A Health State Utility Study to Elicit Societal Values Associated with Pulmonary Hypertension.

Aim: Pulmonary hypertension (PH) is a rare, severe, and progressive pulmonary vascular disease, which includes five subgroups with similar presentation. Symptoms include dyspnea, and fatigue, and can significantly impact one's health-related quality of life (HRQL). Although treatments are mainly medical, PH group 4, chronic thromboembolic pulmonary hypertension (CTEPH), can be managed with procedures, ie pulmonary endarterectomy (PEA) and balloon pulmonary angioplasty (BPA). Currently, drugs in Europe are only approved for pulmonary arterial hypertension (PAH), and CTEPH therefore the aim of this study was to elicit novel societal health state utilities in the UK for PAH and CTEPH based on disease severity, functional class (FC), clinical events, and treatment procedures specifically for CTEPH. Material and Methods: Six health states were defined: World Health Organization (WHO)-FC II, WHO-FC III, and WHO-FC IV [defined by the New York Heart Association (NYHA)]; PH-related hospitalization; and "BPA procedure and recovery" and "PEA surgery and recovery". Health states were based on a targeted literature review and two rounds of interviews with clinical experts (N = 4) and patients (N = 6). Draft health states were validated in cognitive debriefing interviews with clinical experts (N = 3). Health states were valued by the UK general public (n = 200), using a visual analogue scale (VAS) and time trade-off (TTO) assessment with the lead time method. Results: The mean TTO values/utilities were 0.81 (FC II), 0.80 (BPA), 0.78 (PEA), 0.59 (FC III), 0.28 (FC IV), and 0.25 (PH-related hospitalization). Each progression in FC was associated with worse TTO scores. Conclusion: This study reports societal utility values for PAH and CTEPH in the UK. It provides first utility estimates for states such as BPA procedure and recovery, PEA surgery and recovery and PH-related hospitalization for this population. The results show important distinctions between FC, treatment procedures, and hospitalization, and the significant burden of disease on HRQL.

Estimating health state utilities in hemophagocytic lymphohistiocytosis.

BACKGROUND: Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses. METHODS: Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework. RESULTS: Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18-0.35), receiving chemotherapy (0.26, 95%CI 0.17-0.34), undergoing SCT (0.18, 95%CI 0.07-0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67-0.77) and end of life care (- 0.17, 95%CI -0.27- -0.07). CONCLUSIONS: This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting.

Patient Preferences for Metastatic Hormone-Sensitive Prostate Cancer Treatments: A Discrete Choice Experiment Among Men in Three European Countries.

INTRODUCTION: Various treatment options are available for metastatic hormone-sensitive prostate cancer. This study aimed to quantify how men with prostate cancer in the United Kingdom (UK), Germany, and Spain perceive the risks and benefits of hypothetical abiraterone acetate plus prednisone treatment and docetaxel-based chemotherapy treatment options. METHODS: A targeted literature review, exploratory interviews with prostate cancer patients and oncologists, and pre-test interviews were used to develop a discrete choice experiments (DCE). The final DCE included 32 choice sets, selected using a main-effects orthogonal design, divided into two survey blocks. Paired profiles presented hypothetical treatments for prostate cancer through six attributes that could be presented at two or four levels each. Preference estimates were estimated using a conditional logit regression model. Preference results were stratified by cancer stage. RESULTS: A total of 152 participants (mean age 69 years) completed the DCE in the UK, Germany, and Spain. Treatment effectiveness was the main concern for the patients (difference in preference estimates between 8 and 32 months 1.443). Participants wanted to avoid pain that was not well controlled (preference dummy coding estimate - 1.157). Participants valued a change from an oral medication to an intravenous treatment (change in preference estimate - 0.416) more negatively than a change from a 1% to a 5% risk of infection (change in preference estimate - 0.313). CONCLUSIONS: This study shows that treatment effectiveness and pain control were the most important attributes for patients with prostate cancer. These two attributes influenced more than 50% of their decision-making in this study. The risk of fatigue and mode of administration were least prioritised by patients. This study highlights the relative importance that Spanish, German, and British patients place on various aspects of treatment options for prostate cancer. Understanding patient preference and taking them into consideration shall help physicians when developing their treatment strategies for their patients. FUNDING: Janssen.

