Organ damage in patients treated with belimumab versus standard of care: a propensity score-matched comparative analysis.

OBJECTIVES: The study (206347) compared organ damage progression in patients with systemic lupus erythematosus (SLE) who received belimumab in the BLISS long-term extension (LTE) study with propensity score (PS)-matched patients treated with standard of care (SoC) from the Toronto Lupus Cohort (TLC). METHODS: A systematic literature review identified 17 known predictors of organ damage to calculate a PS for each patient. Patients from the BLISS LTE and the TLC were PS matched posthoc 1:1 based on their PS (±calliper). The primary endpoint was difference in change in Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI) score from baseline to 5 years. RESULTS: For the 5- year analysis, of 567 (BLISS LTE n=195; TLC n=372) patients, 99 from each cohort were 1:1 PS matched. Change in SDI score at Year 5 was significantly lower for patients treated with belimumab compared with SoC (-0.434; 95% CI -0.667 to -0.201; p<0.001). For the time to organ damage progression analysis (≥1 year follow-up), the sample included 965 (BLISS LTE n=259; TLC n=706) patients, of whom 179 from each cohort were PS-matched. Patients receiving belimumab were 61% less likely to progress to a higher SDI score over any given year compared with patients treated with SoC (HR 0.391; 95% CI 0.253 to 0.605; p<0.001). Among the SDI score increases, the proportion of increases ≥2 was greater in the SoC group compared with the belimumab group. CONCLUSIONS: PS-matched patients receiving belimumab had significantly less organ damage progression compared with patients receiving SoC.

Test for Respiratory and Asthma Control in Kids (TRACK): clinically meaningful changes in score.

BACKGROUND: The Test for Respiratory and Asthma Control in Kids (TRACK) is the first validated questionnaire to assess respiratory and asthma control exclusively in young children. OBJECTIVE: We sought to determine the minimally important difference (MID) for interpreting meaningful changes in individual patients' TRACK scores. METHODS: In this prospective, nonrandomized, longitudinal study conducted at 20 US pediatric sites, TRACK was administered at 2 separate clinic visits (4-6 weeks apart) to caregivers of children aged less than 5 years with symptoms consistent with asthma. Anchor-based methods were used to determine the MID from mean score differences between patients based on multiple criteria measures: physician guidelines-based respiratory control rating, physician-recommended changes to therapy, episodes of symptoms lasting more than 24 hours in the past 3 months, oral corticosteroid use for respiratory tract illnesses in the past year, physician-assessed change in control status at follow-up, and caregiver-reported change in respiratory status. The MID also was determined from distribution-based methods. RESULTS: TRACK scores were assessed at baseline (426 caregivers) and follow-up (396 caregivers). Mean differences in TRACK scores between patients differing on criteria measures ranged from 3.4 to 16.4 points (mean, 11.1 points). Distribution-based techniques confirmed these findings. Based on logistic regression analyses, scoring 10 or more points less than 80 on TRACK was associated with an approximately 2-fold increased odds of having uncontrolled asthma or respiratory symptoms. CONCLUSION: Changes in TRACK scores of 10 or more points represent clinically meaningful changes in respiratory control status in individual young children with respiratory symptoms consistent with asthma and should alert health care providers to re-evaluate asthma management.

Test for respiratory and asthma control in kids (TRACK): a caregiver-completed questionnaire for preschool-aged children.

