Health state utilities for non small cell lung cancer

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Deriving a Preference-Based Measure for Myelofibrosis from the EORTC QLQ-C30 and the MF-SAF

AbstractBackgroundUtility values are required for economic evaluation using cost-utility analyses. Often, generic measures such as the EuroQol five-dimensional questionnaire are used, but this may not appropriately reflect the health-related quality of life of patients with cancer including myelofibrosis.ObjectiveTo derive a condition-specific preference-based measure for myelofibrosis using appropriate existing measures, the Myelofibrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire.MethodsData from the Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment trial (n = 309) were used to derive the health state classification system. Psychometric and factor analyses were used to determine the dimensions of the classification system. Psychometric and Rasch analyses were then used to select an item to represent each dimension. Item selection was validated with experts. A selection of health states was valued by members of the general population using time trade-off. Finally, health state values were modeled using regression analysis to produce utility values for every state.ResultsThe Myelofibrosis 8 dimensions has eight dimensions: physical functioning, emotional functioning, fatigue, itchiness, pain under ribs on the left side, abdominal discomfort, bone or muscle pain, and night sweats. Regression models were estimated using time trade-off data from 246 members of the general population valuing a total of 33 states. The best performing model was a random effects maximum likelihood model producing utility values ranging from 0.089 to 1.ConclusionsThe Myelofibrosis 8 dimensions is a condition-specific preference-based measure for myelofibrosis. This measure can be used to generate utility values for myelofibrosis for any data set containing the Myelofibrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire data

The ostomy leak impact tool: development and validation of a new patient-reported tool to measure the burden of leakage in ostomy device users

Abstract Background Leakage is a major concern for people who use a stoma, but people’s experience and its impact is not well understood. This study aimed to establish a definition of leakage through clinical and user input. This information was used to develop and validate a new measurement tool to understand the impact of leakage for people using a stoma appliance, in the UK, US, France, and Denmark. Methods Participants were recruited from a panel of users, hosted by Coloplast, that includes people who currently use Coloplast products. Six clinicians and 41 users took part in concept elicitation interviews. The qualitative findings were used to draft items. A panel of clinical experts was organized to develop and validate items (N = 6). Cognitive debrief interviews were conducted with five users in each country, which resulted in removing some items and revising the measure. A psychometric validation was conducted with 340 people in four countries whereby participants were asked to complete a series of measures online. Full psychometric analyses including validity and reliability were conducted. Results A final tool was established consisting of three domains related to the burden of leakage: “Emotional impact,” “Usual and social activities,” and “Coping and control.” Convergent validity was evaluated by benchmarking to existing health-related quality of life instruments (domains of SF-36 and Ostomy-Q). This showed high correlation between domains of the leakage tool and other measures, in particular for the Emotional impact domain when compared with SF-36 Emotional well-being and Ostomy-Q Confidence domain (p  0.92). Conclusion The study highlights how users define leakage and its impact in a way that is meaningful to them. This information has been used to develop an instrument to measure leakage which can potentially be used by clinicians and researchers. The instrument demonstrated evidence supporting its reliability and validity as an outcome measure to assess the impact of leakage in stoma care

Health state utilities in non-small cell lung cancer : an international study

Aim: Quality of life weights (utilities) are an important input in economic evaluation and evidence suggests that there can be important differences between countries. This study was designed to capture utilities for metastatic non-small cell lung cancer and common grade III/IV toxicities associated with treatment from local populations in the United Kingdom, Australia, France, China, Taiwan, and Korea. Toxicities included neutropenia, febrile neutropenia, fatigue, diarrhea, nausea and vomiting, rash, bleeding, hypertension, and hair loss. Methods: Existing health state descriptions of non-small cell lung cancer were adapted to represent descriptions of patients on first-line treatment. Twenty-three states were translated and assessed in cognitive debrief content validation interviews with oncologists in each country. Seventy-five respondents per country completed a time trade-off interview to evaluate the states. Variation between countries for all states was explored with a Generalized Estimating Equations model. Results: The mean utility for stable disease and no side effects (base state) varied between 0.84 (United Kingdom) and 0.54 (Taiwan). The largest utility decrements were found for febrile neutropenia (0.47) and neutropenia (0.35) across all countries. Asian countries regarded bleeds as a severe toxicity whereas non-Asian countries did not and valued diarrhea and fatigue as more severe. Significant differences in utilities between countries emerged with the Taiwanese population in particular rating states as significantly worse than other countries. Conclusion: This study improves our understanding of how utilities for the same states can vary across countries. The study shows the importance of capturing utilities that reflect the preferences of the local population

Willingness to pay for improvements in chronic long-acting insulin therapy in individuals with type 1 or type 2 diabetes mellitus.

BACKGROUND Long-acting insulin treatments with varying clinical benefits are currently available for patients with type 1 diabetes mellitus (T1DM) and type 2 diabetes mellitus (T2DM). The current evidence base demonstrates the efficacy of treatments, but it is critical also to understand patient preferences regarding treatments and how they are determined. OBJECTIVE This study aimed to measure the willingness to pay (WTP) of individuals with diabetes in the United Kingdom for different attributes of long-acting insulin therapy. METHODS A survey based on discrete choice experiment methodology was developed to elicit the preferences and values of adults with T1DM or insulin-dependent T2DM regarding different aspects of their therapy. Participants were presented with a series of 27 paired choices and asked which they preferred. WTP values were calculated for relevant attribute levels. RESULTS A total of 252 participants completed the questionnaire (52% response rate); 143 had T1DM and 109 had insulin-treated T2DM. The highest WTP values in participants with T1DM were avoiding 2-kg or 4-kg weight gain (£29 and £58, respectively), avoiding major difficulties with the injection device (£49), increasing the number of days per week when blood glucose levels are in the target range from 2 to 6 (£40), reducing the number of daily injections from 3 to 1 (£39), and avoiding nighttime hypoglycemia (£33). In participants with T2DM, similar factors had the highest WTP. CONCLUSIONS This is the first study to assess WTP for long-acting insulin therapy and could have implications for future guidelines on diabetes management, however some limitations, notably in sample selection, could affect generalizability of the results. In both T1DM and T2DM, the highest WTP values were for avoidance of weight gain, and reduction in the number of injections and hypoglycemia

Parent preferences regarding stimulant therapies for ADHD: a comparison across six European countries

The objective is to identify attributes of ADHD stimulant medications that influence treatment preferences of parents of children and adolescents with ADHD across six European countries, using a discrete choice experiment (DCE). Different attributes (and associated levels) of stimulant therapies were identified through literature review and clinician input. Attributes included duration and degree of symptom control after each dose, frequency of medication dosing, potential for treatment to be abused, the side effects of vomiting, loss of appetite, and sleep disturbance. Attributes and levels were combined using an orthogonal design to produce a number of discrete hypothetical treatments. Parents were recruited via patient panels in different countries and asked to complete a survey. DCE data were analyzed using conditional logit models to explore the impact of each attribute on participants' choices. Six hundred individuals (220 parents of adolescents and 380 parents of children) participated. All attributes were significant predictors of choice (p < 0.01). 'Degree of symptom control' was the most important attribute whereby the odds of choosing 'very much improved symptoms' compared with 'minimally improved' was 4.85 [95 % confidence interval (CI) = 4.28-5.49] for the adolescent group and 6.37 (95 % CI = 5.79-7.01) for the child group. Some inter-country differences emerged, e.g., achieving the best degree of symptom control was more important to parents in some countries than others. In conclusion, the study showed that duration and degree of symptom control were the most important aspects of treatment for parents in all countries. The findings revealed cultural differences in the relative importance of attributes