CAR-T Treatment Delay Results in Decreased Life Expectancy, QALYs


Improvements in life expectancy and quality-adjusted life years (QALYs) as a result of chimeric antigen receptor (CAR) T-cell therapy is dependent upon immediate access to treatment, according to a study that will be presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting (June 1-5, 2018; Chicago, IL).

While the United States Food and Drug Administration approved tisagenlecleucel—a CAR-T therapy—to treat pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia (ALL) in August 2017, many patients still face barriers to treatment.

Julia Thornton Snider, PhD, Precision Health Economics (Oakland, CA), and colleagues conducted a study to quantify the social value of tisagenlecleucel and the value lost from treatment delays.

The social value of the CAR-T therapy was predefined as its economic value to patients and the manufacturer, relative to clofarabine – the standard of care in this treatment setting. Researchers obtained estimates of life expectancy, QALYs, and health care costs from a cost-effectiveness model for tisagenlecleucel and valued each QALY gained at $150,000.

The social value of tisagenlecleucel was calculated using a price of $475,000. Researchers quantified lost value from monthly treatment delays under the assumption that patients started clofarabine treatment while awaiting access to tisagenlecleucel.

The total study population included 20 incident cohorts of 500 patients. Those treated with immediate tisagenlecleucel gained 37,018 QALYs, which translated to a total social value of $4.6 billion.

However, when treatment was delayed 1-3 months, 3634 to 17,000 QALYs and $452 million to $2.1 billion of social value were lost. When treatment was delayed 4 months, over half the social value, QALYs, and value to patients from tisagenlecleucel were lost.


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“Innovative therapies such as tisagenlecleucel have the potential to provide substantial social value from improvements in life expectancy, QALYs, and productivity” Dr Snider and colleagues concluded. “However, the magnitude of benefit depends upon the ability of patients to access these treatments promptly.”

Researchers added that efficient payment mechanisms and sufficient technological capabilities are needed to minimize treatment delays for these patients.—Zachary Bessette