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Reimbursement agencies are increasingly using patient preference data to evaluate health technologies. Discrete choice experiments (DCE) are commonly used to elicit patient preferences, but they require large sample sizes to obtain meaningful results. For this reason, it is often not possible to use DCE to elicit patient preferences in rare diseases. This study assessed a swing weighting method for eliciting preferences from a small sample: patients with immunoglobulin A nephropathy (IgAN) in the United States (US) and China.
Attributes and levels were selected based on a review of clinical studies and qualitative research on patients. Computer-assisted, interview-based swing weighting exercises were piloted in a focus group with five participants each from the US and China. Preferences were then elicited in interviews with twenty-five patients in the US and fifteen patients in China. Consistency tests were used to assess internal validity. Qualitative data were collected on the reasons for patients’ preferences.
Preference consistency: The weights for one attribute were elicited twice. The difference between initial and consistency test weights was not statistically significant (p < 0.1), although this may partly reflect the small sample sizes. Trade-offs: Qualitative data were used to demonstrate the validity of interpreting participants’ ratings as trade-offs. Using the partial value function for end-stage renal disease as an example, qualitative data demonstrated that patients were able to provide face-valid reasons for different shaped, non-linear preference functions. Robustness of treatment evaluation: Three hypothetical treatment profiles (using the attribute swings) were constructed. Preferences for these treatment profiles were robust to variations in patients’ preferences; all patients preferred one specific profile. This finding was not sensitive to changes in weights.
This study supports the feasibility of collecting valid and robust preference data from small groups of patients using swing weighting. Further work could be done to test the performance of swing weighting in larger sample sizes.
Previous qualitative research analyzing social media and online community discussions highlighted the symptomatic burden of cough and mucus (sputum), alongside shortness of breath, in patients with chronic obstructive pulmonary disease (COPD). The objective of this study was to determine the relative importance of these symptoms and their consequences (for example, disturbed sleep) to COPD patients, compared with conventional COPD endpoints (lung function and exacerbations).
A total of 1,050 patients (at least 40 years of age) with moderate to severe COPD or chronic bronchitis, and regular symptoms of cough and excess mucus production, are to be recruited through patient advocacy groups (PAGs) from five countries (Australia, France, Japan, the United Kingdom, and the United States; 150 to 400 patients per country). A discrete choice experiment was designed with input from clinical experts and the PAGs, plus scientific advice from the National Institute for Health and Care Excellence (NICE) in the United Kingdom. Patients’ preferences for the conditional relative importance of symptoms and impact of COPD will be quantified based on trade-offs they are willing to make among hypothetical COPD disease state profiles, described by differing attributes and levels. Hierarchical Bayesian analysis with effect-coding parameterization will be undertaken on the choice data to estimate (using Gibbs sampling) the relative value each respondent places on an attribute level.
The feedback from NICE informed the selection of screening criteria and the statistical analysis plan, as well as the inclusion of a health status measure, the EQ-5D-3L. Qualitative patient interviews and pilot testing of the attributes and levels grid have been completed, informing finalization of the online survey design.
Patient preference studies evaluating the relative importance of symptom burden through assessment of disease state preference values are an important new form of patient-based evidence for informing value-based decision making in HTA. The present study should facilitate a more patient-centered approach to developing new treatments for and improving the care of patients with COPD.