Evaluating preference weights for the Asthma Symptom Utility Index (ASUI) across countries

BACKGROUND: The Asthma Symptom Utility Index (ASUI) is a preference-based outcome measure used in US clinical trials and cost-effectiveness studies for asthma. This study evaluated ASUI preference weights in Europe to determine whether the multi-attribute utility function, based on preferences from a US population, is generalizable across countries. METHODS: Data were collected from ninety asthma patients from Italy, France, and the United Kingdom using the Asthma Control Questionnaire, the Asthma Quality of Life Questionnaire, and the ASUI. Subjects rated their preferences for 10 asthma health states using a visual analogue scale (VAS) and a standard gamble (SG) interview. RESULTS: All multi-symptom states showed statistically significant differences (p < 0.001) between countries in mean VAS scores. Mean SG utility scores between the US and France and the US and Italy demonstrated statistically significant differences (p < 0.001) for three states: severe wheeze; moderate cough and wheeze; and moderate cough and dyspnea. Because of these differences, the multi-attribute utility functions derived within countries were somewhat different. Despite these differences, country-specific algorithms captured a similar rank ordering of patients by disease severity, were strongly correlated (r = 0.971 to 0.995), and demonstrated similar relationships with symptom and AQLQ scores. CONCLUSION: Results of this study suggest that the ASUI may be a complementary patient-reported outcome for clinical studies and may be useful for applications in cost-effectiveness studies comparing different asthma treatments

Development of the PREMature Infant Index (PREMII™), a clinician-reported outcome measure assessing functional status of extremely preterm infants

Background: Comprehensive measures to evaluate the effectiveness of medical interventions in extremely preterm infants are lacking. Although length of stay is used as an indicator of overall health among preterm infants in clinical studies, it is confounded by nonmedical factors (e.g. parental readiness and availability of home nursing support). Objectives: To develop the PREMature Infant Index (PREMII™), an electronic content-valid clinician-reported outcome measure for assessing functional status of extremely preterm infants (<28 weeks gestational age) serially over time in the neonatal intensive care unit. We report the development stages of the PREMII, including suggestions for scoring. Methods: We developed the PREMII according to US Food and Drug Administration regulatory standards. Development included five stages: (1) literature review, (2) clinical expert interviews, (3) Delphi panel survey, (4) development of items/levels, and (5) cognitive interviews/usability testing. Scoring approaches were explored via an online clinician survey. Results: Key factors reflective of functional status were identified by physicians and nurses during development of the PREMII, as were levels within each factor to assess functional status. The resulting PREMII evaluates eight infant health factors: respiratory support, oxygen administration, apnea, bradycardia, desaturation, thermoregulation, feeding, and weight gain, each scored with three to six gradations. Factor levels are standardized on a 0–100 scale; resultant scores are 0–100. No usability issues were identified. The online clinician survey identified optimal scoring methods to capture functional status at a given time point. Conclusions: Our findings support the content validity and usability of the PREMII as a multifunction outcome measure to assess functional status over time in extremely preterm infants. Psychometric validation is ongoing

Psychometric evaluation of the Osteoporosis Patient Treatment Satisfaction Questionnaire (OPSAT-Q™), a novel measure to assess satisfaction with bisphosphonate treatment in postmenopausal women

BACKGROUND: The Osteoporosis Patient Satisfaction Questionnaire (OPSAT-Q) is a new measure of patient satisfaction with bisphosphonate treatment for osteoporosis. The objective of this study was to evaluate the psychometric characteristics of the OPSAT-Q. METHODS: The OPSAT-Q contains 16 items in four subscales: Convenience, Confidence with Daily Activities, Side Effects, and Overall Satisfaction. All four subscale scores and an overall composite satisfaction score (CSS) can be computed. The OPSAT-Q, Osteoporosis Targeted Quality of Life (OPTQoL), and sociodemographic/clinical questionnaires, including 3 global items on convenience, functioning and side effects, were self-administered to women with osteoporosis or osteopenia recruited from four US clinics. Analyses included item and scale performance, internal consistency reliability, reproducibility, and construct validity. Reproducibility was measured using the intraclass correlation coefficient (ICC) via a follow-up questionnaire completed by participants 2 weeks post baseline. RESULTS: 104 women with a mean age of 65.1 years participated. The majority were Caucasian (64.4%), living with someone (74%), and not currently employed (58.7%). 73% had osteoporosis and 27% had osteopenia. 80% were taking weekly bisphosphonates and 18% were taking daily medication (2% missing data). On a scale of 0–100, individual patient subscale scores ranged from 17 to 100 and CSS scores ranged from 44 to 100. All scores showed acceptable internal consistency reliability (Cronbach's alpha > 0.70) (range 0.72 to 0.89). Reproducibility ranged from 0.62 (Daily Activities) to 0.79 (Side Effects) for the subscales; reproducibility for the CSS was 0.81. Significant correlations were found between the OPSAT-Q subscales and conceptually similar global measures (p < 0.001). CONCLUSION: The findings from this study confirm the validity and reliability of the OPSAT-Q and support the proposed composition of four subscales and a composite score. They also support the use of the OPSAT-Q to examine the impact of bisphosphonate dosing frequency on patient satisfaction

