Oncology
Chronic Lymphocytic Leukemia
The Financial Burden of First-line Targeted Therapy in Chronic Lymphocytic Leukemia
Over the past decade, we have seen a tremendous change in how we treat patients with CLL. Ten to 15 years ago, the standard of first-line treatment was chemoimmunotherapy for 6 cycles, such as fludarabine, cyclophosphamide, and rituximab (the FCR regimen) or bendamustine with rituximab. After chemoimmunotherapy, we would just watch and wait. Patients had a high chance of relapse, and when they did, we would treat them again.
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In 2014, ibrutinib, the first targeted oral therapy for CLL, was approved by the US Food and Drug Administration (FDA). Since then, other oral therapies have become available, including new generations of BTK inhibitors such as acalabrutinib and zanubrutinib. We also have venetoclax, a Bcl-2 inhibitor, and are routinely using anti-CD20 antibodies. The paradigm has shifted from chemoimmunotherapy to targeted therapies. Targeted therapies are improving both progression-free and overall survival compared with chemoimmunotherapy.
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Targeted therapies are expensive compared with historic chemoimmunotherapies. Increased use adds to the treatment costs for patients with CLL, especially because several of the current regimens are continuous oral daily therapies. Patients stay on treatment for as long as it works and they are not experiencing significant side effects—sometimes a daily drug for 5, 7, or 10 or more years. This incurs a financial burden. However, over the past few years, time-limited treatment regimens have been developed in which patients receive treatment for only 1 to 2 years. Time-limited regimens may be more cost-effective than continuous daily therapy even though we may be using 2 or 3 drugs together.
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There have been several published articles looking at the costs of CLL therapies. I was involved in one of the early articles on this topic back in 2017 when the oral agents were first becoming available. There are now many other articles that have examined the costs of CLL therapies.
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Overall, I think that we will find that 1 to 2 years of targeted, time-limited therapy will be the way to go for patients with CLL. I believe that it will ultimately be more cost-effective, and the efficacy data look very, very promising.
Allen JM, Carroll K, Gurfinkel D, et al. Financial toxicity, health-related quality of life, and medication adherence patient-reported outcomes (PROs) in patients with chronic lymphocytic leukemia (CLL) on first-line oral oncolytics. Blood. 2024;144(suppl 1):2354. doi:10.1182/blood-2024-198915
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Chen Q, Jain N, Ayer T, et al. Economic burden of chronic lymphocytic leukemia in the era of oral targeted therapies in the United States. J Clin Oncol. 2017;35(2):166-174. doi:10.1200/JCO.2016.68.2856
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Huntington SF, Manzoor BS, Jawaid D, et al. Real-world comparison of health care costs of venetoclax-obinutuzumab vs Bruton’s tyrosine kinase inhibitor use among US Medicare beneficiaries with chronic lymphocytic leukemia in the frontline setting. J Manag Care Spec Pharm. 2024;30(10):1106-1116. doi:10.18553/jmcp.2024.24049
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Patel KK, Isufi I, Kothari S, Davidoff AJ, Gross CP, Huntington SF. Cost-effectiveness of first-line vs third-line ibrutinib in patients with untreated chronic lymphocytic leukemia. Blood. 2020;136(17):1946-1955. doi:10.1182/blood.2020004922
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Waweru C, Kaur S, Sharma S, Mishra N. Health-related quality of life and economic burden of chronic lymphocytic leukemia in the era of novel targeted agents. Curr Med Res Opin. 2020;36(9):1481-1495. doi:10.1080/03007995.2020.1784120