Disutility Study for Adult Patients with Moderate to Severe Crohn's Disease.

BACKGROUND: Crohn's disease (CD) treatments and associated adverse events (AEs) can be burdensome for patients. However, specific values which quantify the impact on health-related quality of life (HRQL) for economic evaluation are lacking. OBJECTIVES: This study aimed to elicit health utility values for AEs related to biologic treatment and surgical complications for CD in the UK. METHODS: Health states were developed by literature review and interviews with CD patients (n=6) and gastroenterologists (n=3). Draft health states were validated in cognitive debrief interviews with patients (n=4) and gastroenterologists (n=2). Treatment AEs were described with moderate-severe CD (reference state) and included hypersensitivity, injection site reactions, serious infection, lymphoma, and tuberculosis. Surgical complications were described following bowel surgery (reference state) and included anastomotic leak, wound infection, prolonged ileus/bowel obstruction, and intra-abdominal abscess. Health states were valued by 100 members of the general public who completed background questions, EQ-5D-3L, visual analogue scale rating task and time trade-off (TTO) interviews. RESULTS: The mean TTO value for reference states 'moderate to severe CD' and 'bowel surgery' were 0.70 (SD=0.28) and 0.69 (SD=0.28). Participants rated lymphoma as the worst AE/surgical complication state (0.44, SD=0.37), followed by tuberculosis (0.47, SD=0.85) and anastomotic leak (0.48, SD=0.38). Values of other AE/surgical complication states ranged from 0.76 (hypersensitivity) to 0.56 (intra-abdominal abscess). CONCLUSIONS: This study provides utility estimates for AE and surgical complication health states not previously assessed in the context of CD. As new treatments are emerging, it is important to include these influences on quality of life in any economic evaluation of treatments.

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

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 < 0.001). Coping and control correlated moderately well with most PROs tested for except the physical functioning domains, which showed only modest correlation (p < 0.001). Usual and social activities correlated equally well with all domains. Internal consistency was high for Emotional impact and Usual and social activities (> 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.

Evaluation and quantification of treatment preferences for patients with asthma or COPD using discrete choice experiment surveys.

INTRODUCTION: To investigate treatment preferences of patients with asthma or chronic obstructive pulmonary disease (COPD), previously identified influential treatment factors were used to develop a discrete choice experiment (DCE) survey. METHODS: An internet-based survey was conducted with UK-resident adults (recruited using a commercial panel) who were currently receiving asthma/COPD treatment and had not taken part in the previous phase of this study (qualitative interviews to understand patient burden, life impact and treatment preferences). Participants ranked treatment attributes from 0 (extremely important) to 8 (not at all important) and chose between hypothetical treatments for asthma/COPD with differing attributes. Preferences for each condition were assessed separately using a mixed logit regression model. RESULTS: Most of the 302 participants had not well-controlled asthma (Asthma Control Test™ scores ≤19/25) or experienced a high impact of COPD (COPD Assessment Test™ scores >20/40). Participant views were generally similar for both conditions; having well-controlled symptoms all day was considered most important. All treatment attributes significantly influenced preferences; the most preferred were no sleep disturbance (versus waking up often) and low cost. Subsequent preferences (with some variation between asthma/COPD) were for treatments with easy/convenient use, no flare ups/exacerbations, that enabled desired physical activities, well-controlled symptoms all day, that enabled desired social activities, and low medication frequency. CONCLUSIONS: These eight treatment attributes, valued by patients with asthma or COPD, are important for healthcare professionals to consider regarding treatment options and for future therapy development. Our DCE results broadly reinforce the findings from qualitative interviews in the first study phase.

The Ostomy-Q: Development and Psychometric Validation of an Instrument to Evaluate Outcomes Associated with Ostomy Appliances.