Encuesta de la perspectiva del cuidador en el TDAH (CAPPA): Analisis de las características sociodemográficas y clínicas, el uso del tratamiento y el impacto del TDAH en Europa

Background Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder that affects the lives of patients and their families. The Caregiver Perspective on Paediatric ADHD (CAPPA) survey was conducted to evaluate the burden associated with ADHD in Europe and to identify unmet needs. Here, we describe sociodemographic and clinical characteristics, treatment use and impact of ADHD. Methods The cross-sectional web-based CAPPA survey was fielded in 10 European countries among caregivers of children/adolescents (aged 6–17 years) with ADHD who were currently receiving or had received pharmacotherapy in the previous 6 months. Results Data on 3688 completed CAPPA surveys were evaluated. Children/adolescents were diagnosed with ADHD at a mean age of 6.9 years; 80% were male. Most children/adolescents (56%) had undergone behavioural therapy. Overall, 78% of children/adolescents currently received ADHD pharmacotherapy; high rates of atypical antipsychotic use were reported in some countries. Overall, 23% of children/adolescents had repeated a school year and 4% had been expelled recently. Most caregivers (68–88%) reported difficulty with schoolwork, social interactions/activities and family relationships, even when the child/adolescent was receiving ADHD medication. Almost one third (31%) of caregivers felt the need to change employment status despite their child/adolescent receiving ADHD medication in 53% of these cases. Limitations Information was reported by caregivers recruited through market research panels; reporting, recall and selection biases may be present. Conclusion Variation across Europe was observed in characteristics of caregivers and children/adolescents with ADHD, and treatment use. Even with medication, ADHD compromised or negatively impacted caregivers’ work and children/adolescents’ schoolwork, their social interactions and family relationships.hire Development, LLCDepto. de Medicina Legal, Psiquiatría y PatologíaFac. de MedicinaTRUEpu

Bipolar Disorder and Health-Related Quality of Life: Review of Burden of Disease and Clinical Trials

Bipolar disorder is a chronic psychiatric disorder with a variable course and significant impact on patients' social, occupational, and general functioning and wellbeing. Although there are effective pharmaceutical and psychosocial interventions for patients with bipolar disorder, many patients receive poor-quality care. Prospective longitudinal studies demonstrate that less than half of bipolar disorder patients have a good long-term response to treatment, long-term outcome is highly variable, and many patients do not fully recover. There is substantial evidence that bipolar disorder is associated with significant impairment to functioning and wellbeing. However, few clinical trials comparing treatments for bipolar disorder have incorporated health-related quality-of-life (HR-QOL) assessments. Existing studies suggest that, while treatment improves HR-QOL, there is limited evidence for differences between the mood stabilisers in terms of HR-QOL outcomes. Additional clinical trials are needed to evaluate patient-reported outcomes associated with the most frequently used pharmacological treatments to determine whether there are meaningful differences between treatments. There are challenges in measuring HR-QOL in patients with acute mania, and future studies should assess the psychometric qualities of HR-QOL instruments in these and other bipolar disorder patients. HR-QOL outcome data may be useful in informing psychiatrists, patients and patient family members of the effects of treatment for bipolar disorder on patients' everyday lives, functioning and wellbeing.Bipolar-disorders, Quality-of-life

Development of the pediatric daily ulcerative colitis signs and symptoms scale (DUCS): qualitative research findings

Abstract Background The purpose of this study is to develop patient-reported (PRO) and observer-reported (ObsRO) outcome measures of ulcerative colitis (UC) signs/symptoms in children aged 5–17 with mild/moderate UC. The daily ulcerative colitis signs and symptoms scale (DUCS) was developed in two phases. Phase I involved concept elicitation interviews with patients and healthcare providers, review of website posts and item generation. Phase II involved cognitive debriefing and assessment of usability and feasibility of the eDiaries. Participants were recruited from five US clinical sites, a research recruitment agency, and internet advertising. Thematic and content analysis was performed to identify concepts from Phase I. The Phase II cognitive debriefing interviews were analyzed iteratively to identify problems with clarity and relevance of eDiary content. The US Food and Drug Administration (FDA) also reviewed and provided feedback on the eDiaries. Results Phase I included 32 participants (22 remission; 10 active disease). Phase II included 38 participants (22 remission; 16 active disease). A core set of seven signs and symptoms emerged that were reported by at least 30% of the patients interviewed: abdominal pain, blood in stool, frequent stools, diarrhea, stool urgency, nighttime stools, and tiredness. Participant input influenced changes such as refinement of item wording, revision of graphics, and selection of response scales. Revisions suggested by FDA included simplifying the response scale and adding questions to capture symptoms during sleeping hours. Conclusions The findings of instrument development suggest that the DUCS PRO and ObsRO eDiaries are content-valid instruments for capturing the daily signs and symptoms of pediatric patients with mild to moderate UC in a clinical trial setting

