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Objectives: To adapt a patient-reported outcome (PRO) measure, the Western Ontario McMaster Universities Osteoarthritis Index (WOMAC), into efficacy attributes for a discrete choice experiment (DCE) survey designed to quantify the relative importance of endpoints commonly used in knee osteoarthritis (KOA) trials. Methods: The adaptation comprised four steps: (1) selecting domains of interest; (2) determining presentation and framing of selected attributes; (3) determining attribute levels; and (4) developing choice tasks. This process involved input from multiple stakeholders, including regulators, health preference researchers, and patients. Pretesting was conducted to evaluate if patients comprehended the adapted survey attributes and could make trade-offs among them. Results: The WOMAC pain and function domains were selected for adaption to two efficacy attributes. Two versions of the discrete choice experiment (DCE) instrument were created to compare efficacy using (1) total domain scores and (2) item scores for "walking on a flat surface." Both attributes were presented as improvement from baseline scores by levels of 0%, 30%, 50%, and 100%. Twenty-six participants were interviewed in a pretest of the instrument (average age 60 years; 58% female; 62% had KOA for ≥ 5 years). The participants found both versions of attributes meaningful and relevant for treatment decision-making. They demonstrated willingness and ability to tradeoff improvements in pain and function separately, though many perceived them as inter-related. Conclusions: This study adds to the growing literature regarding adapting PRO measures for patient preference studies. Such adaptation is important for designing a preference study that can incorporate a clinical trial's outcomes with PRO endpoints. [ABSTRACT FROM AUTHOR]

Objective: To understand treatment preferences of people with migraine and the relative importance of improvements in efficacy and avoiding adverse events (AEs), such as cognition problems or weight gain. Background: Current preventive migraine medicines are associated with poor adherence and tolerability. There is an unmet need for effective migraine‐specific preventive treatments with fewer AEs. Methods: In a web‐based discrete‐choice experiment survey, respondents who self‐reported having ≥6 migraine days/month were offered choices between pairs of hypothetical preventive migraine medicines. Six attributes, each with 3 levels, defined the medicines: reduction in headache days per month (10%, 25%, or 50%), frequency of limitations with physical activities (none, 1‐category improvement, or 2‐category improvement), cognition problems (no problems, thinking problems, or memory problems), weight gain (none, 5% body weight gain, or 10% body weight gain), how the medicine is taken (daily oral pill, once‐monthly injection, or twice‐monthly injection), and monthly out‐of‐pocket cost ($5, $60, or $175). The attributes and levels were informed by clinician input, the clinical literature, and 2 focus groups. An experimental design was used to create the pairs of hypothetical medicines for the discrete‐choice experiment questions. Random‐parameters logit was used to estimate the relative importance of the medicine attributes, and the results were used to predict the percentage of respondents who would select one medicine profile over another and to calculate willingness to pay for changes in attribute levels. Results: The sample included 300 respondents; 72% indicated that migraines make physical activities difficult all or most of the time, and 81% had taken a prescription medicine to prevent migraine in the last 6 months. Respondents reported having, on average, approximately 16 headache days per month. Among noncost attributes, respondents valued a change from a 10% reduction in migraine days to a 50% reduction more highly than avoiding the worst levels of AEs, but were willing to trade off efficacy for less‐severe AEs. Avoiding memory problems was more important than avoiding thinking problems. Avoiding a 10% weight gain was more important than avoiding thinking and memory problems. Respondents preferred a once‐monthly injection or daily pill to twice‐monthly injection. Respondents, on average, were willing to pay $84 (95% confidence interval [CI], $64‐$103) per month to avoid a 10% weight gain, $59 (95% CI, $42‐$76) per month to avoid memory problems, $35 (95% CI, $20‐$51) per month to avoid a 5% weight gain, and $32 (95% CI, $18‐$46) per month to avoid thinking problems. Conclusions: A preventive migraine medicine with improved efficacy and AE profile and a favorable mode of administration would be valuable to migraine sufferers. Patients may be willing to trade off efficacy for better AE profiles. Clinicians should work with patients to select treatments that meet each patient's needs. [ABSTRACT FROM AUTHOR]

