ORLANDO, FLA “more sustainable pricing” for first-line oral targeted therapies—for both current and future agents—is needed so that all patients with chronic lymphocytic leukemia may benefit from this significant advance, attendees at the 57th American Society of Hematology (ASH) Annual Meeting were told.1

An increasing number of patients are prescribed agents such as ibrutinib and idelalisib, leading to improved survival. “However, their high cost, approaching more than $130,000/year for an indefinite duration of treatment, has raised concerns about their affordability and cost to the society,” said Nitin Jain, MD, of The University of Texas MD Anderson Cancer Center in Houston, TX.

Dr. Jain and colleagues developed a Markov micro-simulation model that represented the population with chronic lymphocytic leukemia and projected the prevalence and total cost of the disease in the United States for each year from 2011 to 2035.

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“Our model was calibrated to the Surveillance, Epidemiology, and End Results (SEER) data and closely predicted the chronic lymphocytic leukemia prevalence in 2011,” Dr. Jain said. “The model included new incidences every year, and considered the individual patient’s aging, disease progression, and treatment with the available therapies in the given year.”

They estimated disease progression from 10 published clinical trials that documented progression-free and overall survival data in the first-line or relapse settings. Treatment was assigned based on fitness status, determined by age, and presence of del(17p).

“Cost estimation included the cost of drug, treatment administration, and management of adverse events,” he said.

The investigators then simulated changes in prevalence and cost of chronic lymphocytic leukemia. From 2014, they assumed oral targeted therapies to be standard of care for relapsed and del(17p) disease and, from 2017 onwards, as first-line treatment. A scenario that assumed chemoimmunotherapy would remain standard of care from 2011 onwards was also modeled.

Results showed the CLL population is projected to increase substantially over the next decade “due to significantly better progression-free survival and overall survival” observed with the oral tyrosine kinase inhibitors compared with chemoimmunotherapy, Dr. Jain said.

Specifically, the use of oral targeted therapies is estimated to result in an additional 172,000 person-years of life. The cumulative cost of treatment for CLL from 2011 to 2025 would be $54.24 billion for the oral targeted therapies and $19.08 billion for chemoimmunotherapy, an increment of $35.16 billion.

The average lifetime cost per person by year of starting first-line therapy is projected to grow from $180,000 in 2011 to $700,000 in 2017.

This “significant economic impact” presents new challenges related to financial toxicity, such as decreased adherence and limited access, he concluded.


1. Jain N, Chen Q, Ayer T, et al. Prevalence and economic burden of chronic lymphocytic leukemia (CLL) in the era of oral targeted therapies. Oral presentation at: 57th American Society of Hematology (ASH) Annual Meeting & Exposition; December 7, 2015; Orlando, FL.