In late November 2016, Qiushi Chen from the Georgia Institute of Technology, GA, USA, and colleagues published in the Journal of Clinical Oncology, a projection of the economic costs of treating CLL going forwards in the current era of oral targeted therapies, such as ibrutinib.
The authors developed a simulation model that included data from 2011, and projected foreword to 2025. The model assumed that the standard of care from 2011-2014 was chemoimmunotherapy (CIT), from 2014 was oral targeted therapies for patients with del(17p) or relapsed CLL, and from 2016 was oral targeted therapy in the first-line setting. They compared these model predictions with another model, which assumed the standard of care remains the same throughout the period of 2011-2025.
- Incidence of patients with CLL predicted to increase by 55% in the US, by 2025
- Cost changes 2011 vs 2025 (2015 USD):
- Annual cost overall: $740 million vs. $5.13 billion, compared with $1.12 billion if CIT remained the standard
- Average per patient lifetime cost: 2011= $147,000, 2014= $331,000, 2016= $604,000
- Average per patient out-of-pocket cost with Medicare: 2011= $9,200, 2014= $27,000, 2016= $57,000
- Cost-effective analysis:
- Oral targeted therapies provided 107,000 additional person-quality-adjusted-life-years (QALYs) over CIT alone and 149,000 person-life-years (LY)
- Estimated incremental cost-effectiveness ratio of oral targeted therapies was $189,000/QALY and $136,000/LY
- Not all treatment scenarios were analyzed
- Only the recommended standard of care was used in this analysis
- Future drug price fluctuations were not factored into the analysis
- Drug prices would need to be 69% lower than modeled in order for the cost to be classified as cost-effective
- Rising cost may result in disparity of access and treatment choice due to affordability
In discussing the study, the authors stated that oral targeted therapies were a major advance in the treatment of CLL, and that the predicted number of people with CLL will increase as a result of improved survival. However, the cost of these therapies is not deemed cost-effective as the cost exceeds the authors’ willingness to pay threshold of $100,000/QALY.
The authors proposed solution is the reduction of prices charged for oral targeted therapies. Failing that, the authors suggested that further clinical trials designed to optimize treatment regimen and assess whether minimal residual disease could be used as a metric by which discontinuation of therapies could be recommended. The alternative, the authors proposed, may result in increased 'financial toxicity', reduced access and lower regimen adherence, which may undermine the efficacy of this new generation of treatments for CLL patients.
- Chen, Q. et al. Economic Burden of Chronic Lymphocytic Leukemia in the Era of Oral Targeted Therapies in the United States. Journal of Clinical Oncology. Nov 2016. DOI: 10.1200/JCO.2016.68.2856
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.