Red Stethoscope with a Pile of Hundred Dollar Bills.

A study published online in Blood Advances, a Journal of the American Society of Hematology (ASH) shows that when choosing their preferred treatment, patients with Chronic Lymphocytic Leukemia (CLL), the most prevalent type of leukemia, place the highest value on medicines that deliver the longest progression-free survival. However, the study results also suggest that patients are willing to swap some drug efficacy for a reduced risk of serious adverse events.

The researchers also found that factoring out-of-pocket costs into their decision-making process may significantly influence a patient?s choice of treatment.

Cost of treatment is a factor
Chronic lymphocytic leukemia affects approximately 130,000 people in the United States, and more than 20,000 new cases are diagnosed each year. As more treatment options have been approved in recent years, each with its own associated benefits, side effects, and price tag, patients are forced to weigh many factors when determining their treatment plan.

To gain insight into how patients with CLL balance these differences, lead study author Carol Mansfield, PhD, of RTI Health Solutions, collaborated with the Leukemia & Lymphoma Society, the Lymphoma Research Foundation, and Genentech to conduct a survey among 384 people with CLL.

In this discrete-choice-experiment (DCE), patients were asked to choose between hypothetical treatment options, each of which was defined by five variable attributes, including:

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  • Progression-free survival (PFS),
  • Mode of administration,
  • Typical severity of diarrhea,
  • Chance of serious infection, and
  • Chance of organ damage.

?While every patient wants the most effective drug with the fewest side effects, most people don?t have that option available,? Mansfield said.

?By asking patients to make tradeoffs and rank their preference, we can form an understanding of how patients approach their treatment,? she added

Effectiveness most important
The results of the study show that effectiveness of the drug was the most important factor for participants. Avoiding serious adverse events was also highly valued. On average, a gain in 36 months of Progression-Free Survival or PFS was needed for patients to accept a 30% risk of serious infection. Least important to respondents was the mode of administration. The researchers also noted that patients were only willing to trade a few months of efficacy for a more convenient dosing method such as oral administration.

Unique circumstances
“This research brings home the point that each patient has unique circumstances and choosing the right treatment means weighing the different efficacy profiles and side effects against the patient?s priorities,? Mansfield explained.

?A successful outcome can be different from one individual to the next, and the outcome they desire depends on their circumstances,”she further noted.

Cost analysis
When the researchers conducted a supplemental cost analysis, they found that out-of-pocket cost had a dramatic impact on the choices respondents made. In a follow-up question giving participants the choice between two medicines with different costs, up to 65% of respondents changed their choice of medicine.

?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,? Mansfield observed.

?Cost is clearly something that has an impact. When patients get prescribed something they can?t afford, they have to make very difficult choices,” she said.

Ultimately, findings from discrete-choice experiments like this one 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. 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,? Mansfield concluded.

Overall, this relatively large-scale study among patients with CLL?is, according to the study’s authors, expected to fill a knowledge gap on patient preferences.

Last Editorial Review: November 21, 2017

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