Oral targeted therapies will increase survival rates dramatically in chronic lymphocytic leukemia (CLL), though they will also substantially increase the cost of CLL management for both patients and payers, according to a study published in the Journal of Clinical Oncology.1

Although the introduction of oral targeted therapies like imatinib, dasatinib, and nilotinib has altered the treatment landscape of CLL and improved survival for patients with CLL, the high cost of these agents raises concerns surrounding affordability and the economic impact on the US health care system. For the present study, researchers estimated the future prevalence and cost burden of CLL in the era of targeted therapies.

To evaluate the evolving management of CLL from 2011 to 2025, researchers developed a simulation model with chemoimmunotherapy as the standard of care before 2014, oral targeted therapies as standard treatment for patients with del(17p) and relapsed CLL from 2014, and as first-line treatment from 2016 onward. For comparison, researchers used a simulation model where chemoimmunotherapy remained the standard of care throughout the 14-year period.

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The number of people living with CLL in the United States is projected to increase from 128,000 in 2011 to 199,000 by 2025, representing a 55% increase, due to prolonged survival as a result of treatment with oral targeted therapies.

The researchers estimated that the annual cost of CLL management will increase from $740 million to $5.13 billion, a 590% increase in annual costs. The per-patient lifetime cost of CLL treatment will increase from $147,000 to $604,000 (a 310% increase) as oral targeted therapies become frontline therapy for CLL.

Out-of-pocket costs will increase 520% from $9200 to $57,000 for patients enrolled in Medicare.

Oral targeted therapies resulted in an incremental cost-effectiveness ratio of $189,000 per quality-adjusted life-year (QALY) compared with the chemoimmunotherapy scenario. Researchers determined that oral therapies at the current price are not cost effective based on a willingness-to-pay threshold of $100,000 per QALY.

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The findings suggest that this implied impact on the health care system could increase the risk for financial toxicity, limited access, and lower adherence to treatment, which may reduce overall clinical effectiveness. The authors concluded that a more sustainable pricing strategy is needed for oral targeted treatments for patients with CLL.


  1. 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. 2016 Nov 21. doi: 10.1200/JCO.2016.68.2856 [Epub ahead of print]