Economic Burden of Chronic Lymphocytic Leukemia in the Era of Oral Targeted Therapies in the United States

Abstract

Purpose Oral targeted therapies represent a significant advance for the treatment of patients with chronic lymphocytic leukemia (CLL); however, their high cost has raised concerns about affordability and the economic impact on society. Our objective was to project the future prevalence and cost burden of CLL in the era of oral targeted therapies in the United States. Methods We developed a simulation model that evaluated the evolving management of CLL from 2011 to 2025: chemoimmunotherapy (CIT) as the standard of care before 2014, oral targeted therapies for patients with del(17p) and relapsed CLL from 2014, and for first-line treatment from 2016 onward. A comparator scenario also was simulated where CIT remained the standard of care throughout. Disease progression and survival parameters for each therapy were based on published clinical trials. Results 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 (55% increase) due to improved survival; meanwhile, the annual cost of CLL management will increase from $0.74 billion to $5.13 billion (590% increase). The per-patient lifetime cost of CLL treatment will increase from $147,000 to $604,000 (310% increase) as oral targeted therapies become the first-line treatment. For patients enrolled in Medicare, the corresponding total out-of-pocket cost will increase from $9,200 to $57,000 (520% increase). Compared with the CIT scenario, oral targeted therapies resulted in an incremental cost-effectiveness ratio of $189,000 per quality-adjusted life-year. Conclusion The increased benefit and cost of oral targeted therapies is projected to enhance CLL survivorship but can impose a substantial financial burden on both patients and payers. More sustainable pricing strategies for targeted therapies are needed to avoid financial toxicity to patients.

Fig 1.

Management strategies for patients with chronic lymphocytic leukemia (CLL). (A) The oral targeted therapy scenario with evolving therapeutic options for patients with CLL. (B) The chemoimmunotherapy scenario, which continues to use chemoimmunotherapy as the standard of care. We assumed equal allocation to multiple therapies if more than one therapy is considered available for patients in the same condition. For example, for fit patients in the relapse setting during 2014 to 2016, 50% of patients received ibrutinib and 50% received idelalisib plus rituximab. Moreover, 5% of patients in the relapse setting were assumed to receive hematopoietic stem-cell transplantation (data not shown). BR, bendamustine plus rituximab; Clb, chlorambucil; FCR, fludarabine, cyclophosphamide, and rituximab; GClb, obinutuzumab plus chlorambucil; Idel+R, idelalisib plus rituximab.

Fig 2.

Trend in disease and cost burden of chronic lymphocytic leukemia (CLL) for the chemoimmunotherapy and the oral targeted therapy scenarios. (A) The number of patients with CLL under the chemoimmunotherapy and oral targeted therapy scenarios. The use of oral targeted therapies is projected to increase the number of people living with CLL from 128,000 in 2011 to 199,000 (55% increase) in 2025 due to improved survival with the use of oral targeted therapies. (B) Annual management cost of CLL for the chemoimmunotherapy and the oral targeted therapy scenarios. The use of oral targeted therapies is projected to increase the annual cost in CLL management from $0.74 billion in 2011 to $5.13 billion (593% increase) in 2025, which is mainly driven by high drug prices, prolonged treatment duration of oral agents, and increased number of patients living with CLL.

Fig 3.

Lifetime treatment cost grouped by the year of initiating first-line treatment of the oral targeted therapy scenario. (A) Lifetime treatment cost to payers. (B) Lifetime out-of-pocket cost for Medicare patients.

Fig 4.

Sensitivity analysis of the cost-effectiveness of the oral targeted therapy (OTT) scenario compared with the chemoimmunotherapy (CIT) scenario. (A) Tornado diagram for one-way sensitivity analysis of incremental cost-effectiveness ratio. (B) Cost-effectiveness acceptability curves from the probabilistic sensitivity analysis. HR, hazard ratio; QALY, quality-adjusted life-year.