Maintenance inhaler therapy preferences of patients with asthma or chronic obstructive pulmonary disease: a discrete choice experiment.

BACKGROUND: A variety of maintenance inhaler therapies are available to treat asthma and COPD. Patient-centric treatment choices require understanding patient preferences for the alternative therapies. METHODS: A self-completed web-based discrete choice experiment was conducted to elicit patient preferences for inhaler device and medication attributes. Selection of attributes was informed by patient focus groups and literature review. RESULTS: The discrete choice experiment was completed by 810 patients with asthma and 1147 patients with COPD. Patients with asthma most valued decreasing the onset of action from 30 to 5 min, followed by reducing yearly exacerbations from 3 to 1. Patients with COPD most and equally valued decreasing the onset of action from 30 to 5 min and reducing yearly exacerbations from 3 to 1. Both patients with asthma and patients with COPD were willing to accept an additional exacerbation in exchange for a 15 min decrease in onset of action and a longer onset of action in exchange for a lower risk of adverse effects from inhaled corticosteroids. Patients with asthma and COPD valued once-daily over twice-daily dosing, pressurised inhalers over dry powder inhalers and non-capsule priming over single-use capsules, although these attributes were not valued as highly as faster onset of action or reduced exacerbations. CONCLUSIONS: The most important maintenance inhaler attributes for patients with asthma and COPD were fast onset of symptom relief and a lower rate of exacerbations. Concerns about safety of inhaled corticosteroids and device convenience also affected patient preferences but were less important.

Health Preference Research in Europe: A Review of Its Use in Marketing Authorization, Reimbursement, and Pricing Decisions-Report of the ISPOR Stated Preference Research Special Interest Group.

OBJECTIVE: This study examines European decision makers' consideration and use of quantitative preference data. METHODS: The study reviewed quantitative preference data usage in 31 European countries to support marketing authorization, reimbursement, or pricing decisions. Use was defined as: agency guidance on preference data use, sponsor submission of preference data, or decision-maker collection of preference data. The data could be collected from any stakeholder using any method that generated quantitative estimates of preferences. Data were collected through: (1) documentary evidence identified through a literature and regulatory websites review, and via key opinion leader outreach; and (2) a survey of staff working for agencies that support or make healthcare technology decisions. RESULTS: Preference data utilization was identified in 22 countries and at a European level. The most prevalent use (19 countries) was citizen preferences, collected using time-trade off or standard gamble methods to inform health state utility estimation. Preference data was also used to: (1) value other impact on patients, (2) incorporate non-health factors into reimbursement decisions, and (3) estimate opportunity cost. Pilot projects were identified (6 countries and at a European level), with a focus on multi-criteria decision analysis methods and choice-based methods to elicit patient preferences. CONCLUSION: While quantitative preference data support reimbursement and pricing decisions in most European countries, there was no utilization evidence in European-level marketing authorization decisions. While there are commonalities, a diversity of usage was identified between jurisdictions. Pilots suggest the potential for greater use of preference data, and for alignment between decision makers.

Patient preferences for dry powder inhaler attributes in asthma and chronic obstructive pulmonary disease in France: a discrete choice experiment.

BACKGROUND: Dry powder inhalers (DPIs) are often used in asthma and chronic obstructive pulmonary disease (COPD) therapies. Using the discrete choice experiment (DCE) methodology, this study conducted in France was designed to assess patients' preferences for different attributes of DPIs. METHODS: Attributes of DPIs were defined based on a literature review, patient focus group discussions and interviews with healthcare professionals (qualitative phase of the study). An online survey was then conducted among French patients with asthma or COPD to elicit patient preferences and willingness to pay (WTP) for these attributes using the DCE methodology (quantitative phase). A fractional factorial design including three blocks of 12 choice sets was created. Each choice set comprised three alternatives: two fictitious inhalers and the patient's current inhaler. Marginal utilities were estimated using a ranked ordered logit model. Interactions between attributes and disease (asthma or COPD) were tested. RESULTS: Six DPI attributes were defined based on the qualitative phase: ease of use/fool-proof priming; accurate and easy-to-read dose counter; dose confirmation; hygiene of the mouthpiece; flexibility of the device handling; ability to use the inhaler with breathing difficulties. Overall, 201 patients with asthma and 93 with COPD were included in the online survey. Patients with asthma placed most value on an inhaler that requires one step for dose preparation (WTP €4.83 [95% CI: €3.77-€5.90], relative to an inhaler requiring four steps) and one that could be used during episodes of breathing difficulties (WTP €4.49 [95% CI: €2.95-€6.02]). Patients with COPD placed most value on an inhaler that could be used during episodes of breathing difficulties (WTP €7.70 [95% CI: €5.65-€9.76]) and on the accuracy of the dose counter (WTP €5.87 [95% CI: €3.98-€ 7.77]). CONCLUSION: This study suggests that asthma and COPD patients would be willing to change their inhaler if they were offered the option of a new inhaler with improved characteristics and they place a high value on an inhaler with ease of use during breathing difficulty episodes.

Patient-centered benefit-risk analysis of transcatheter aortic valve replacement.