BACKGROUND: A validated questionnaire is needed to monitor respiratory control in preschool-aged children. OBJECTIVE: We sought to develop and validate a caregiver-completed questionnaire that measures respiratory control in young children. METHODS: A 33-item questionnaire that included asthma impairment and risk items was administered to 486 caregivers of children aged younger than 5 years with a current, recent, or past history of respiratory symptoms. Stepwise regression was used to select a subset of items with the greatest discriminant validity in relation to guidelines-defined asthma control in a random two-thirds development sample. Reliability, validity, and ability to screen for respiratory control problems were tested in development and validation samples (remaining one-third sample). RESULTS: The content of the 5 items selected, the Test for Respiratory and Asthma Control in Kids (TRACK), included frequency of respiratory symptoms (wheeze, cough, shortness of breath), activity limitation, and nighttime awakenings in the past 4 weeks; rescue medication use in the past 3 months; and oral corticosteroid use in the previous year. Reliability was greater than 0.70 in both samples. ANOVA showed that mean scores differed significantly (P < .001) in the expected direction across both samples for 3 levels of guidelines-based respiratory control, physician-recommended change in therapy, and symptom status. In the development and validation samples, screening analyses revealed areas under the receiver operating characteristic curve of 0.88 and 0.82, respectively; control status was correctly classified in 81% and 78% of cases. CONCLUSION: TRACK is a valid, easy-to-administer, caregiver-completed questionnaire of respiratory control in preschool-aged children with symptoms consistent with asthma.

Comparative analysis of long-term organ damage in patients with systemic lupus erythematosus using belimumab versus standard therapy: a post hoc longitudinal study.

OBJECTIVE: Long-term extension (LTE) studies of belimumab in SLE do not include a comparator arm, preventing comparisons between belimumab plus standard therapy and standard therapy alone for organ damage accrual. Propensity score matching can be used to match belimumab-treated patients from LTE studies with standard therapy-treated patients from observational cohort studies. This analysis was designed to compare organ damage progression between treatment groups (belimumab plus standard therapy vs standard therapy alone) in patients with SLE with ≥5 years of follow-up, reproducing our previous study with more generalisable data. METHODS: This exploratory post hoc analysis used a heterogeneous population of US and non-US patients receiving monthly intravenous belimumab from pooled BLISS LTE trials (BEL112234/NCT00712933) and standard therapy-treated patients from the Toronto Lupus Cohort. Sixteen clinical variables were selected to calculate the propensity score. RESULTS: The 592 LTE and 381 Toronto Lupus Cohort patients were highly dissimilar across the 16 variables; an adequately balanced sample of 181 LTE and 181 matched Toronto Lupus Cohort patients (mean bias=3.7%) was created using propensity score matching. Belimumab treatment was associated with a smaller increase in Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI) over 5 years than standard therapy alone (mean treatment difference=-0.453 (95% CI -0.646 to -0.260); p<0.001). Patients treated with belimumab were 60% less likely to progress to a higher SDI score over any given year of follow-up, compared with standard therapy alone (HR (95% CI) 0.397 (0.275 to 0.572); p<0.001). CONCLUSION: Using propensity score matching, this highly heterogeneous sample was sufficiently matched to the Toronto Lupus Cohort, suggesting that patients treated with intravenous belimumab may have reduced organ damage progression versus standard therapy alone. This analysis of a large and diverse pooled SLE population was consistent with our previously published US-focused study.

Efficacy and tolerability of budesonide/formoterol in one hydrofluoroalkane pressurized metered-dose inhaler in patients with chronic obstructive pulmonary disease: results from a 1-year randomized controlled clinical trial.

BACKGROUND: Combination therapy with a long-acting bronchodilator and an inhaled corticosteroid (ICS) is recommended in patients with chronic obstructive pulmonary disease (COPD) who have frequent exacerbations. The efficacy and tolerability of the combination of budesonide/formoterol have been demonstrated in patients with COPD when administered via the dry powder inhaler (DPI) in a 1-year study and when administered via the hydrofluoroalkane (HFA) pressurized metered-dose inhaler (pMDI) in a 6-month study. OBJECTIVE: This study assessed the long-term efficacy and tolerability of budesonide/formoterol HFA pMDI in patients with moderate to very severe COPD. METHODS: This was a 12-month, randomized, double-blind, double-dummy, parallel-group, active- and placebo-controlled, multicentre study (NCT00206167) of 1, 964 patients aged >or =40 years with moderate to very severe COPD conducted from 2005 to 2007 at 237 sites in the US, Europe and Mexico. After 2 weeks of treatment based on previous therapy (ICSs, short-acting bronchodilators allowed), patients received one of the following treatments twice daily: budesonide/formoterol pMDI 160/4.5 microg x two inhalations (320/9 microg); budesonide/formoterol pMDI 80/4.5 microg x two inhalations (160/9 microg); formoterol DPI 4.5 microg x two inhalations (9 microg); or placebo. MAIN OUTCOME MEASURES: The co-primary efficacy variables were pre-dose forced expiratory volume in 1 second (FEV1) and 1-hour post-dose FEV1. . RESULTS: Budesonide/formoterol 320/9 microg demonstrated greater improvements in pre-dose FEV1 versus formoterol (p = 0.008), and both budesonide/formoterol doses demonstrated greater improvements in 1-hour post-dose FEV1 versus placebo (p < 0.001). The rate of COPD exacerbations was lower in both budesonide/formoterol groups compared with formoterol and placebo (p

