The Lynx Group

Balancing Cost and Cost-Effectiveness in Multiple Myeloma Therapy

Faculty Perspectives: The Evolving Clinical and Pharmacoeconomic Impact of Isatuximab in Multiple Myeloma Management
Ryan Haumschild, PharmD, MS, MBA
Director of Pharmacy
Winship Cancer Institute
Atlanta, GA

As providers and payers, we recognize that the costs of treating multiple myeloma are some of the highest among all disease states. This high cost is inherent to the disease because it is a plasma-cell disorder where patients continue to progress to subsequent lines of therapy as there is currently no cure. As treatments improve in their effectiveness, we utilize triplet and quadruplet medication combinations in the frontline setting. These combinations include medications that are branded and drive high costs. It is essential as providers that we manage these costs appropriately and look to achieve optimal progression-free survival with cost-effectiveness.

As these major advances in multiple myeloma treatment continue to occur, we must select cost-effective therapies that still demonstrate efficacy and safety. As patients progress on further lines of therapy, they not only incur the cost of new treatments, but they also experience a higher total cost of care. This means that patients will require more symptom management, increased healthcare resource utilization, and greater utilization of urgent care or emergency department services. It is important that providers select therapies in later lines that can be well-tolerated and continue to provide durable responses.

As a healthcare provider, I am interested in evaluating the cost of a specific therapy, but in multiple myeloma I am more concerned about the cost of the regimen and the total cost of care. I want to understand the cost impact of a new therapeutic regimen and ultimately how this will impact the patient’s quality of life. A great way of evaluating this impact in totality is looking at the quality-adjusted life-years (QALYs). This allows us to recognize how costs intersect with activities of daily living. This new way of evaluating pharmacoeconomics takes into consideration not just treatment cost but total cost over the continuum of the patient journey whether it be second-, third-, or fourth-line therapy and beyond.

As patients progress to the relapsed and refractory setting by adding additional agents such as daratumumab and isatuximab, we may increase initial acquisition cost, but may provide prolonged efficacy. This means better QALYs and improved efficacy with longer response rates. This challenges the superficial perception that quadruplet medications will only increase costs and solidifies this therapeutic approach as a cost-effective option. Although both therapies are important, we see clinically that isatuximab plus pomalidomide plus dexamethasone is associated with incremental survival gains of approximately 1 month and quality-adjusted survival gains of 0.5 months compared with daratumumab plus pomalidomide plus dexamethasone when patients are treated for 1 year. The benefits increase with treatment duration to reach approximately 7-month life-year gains and 4-month QALY gains if patients are treated for 5 years. Due to its lower total costs, an isatuximab-based regimen yielded decreasing incremental cost-effectiveness ratios (ICERs) at 1 and 3 years. However, the isatuximab plus pomalidomide plus dexamethasone cost exceeded daratumumab plus pomalidomide plus dexamethasone at the 5-year time horizon to yield an ICER above the willingness-to-pay threshold.1

Looking to the future, we must incorporate CD38-directed agents into therapy to ensure strong responses and aim for low minimal residual disease (MRD). By establishing MRD, we may allow the patient to have the potential for drug-free treatment periods. This would reduce the overall drug expense and allow patients to improve their activities of daily living. The emerging areas of treatment, which include cellular therapy and bispecific agents, will introduce strong overall response rates, but also significant costs. We must continue to review and demonstrate cost-effectiveness among treatment strategies. Leveraging patient-reported outcomes, QALYs and ICERs will provide statistically significant platforms to demonstrate value. The combination of clinical efficacy, safety, and cost will be the lens providers must consider as they select and prioritize therapies across patient types.


  1. Alrawashdh N, Almutairi A, McBride A, Abraham I. Economic evaluation of daratumumab and pomalidomide and dexamethasone versus isatuximab and pomalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma. Presented at: 62nd ASH Annual Meeting and Exposition; December 5-8, 2020. Abstract 3419.

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