Using an ostomy appliance can affect many aspects of a person's health-related quality of life (HRQL). A 2-part, descrip- tive study was designed to develop and validate an instrument to assess quality-of-life outcomes related to ostomy ap- pliance use. Study inclusion/exclusion criteria stipulated participants should be 18 to 85 years of age, have an ileostomy or colostomy, used an appliance for a minimum of 3 months without assistance, and able to complete an online survey. All participants provided sociodemographic and clinical information. In phase 1, a literature search was conducted and existing instruments used to measure HRQL in persons with an ostomy were assessed. Subsequently, the Ostomy-Q, a 23-item, Likert-response type questionnaire, divided into 4 domains (Discreetness, Comfort, Confidence, and Social Life), was developed based on published evidence and existing ostomy-related HRQL tools. Seven (7) participants re- cruited from a manufacturer user panel took part in exploratory/cognitive qualitative interviews to refine the new quality- of-life questionnaire. In phase 2, the instrument was tested to assess item variability and conceptual structure, item-total correlation, internal consistency, test-retest reliability, sensitivity, and minimal important difference (MID) in an online validation study among 200 participants from the manufacturer's user panel (equally divided by gender, 125 [62.5%] >50 years old, 128 [64%] with an ileostomy). This exercise also included completion of the Stoma Quality of Life Question- naire and 2 domains from the Ostomy Adjustment Inventory-23 to assess convergent validity. Eighty-two (82) participants recompleted these study instruments 2 weeks later to assess test-retest reliability. Sociodemographic and clinical data were assessed using descriptive statistics; Cronbach's alpha was used for internal consistency (minimum 0.70), principle component analysis for item variability/conceptual structure, and item-total correlation; intraclass correlation coefficient was used for test-retest reliability; and standard error of measurement was applied to MID. All domains demonstrated good internal consistency (between 0.69 and 0.78). All scales showed stability, with a minimum intraclass correlation coefficient of 0.743 (P <.001). The Ostomy-Q showed good convergent validity with other instruments to which it was compared (P <.01). In this study, the Ostomy-Q was found to be a reliable and valid outcome measure that can enhance understanding of the impact of ostomy appliances on users. Some items for social relationships and discreetness may need more exploring in the future with other patient groups.

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.

Estimating minimal important differences for several scales assessing function and quality of life in patients with attention-deficit/hyperactivity disorder.

OBJECTIVE: Defining minimal important difference (MID) is critical to interpreting patient-reported outcomes data and treatment efficacy in clinical trials. This study estimates the MID for the Weiss Functional Impairment Rating Scale-Parent Report (WFIRS-P) and the Child Health and Illness Profile-Parent Report (CHIP-CE-PRF76) among parents of young people with attention-deficit/hyperactivity disorder (ADHD) in the UK. METHODS: Parents of children (6-12 years; n=100) and adolescents (13-17 years; n=117) with ADHD completed a socio-demographic form, the CHIP-CE-PRF76, the WFIRS-P, and the Pediatric Quality of Life scale at baseline and 4 weeks later. At follow-up, a subset of parents completed anchor questions measuring change in the child/adolescent from baseline. MIDs were estimated using anchor-based and distribution-based methods, and separately for children and adolescents. RESULTS: The MID estimates for overall change in the WFIRS-P total score ranged from 11.31 (standard error of measurement) to 13.47 (anchor) for the total sample. The range of MID estimates for the CHIP-CE-PRF76 varied by domain: 6.80-7.41 (satisfaction), 6.18-7.34 (comfort), 5.60-6.72 (resilience), 6.06-7.57 (risk avoidance), and 4.00-5.63 (achievement) for the total sample. Overall, MID estimates for WFIRS-P MID and CHIP-CE-PRF76 were slightly higher for adolescents than for children. CONCLUSION: This study estimated MIDs for these instruments using several methods. The observed convergence of the MID estimates increases confidence in their reliability and could assist clinicians and decision makers in deriving meaningful interpretations of observed changes in the WFIRS-P and CHIP-CE in clinical trials and practice.

The real-world patient experience of fingolimod and dimethyl fumarate for multiple sclerosis.

