Report: CAR-T for multiple myeloma clinically beneficial, but value low at current cost
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An independent panel unanimously agreed that chimeric antigen receptor T-cell therapy provides clinical benefit superior to the current standard of care for patients with relapsed or refractory multiple myeloma.
Nevertheless, the panel concluded that two therapies it reviewed represented low long-term value at current prices. These included idecabtagene vicleucel (Abecma; Bristol Myers Squibb, bluebird bio), the only commercially available CAR T-cell therapy, which would require a discount of 37% to 54% to meet a recommended health-benefit price benchmark, according to a final report by the Institute for Clinical and Economic Review.
The evidence report, finalized after a virtual public meeting April 11, 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 and ciltacabtagene autoleucel (JNJ-4528; Janssen, Legend Biotech Corp.) — and the antibody-drug conjugate belantamab mafodotin-blmf (Blenrep, GlaxoSmithKline) with current treatment options for this patient population.
“Many people with multiple myeloma develop resistance to existing treatments, so these three new therapies with new mechanisms of action represent a very important expansion of the clinical options available to patients and oncologists,” Steven D. Pearson, MD, MSc, president of ICER, said in a press release. “Data are extremely limited at this time for the two CAR-Ts, and important evidence gaps remain to be filled, but having a new innovative approach become available for patients with multiple myeloma is something to celebrate.”
The Midwest Comparative Effectiveness Public Advisory Council voted 15-0 that adequate evidence existed to show idecabtagene vicleucel, also known as ide-cel, provided a net clinical benefit over standard-of-care treatments for relapsed or refractory multiple myeloma. A majority (13-2) of the panelists agreed that ciltacabtagene autoleucel, or cilta-cel, provided the same benefit over currently available standard treatments.
The panel voted unanimously that adequate evidence did not exist to determine which of the two CAR T-cell therapies provided more of a clinical benefit when compared head-to-head.
In a 10-5 vote, panelists concluded that evidence also was lacking to demonstrate a net health benefit of belantamab when compared with standard-of-care treatments.
The panel voted on long-term value only for belantamab and ide-cel because both have been approved for commercial use and have established prices.
Nine panelists voted that ide-cel, when compared with standard care, provided low long-term value at its current price. Five panelists voted that it provided intermediate long-term value and none voted that it provided high long-term value.
The voting was similar for belantamab, with eight panelists voting that it provided low long-term value at current pricing, six citing intermediate long-term value and one panelist voting that it provided high long-term value.
A majority of the panel agreed that both CAR T-cell therapies had a negative effect on reducing heath care inequities, with five panelists saying it would have a major negative effect and nine voting that it would have a minor negative effect.
“The panel voted that the CAR-T therapies could have a minor or major negative effect on reducing health inequities due to these being therapies with high cost and a high side effect burden,” the ICER report stated. “Furthermore, CAR-T therapies require treatment at specialized academic medical centers, which are often utilized at lower rates among historically disadvantaged populations.”
Results of the analysis prompted ICER to issue an Access and Affordability Alert for both CAR T-cell therapies evaluated in the report. It noted that approximately 43% (ide-cel) and 50% (cilta-cel) of eligible patients with relapsed or refractory multiple myeloma could be treated within 5 years before crossing ICER’s budget impact threshold of $819 million per year.
The report concluded that 100% of this patient population could be treated with belantamab within 5 years based on its current wholesale acquisition price.
“Shadowing these new treatments are concerns that the pricing for the first approved CAR-T agent in multiple myeloma exceeds a reasonable level for its given benefit,” Pearson said in the statement. “Manufacturers should restrain their pricing and work with payers to ensure that payment mechanisms and overall benefit coverage can help patients from all walks of life get affordable access to these treatments.”
References:
Institute for Clinical and Economic Review. ICER publishes final evidence report and policy recommendations on therapies for multiple myeloma [press release]. May 11, 2021. Available at: icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-and-policy-recommendations-on-therapies-for-multiple-myeloma. Accessed May 13, 2021.
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; final evidence report. May 11, 2021.