WASHINGTON,
Nov. 21, 2017
/PRNewswire/ -- When choosing their preferred treatment,
people with chronic lymphocytic leukemia (CLL) place the highest
value on medicines that deliver the longest progression-free
survival, but are willing to swap some drug efficacy for a reduced
risk of serious adverse events according to a study
published online in Blood
Advances, a Journal of the American
Society of Hematology (ASH). The study also suggests
that factoring out-of-pocket costs into this decision-making
process may significantly influence a patient's choice of
treatment.
The most prevalent form of leukemia, CLL 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, patients were asked to
choose between hypothetical treatment options, each of which was
defined by five variable attributes — progression-free survival,
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," said Dr. Mansfield. "By asking patients to make
tradeoffs and rank their preference, we can form an understanding
of how patients approach their treatment."
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 was needed for patients
to accept a 30 percent risk of serious infection. Least important
to respondents was the mode of administration: patients were only
willing to trade a few months of efficacy for a more convenient
dosing method (oral administration).
"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," said Dr. Mansfield. "A successful
outcome can be different from one individual to the next, and the
outcome they desire depends on their circumstances."
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 percent 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," said Dr. Mansfield. "Cost is
clearly something that has an impact. When patients get prescribed
something they can't afford, they have to make very difficult
choices."
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," said
Dr. Mansfield. "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."
Blood Advances
(www.bloodadvances.org), is a
peer-reviewed, online only, open access journal of the American
Society of Hematology (ASH)
(www.hematology.org), the world's
largest professional society concerned with the causes and
treatment of blood disorders.
ASH's mission is to further the understanding,
diagnosis, treatment, and prevention of disorders affecting blood,
bone marrow, and the immunologic, hemostatic, and vascular systems
by promoting research, clinical care, education, training, and
advocacy in hematology.
Blood Advances® is a registered trademark of the
American Society of Hematology.
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SOURCE Blood Advances