Costs of Bipolar Disorder

Bipolar disorder is a chronic affective disorder that causes significant economic burden to patients, families and society. It has a lifetime prevalence of approximately 1.3%. Bipolar disorder is characterised by recurrent mania or hypomania and depressive episodes that cause impairments in functioning and health-related quality of life. Patients require acute and maintenance therapy delivered via inpatient and outpatient treatment. Patients with bipolar disorder often have contact with the social welfare and legal systems; bipolar disorder impairs occupational functioning and may lead to premature mortality through suicide. This review examines the symptomatology of bipolar disorder and identifies those features that make it difficult and costly to treat. Methods for assessing direct and indirect costs are reviewed. We report on comprehensive cost studies as well as administrative claims data and program evaluations. The majority of data is drawn from studies conducted in the US; however, we discuss European studies when appropriate. Only two comprehensive cost-of-illness studies on bipolar disorder, one prevalence-based and one incidence-based, have been reported. There are, however, several comprehensive cost-of-illness studies measuring economic burden of affective disorders including bipolar disorder. Estimates of total costs of affective disorders in the US range from $US30.4-43.7 billion (1990 values). In the prevalence-based cost-of-illness study on bipolar disorder, total annual costs were estimated at$US45.2 billion (1991 values). In the incidence-based study, lifetime costs were estimated at $US24 billion. Although there have been recent advances in pharmacotherapy and outpatient therapy, hospitalisation still accounts for a substantial portion of the direct costs. A variety of outpatient services are increasingly important for the care of patients with bipolar disorder and costs in this area continue to grow. Indirect costs due to morbidity and premature mortality comprise a large portion of the cost of illness. Lost workdays or inability to work due to the disease cause high morbidity costs. Intangible costs such as family burden and impaired health-related quality of life are common, although it has proved difficult to attach monetary values to these costs.Affective-disorders, Antipsychotics, Bipolar-disorders, Cost-of-illness, Mood-stabilisers, Pharmacoeconomics

Using qualitative interviews to identify patient-reported clinical trial endpoints and analyses that are the most meaningful to patients with advanced breast cancer.

BackgroundDesigning clinical trials with the emphasis on the patient-centered approach and focusing on clinical outcomes that are meaningful to patients is viewed as a priority by drug developers, regulatory agencies, payers, clinicians, and patients. This study aimed to capture information on clinical trial endpoints that would be most important and relevant for patients with advanced breast cancer, based on patient-reported outcomes.MethodsPatients with either advanced triple-negative breast cancer [TNBC] and a maximum of two lines of systemic therapy or hormone receptor-positive/human epidermal growth factor receptor 2-negative [HR+/HER2-] breast cancer and a maximum of three lines of systemic therapy, participated in semi-structured concept elicitation interviews. Concept saturation was assessed. A sign, symptom, or impact was defined as "salient" if mentioned by ≥ 60% of participants, with an average bother rating of ≥ 5 (0-10 Scale). Participants were also asked about treatment priorities and to evaluate hypothetical scenarios showing different health-related functioning and quality-of-life treatment outcomes, using graphical representations.ResultsThirty-two participants (97% women; aged 29+ years) with TNBC (n = 17) or HR+/HER2- breast cancer (n = 15) provided generally similar reports on symptom experience, with fatigue and pain being most salient, though importance of certain treatment-related symptoms varied between the two groups. Patients reported consistent perspectives on the importance of treatment outcomes: when considering a new treatment, they prioritized efficacy of the therapy, acceptable tolerability, stability, predictability of symptoms over time, and the duration of preserved health-related quality of life and physical functioning. The meaningful difference in preserved physical functioning was 2-3 months for 46% of participants with TNBC, whereas for most participants with HR+/HER2- breast cancer it started from 6-7 months. Both groups of participants found it easier to accept some toxicity at the beginning of therapy if it was followed by improvement, as opposed to improvement followed by deterioration.ConclusionThe results may help to inform the design of patient-centered clinical trials, to interpret health-related quality of life and/or patient-reported outcomes, and to optimize care for patients with advanced breast cancer