BACKGROUND: Oral disease-modifying therapies offer equivalent or superior efficacy and greater convenience versus injectable options. OBJECTIVES: To compare patient-reported experiences of fingolimod and dimethyl fumarate. METHODS: Adult relapsing-remitting multiple sclerosis patients treated with fingolimod or dimethyl fumarate were recruited from an online patient community and completed an online survey about treatment side effects, discontinuation, and satisfaction. RESULTS: 281 patients in four groups completed the survey: currently receiving fingolimod (CF, N = 61), currently receiving dimethyl fumarate (CDMF, N = 129), discontinued fingolimod (DF, N = 32) and discontinued dimethyl fumarate (DDMF, N = 59). Reasons for treatment switch were to take oral treatment (CF: 63.3 %, CDMF: 61.8 %), side effects of prior medication (CF: 67.3 %, CDMF: 44.1 %) and lack of effectiveness of prior medication (CF: 38.8 %, CDMF: 31.4 %). Main reasons for discontinuation were side effects (DF: 46.9 %, DDMF: 67.8 %) and lack of effectiveness (DF: 25.0 %, DDMF: 15.3 %). CDMF patients had an increased risk of abdominal pain, flushing, diarrhea, and nausea. Treatment satisfaction was highest among CF patients followed by CDMF, DF, and then DDMF patients. CONCLUSIONS: Discontinuation was driven by experience of side effects. Patients currently taking dimethyl fumarate were more likely to experience a side effect versus patients currently taking fingolimod. Examination of the relationship between tolerability and adherence/persistence is needed.

Managing neurogenic bowel dysfunction: what do patients prefer? A discrete choice experiment of patient preferences for transanal irrigation and standard bowel management.

BACKGROUND: Most patients with bowel dysfunction secondary to neurological illness are managed by a range of nonsurgical methods, including dietary changes, laxatives, and suppository use to transanal irrigation (TAI). The aim of the present study was to explore individuals' preferences regarding TAI devices and furthermore investigate willingness to pay (WTP) for attributes in devices in the UK. METHODS: A discrete choice experiment survey was conducted to evaluate the patients' perceived value of TAI devices. Attributes were selected based upon a literature review and input from clinicians. Interviews were conducted with three clinicians and the survey was developed and finalized with the input from both patients and professionals. The final attributes were "risk of urinary tract infections" (UTIs), "risk of fecal incontinence" (FI), "frequency of use", "time spent on toilet", "ease of use", "level of control/independence", and "cost". Participants were recruited by a patient panel of TAI device users in the UK. Data were analyzed using the conditional logit model whereby the coefficients obtained from the model provided an estimate of the (log) odds ratios (ORs) of preference for attributes. WTP was also estimated for each attribute. RESULTS: A total of 129 participants were included in the final analyses. Sixty two percent of the participants had suffered from three UTIs in the preceding year and 58% of patients reported currently experiencing FI using their current device. All attributes were significant predictors of choice. The most important attributes for participants were the "risk of FI", "frequency of use", and "risk of UTIs". CONCLUSION: Participants with bowel dysfunction regarded "risk of FI", "frequency of use", and "avoiding UTIs" as the most important features of a TAI device. These preferences are valuable in informing decision makers and clinicians regarding different bowel management solutions as well as for development of future devices.

What are the preferences of health care professionals in Germany regarding fully liquid, ready-to-use hexavalent pediatric vaccine versus hexavalent pediatric vaccine that needs reconstitution?

Diphtheria, tetanus and acellular pertussis (DTaP) based combined vaccines have led to significant reduction in incidence of several serious pediatric infectious diseases. A new, fully liquid combined hexavalent vaccine has been introduced and has been shown to reduce administration time. This fully liquid vaccine may also be simpler to administer and could reduce handling errors. The present study was designed to understand the value that health care providers (HCPs) place on aspects of injection devices for combined hexavalent vaccine programs in Germany. A discrete choice experiment (DCE) was designed to elicit the views of HCPs regarding hexavalent vaccines. The key attributes of injection devices were identified through a focused literature search and interviews with HCPs. Five key attributes, each with two or three levels were described which included: type of device, experience of this hexavalent vaccine on the German market, preparation time, probability of handling errors, and dosage errors. Physicians (n=150) and nurses (n=150) who administered hexavalent vaccines in Germany completed the survey. Choice data were analyzed using the conditional logit procedure. All attributes were significant and important independent influences on physicians' and nurses' choices. Reducing any "probability of dosage errors" was the most important attribute. Both physicians and nurses had a strong preference to reduce preparation time. All other things equal both groups also significantly preferred a fully liquid hexavalent vaccine. They also preferred vaccines that had been on the market for a few years compared to ones that had not (especially the physicians). Additional analyses explored participants' preferences in more detail through interaction terms. The DCE choice data provide useful insights into how HCPs view each aspect of the vaccination device. Overall, the HCPs preferred fully liquid vaccines. The survey also highlighted the importance of handling and dosage errors, reducing preparation time, and also experience of the HCPs with the use of a vaccine. The survey work included physicians and nurses and explored their views separately.