Better Asthma Management with Advanced Technology: Creation of an Asthma Utilization Rx Analyzer (AURA) Tool.

With nearly 23 million people affected by asthma each year, optimizing care among patients with persistent disease is a constant challenge for health care providers. The Asthma Utilization Rx Analyzer (AURA) tool enables health plan managers to evaluate quality and resource utilization for its members with asthma by analyzing medical and pharmacy claims. Customizable quality measures allow users of the tool to generate results from specific plans in order to optimize asthma disease management.

Long-Term Impact of Belimumab on Health-Related Quality of Life and Fatigue in Patients With Systemic Lupus Erythematosus: Six Years of Treatment.

OBJECTIVE: To report long-term health-related quality of life (HRQoL) and fatigue outcomes in patients with systemic lupus erythematosus (SLE) receiving belimumab. METHODS: Patients with SLE who completed the Study of Belimumab in Subjects with SLE 76-week trial (BLISS-76) were enrolled in this continuation study (BEL112233 [ClinicalTrials.gov identifier: NCT00724867]). The belimumab groups continued to receive the same dose (1 mg/kg or 10 mg/kg) intravenously. After March 2011, all patients received belimumab 10 mg/kg every 28 days plus standard therapy. The placebo group switched to belimumab 10 mg/kg. HRQoL and fatigue assessments included the Short Form 36 (SF-36) health survey and the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue subscale. Post hoc subgroup analyses (BEL206350) assessed clinical characteristics associated with improved HRQoL and fatigue. RESULTS: Of the 268 patients enrolled, 140 completed the study. Patients receiving long-term belimumab treatment reported continued improvements in HRQoL and fatigue. At study year 6, the mean ± SD SF-36 physical component summary (PCS) score and the mental component summary (MCS) score increased from 37.0 ± 9.9 at baseline to 41.7 ± 10.0 (mean ± SD change 4.8 ± 9.4) and from 44.3 ± 11.3 to 47.0 ± 11.6 (mean ± SD change 2.7 ± 11.3) for the PCS and MCS, respectively, exceeding the minimum clinically important difference (MCID) for improvement (2.5 units). The mean ± SD FACIT-Fatigue score exceeded the MCID of 4 at study years 1-5; at study year 6, the mean ± SD change was 3.7 ± 11.8. Statistically significant associations were observed between parent trial treatment groups and change from baseline in PCS, MCS, and FACIT-Fatigue scores (P < 0.01). CONCLUSION: Long-term control of SLE disease activity with belimumab plus standard therapy translates into meaningful improvements in patient-reported fatigue and HRQoL.

Efficacy and safety of budesonide and formoterol in one pressurized metered-dose inhaler in patients with moderate to very severe chronic obstructive pulmonary disease: results of a 6-month randomized clinical trial.

