People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study

Abstract

IntroductionThis patient preference study (PPS) sought to quantify the preferences of people living with chronic obstructive pulmonary disease (COPD) regarding symptom improvement in the United Kingdom, United States, France, Australia, and Japan.MethodsInclusion criteria: People living with COPD aged 40 years or older who experienced ≥1 exacerbations in the previous year with daily symptoms of cough and excess mucus production. Study design: (I). development of an attributes and levels (A&L) grid through qualitative patient interviews; (II). implementation of the main online quantitative survey which included a discrete choice experiment (DCE) to allow assessment of attributes and levels using hypothetical health state profiles. Preference weights (utilities) were derived from the DCE using Hierarchical Bayesian analysis. A preference simulator was developed which enabled different health state scenarios to be evaluated based on the predicted patient preferences.Results1050 people living with moderate-to-severe COPD completed the survey. All attributes were considered important when patients determined their preferences in the DCE. In a health-state preference simulation, two hypothetical health states (comprising of attribute levels) with qualitatively equivalent improvements in A) cough and mucus and B) shortness of breath (SOB) resulted in a clear preference for cough and mucus-improved profile. When comparing two profiles with C) daily symptoms improved and D) exacerbations improved, there was a clear preference for the daily symptoms improved profile.ConclusionsPeople living with moderate-to-severe COPD prefer to reduce cough and mucus production together over improvement of SOB and would prefer to reduce combined daily symptoms over an improvement in exacerbations.

Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study

“…Most healthcare DCEs are designed as a choice between two or more hypothetical product profiles, with the attributes covering a range of efficacy, safety, and convenience factors for the product profiles in question. Our study differed in that the attributes were based on the disease symptoms that matter most to COPD patients (25–27), allowing the patient preferences for different disease health states to be investigated. Symptom-based preference studies of this kind can be important when conducted early in the medical product lifecycle to define the important clinical endpoints for inclusion in pivotal clinical trials (3;31).…”

“…Symptom-based preference studies of this kind can be important when conducted early in the medical product lifecycle to define the important clinical endpoints for inclusion in pivotal clinical trials (3;31). Scientific advice was sought from NICE during the design phase of our COPD patient preference study processes (10;25), the outputs of which both led to improvements in the study design and enabled an alignment of stakeholder perspectives around the endpoints that matter most and whose alleviation would constitute the greatest value to the patient (26;27).…”

“…The study used an online DCE supplemented with patient-reported questionnaires, including self-perceived severity of COPD and EQ-5D-3L. We enrolled 1050 COPD patients from five countries: Australia, France, Japan, United Kingdom, and United States, with sample sizes of 150, 150, 150, 200, and 400, respectively (see (25) for more details of the DCE design). In summary, attributes and levels (shown in columns 1 and 2 of Table 1) were derived by qualitative patient research (social media listening (26), use of online bulletin boards, (27) and published literature), with input from patient groups, clinical experts, and scientific advice from NICE (10;25).…”

“…We enrolled 1050 COPD patients from five countries: Australia, France, Japan, United Kingdom, and United States, with sample sizes of 150, 150, 150, 200, and 400, respectively (see (25) for more details of the DCE design). In summary, attributes and levels (shown in columns 1 and 2 of Table 1) were derived by qualitative patient research (social media listening (26), use of online bulletin boards, (27) and published literature), with input from patient groups, clinical experts, and scientific advice from NICE (10;25). Experimental design methods were used to derive 11 choice sets; each choice presented two hypothetical COPD patients (A and B) (28).…”

“…Our previously reported patient preference study used a discrete choice experiment (DCE) to explore the relative importance COPD patients place on six symptoms of their disease: shortness of breath, exacerbations, cough, excess mucus, sleep disturbance, and urinary incontinence (25). We use these DCE data to develop a COPD health state utility score, assess the convergent validity with self-perceived severity of COPD, and compare this disease-specific utility score with that of the generic HRQOL instrument, EQ-5D-3L (14).…”