To develop and validate mapping algorithms that estimate EQ-5D health state utility values (HSUVs) from two diabetes-specific patient-reported outcome measures, the Problem Areas in Diabetes (PAID) and Diabetes Distress Scale (DDS), to support economic evaluations when EQ-5D data are unavailable. Data from 662 adults with diabetes in Germany included PAID-20, DDS-17, and EQ-5D-5L responses. Direct mapping models predicted EQ-5D utilities using Tobit and censored least absolute deviation (CLAD) regressions, while indirect mapping models used proportional and partial proportional odds regressions to predict EQ-5D dimensions. Six model specifications were tested, incorporating PAID and DDS items with covariates (age, sex, diabetes type), with selection of predictors guided by item correlations and backward stepwise procedure to retain statistically relevant variables. Model performance was assessed using a selection of performance metrics including root mean square error (RMSE) and mean absolute error (MAE). Internal validation of the models employed 10-fold cross-validation. The strongest-performing direct mapping models consisted of a model with a selection of PAID-20 items and covariates (Tobit regression; RMSE: 0.159; MAE: 0.117) and a model that incorporated PAID-20, DDS-17, and covariates (CLAD regression; RMSE: 0.146; MAE: 0.09), and a model based on DDS-17 items and covariates (Tobit regression; RMSE: 0.164; MAE: 0.121). Indirect mapping models demonstrated higher prediction errors overall (RMSE: 0.187–0.21; MAE: 0.124–0.141) than their counterparts. We present novel algorithms that enable estimation of EQ-5D utilities from PAID and DDS scores, facilitating economic evaluation in diabetes. Preferred models demonstrated predictive accuracy comparable to published mapping studies. Many people with diabetes experience emotional strain, worries, and frustrations while managing their condition. Two widely used tools that serve to measure how the condition affects their daily life are the Problem Areas in Diabetes (PAID) scale and the Diabetes Distress Scale (DDS). To assess the value of new treatments, researchers and health agencies commonly rely on a measure called EQ‑5D, which summarizes overall health and is used to calculate quality‑of‑life scores important for economic evaluations and treatment choices. However, many diabetes studies collect PAID or DDS but do not collect EQ‑5D, making it difficult to judge the wider impact of treatments. This study set out to solve that problem by creating new algorithms that can estimate EQ‑5D scores from PAID and DDS responses when EQ‑5D was not included in a study. Using data from 662 adults with diabetes in Germany, several versions of these algorithms were tested, and the best ones closely matched people’s actual EQ‑5D scores. Among the different statistical approaches we tested, the models that directly linked both PAID and DDS responses to EQ-5D produced the most accurate predictions. These models performed well across a wide range of health states and closely matched the EQ-5D scores that participants reported. In practice, this enables researchers to generate the health-related quality-of-life information required for economic evaluations, even in studies that collect PAID or DDS but not EQ-5D.

Background: As the available treatments for moderate-to-severe atopic dermatitis (AD) expand, understanding patient and physician preferences becomes crucial for informed decision-making.Objective: To quantify patient and physician preferences for biologics and oral systemic AD treatment attributes.Materials and methods: We conducted a cross-sectional, online discrete choice experiment (DCE) involving 306 AD patients and 206 physicians throughout the United Kingdom and Germany. Qualitative interviews identified the key attributes for inclusion in the DCE. Each choice task comprised two hypothetical patient profiles. Data were analyzed using a random-parameters logit model.Results: Results indicated a significant emphasis on efficacy, with reducing sleep disturbance and itch ranking first and second among patients, and the reverse for physicians. Time to itch relief was the third most important efficacy attribute for both groups, but relatively more important for patients than for physicians. For both groups, the risk of eye problems was the most important safety concern of those included. Mode of administration was not of great importance compared to efficacy and safety attributes.Conclusions: Our findings suggest patients prioritize sleep disturbance, an attribute not captured in prior preference studies in AD, time to itch relief and itch. These findings emphasize the importance of addressing sleep-related issues, whilst also targeting fast itch control, to enhance patients’ well-being.

A growing number of treatment options are becoming available for moderate-to-severe atopic dermatitis (AD), including biologic therapies. As these treatments have different efficacy, safety, and tolerability profiles, it is important to understand patient and physician preferences for informed treatment decision making. To discover the importance of different attributes, including efficacy, safety, and mode of administration, and to provide insights into whether patient preferences concur or conflict with those of physicians. We conducted a cross-sectional, online discrete choice experiment involving 306 patients with AD and 206 physicians in the UK and Germany. Qualitative interviews identified key attributes influencing treatment preferences, including efficacy (itch relief, sleep disturbance, time-to-itch relief, and total body surface area affected by eczema), safety (special warnings and risks of eye problems and shingles), and treatment administration. Data were analyzed using a random parameters logit model to calculate the conditional relative importance of each attribute. Both groups placed significant emphasis on efficacy, with reducing sleep disturbance ranking first for patients and second for physicians and itch ranking first for physicians and second for patients. Time to itch relief was the third most important efficacy attribute for both groups, although it was more important for patients. For both groups, the risk of eye problems was the most important safety concern. The mode of administration was not considered of great importance when compared with efficacy and safety attributes. Our findings suggest patients prioritize sleep disturbance, an attribute not captured in other preference studies in AD, and itch. This emphasizes the importance of addressing sleep-related issues and itching to enhance patients’ well-being. The findings offer insights for prioritization strategies among healthcare providers, aiming to improve patient outcomes amidst a growing number of AD treatments.