BACKGROUND: The combination of an inhaled corticosteroid (ICS) and a long-acting bronchodilator is recommended in the treatment of patients with chronic obstructive pulmonary disease (COPD) who have frequent exacerbations. Budesonide/formoterol dry powder inhaler (DPI) has demonstrated efficacy and tolerability in patients with COPD. OBJECTIVE: To evaluate the efficacy and tolerability of budesonide/formoterol administered via one hydrofluoroalkane pressurized metered-dose inhaler (pMDI) in patients with COPD. METHODS: This was a 6-month, randomized, double-blind, double-dummy, placebo-controlled, parallel-group, multicentre study (NCT00206154) of 1704 patients aged > or =40 years with moderate to very severe COPD conducted in 194 centres in the US, Czech Republic, the Netherlands, Poland and South Africa. After 2 weeks of treatment based on previous therapy (ICSs and short-acting bronchodilators allowed during the run-in period), patients received one of the following treatments administered twice daily: budesonide/formoterol pMDI 160/4.5 microg x two inhalations (320/9 microg); budesonide/formoterol pMDI 80/4.5 microg x two inhalations (160/9 microg); budesonide pMDI 160 microg x two inhalations (320 microg) plus formoterol DPI 4.5 microg x two inhalations (9 microg); budesonide pMDI 160 microg x two inhalations (320 microg); formoterol DPI 4.5 microg x two inhalations (9 microg); or placebo. MAIN OUTCOME MEASURES: The co-primary efficacy variables were pre-dose forced expiratory volume in 1 second (FEV(1)) and 1-hour post-dose FEV(1). RESULTS: Budesonide/formoterol 320/9 microg demonstrated significantly greater improvements in pre-dose FEV(1) versus formoterol (p = 0.026; pre-specified primary comparator) and 1-hour post-dose FEV(1) versus budesonide (p < 0.001; pre-specified primary comparator); budesonide/formoterol 160/9 microg demonstrated significantly greater improvements versus budesonide (p < 0.001) for 1-hour post-dose FEV(1) but not versus formoterol for pre-dose FEV(1). Dyspnoea (measured using the Breathlessness Diary) and health-related quality-of-life (HR-QOL) scores (based on the St George's Respiratory Questionnaire total score) were significantly improved with both dosage strengths of budesonide/formoterol compared with budesonide, formoterol and placebo (p < or = 0.044 for all). Although not powered a priori for comparisons, the number of exacerbations per patient-treatment year requiring treatment with oral corticosteroids and/or hospitalization was numerically (20-25%) lower with the budesonide-containing treatments (0.710-0.884) versus formoterol (1.098) and placebo (1.110). This result was driven by the exacerbations requiring treatment with oral corticosteroids (79-120 events). The number of exacerbations resulting in hospitalization was very low across treatment groups (11-22); the number per patient-treatment year was significantly different for budesonide/formoterol 320/9 microg (0.158) versus other treatment groups (0.081-0.108) except budesonide/formoterol 160/9 microg (0.139), and for budesonide/formoterol 160/9 microg versus formoterol (0.081) [p < or = 0.05]. All treatments were generally well tolerated. The incidence of individual non-fatal serious adverse events was similar across all treatment groups, except COPD, which was highest in the budesonide/formoterol 320/9 microg group (6.1%) and lowest in the budesonide (3.6%) and formoterol (3.9%) groups, with a range of 4.3-4.6% in the budesonide/formoterol 160/9 microg, budesonide plus formoterol and placebo groups. Budesonide/formoterol had a safety profile comparable with that of the monocomponents and placebo. There was no increase in the incidence of pneumonia in the active treatment groups relative to placebo. CONCLUSIONS: Budesonide/formoterol pMDI 320/9 microg demonstrated significantly greater efficacy for pulmonary function on both co-primary endpoints versus the pre-specified comparators (formoterol DPI 9 microg for pre-dose FEV(1) and budesonide pMDI 320 microg for 1-hour post-dose FEV(1)). Budesonide/formoterol pMDI 160/9 microg demonstrated significantly greater efficacy for 1-hour post-dose FEV(1) versus budesonide pMDI 320 microg. Dyspnoea scores and HR-QOL were significantly improved with both budesonide/formoterol pMDI dosage strengths versus both monocomponents and placebo. Both budesonide/formoterol pMDI dosage strengths were well tolerated relative to the monocomponents and placebo.

