Do Patient Preferences Align with Value Frameworks? A Discrete-Choice Experiment of Patients with Breast Cancer

The study assessed patient preferences for aspects of breast-cancer treatments to evaluate the usual assumptions in scoring rubrics for value frameworks.

Authors: Hollin L, González J, Buelt L, Ciarametaro M, Dubois R.
Publication: MDM Policy and Practice
June 15, 2020

Purpose:

The study assessed patient preferences for aspects of breast-cancer treatments to evaluate the usual assumptions in scoring rubrics for value frameworks. The goal was to test an assumption in the most commonly-used oncology value frameworks – that patients view treatment outcomes the same way – and to test whether the assumption is close in terms of disease stage and background. 

Researchers noted that current frameworks use formulaic or expert-driven scoring procedures for specific treatment attributes that do not resemble the tradeoffs that patients would be willing to make between these outcomes. As a result, patient preferences do not play a key role in current value assessments tools because:

  • None of the most notable frameworks focused on cancer-related treatments pay much attention to how patients view treatments.
  • These frameworks have mainly focused on finding relevant treatment outcomes and the relevant weights the outcomes should have based on their supporting medical evidence.
  • The frameworks estimate the value of oncology treatments through health benefits, side effects, and improvements in patient symptoms or quality of life in the context of cost. 

Researchers wanted to learn what treatment outcomes matter the most to breast cancer patients to better understand the importance of these outcomes. This information can help doctors and health care providers with treatment decisions because they will have more insight into what treatment options breast cancer patients care about the most.

Methods:

The researchers used a discrete-choice experiment (DCE) to collect numerical data and evidence on patient preferences to accomplish the following: 

  1. Figure out what aspects of treatment patients with breast cancer consider to be the most important.
  2. Assess whether patients with breast cancer view treatment outcomes the same way.
  3. Help improve the way health treatments are rated in future value frameworks.

Each DCE question drew on a patient’s preference for treatments that would receive different scores in a value framework. Choices between options reveal how often patients decide whether to go through with a treatment that they like or do not like. 

Study participants eligibility:

  • Female adults with a self-reported physician diagnosis of stage 3 or stage 4 breast cancer.
  • Diagnosis had to be received at least 1 month before the patient completed the survey to avoid some of the emotional distress expected during a new diagnosis.
  • Patients had to be currently under the care of a physician and receiving treatment.

Results:

  • Better clinical outcomes are associated with treatments that score higher in preference.
    • Treatments that make it possible for patients to live longer are considered most important by patients  
    • Other important attributes were:
      • out-of-pocket cost of treatment
      • the method used to administer a medicine to a patient
      • a test that could show how effective a treatment actually is 
  • Scores for some of the value frameworks are similar to how breast cancer patients score their treatment preferences.
    • the treatment’s effectiveness is very important to breast cancer patients.
    • how much a patient values a treatment changes based on the patient’s cancer stage.
  • Results also show that it is wrong to use a generic scoring system for breast cancer patients, even if their cancer stages are different.
    • Although preferences for breast cancer treatments may differ based on the disease stage, our results show that patients choose only one preferred treatment when it comes to effectiveness. This is important because scores for treatment effectiveness carry the most weight in the rating system used by the American Society of Clinical Oncology (ASCO).
    • Toxicity does not seem to be nearly as important as seen in some of the value frameworks. A 10% increase in major side effects, for example, would cause a significant decrease with the ASCO and the National Comprehensive Cancer Network (NCCN) rating system, but the same changes appeared to barely draw the patents’ attention in the application.

Conclusions:

Researchers found that although value frameworks are an important step in the routine evaluation of medications for a complex treatment plan, frameworks are still largely driven by the views of medical experts and fail to consider how patients view the effectiveness of a treatment. This might make it seem that the benefit of a treatment for a specific patient could be to low or too high. If patients’ treatment outcomes are all thought to be either the same or different from the scores used in frameworks, the use of such tools may not be meaningful or enough for some patients.