Multiple myeloma (MM) is a relatively rare hematologic cancer with no curative treatments. New treatments introduced within the past 15 years have led to improvements in survival for patients with MM. 

The Institute for Clinical and Economic Review (ICER) analyzed the effectiveness of new treatments and their costs and impact on quality of life to develop recommendations for stakeholders seeing patients with MM. 

ICER reviewed 3 new treatments for heavily pre-treated patients with relapsed/refractory MM that target B-cell maturation antigen (BCMA): belantamab mafodotin-blmf, idecabtagene vicleucel (ide-cel), and ciltacabtagene autoleucel (cilta-cel). They compared these treatments to usual care, which consists of immunomodulatory agents, proteasome inhibitors, and anti-CD38 monoclonal antibodies.


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Ide-cel and cilta-cel are chimeric antigen receptor (CAR) T-cell therapies for patients with MM who are triple- or quad-refractory. The report found that these therapies likely have a small to substantial net benefit over usual care, including longer survival and improved quality of life.

A review of the evidence suggests belantamab is promising but inconclusive compared to usual care for patients with relapsed or refractory MM.

All therapies carry substantial costs that currently fall outside the cost-effectiveness range. The ICER final report provided recommendations targeted to different audiences that interact with patients with MM.

All stakeholders should ensure that patients with MM have access to treatment equally. African Americans are more likely to have MM and are also at higher risk of not being adequately educated about their disease. Manufacturers, payers, clinicians, and patient organizations should all work to reduce health inequalities for these patients.

Manufacturers should set prices that allow for affordability and access for all patients. Current therapies have a significant cost burden, and drug prices set outside the cost-effectiveness range limit access and cause financial toxicity for patients and families. ICER recommends that prices should be set in accordance with the benefits to patients.

Clinicians and clinical specialty societies should be aware of the costs of treatment and the financial burden it places on patients. Clinicians should take a shared decision-making approach with patients and recommend the less expensive option after discussing side effect profiles and when efficacy is similar.

Payers should determine coverage policy based on FDA labels. Anti-BCMA therapy is still new with uncertainty about effectiveness. Payers should provide clear guidance on the prior authorization process for these therapies. Medicare should evaluate its reimbursement system to provide access to high-cost, novel, one-time therapies.

Future clinical research should focus on when and if patients can stop therapy while in response. Existing studies continue treatment until disease progression. In practice, this leads to exposure to side effects and continuing costs. Public funding will likely be necessary for studies that provide evidence for less drug use. 

Disclosure:  Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.

Reference

Lee SJ, McQueen RB, Beinfeld M, 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. https://icer.org/wp-content/uploads/2020/10/ICER_Multiple-Myeloma_Final-Report_051121.pdf