Clinical and Cost Burden of Primary Sjögren's Syndrome: Descriptive Analysis Using a US Administrative Claims Database.

Background: Current knowledge of the disease burden of primary Sjögren's syndrome (pSS) is limited. Objectives: The primary objective of this study was to describe the demographic and clinical characteristics of patients with pSS. The secondary objective was to describe the treatment patterns and healthcare resource utilization of patients with pSS. Furthermore, clinical characteristics of interest were described and the proportions of patients with glandular versus extra-glandular disease were reported. Methods: This was a retrospective cohort study (HO-15-16077) conducted in the US Truven MarketScan Commercial database. We report descriptive data from employees and their families, as covered by employer-sponsored medical insurance. The primary cohort comprised patients with pSS (with ≥1 diagnosis of sicca syndrome prior to January 1, 2013), with continuous enrollment for ≥24 months (January 1, 2012-December 31, 2013). Patients with conditions mimicking sicca symptoms not due to SS were excluded, as were those with connective tissue disease that may have suggested secondary SS. To compare the healthcare burden of patients with and without sicca symptoms, a 1:1 matched comparator population comprising subjects without a previous diagnosis of sicca syndrome (sicca-free) was also identified. Results: There were 12 717 eligible patients in the primary cohort. The majority (86%) was female and the mean age was 51 years. Overall, 60.7% of patients had claims associated with pSS extra glandular disease manifestations. These patients experienced a higher disease burden, and more commonly reported pain, fatigue or insomnia and any combination of pain, fatigue or insomnia (41.3%) compared with patients with glandular disease only (12.4%). Patients in the primary cohort incurred greater annual healthcare service costs (1.6 times greater, all causes) and healthcare resource utilization compared with the sicca-free comparator cohort. Patients with extra glandular disease also incurred greater average annual costs (2.9 times) contributing to ≥2 times/year more resource use for outpatient services than patients with glandular disease only. Conclusion: Patients with pSS experience a high disease burden despite treatment. This study provides novel insights in to the extent of the burden on healthcare resources among patients with pSS, in particular for patients with extra-glandular disease manifestations, when compared with sicca-free subjects.

Patient Evaluation of Satisfaction and Outcomes with an Autoinjector for Self-Administration of Subcutaneous Belimumab in Patients with Systemic Lupus Erythematosus.

OBJECTIVE: This study assessed patient experiences of using an autoinjector device to self-administer subcutaneous belimumab for the treatment of systemic lupus erythematosus (SLE). Satisfaction, ease and convenience of use, and confidence with the device were assessed, in addition to overall experience with belimumab. METHODS: This cross-sectional study was conducted among patients who completed a phase IIb open-label, multi-dose usability, tolerability, and safety study of subcutaneous belimumab (NCT02124798), in which patients receiving intravenous belimumab or subcutaneous belimumab using a prefilled syringe were switched to eight weekly self-administered doses of subcutaneous belimumab using the autoinjector. This follow-up study comprised an online/paper questionnaire and qualitative telephone interviews. RESULTS: In total, 43 patients receiving belimumab completed the questionnaire, 21 of whom also completed a follow-up telephone interview. Qualitative interviews indicated that 17 of 21 (81%) patients had a positive experience using the autoinjector; all patients considered the autoinjector to be convenient. Of the 42 patients who switched from intravenous belimumab to the autoinjector, 32 (76%) expressed a preference for the autoinjector over intravenous administration; reasons included convenience, time saved, cost, and reduced injection pain. The most commonly reported disadvantage of the autoinjector was injection discomfort (n = 5 [24%]; qualitative interview). Compared with intravenous administration, the autoinjector improved ability to work (17 of 29 [59%] of those employed) and carry out daily activities (40%). CONCLUSION: Patients with SLE reported high levels of satisfaction with the belimumab autoinjector and preferred the autoinjector to intravenous administration, citing advantages such as time saved, cost, and improved ability to work and carry out daily activities.