BackgroundIn childhood autism spectrum disorder (ASD), the Childhood Autism Rating Scale–2nd edition (CARS2) is a condition-specific instrument to be filled out by clinicians, resulting in a score for diagnosis and severity. Our aim is to estimate a preference-based scoring of CARS2 to better understand the value of changes in the CARS2 score. Using caregivers and clinicians as proxies for autistic children, we assessed the feasibility of establishing preferences for CARS2-based attributes.MethodsThe 15 CARS2 items were assessed regarding their relevance as attributes and the appropriateness of their wording. Best-worst scaling (BWS) and discrete choice experiment (DCE) choice task designs were developed, as well as a mapping task between CARS2 and the EQ-5D-Y. Individual qualitative interviews with caregivers, clinicians, and autistic adults were conducted (N=10). A committee of experts advised on the study, including caregivers, clinicians, and specialists in CARS2, health technology assessment, and preference research methodology. ResultsThirteen of the 15 CARS2 items were deemed appropriate attributes for the preference study. Caregivers and autistic adults identified with the attributes and perceived them as comprehensive. The attribute definitions and level descriptions were shortened and refined to ensure comprehension for non-experts. The choice tasks were challenging for participants; however, 9/10 respondents could make a choice. Interviewees favored a two-profile DCE over a three-profile BWS design. In a subsequent internal pilot (N=13), participants favored a stacked layout, in which attributes with overlapping levels were clustered. The CARS2/EQ-5D-Y mapping task was feasible using step-by-step instructions. ConclusionA preference study in childhood ASD based on the CARS2 instrument is feasible with caregivers and clinicians as proxies for children’s preferences.

Abstract BackgroundIn childhood autism spectrum disorder (ASD), the Childhood Autism Rating Scale–2nd edition (CARS2) instrument is used for diagnosis and assessment of severity and change. Following a feasibility study, we conducted 2 discrete choice experiments (DCEs), each of which used caregivers and clinicians as proxies for autistic children, to assess preferences for CARS2-based attributes. MethodsCaregivers and clinicians from 5 European countries received an online DCE corresponding to either the standard or the high-functioning version of the CARS2. Participants completed 14 choice tasks with 2 hypothetical profiles composed of 13 attributes set at 4 varying levels. To reduce task complexity, the 2 profiles of each choice task had at least overlap in 7 attributes, i.e., attributes were set at the same level, and presented in a stacked layout. Multinomial, mixed and scale-adjusted logit models were used to estimate preference weights. Explorative anchoring to the EQ-5D-Y was undertaken and a rescaled set of DCE coefficients is provided. ResultsModels were fit separately for caregivers and clinicians in each experiment. The final models included 563 caregivers and 666 clinicians for the standard experiment, and 346 caregivers and 310 clinicians for the high-functioning one. Caregivers and clinicians, as expected, prioritized some of the same attributes but not all. For example, in the standard experiment, Verbal communication, Non-verbal communication, and Activity-level were highly important attributes to both groups, whilst Taste, smell, and touch response and use was more important to caregivers than it was to clinicians. In the high-functioning experiment, preferences for caregivers were highest for Thinking/cognitive integration skills, Verbal communication and Fear or anxiety. For clinicians, the most important attributes were Thinking/cognitive integration skills, Fear or anxiety and Social-emotional understanding. ConclusionCaregiver and clinician preferences indicated some disparity in what constitutes the greatest unmet need for autistic children. These findings can support clinicians and caregivers in creating mutual understanding and agreement on therapeutic goals, which can be very direct and near (e.g., Taste, smell, and touch response and use) or future-oriented and developmental (e.g., Relating to people).

Introduction Venetoclax in combination with rituximab (VEN + R) demonstrated prolonged overall survival (OS) and progression-free survival (PFS) for patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) in comparison to standard chemoimmunotherapy [bendamustine + rituximab (BR)]. We conducted a cost-effectiveness and budget impact analysis comparing VEN + R versus six comparators from the Swiss healthcare payer perspective. Methods A three-state partitioned survival model, developed in accordance with NICE and ISPOR decision modelling guidelines, was adapted to Switzerland. Model inputs were informed by the MURANO trial (survival data, patient characteristics), publicly available Swiss sources (drug prices, inpatient and outpatient costs), Swiss National Institute of Cancer Epidemiology and Registration data (incidence and prevalence values), and Swiss medical expert feedback. We used published (dis-)utility values and adverse event probabilities. Results Over a lifetime, VEN + R resulted in an expected gain of 2.60 quality-adjusted life years (QALYs) per patient and incremental costs of Swiss Francs (CHF) 147,851 compared to BR, leading to an incremental cost-effectiveness ratio of CHF 56,881/QALY gained. Other treatment strategies (for example ibrutinib versus VEN + R) resulted in higher costs and lower QALYs. Results were not different for subgroups of patients with/without deletion of chromosome 17p/tumour protein 53 mutation. In scenario analysis, changes in post-progression treatment costs demonstrated a high impact on results. We estimated an expected value of perfect information of CHF 3,318/patient. A moderate VEN + R uptake was estimated to save CHF 12.3 million during 5 years. Conclusions Using a threshold of CHF 100,000 per QALY, VEN + R was projected to be cost-effective vs BR.