CAR T-cell therapy benefit for pediatric leukemia dependent on prompt treatment

Julia Thornton Snider

CHICAGO — Treatment delays significantly decreased value derived from treatment with tisagenlecleucel among pediatric patients with acute lymphoblastic leukemia, according to data presented at the ASCO Annual Meeting.

Tisagenlecleucel (Kymriah, Novartis) — the first FDA-approved chimeric antigen receptor (CAR) T-cell therapy — is indicated to treat patients aged younger than 25 years with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse.

“CAR T therapies have demonstrated impressive remission rates and survival gains relative to the previous standard of care, but we started to hear about delays in access,” Julia Thornton Snider, PhD, senior director of policy and economics at Precision Health Economics, told HemOnc Today. “Therefore, we wanted to do a study to understand the social value of CAR T and the consequences of delayed access to treatment.”

Snider and colleagues assessed the social value of tisagenlecleucel by estimating life expectancy, quality-adjusted life years (QALYs) and health care costs. Each QALY was valued at $150,000.

For this analysis, researchers used a price of $475,000 for tisagenlecleucel and adjusted for inflated 2016 U.S. dollars. They also discounted health and monetary values at 3%.

To assess for lost value from monthly treatment delays, the researchers assumed patients would start clofarabine — the standard of care for this population — while awaiting access to tisagenlecleucel.

The analysis included 20 incident cohorts comprised of 500 patients with pediatric ALL.

Patients treated with tisagenlecleucel gained 37,018 QALYs, and the total social value was $4.6 billion.

However, when treatment was delayed 1 to 3 months, social value decreased by $452 million to $2.1 billion. Additionally, with treatment delays, researchers observed 3,634 to 17,100 lost QALYs.

With a 4-month treatment delay, patients lost over half of the value and QALYs gained by treatment from tisagenlecleucel.

“Patients taking older therapies while waiting for approval for CAR T have very high mortality rates so, the longer they wait, the less likely they live to try CAR T,” Snider said. “The current data suggests the potential for long-term survival gains for many patients treated with CAR T, and our results show that these gains in survival and quality of life translate to a large social value. The social value stems from the health benefits but also from productivity gains. CAR T allows adults the possibility to continue in their jobs and children the possibility of growing up to have a career.”– by Cassie Homer

Reference:

Thornton Snyder J, et al. Abstract 10529. Presented at: ASCO Annual Meeting; June 1-5, 2018; Chicago.

Disclosures: Thornton Snyder reports employment with Precision Health Economics, stock/other ownership interests in Precision Advisors and institutional research funding from Precision Health Economics. Please see the abstract for all other authors’ relevant financial disclosures.