel may help curb costs; however, the impact on the health care system is large, Hernandez said.

“I think we can all agree that health insurance is already quite expensive in the U.S., and nobody likes to see premiums increase,” she added. “But if we don’t want premiums to keep increasing, it is necessary to evaluate new therapies thoroughly and assess the benefit they bring for their cost, and how that ratio compares to that of other health services.” – by Cassie Homer


For more information:

Inmaculada Hernandez, PharmD, PhD, can be reached at


Disclosures: Hernandez reports no relevant financial disclosures. One author reports research funding from the Laura and John Arnold Foundation and royalties from his book, Ending Medical Reversal.