Micrograph showing CLL
New research suggests patients with chronic lymphocytic leukemia (CLL) are willing to trade treatment efficacy for a reduced risk of side effects, but the cost of treatment may trump other factors.
The patients studied placed the highest value on treatments that deliver the longest progression-free survival (PFS), but the patients were also willing to swap some efficacy for a reduced risk of serious adverse events (AEs).
Study results also indicated that factoring out-of-pocket costs into the decision-making process can significantly influence a patient’s choice of treatment.
Carol Mansfield, PhD, of RTI Health Solutions in Research Triangle Park, North Carolina, and her colleagues conducted this study and reported the results in Blood Advances. The study was supported by funding from Genentech, Inc., to RTI Health Solutions.
The researchers surveyed 384 patients with CLL. Patients were asked to choose between hypothetical treatment options, each of which was defined by 5 variable attributes—PFS, mode of administration, typical severity of diarrhea, chance of serious infection, and chance of organ damage.
The attribute patients ranked highest was a change in PFS from 10 months to 60 months. This was followed by a change in infection risk from 30% to 0%, a change in the risk of organ damage from 8% to 0%, a change in diarrhea from severe to none, and a change in the mode of administration from intravenous to oral.
On average, a gain in PFS of 35.9 months was needed for patients to accept a 30% risk of serious infection. A gain in PFS of 26.3 months was needed for patients to accept an 8% risk of organ damage.
A gain in PFS of 21.6 months was needed for patients to accept severe diarrhea. And a gain in PFS of 3.5 months was needed for patients to accept the change from a daily pill to intravenous administration for 6 months.
There were no significant differences in preferences among treatment-naïve patients, first-line patients, and relapsed/refractory patients.
Impact of cost
When the researchers conducted a supplemental cost analysis, they found that out-of-pocket cost had a substantial impact on treatment choice.
The cost analysis included 2 treatments—medicines A and B. Based on the prior analysis, the researchers predicted that 91% of patients would choose medicine B if cost were not a concern because B offered longer PFS than A.
“We used the results from the discrete-choice experiment to forecast the probability that a respondent would pick each hypothetical drug without any mention of cost and then compared that to the choices people made when out-of-pocket costs for these medicines were included,” Dr Mansfield explained.
Patients were asked to choose between medicines A and B under 2 circumstances in which B cost more than A.
When medicine B had a monthly out-of-pocket cost that was $75 more than medicine A, 50% of patients chose medicine A.
When medicine B had a monthly out-of-pocket cost that was $400 more than medicine A, 74% of patients chose medicine A.
“Cost is clearly something that has an impact,” Dr Mansfield said. “When patients get prescribed something they can’t afford, they have to make very difficult choices.”
Dr Mansfield and her colleagues believe their findings will help doctors and patients focus on treatments that account for a patient’s unique circumstances and goals.
“Patients don’t always know that they could be making these tradeoffs,” Dr Mansfield said. “We hope that our findings can help doctors to have frank discussions with their patients about the differences between treatments and how these might affect their lives.”
