The chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel may be a cost-effective second-line treatment option in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), according to a recent study published by Choe et al in Blood Advances.
Background
In cases where DLBCL—the most prevalent form of non-Hodgkin lymphoma affecting the lymphocytes—does not respond to initial therapy or recurs within 12 months posttreatment, the standard-care protocol typically includes platinum-based chemotherapy followed by high-dose chemotherapy and autologous stem cell transplantation.
In 2022, the U.S. Food and Drug Administration granted approval to lisocabtagene maraleucel as a second-line treatment for patients with DLBCL. However, the cost effectiveness of lisocabtagene maraleucel is currently a topic of debate among oncologists—especially considering the drug’s cost increased from $410,300 to $447,227 between 2022 and 2023.
"In our study, we incorporated the often-overlooked societal costs associated with cancer treatment, which are typically neglected in cost-effectiveness analyses that focus solely on the health-care sector–related expenses," explained senior study author Mohamed Abou-el-Enein, MD, PhD, MSPH, Associate Professor of Medicine at the Keck School of Medicine at the University of Southern California. “Cancer treatments can diminish quality of life, causing work absences and challenges in managing everyday activities, especially among … elderly [patients]. Treatments that improve quality of life not only benefit the patients but also reduce these broader societal costs, which is an important aspect of our cost-effectiveness evaluation,” he added.
Study Methods and Results
In the new study, investigators used a partitioned survival model to perform a cost-effectiveness analysis of second-line lisocabtagene maraleucel or standard care in patients with relapsed or refractory DLBCL. The modeling was based on outcomes from patients who participated in the TRANSFORM trial. The investigators also incorporated health-care expenses, societal productivity losses, and patient quality of life in assessing lisocabtagene maraleucel's cost-effectiveness. They noted that the direct medical costs included in the analysis were comprehensive, covering CAR T-cell therapy procedures, chemotherapy, stem cell transplant, hospital admissions, ongoing monitoring, management of disease progression, and end-of-life care—as well as costs associated with treating adverse events. When analyzing societal costs, the investigators considered lost productivity, including lost labor market earnings, the value of unpaid productivity and household activities, and out-of-pocket travel expenses.
The investigators found that the patients who received lisocabtagene maraleucel had an average life expectancy of 5.34 years and gained 3.64 quality-adjusted life-years compared with the 2.47 years and 1.62 quality-adjusted life-years among those who received standard care. The investigators then utilized the incremental cost-effectiveness ratio to evaluate the additional costs required for each quality-adjusted life-year gained, assuming a societal willingness to pay up to $100,000 per quality-adjusted life-year. The cost-effectiveness of lisocabtagene maraleucel was $99,669 per quality-adjusted life-year from a health-care sector perspective and $68,212 per quality-adjusted life-year from a societal perspective—indicating that lisocabtagene maraleucel may be a cost-effective treatment and stay below the $100,000 per quality-adjusted life-year threshold.
Further, lisocabtagene maraleucel was associated with a loss of $110,608 in mortality-related labor earnings, which was lower than the $156,362 associated with standard care. Both unpaid productivity losses and uncompensated household production costs were comparatively lower for lisocabtagene maraleucel. As a result, the investigators suggested that lisocabtagene maraleucel could offer economic benefits by reducing losses in both paid and unpaid labor sectors, compared with the benefits of standard care.
Conclusions
The new findings underlined the critical need to manage lisocabtagene maraleucel's costs effectively to ensure that the drug will be affordable and economically sustainable and to continue the dialogue among clinicians, researchers, pharmaceutical manufacturers, and patients concerning the escalating costs of CAR T-cell therapy.
The investigators stressed that their strict adherence to outcomes from the TRANSFORM trial may have brought about limitations caused by a lack of long-term follow-up data to be incorporated in their cost-effectiveness analysis. Moreover, the scenario analyses conducted— which looked at various time horizons, utility values, and list prices—revealed that maintaining lisocabtagene maraleucel's cost-effectiveness at the $100,000 per quality-adjusted life-year threshold might pose a challenge.
The investigators plan to continue assessing the cost effectiveness of various CAR T-cell therapies. They hope their new findings can help inform decisions about reimbursements and their integration into the health-care system.
“This study demonstrates that CAR T-cell therapy is worth considering as a second-line treatment for [relapsed or refractory] DLBCL,” Dr. Abou-el-Enein underscored. “When deciding on what therapies to use in clinical practice, I think it is important that we do not shy away from these therapies solely because of their list price. We must keep our patients front and center in these decisions and continue to have conversations about how we can lower the price of CAR T-cell therapy and increase patient access to this life-saving treatment,” he concluded.
Disclosure: For full disclosures of the study authors, visit ashpublications.org.