An indirect comparison of intravenous and subcutaneous belimumab efficacy in patients with SLE and high disease activity.

AIM: To compare the efficacy of intravenous (IV) and subcutaneous (SC) belimumab plus standard therapy in patients with active, autoantibody-positive systemic lupus erythematosus and high disease activity (HDA). PATIENTS & METHODS: An indirect treatment comparison using patient-level data of patients with HDA from three belimumab IV Phase III randomized controlled trials (BLISS-52 [BEL110751]; BLISS-76 [BEL110752]; Northeast Asia study [BEL113750]) and one belimumab SC randomized controlled trial (BLISS-SC [BEL112341]). RESULTS: Similar efficacy results were identified between the belimumab formulations and greater improvements in efficacy endpoints were observed for both formulations compared with placebo. CONCLUSION: This indirect treatment comparison provides further evidence of the efficacy of belimumab IV and SC in patients with systemic lupus erythematosus and HDA, compared with standard therapy.

Association between common asthma therapies and recurrent asthma exacerbations in children enrolled in a state Medicaid plan.

PURPOSE: To evaluate the effectiveness of budesonide inhalation suspension relative to other common asthma therapies in a high-risk population, a study was conducted to compare the risk of having a repeat asthma-related hospitalization or emergency department (ED) visit in a Medicaid population of children; the relationship between asthma medication adherence level and repeat asthma hospitalizations or ED visits was also evaluated. METHODS: Children eight years of age or younger, with a hospitalization or ED visit for asthma between January 1999 and June 2001 (index event), were identified in a Florida Medicaid database. Claims data for each child were examined 12 months before and after the index event. Cox proportional hazards regression was used to model the risk of subsequent asthma exacerbation according to the asthma medication received during the first 30 days after the index event. Logistic regression was used to model the relationship between medication adherence as measured by the medication possession ratio (MPR) and the likelihood of a subsequent asthma exacerbation. RESULTS: There were 10,976 children in the study. Patients who had a claim for budesonide inhalation suspension had a lower risk of a subsequent hospitalization or ED visit (hazard ratio, 0.55; 95% confidence interval, 0.41-0.76; p < 0.001) than patients who did not have budesonide inhalation suspension claims. Other controller medications were not associated with a reduction in the risk of subsequent asthma exacerbations. Adherence to medication was poor (a median MPR of 0.08 for budesonide inhalation suspension and a median MPR of 0.16 for any asthma controller medication). The odds of a repeat hospitalization or ED visit were significantly lower for children who were adherent to their asthma controller medication. CONCLUSION: Children with asthma and insured by Medicaid were at a high risk of repeat exacerbations leading to increased hospitalizations and ED visits. Treatment with budesonide inhalation suspension in the first 30 days after a hospitalization or ED visit for asthma was associated with a significant reduction in the risk of repeat asthma-related hospitalizations or ED visits during the following year. Children who were adherent to their asthma controller medication had significantly lower odds of having a subsequent asthma exacerbation.

Caregiver preferences for pediatric asthma treatment delivery systems.

This study was conducted to assess caregiver preferences for pediatric asthma treatment delivery systems. A total of 186 caregivers of children with asthma who ranged in age from 1 to 4 y completed a stated-preference questionnaire to assess the importance of specific treatment attributes in terms of caregivers' preferences, and to determine the percentages of caregivers who preferred specific treatments. Other outcomes assessed included caregivers' likelihood of adhering to pediatric asthma treatment. Most respondents (75%) preferred treatments that have been approved by the US Food and Drug Administration (FDA) in children as young as 12 mo, that require minimal effort in coordination and inhalation on the child's part, and that take up to 10 min to administer. A greater proportion of respondents indicated that they would be able to follow the physician's advice (increased likelihood of adherence) if given this treatment (54%) versus an alternative (35%). This difference did not achieve statistical significance, however. The conjoint analysis method used in this study enabled investigators to assess caregivers' relative preferences for specific attributes of various pediatric asthma treatments. Overall, FDA approval in children as young as 12 mo was the most preferred attribute. Caregiver preference for pediatric asthma treatment alternatives is an important consideration and should be a component of discussions between healthcare professionals and caregivers.