Background: Aortic stenosis (AS) treatments include surgical aortic valve replacement (SAVR) and transcatheter aortic valve replacement (TAVR). Choosing between SAVR and TAVR requires patients to trade-off  benefits and risks. The objective of this research was to determine which  TAVR and SAVR outcomes patients consider important, collect quantitative data about how patients weigh benefits and risks, and evaluate patients' preferences for SAVR or TAVR. Methods: Patients  were recruited from advocacy organization databases. Patients self-reported as being diagnosed with AS, and as either having received AS treatment or as experiencing AS-related physical activity limitations. An online adapted swing weighting (ASW) method - a pairwise comparison of attributes - was used to elicit attribute trade-offs from 219 patients. Survey data were used to estimate patients' weights for AS treatment attributes, which were incorporated into a quantitative benefit-risk analysis (BRA) to evaluate patients' preferences for TAVR and SAVR. Results: On average, patients put greater value on attributes that favored TAVR than SAVR. Patients' valuation of the lower mortality rate, reduced procedural invasiveness, and quicker time to return to normal quality of life associated with TAVR, offset their valuation of the time over which SAVR has been proven to work. There was substantial heterogeneity in patients' preferences. This was partly explained by age, with differences in preference observed between patients <60 years to those ≥60 years. A Monte Carlo Simulation found that 79.5% of patients prefer TAVR. Conclusions: Most AS patients are willing to tolerate sizable increases in clinical risk in exchange for the benefits of TAVR, resulting in a large proportion of patients preferring TAVR to SAVR. Further work should be undertaken to characterize the heterogeneity in preferences for AS treatment attributes. Shared decision-making tools based on attributes important to patients can support patients' selection of the procedure that best meets their needs.

Elicitation of Health-related Quality-of-life Concepts Associated with Overactive Bladder: A Qualitative Study.

Background: Overactive bladder (OAB) is a symptom-defined disorder. A range of instruments are available for assessing OAB symptom bother, urinary urgency and the effects of symptoms on health-related quality of life (HRQoL), but few have been specifically designed and validated for this condition. HRQoL instruments should capture the concepts that are most relevant to patients. To our knowledge, there is no existing published conceptual framework for OAB. Objectives: We performed a qualitative study to explore the impact of symptoms of OAB on affected patients and to develop a conceptual framework for OAB. Methods: Patients diagnosed with OAB living in the United Kingdom were interviewed on the telephone by a trained psychologist using an interview discussion guide. Interview transcripts were analyzed thematically by two psychologists. Data collection and analysis was completed when data saturation, i.e. when little or no new information was obtained, was achieved. Results: A total of 30 patients were interviewed. Fifteen patients (50%) had urge incontinence (i.e. OAB-wet). Interview data showed that OAB affected role functioning, sleep quality, social functioning, and emotional/mental functioning. In addition, patients often adopted non-medical coping strategies to manage their symptoms (e.g. planning activities). Factors which affected more than 50% of patients were going for a short walk, waking up at night, travelling/holidays, socializing/going out, embarrassment/shame, need to plan activities, and restriction of places visited. More patients with OAB-wet reported impairment of social and emotional/mental functioning than patients with OAB-dry. A conceptual framework for adults with OAB depicting the relationships between OAB concepts (or outcomes) was developed. Conclusions: OAB has a profound effect on patient HRQoL and negatively affects a broad range of functions, including daily and work activities, leisure and social activities, psychological well-being, and sleep capacity. The conceptual framework emerging from this study supports the utilization of existing disease-specific HRQoL instruments, but identifies that work-related effects, which are relevant for OAB patients, are missing from currently available measures.

Patient survey to identify reasons for non-adherence and elicitation of quality of life concepts associated with immunosuppressant therapy in kidney transplant recipients.

BACKGROUND: Renal transplantation (RT) is considered the treatment of choice for end-stage renal disease compared to dialysis, offering better health-related quality of life (HRQoL) and higher survival rates. However, immunosuppressants are essential for the long-term survival of kidney grafts and patients' non-adherence to their medication leads to poor outcomes. Immunosuppressants can also significantly alter patients' HRQoL because of their side effects and the complex chronic medication regimen they represent. PURPOSE: To elicit key concepts related to adherence to immunosuppressant therapy (IT) and reasons for non-adherence in terms of patient reported outcomes, side effects, and the impact of the medication on HRQoL in RT population, including patient preference of once daily over twice-daily immunosuppressive regimen. Results were used to develop an IT-specific conceptual framework and provide suggestions for improving patients' adherence to IT. MATERIALS AND METHODS: Interviews were conducted with three clinical experts to determine key concepts related to RT and immunosuppressants. Thirty-seven participants in four focus groups were asked to cite important concepts related to adherence and impact of IT on HRQoL and to rate them. Qualitative analysis was conducted to code participants' responses. RESULTS: Non-adherence among participants where admitted was unintentional. The reason for this included forgetfulness, interference with lifestyle, being asleep at the time the medication should be taken, change in routine, and impact of side effects. Overall, participants reported that the evening dose was more problematic to remember and that the exclusion of this dose could make them more adherent. Participants also reported that IT impacted on their HRQoL in a number of ways including: placing restrictions on their lifestyle, causing anxiety, or impairing their ability to work. CONCLUSION: This study provides qualitative evidence about the barriers to IT adherence and the components of HRQoL that are important from the perspective of RT patients. The developed conceptual framework of IT-HRQoL in RT transplants, including social, psychological, and work life domains, can be used to inform the development of a new IT-specific measure of HRQoL in RT patients for use in head-to-head clinical trials or observational studies. Despite limitations associated with the number and the age range of patients recruited, this study suggests that a change in the regimen from twice-daily to once daily among other measures could improve their adherence to IT and their HRQoL by placing less restrictions on their lifestyles.