ICER Publishes Evidence Report on Therapies for Multiple Myeloma

— The evidence suggests CAR-Ts ide-cel and cilta-cel both have the potential to help patients live substantially longer with manageable side effects, although ide-cel’s recently announced price would still require at least a 37% discount to reach ICER’s recommended health-benefit price benchmark —

— The evidence suggests belantamab appears to be equivalent or slightly superior to alternative three-drug regimens, and it achieves commonly cited thresholds for cost-effectiveness due in part to discontinuation rates and the high prices of the alternative combinations —

— At April 16 virtual meeting, the Midwest Comparative Effectiveness Public Advisory Committee will review the evidence, hear further testimony from stakeholders, and deliberate over these treatments’ comparative clinical effectiveness, other potential benefits, and long-term value for money —

BOSTON, April 5, 2021 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of three new treatments targeting the B-cell maturation antigen (BCMA) for heavily pre-treated patients with triple class refractory multiple myeloma (TCRMM) who have cycled through numerous previous lines of therapy:

  • idecabtagene vicleucel (“ide-cel,” Abecma®, Bristol-Myers Squibb and bluebird bio), a chimeric antigen receptor (CAR) T-cell therapy that the FDA approved in March 2021 to treat TCRMM patients after four or more prior lines of therapy;
  • ciltacabtagene autoleucel (“cilta-cel,” Janssen and Legend Biotech), an investigational CAR T-cell therapy for which a rolling biologic license application (BLA) was submitted to the FDA in December 2020; and
  • belantamab mafodotin-blmf (“belantamab,” Blenrep™, GlaxoSmithKline), which the FDA approved in August 2020 to treat TCRMM patients after four or more prior lines of therapy.

“Many people with multiple myeloma develop resistance to existing treatments, so these three new therapies that target BCMA — a new method of action — represent an important expansion of the clinical armamentarium available to patients and oncologists,” said Steven D. Pearson, MD, MSc, ICER’s President. “Data are extremely limited for the two CAR-Ts, but those data suggest the potential for patients to live longer with manageable side effects. At our public meeting, our policy roundtable will likely discuss the potential for certain patients to require a second dose of these CAR-Ts, and what the implications will be not only for how T-cells should be initially harvested, but also on the treatments’ overall pricing, coverage, and long-term value. Belantamab appears to deliver more modest clinical benefit, along with some safety concerns that have required the drug to be stopped or reduced in certain instances. At its current pricing, belantamab appears to meet commonly cited thresholds for cost-effectiveness, but this favorable result is due in part to early discontinuation among many patients, as well as the high prices of the three-drug regimens to which belantamab was compared.”   

This Evidence Report will be reviewed at a virtual public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) on April 16, 2021. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

While the data are limited and toxicities are common, both ide-cel and cilta-cel appear to deliver relatively sizeable gains in both progression-free survival and overall survival for TCRMM patients exposed to three or more prior lines of treatment, with higher rates of response and longer survival than treatment with current therapies. Based on this evidence, ICER determined that there is high certainty that both therapies provide at least a small net health benefit compared to usual care, with the possibility of a substantial benefit (“B+”). The evidence remains insufficient (“I”) to compare ide-cel and cilta-cel to each other.

Belantamab appears to be equivalent or slightly superior to current treatments for TCRMM patients exposed to four or more prior lines of treatment. However, visual disturbances and other toxicities are also common with belantamab, requiring dose reduction or discontinuation in some circumstances. ICER rated the current evidence promising but inconclusive (“P/I”), as the balance of potential benefits and risks did not rule out a small possibility of overall net harm.

Key Cost-Effectiveness Findings

ICER’s recommended health-benefit price benchmark (HBPB) range for ide-cel is between $192,000-$265,000 per dose, which would require a 37-54% discount off the treatment’s recently announced wholesale acquisition cost (WAC) of $419,500. ICER’s HBPB assumes that there would be a second charge for individuals requiring retreatment; if a different pricing model were to be adopted, ICER’s HBPB for ide-cel would change.

ICER’s HBPB for cilta-cel is between $317,000-$475,000, with the assumption that a second dose will require payment. These results should be considered preliminary, as the evidence base is extremely limited and unpublished. Cilta-cel’s manufacturers have not yet announced an estimated or actual price for the therapy.

ICER’s HBPB for belantamab is between $8,300-$9,500 per vial, which would require no discount off the therapy’s WAC of $8,277 per vial. Belantamab is dosed based on a patient’s weight, and the manufacturer has projected that the treatment’s monthly cost will average $23,900 per patient; however, the monthly cost may be above $32,000 for those requiring three vials. The cost-effectiveness results for belantamab are relatively favorable due in part to patients discontinuing therapy because of side effects, as well as the high prices for the three-drug alternatives.

The HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.

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