Background: The growing number of treatments for relapsing multiple sclerosis (MS) provides opportunities to consider patient preferences in treatment decisions. Methods: We designed a Web-based, discrete-choice experiment survey to analyze treatment preferences in patients with relapsing-remitting MS (RRMS). The survey presented hypothetical MS treatments defined by six attributes: risk of MS progression, time between relapses, risk of serious infection, treatment-related flu-like symptoms and gastrointestinal symptoms, and route and frequency of administration. Preference weights estimated with random-parameters logit were used to calculate importance scores and preference shares among three pairs of subsamples. Results: Patients with a self-reported physician diagnosis of RRMS (N = 301) completed the survey: 56% rated their disability level as normal or mild; 43% currently used a self-injectable treatment. Respondents with normal or mild disability levels placed greater weight on avoiding injections with fu-like symptoms and risk of progression, whereas patients with worse disability placed greater weight on reducing risk of progression and risk of serious infection. Patients taking injectables placed the most weight on risk of progression and risk of serious infection, whereas respondents not taking injectables placed the most weight on route and frequency of administration. Differences in preferences between subgroups were significant (P < .05). The presence of common adverse events associated with daily pills and injectables altered predicted preferences for route of administration. Conclusions: Preferences of patients with RRMS varied depending on current treatment and disability level, especially regarding mode of administration. Considering patient preferences for treatment features may lead to higher treatment satisfaction and adherence. [ABSTRACT FROM AUTHOR]

This article reports on an evaluation of the Merck Childhood Asthma Network, Inc. (MCAN) initiative using pooled cross-site data on patient-reported outcomes pre- and postintervention to quantify the changes experienced by children in five program sites supported by the network. The results show a consistent pattern of improvement across all measured outcomes, including symptoms, hospital and emergency department use, school absences, and caregiver confidence. Children who started with uncontrolled asthma experienced larger improvements than children with controlled asthma at baseline. However, even considering the significant gains made by children with uncontrolled asthma at baseline, after 12 months, most of the outcomes for these children were significantly worse than the 12-month outcomes for children with controlled asthma at baseline. The evaluation of the MCAN initiative offers a model that can be used in cases where resources must be balanced between evaluation and delivering services to children. The design process and results from the common survey instrument provide information for future initiatives seeking to translate evidence-based interventions in a community-based setting. [ABSTRACT FROM PUBLISHER]

Care coordination programs have been used to address chronic illnesses, including childhood asthma, but primarily via practice-based models. An alternative approach employs community-based care coordinators who bridge gaps between families, health care providers, and support services. Merck Childhood Asthma Network, Inc. (MCAN) sites developed community-based care coordination approaches for childhood asthma. Using a community-based care coordination logic model, programs at each site are described along with program operational statistics. Four sites used three to four community health workers (CHWs) to provide care coordination, whereas one site used five school-based asthma nurses. This school-based site had the highest caseload (82.5 per year), but program duration was 3 months with 4 calls or visits. Other sites averaged fewer cases (35 to 61 per CHW per year), but families received more (7 to 17) calls or visits over a year. Retention was 43% to 93% at 6 months and 24% to 75% at 12 months. Pre–post cross-site data document changes in asthma management behaviors and outcomes. After program participation, 93% to 100% of caregivers had confidence in controlling their child’s asthma, 85% to 92% had taken steps to reduce triggers, 69% to 100% had obtained an asthma action plan, and 46% to 100% of those with moderate to severe asthma reported appropriate use of controller medication. Emergency department visits for asthma decreased by 36% to 63%, and asthma-related hospitalizations declined by 26% to 78%. More than three fourths had fewer school absences. In conclusion, MCAN community-based care coordination programs improved management behaviors and decreased morbidity across all sites. [ABSTRACT FROM PUBLISHER]