CAR-T provides superior efficacy for multiple myeloma at higher cost, report states
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Chimeric antigen receptor T-cell therapies demonstrated greater clinical effectiveness than standard care among patients with heavily pretreated multiple myeloma, according to a report by the Institute for Clinical and Economic Review.
However, despite the superior net health benefits they confer, the CAR T-cell therapies would require steep discounts from their current list prices to meet the nonprofit research institute’s recommended health-benefit price benchmark.
“[Because] anti-[B-cell maturation antigen] treatments represent a novel mechanism of action, these treatments may provide efficacy for patients who currently have few alternatives,” the report stated. “However, CAR-T therapies are complex and high-cost, with significant side effects. Treatments with these characteristics have been underutilized by disadvantaged populations, suggesting that disparities may be worsened.”
The evidence report assessed the comparative clinical effectiveness and value of B-cell maturation antigen (BCMA)-directed therapies for heavily pretreated multiple myeloma.
The institute compared two CAR T-cell therapies — idecabtagene vicleucel (Abecma; Bristol Myers Squibb, bluebird bio) and ciltacabtagene autoleucel (JNJ-4528; Janssen, Legend Biotech Corp.) — in addition to the antibody-drug conjugate belantamab mafodotin-blmf (Blenrep, GlaxoSmithKline) with current treatment options for this patient population.
Ciltacabtagene autoleucel, also known as cilta-cel, is an investigational therapy available only through clinical trials. Idecabtagene vicleucel, also known as ide-cel, received FDA approval in March for commercial use among adults with relapsed or refractory multiple myeloma.
The report concluded that both CAR T-cell therapies provided superior clinical benefits in terms of OS and PFS when compared with current therapies for patients with triple- or penta-refractory multiple myeloma. Both therapies received a B+ grade for level of evidence, which indicates a “high certainty that both therapies provide at least a small net health benefit compared to usual care, with the possibility of a substantial benefit,” according to an ICER-issued press release.
Belantamab mafodotin-blmf appeared to be “equivalent or slightly” superior to currently available standard therapies in terms of clinical effectiveness, according to the report. Questions about treatment-related toxicities and the frequency of treatment discontinuation for belantamab mafodotin-blmf led the institute to deem the available evidence “promising but inconclusive, as the balance of potential benefits and risks did not rule out a small possibility of overall net harm,” the release stated.
The report estimated ide-cel would need to be priced between $192,000 and $265,000 per dose to meet its recommended health-benefit price benchmark. This would require a discount of 37% to 54% off the current wholesale acquisition cost of $419,500, according to the analysis.
Additionally, cilta-cel would need to be priced around $300,000 per dose to meet the institute’s $100,000 per quality-adjusted life-year threshold.
The price of belantamab mafodotin-blmf was considered “within commonly cited cost-effectiveness thresholds” for patients with heavily pretreated and refractory multiple myeloma, the report stated. However, investigators noted uncertainties about “the PFS-OS relationship and other parameters in the belantamab model,” suggesting small changes to the model could result in a significant change in the cost-effectiveness findings.
The evidence report is the penultimate step in the organization’s assessment of the three treatments. The institute will consider the evidence report as part of a virtual public meeting April 16, when an independent council will vote on key questions raised in the report. Registration for the virtual meeting is still open.
The institute will issue its final evidence report and policy recommendations May 11.
Reference:
Lee SJ, et al. Institute for Clinical and Economic Review. Anti B-cell maturation antigen CAR T-cell and antibody drug conjugate therapy for heavily pre-treated relapsed and refractory multiple myeloma; evidence report. April 5, 2021. Available at: 34eyj51jerf417itp82ufdoe-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/ICER_Multiple-Myeloma_Evidence-Report_040521-1.pdf.
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