Benefits of 2 CAR T-cell Therapies May Justify Their High Price, ICER Report Argues

Benefits of 2 CAR T-cell Therapies May Justify Their High Price, ICER Report Argues

The clinical benefits of two recently approved CAR T-cell cancer therapies appear to justify their hefty price, a draft report by University of Colorado researchers suggests.

The researchers — Melanie Whittington, R. Brett McQueen, and Jon Campbell, all with the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences — were appointed to lead a study estimating the cost-effectiveness of approved CAR T-cell therapies.

Their report, “Chimeric Antigen Receptor T-Cell Therapy for B- Cell Cancers: Effectiveness and Value,” was conducted at the request of the non-profit Institute for Clinical and Economic Review (ICER), to generate evidence on whether the treatments, already approved by the U.S. Food and Drug Administration (FDA), are cost-effective. Its findings will be the focus of a March public meeting sponsored by ICER.

Two CAR T-cell therapies are now FDA-approved, Yescarta (axicabtagene ciloleucel) for people with advanced large B-cell lymphoma, and Kymriah (tisagenlecleucel) to treat children and young adults with advanced B-cell acute lymphoblastic leukemia. Yescarta is marketed by Kite Pharma and Kymriah by Novartis.

CAR, or chimeric antigen receptor, T-cell treatments involve harvesting a patient’s own T-cells and then genetically engineering them to recognize cancer cells, before returning the engineered to the patient’s body.

The researchers argued in their study that evidence suggests there may be potentially great benefits from these therapies, even though they are costly. Kymriah costs $475,000 per patient, while Yescarta costs $373,000 per patient.

In their report, the researchers compared CAR-T therapies to chemotherapy, taking into account patient survival, quality of life, and healthcare costs, from the healthcare system’s point of view, over the lifetime of a patient receiving these therapies.

“We take into account the clinical evidence, quality of life data, and health system costs to generate cost-effectiveness evidence,” said Whittington in a press release.

The cost-effectiveness findings for both CAR t-cell therapies were “promising and suggested that they may be a good use of our healthcare resources toward improving health,” Campbell said. Both therapies significantly extended the lives of some patients, much more on average than traditional chemotherapy, he noted.

Their cost-effectiveness model showed that patients receiving Kymriah had a total cost of $679,000 but gained 10.34 life-years (LY) and 9.28 quality-adjusted life years (QALYs), compared to 2.43 LYs and 2.10 QALYs among those receiving chemotherapy, whose cost was put at $269,000 per patient. QALY is a measure of the years a patient lives in good health.

Yescarta had a total cost of $625,000, and patients gained on average 7.57 LYs and 6.06 QALYs. Chemotherapy was priced for this group at $105,000 and associated with 3.23 LYs and 2.48 QALYs gained.

“The CAR-T science is beyond whether the therapies work for certain patients and is now questioning its value,” Campbell said. “CAR-T is promising on the clinical side but there is some feeling of sticker shock related to the price. Is it worth it? Yes, it seems to be.”

Measures of cost-effectiveness is important in U.S. healthcare, McQueen said, because “insurance companies have a higher likelihood of providing access and payment for therapies that are considered good value for money.”

The draft evidence report is open to public comment through 5 p.m. on Jan. 24, 2018, as are draft voting questions, ICER announced in a release. Findings of the report are preliminary and may be subject to change based on public comment and further analysis.

The public is invited to submit comments by email to Guidelines for submission are available on ICER’s website.

ICER’s Manufacturer Engagement Guide and Patient Participation Guide provide additional information on what types of comments might be most informative to the report. Comments will be reviewed and possibly incorporated into an evidence report to be posted on its website on or around Feb. 15.

That February report will be the topic of a public meeting of the California Technology Assessment Forum set for March 2, where an independent council will vote on key questions raised in the report, and an expert policy roundtable will discuss recommendations to apply to policy and practice. That meeting will take place in Oakland, California.

Requests to submit oral comments at the March meeting must be made by Jan. 24. Registration is required to both attend the meeting in person, or to watch the live webcast.

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