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

BACKGROUND: Utility 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. OBJECTIVE: To 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. METHODS: Data 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. RESULTS: The 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. CONCLUSIONS: The 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.

Patient preferences and willingness to pay for innovations in intermittent self-catheters.

BACKGROUND: Intermittent catheterization is the gold standard for bladder management in Europe in people with spinal cord injuries. The aim of the present study was to identify and investigate individuals' preferences regarding intermittent self-catheterization (ISC) devices and furthermore investigate the willingness to pay for attributes in ISC devices in the UK, France, and the Netherlands. METHODS: A discrete choice experiment survey was conducted to evaluate the patients' perceived value of catheter features. Attributes were selected based upon a literature review of the most important characteristics of catheters and the survey was developed and validated with input from patients and medical experts. Data were analyzed using the conditional logit model whereby the coefficients obtained from the model provided an estimate of the (log) odds ratios of preference for attributes. Willingness to pay was estimated for all levels of the attributes. RESULTS: Two-hundred and eighty-three participants completed the questionnaire and were included in data analysis. Risk of infection had the highest odds ratios as preferred important attribute for all three countries followed by ease of insertion. "Pre-coated catheters" was found to be valued as the most preferred coating technology across all countries. Out of pocket cost was a significant influence on patients' choice. CONCLUSION: Users of ISC perceive the value of convenience (size of catheter), ease of insertion, and reduced risk of infection as the most important features attached to an intermittent catheter. These results are applicable both for the "classic" ISC user as well as for another broad group of catheter dependent individuals.

To explore preferences and willingness to pay for attributes regarding stoma appliances amongst patients in the UK, France and Germany.

OBJECTIVES: To explore patient preferences regarding stoma appliances in the UK, France and Germany and to estimate willingness to pay (WTP) for attributes of stoma appliances. RESEARCH DESIGN AND METHODS: A discrete choice (DCE) survey was developed based on published literature, attributes of current available appliances and qualitative interviews with patients from the UK (N = 3), France (N = 2) and Germany (N = 2). Members from a patient panel in the UK, France and Germany were asked to participate in the DCE survey and to fill out two quality of life (QoL) questionnaires. Data were analyzed using the conditional logit model whereby the coefficients obtained from the model provided an estimate of the (log) odds ratios (ORs) of preference for attributes. WTP was estimated for each level of a given identified attribute. RESULTS: Seven key attributes were identified for the DCE survey: comfort and elastic flexibility, skin problems, early detection of leakage, leakage, filter performance, service/help after hospital discharge and out-of-pocket cost. A total of 415 participants (166 patients in UK, 99 in France, and 150 in Germany) completed the questionnaires. All attributes were significant predictors of choice. The two most important drivers of preference were the attributes comfort and elastic flexibility and skin problems which resulted in high WTP values. Appliances which were able to detect episodes of leakage were also of high importance to participants' appliances. CONCLUSIONS: The results show the importance of different attributes of stoma appliances for patients. Improving comfort and elastic flexibility, and risk of skin problems were the most important aspects of appliances. The WTP values indicate the value people place on improvement in each attribute of appliances.

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.

Psychometric evaluation of the Sinonasal Outcome Test-16 and activity impairment assessment in acute bacterial sinusitis.