Patient- and Physician-reported Satisfaction With Systemic Lupus Erythematosus Treatment in US Clinical Practice.

PURPOSE: This two-part study comprised two descriptive, cross-sectional surveys to evaluate treatment satisfaction among patients with systemic lupus erythematosus (SLE) and their physicians from US clinical practices. The Lupus Plus Project (LPP; part one) involved belimumab-containing regimens; the Disease Specific Program (DSP; part two) included all treatments and was designed to build on the body of evidence from part one. METHODS: The LPP recruited patients receiving belimumab, and comprised 2 paper questionnaires: a patient self-completion questionnaire (PSC) and a patient record form (PRF) completed by the physician. The DSP enrolled patients with SLE receiving any treatment and comprised four parts: a PSC, a PRF completed by the physician after patient consultation, face-to-face physician interviews, and a workload form completed by the physicians to indicate their total SLE patient workload. The key objective of this study was to assess physician and patient satisfaction with current treatment. FINDINGS: From the PSCs, data regarding patient-reported satisfaction with current treatment were available for 263 patients who were receiving belimumab combination therapy (LPP) and 250 patients who were receiving non-belimumab treatment (DSP). The majority of patients (belimumab, 86.3% [227/263]; non-belimumab, 78.4% [196/250]) responded positively (at least "somewhat satisfied") when asked about current treatment satisfaction, as did physicians (belimumab, 82.9% [311/375]; non-belimumab, 74.3% [326/439]). In multivariate analysis, factors most strongly associated with patient-reported satisfaction for patients receiving belimumab were patient-reported improvements in leisure activities since taking belimumab (odds ratio [OR] = 4.66), physician-reported improvements in fatigue (OR = 3.72), patient-reported improvements in general symptoms (OR = 3.02), and pain/achiness (OR = 2.71). Physician satisfaction was associated with clinical outcome such as improvements in pain/achiness (OR = 6.16), fatigue (OR = 3.76), and patient-reported satisfaction with treatment frequency (OR = 3.91). In patients receiving other SLE treatments, dosing frequency of current treatment (OR = 3.64) and a reduction in fatigue severity (OR = 3.61) were most strongly associated with patient-reported satisfaction; physician satisfaction was most strongly associated with a reduction in fatigue (OR = 6.22) and current remission status (OR = 6.05). IMPLICATIONS: When considering SLE treatment satisfaction patients tend to consider impact on daily functioning, whereas physicians take into account a wider range of clinical outcomes; however, both strongly consider improvements in fatigue. These surveys provide insights into treatment satisfaction among prescribers and patients with SLE. GSK-ClinicalStudyRegister.com identifiers: GSK study 202146 [HO 15-15509] and 205086 [HO 15-16709].

Patients with Gout Treated with Conventional Urate-lowering Therapy: Association with Disease Control, Health-related Quality of Life, and Work Productivity.

