Research from ASH ’ s online peer-reviewed journal , Blood Advances in a Different Vein
Patients With CLL Willing to Trade Treatment Efficacy for Reduced Costs and Adverse Events
Some patients living with chronic lymphocytic leukemia ( CLL ) are willing to accept drugs that are less effective in exchange for reduced adverse events ( AEs ) when making treatment decisions , according to results from a discrete-choice experiment published in Blood Advances . Findings from the study , which surveyed patients about treatment preferences , also suggest that out-of-pocket costs can override both safety and efficacy .
Although participants placed the greatest importance on longer progression-free survival ( PFS ), “ any increase in AEs or the risk of AEs would require a potentially large improvement in efficacy to offset the disutility to respondents associated with the AEs ,” noted Carol Mansfield , PhD , of RTI Health Solutions in Research Triangle Park , North Carolina , and co-authors .
The researchers , along with representatives from The Leukemia & Lymphoma Society ( LLS ), Lymphoma Research Foundation , and Genentech , designed a patient-preference survey that presented participants with a choice between pairs of hypothetical CLL treatments . Each hypothetical treatment was defined by five attributes : PFS , mode of administration , typical severity of diarrhea , chance of serious infection , and chance of organ damage . Patients were asked to rank the importance of each attribute in their treatment choice .
Respondents were recruited between March and April 2016 from the LLS database of 4,420 adult patients with a self-reported physician diagnosis of CLL . They were offered a $ 20 incentive for survey completion .
A total of 384 patients ( median age = 64.6 years ; range = 30-89 ayears ) completed the study ; 53 percent were women and 94 percent were white . Most patients ( 69 %) had a form of public insurance ( including Medicare , Medicaid , and / or Veterans Health Insurance ), and 48 percent were retired .
Respondents ranked change in PFS ( from 10 to 60 months ) as the most important attribute in their treatment decisions , followed by ( in order of importance ):
• a decreased risk of infection ( preferring a medication with no risk of infection over one with a 30 % risk )
• a lower chance of organ damage ( preferring a medication with no risk over one with an 8 % risk )
• a lower grade of diarrhea ( preferring one with no risk over one with a severe risk )
• mode of administration ( preferring oral over intravenous routes )
Although the risk of AEs was important , results from the minimum acceptable benefit calculation showed that , on average , patients would choose a treatment that carried a 30 percent risk of serious infection if it extended PFS by 36 months .
Prior exposure to treatment did not appear to influence decisions , according to a subgroup analysis in which the researchers compared patients who were receiving firstline therapy ( n = 23 ), had relapsed / refractory disease ( n = 39 ), or were treatment naïve ( n = 20 ) at the time they completed the survey . “ There were no statistically significant differences between the preferences of any subgroup and the full sample ,” the authors wrote . “ This suggests that patient preferences may be stable over the stages of the disease ; however , it is possible that sample size was not large enough to estimate the difference in preferences across groups .”
More than half ( 53 %) of the entire group reported receiving financial aid from patient-support programs and 40 percent reported difficulty paying out-of-pocket costs , indicating that cost played a major role in treatment decisions .
When participants were allowed to choose between a medication with a shorter PFS ( Medicine A ) or a longer PFS ( Medicine B ) without knowing the cost , 91 percent chose Medicine B ; however , when they were presented with scenarios in which the monthly out-of-pocket costs for Medicine B were $ 75 higher than Medicine A , 50 percent of patients chose the lower-cost option . As price increased , so did the number of patients choosing lower cost over improved efficacy ( see FIGURE ). “ Cost is clearly something that has an impact ,” said Dr . Mansfield . “ When patients [ are ] prescribed something they can ’ t afford , they have to make very difficult choices .”
Given the value of testing minimal residual disease ( MRD ) to guide treatment decisions for CLL , the authors also asked respondents about MRD monitoring . Approximately 82 percent were interested in the testing , of which 58 percent were very interested . Interest in MRD testing decreased with age , was lower in women , and was higher in those with public insurance .
“ 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 ,” Dr . Mansfield concluded .
The study is limited by its use of a patient advocacy organization for recruitment , which may not be representative of the overall CLL population . In addition , discrete-choice experiments rely on hypothetical scenarios and only a limited number of treatment attributes can be included . “ The answers do not carry the weight of real choices ,” the authors explained . Disease stage and prior treatment regimens were not defined , which could have further limited the implications of the study ’ s findings .
The authors report receiving funding from Genentech , Inc ., which provided funding support for this study .
REFERENCE
Mansfield C , Masaquel A , Sutphin J , et al . Patients ’ priorities in selecting chronic lymphocytic leukemia treatments . Blood Advances . 2017 November 14 .
FIGURE . Impact of Cost on Medicine Choice *
Respondents who preferred medicine (%)
100 90 80 70 60 50 40 30 20 10 0
9 %
91 %
Exclude cost considerations
50 % 50 %
Include cost difference of $ 75 / month more for Medicine B
74 %
Medicine A Medicine B
26 %
Include cost difference of $ 400 / month more for Medicine B
* The bars indicate the percentage of the sample that selected medicine A or B when cost was or was not considered in the prediction model .
36 ASH Clinical News January 2018