OBJECTIVE: To validate the Sinonasal Outcome Test-16 and Activity Impairment Assessment in patients with acute bacterial sinusitis. STUDY DESIGN: Data were used from a phase III clinical trial designed to evaluate the efficacy and safety of moxifloxacin 400 mg once daily for 5 consecutive days in the treatment of acute bacterial sinusitis. The psychometric properties and factor structure of the 2 measures were assessed. SETTING: Participants were given the measures to self-complete using either a telephone voice response system or a paper-and-pencil format. SUBJECTS AND METHODS: Three hundred seventy-four patients with acute bacterial sinusitis were used in the analysis. Patients received either a placebo or 400 mg moxifloxacin once daily. Patients were then reviewed at test of cure and follow-up. All analyses were conducted on a combined sample of placebo and active treatment patients. RESULTS: The Sinonasal Outcome Test-16 was associated with minimal missing data at baseline but a higher proportion by test of cure. There was no evidence of floor or ceiling effects and no significant skew. The Activity Impairment Assessment also had low missing data at baseline and no obvious floor or ceiling effects, but the data were not normally distributed. Both measures had good internal consistency. Convergent and divergent validity as well as sensitivity and the minimally important difference are also reported. CONCLUSION: The measures both have good psychometric properties and are suitable for use with patients with acute bacterial sinusitis. Both instruments are sensitive. The minimal important difference estimates for the Sinonasal Outcome Test-16 are quite high but are similar to estimates reported previously.

Reliability and validity of the Personal and Social Performance scale in patients with schizophrenia.

OBJECTIVE: The construct validity and test-retest reliability of the Personal and Social Performance (PSP) scale were used to assess social functioning in a cohort of ethnically diverse UK patients with schizophrenia. METHODS: A total of 73 patients with schizophrenia took part in the study. At baseline, the PSP, two symptomatology scales and two other functioning scales were administered. A subset of the sample (N=40) took part in a retest where the Clinical Global Impression-Severity (CGI-S) and PSP scales were administered 8-10 days later. RESULTS: PSP significantly correlated with all other measures, Positive and Negative Syndrome Scale (PANSS), Global Assessment of Functioning (GAF) and Quality of Life Scale (QLS) at baseline (p<0.02) and with CGI-S at follow-up (p<0.01). In addition, the PSP scale was moderately sensitive to the severity of illness. Test-retest reliability for the PSP score was 0.45 and the scale was able to discriminate between known groups (mild and severe patients). CONCLUSION: The PSP was easy to administer in this predominantly inpatient cohort and was moderately correlated with all other functioning measures tested. Due to patient homogeneity, the test-retest reliability statistic of the PSP was lower than that observed in other studies.

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.

Health state utilities for non small cell lung cancer.

BACKGROUND: Existing reports of utility values for metastatic non-small cell lung cancer (NSCLC) vary quite widely and are not all suitable for use in submissions in the UK. The aim of this study was to elicit UK societal based utility values for different stages of NSCLC and different grade III-IV toxicities commonly associated with chemotherapy treatments. Toxicities included neutropenia, febrile neutropenia, fatigue, diarrhoea, nausea and vomiting, rash and hair loss. METHODS: Existing health state descriptions of metastatic breast cancer were revised to make them suitable as descriptions of metastatic NSCLC patients on second-line treatment. The existing health states were used in cognitive debrief interviews with oncologists (n = 5) and oncology specialist nurses (n = 5). Changes were made as suggested by the clinical experts. The resulting health states (n = 17) were piloted and used in a societal based valuation study (n = 100). Participants rated half of the total health states in a standard gamble interview to derive health state utility scores. Data were analysed using a mixed model analysis. RESULTS: Each health state described the symptom burden of disease and impact on different levels of functioning (physical, emotional, sexual, and social). The disutility related to each disease state and toxicity was estimated and were combined to give health state values. All disease states and toxicities were independent significant predictors of utility (p < 0.001). Stable disease with no toxicity (our base state) had a utility value of 0.653. Utility scores ranged from 0.673 (responding disease with no toxicity) to 0.473 for progressive disease. CONCLUSION: This study reflects the value that society place on the avoidance of disease progression and severe toxicities in NSCLC.