While every patient wants the most effective drug with the fewest side effects, most people don’t have that option available. By asking patients to make trade-offs and rank their preference, we can form an understanding of how patients approach their treatment
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Treatment efficacy versus quality of life - would you make the trade-off?
Research, published by the American Society of Hematology (ASH) in their journal Blood Advances, indicates that patients in the United States are more likely to choose a treatment which has reduced side effects than a treatment with a stronger chance of efficacy, and that before both these factors some patients rank the cheapest treatment as their preferred option.
Patients with the most prevalent type of leukaemia, Chronic Lymphocytic Leukaemia (CLL), are often opting for a treatment path which not the most effective because of a trade-off with the chances of a better quality of life. An estimated 130,000 individuals in the United States are affected by CLL, with a further 20,000 new cases being diagnosed each year.
The cancer therapy ranked highest by these patients often has a reduced efficacy but also reduced risk of serious adverse events. The study also highlights that efficacy of treatment and reduced side effects are factors which can also rank below the factor of budget for some CLL sufferers.
384 patients with CLL were given hypothetical treatment option to select with five variable attributes:
- Progression-free survival
- Mode of administration
- Typical severity of diarrhoea
- Chance of serious infection
- Chance of organ damage
The study showed that patients valued treatment efficacy most highly, with reduced risk of side effects also highly valued. A 30% risk of serious infection was only accepted (on average) with a 36 month gain of progression-free survival. Patients rated the mode of administration as the least important factor in their decision-making, with patients willing to trade only a few months of efficacy for a therapy option with 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. A successful outcome can be different from one individual to the next, and the outcome they desire depends on their circumstances
When the study brought in a supplemental cost analysis, up to 65% of respondents altered their therapy preferences based on the price of the treatment.
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. Cost is clearly something that has an impact. When patients get prescribed something they can’t afford, they have to make very difficult choices
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