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Targeted Leukemia Drugs Sharply Raising Costs of Care

Jagpreet Chhatwal

Jagpreet Chhatwal (Harvard University)

22 November 2016. Drugs directly attacking cancer cell growth show high remission rates with a common form of leukemia, but a new study suggests the high costs of these drugs could impose difficult economic burdens on patients and the health care system. The analysis led by Jagpreet Chhatwal, a radiologist at Massachusetts General Hospital and Harvard Medical School in Boston, appears in yesterday’s (21 November) issue of the Journal of Clinical Oncology.

Chhatwal, a decision scientist in Mass. General’s Institute for Technology Assessment, applies tools of operations research, such as computer modeling and statistical analysis, to clinical decision-making and health care policies. In this study Chhatwal and colleagues analyze the costs of new small-molecule drugs designed to treat chronic lymphocytic leukemia that block protein signals supporting cancer cell growth.

Chronic lymphocytic leukemia, or CLL, is a disorder of the blood and bone marrow, where the bone marrow makes too many blood stem cells that fail to mature into healthy white blood cells. The overabundance of these leukemia cells crowd out the healthy blood cells, including other white blood cells that support the immune system as well as platelets and red blood cells. CLL is the most common type of leukemia in adults.

Earlier in 2016, Food and Drug Administration approved two small-molecule drugs — the kind that can be formulated into tablets or capsules — as treatments for CLL: ibrutinib as a first-line therapy, marketed as Imbruvica by Pharmacyclics and Janssen Biotech, and venetoclax, marketed as Venclexta by Genentech for relapsed or unmanageable CLL. Ibrutinib was originally approved in 2014 as a treatment for a specific type of CLL.

Also in 2014, FDA approved a combination of idelalisib, marketed as Zydelig by Gilead Sciences and rituximab, marketed as Rituxan by Genentech and Biogen, for relapsed and unmanageable CLL. (In March 2016, Gilead Sciences stopped 6 clinical trials testing idelalisib in combination with other drugs due to high rates of serious adverse effects.)

While these newer targeted drugs achieve higher remission rates and extended survival times, they come at a high financial price tag. The cost for one year of ibrutinib and idelalisib treatments, for example, is $130,000 and can continue for many years. In contrast, chemo-immunotherapies, the previous standard of care for CLL, range in cost from $60,000 to $100,000 per year, and the course of treatments last about 6 months at a time.

The researchers sought to better understand the economic implications of these new drugs for individuals and the U.S. health care system that pays for a large part of the drugs’ costs. To make these projections, the team developed a computer model that covered factors affecting use of the new drugs: patient characteristics and clinical course of treating patients, including different stages of therapy for CLL, based on outcomes of clinical trials. The researchers validated their model against published tables with survival and mortality data.

The model shows the number of people living with CLL in 2025 to increase, but the costs of the new drugs over this period to rise even faster. The authors project the number of people in the U.S. living with CLL to increase by 55 percent from 2011 to 199,000 by 2025. In that time, however the total annual cost of drugs to treat CLL is expected to rise nearly 7 times, from $740 million in 2011 to $5.13 billion in 2025.

As the new targeted therapies become the first-line standard of care, the lifetime cost per patient for drugs is projected to more than quadruple from $147,000 to $604,000. Out-of-pocket costs of these drugs for Medicare patients is expected to jump more than 6 times from $9,200 to $57,000.

The researchers applied a standard measure of economic valuation in health care known as quality-adjusted life year, an index that runs from 0.0 (death) to 1.0 (excellent health). The index considers number of years of life, as well as factors such as mobility, pain, and ability to carry on usual activities. The authors then calculated incremental cost-effectiveness ratios per quality-adjusted life year, for the newer targeted drugs and previous chemo-immunotherapies, converted to standard 2015 dollars.

The team calculated an incremental cost-effectiveness ratio for the newer targeted rugs of of $189,000 per quality-adjusted life-year, compared to chemo-immunotherapies, which far exceeds the benchmark willingness to pay threshold of $100,000 per quality-adjusted life-year. The authors urge more efforts to secure discounts of 50 to 70 percent for the new drugs rather than abandoning these promising treatments. Chhatwal notes in a Mass. General statement that, “high out-of-pocket costs for cancer care not only cause financial hardships for patients but also reduce their quality of life through anxiety, stress and worry about paying those large bills. Lower-income patients may be unable to afford these therapies at all.”

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