CAR T-Cell Therapy for Pediatric Leukemia Is Cost-effective

Roxanne Nelson, BSN, RN

October 09, 2018

Chimeric antigen receptor (CAR) T-cell therapy has been hailed as a major advance and a veritable game changer for pediatric leukemia, but it comes with an exceedingly hefty price tag.

Tisagenlecleucel (Kymriah, Novartis) is priced at $475,000 for one treatment; but when it works, it offers a cure for pediatric and young adult patients (aged 3 - 25 years) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) who have run out of options.

A new analysis concludes that, despite its hefty price tag, the treatment is cost-effective.

The authors found that more than 40% of pediatric patients who undergo leukapheresis in preparation for tisagenlecleucel therapy will become long-term survivors, as compared to only 10% who receive comparator therapy.

Therefore, they conclude that, "with the evidence available at this time, tisagenlecleucel seems to be priced in alignment with benefits observed over a patient lifetime horizon."

The study was published online October 9 in JAMA Pediatrics.

Lead author Melanie D. Whittington, PhD, from the University of Colorado Anschutz Medical Campus, Aurora, and colleagues set out to study long-term survival in patients who have received tisagenlecleucel. They point out that in their study, follow-up was limited; the maximum duration of follow-up was less than 4 years.

Modeling Shows Cost-effectiveness

The team designed a decision analytic model by which they could extrapolate trial evidence to a patient lifetime horizon. Three studies were used for their analysis: B2202, which enrolled patients from April 8, 2015, to November 23, 2016; B2205J, which enrolled patients from August 14, 2014, to February 1, 2016; and B2101J, which enrolled patients from March 15, 2012, to November 30, 2015.

The modeled cohort that was created included only patients with B-ALL who were younger than 25 years and who had refractory disease or had experienced two or more relapses. The primary intervention of interest was tisagenlecleucel; the comparator of interest was the chemoimmunotherapeutic agent clofarabine (Clolar, Genzyme).

Their analysis showed that 40% of patients who were treated with tisagenlecleucel were expected either to be long-term survivors or to be alive and responding to treatment after 5 years. The total discounted cost of tisagenlecleucel was $667,000. The researchers projected that treatment with tisagenlecleucel would result in a gain of 10.34 discounted life-years and 9.28 quality-adjusted life-years (QALYs).

In comparison, the total discounted cost of clofarabine was approximately $337,000, with a discounted life-year gain of 2.43 years and a gain of 2.10 QALYs. This difference, say the authors, was an "incremental cost-effectiveness ratio of approximately $42,000 per life-year gained and approximately $46,000 per QALY gained for tisagenlecleucel vs clofarabine."

On analyses involving a variety of scenarios, including those that were more conservative in regard to long-term relapse and survival, the range of incremental cost-effectiveness ratio varied from $37,000 to $78,000 per QALY gained.

"As payers are negotiating their coverage and reimbursement of tisagenlecleucel, they should consider the uncertainties in the evidence and the assumptions made in this analysis," Wittington and coauthors conclude. "Novel payment models consistent with the present evidence may reduce the risk and uncertainty in long-term value and be more closely aligned with ensuring high-value care."

Importance of Cost-effectiveness Analyses

In an accompanying editorial, Lisa A. Prosser, PhD, from the University of Michigan, Ann Arbor, believes that this study "is exemplary in addressing many of these issues," in that it has carefully considered the influence of time horizon, discount rate, duration of benefit, and treatment initiation.

It can be difficult to extrapolate the benefits beyond the time frames used in the study, because the researchers based the effectiveness of tisagenlecleucel on pooled data from three trials in which the combined sample was small, at less than 200 patients, and the median follow-up period was less than 2 years.

"Scenario analyses yield cost-effectiveness results that would still be considered favorable, as long as the duration of benefit endured for 7 to 12 years," Prosser writes.

She notes that the cost-effectiveness ratio that was projected in this analysis is from $37,000 to $78,000 per QALY, which falls within conventional thresholds that have been proposed in the United States. "Understanding the drivers of value for tisagenlecleucel is useful when considering value and affordability of these new high-cost treatments," she says.

Modeling can provide insights for decision making when new treatments are being moved through the pipeline as quickly as possible and when trying to meet the needs of patients with rare conditions.

"This analysis demonstrates that costly interventions can be cost-effective if the clinical utility is high, which underscores the importance of using an economic evaluation framework, such as cost-effectiveness analysis," Prosser concludes.

The study was funded by the Institute for Clinical and Economic Review. Coauthors Daniel A. Ollendorf, PhD, Varun M. Kumar, MPH, MSc, Richard H. Chapman, PhD, and Steven D. Pearson, MD, are employees of the Institute for Clinical and Economic Review, an independent organization that evaluates the evidence on the value of health care interventions, which is funded by grants from the Laura and John Arnold Foundation, Blue Shield of California Foundation, and the California HealthCare Foundation. Dr Prosser has disclosed no relevant financial relaitonships.

JAMA Pediatr.Published online October 8, 2018. Abstract, Editorial

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