OBJECTIVE: Implications of inadequate gout control were assessed through health-related quality of life (HRQOL) and work productivity of patients with gout adequately controlled while taking conventional urate-lowering therapy (ULT) for ≥ 3 months vs those whose gout was inadequately controlled. METHODS: Retrospective data were drawn from the Adelphi Disease Specific Programme (DSP), a cross-sectional survey of patients with gout in France, Germany, the United Kingdom, and the United States. Patients completed these questionnaires: EQ-5D (3L), Patient Reported Outcomes Measurement Information System (PROMIS) Health Assessment Questionnaire (HAQ), and Work Productivity and Activity Impairment. Inadequate control was defined as the most recent serum uric acid (SUA) level > 6 mg/dl (> 360 µmol/l) or ≥ 2 flares in the last 12 months; adequate control as SUA level ≤ 6 mg/dl (≤ 360 µmol/l) and 0 flares. Appropriate statistical tests were used to assess differences between groups. RESULTS: There were 836 (69%) inadequately and 368 (31%) adequately controlled gout cases. Mean age was 61 and 63 years and duration of current ULT was 32 and 57 months, respectively. Patients experiencing inadequate control reported significantly worse functioning and HRQOL, as measured by the EQ-5D (0.790 vs 0.877; difference: -0.087; p < 0.001) and PROMIS HAQ (13.21 vs 6.91; difference: 6.30; p < 0.001) scales. Productivity was also more impaired (work time missed: 4.5% vs 1.3%; impairment while working: 19.1% vs 5.2%; overall work impairment: 20.4% vs 5.6%; activity impairment: 20.3% vs 5.3%; all p < 0.001). CONCLUSION: Less than one-third of patients had gout that was adequately controlled. Those experiencing inadequately controlled gout reported significantly worse functioning, quality of life, and work productivity. Gout treatment strategies to improve disease control may lead to improvements in HRQOL and productivity.

An economic and outcomes assessment of first-line monotherapy in the treatment of community-acquired pneumonia within managed care.

OBJECTIVE: To evaluate the resource consumption and outcomes associated with first-line monotherapy for community-acquired pneumonia, focusing specifically on the use of erythromycin, azithromycin, clarithromycin, and levofloxacin. STUDY DESIGN: Retrospective managed care database analysis. PATIENTS AND METHODS: Subjects included patients within a managed care setting over 18 years of age with an initial diagnosis of community-acquired pneumonia from January 1995 to April 2002. Multivariate linear and logistic regression models were used to examine associations with treatment success rates and direct medical costs between antibiotic treatments after controlling for patient demographics and pneumonia risk factors. MAIN RESULTS: Overall, treatment success rates were high (95.8%), the use of second antibiotics was un common (2.3%), and hospitalizations were infrequent (2.0%) among the 1952 subjects studied. After controlling for patient characteristics and risk factors, significantly lower total costs were associated with erythromycin (92.7% lower, p < 0.001), azithromycin (48.7% lower, p < 0.001), and clarithromycin (21.3% lower, p = 0.015) relative to levofloxacin, with no difference in treatment success between groups. Among newer agents, azithromycin (49.2% lower, p < 0.001) and clarithromycin (21.7% lower, p = 0.013) treatment groups were associated with significantly lower total costs than levofloxacin in the full sample. However, in subjects with a chronic disease score above the sample's mean, only azithromycin was associated with significantly lower total costs (47.9% lower, p < 0.001) relative to levofloxacin. CONCLUSION: Erythromycin, azithromycin, and clarithromycin were associated with significantly lower total costs than levofloxacin, although treatment success rates did not differ between groups. Following stratification based upon various subset criteria, erythromycin and azithromycin were observed to have significantly lower total costs than levofloxacin. Although these findings may augment clinical guidelines and evidence-based approaches, health plans should consider evaluating their own patient data to see if similar differences exist in their populations.

Using community indicators to assess nutrition in Arizona-Mexico border communities.

INTRODUCTION: Community indicators are used to measure and monitor factors that affect the well-being of a community or region. Community indicators can be used to assess nutrition. Evaluating nutrition in communities along the Arizona-Mexico border is important because nutrition is related to an individual's risk of overweight or obesity; obesity is a risk factor for developing type 2 diabetes. METHODS: Local grocery store purchases were selected as a community indicator for nutrition. A structured 26-question interview was developed and administered to grocery store managers in communities along the Arizona-Mexico border that were targeted by the Border Health Strategic Initiative, a program implemented by community groups and the University of Arizona. In addition, data from milk distributors serving the border communities were collected. RESULTS: Residents of these communities favor food items with a higher fat and higher caloric content. This trend held across several food categories. Major barriers to customer acceptance of healthier food items include lack of knowledge concerning healthy foods and their prices. CONCLUSION: The demand for healthy food items is relatively low along the Arizona-Mexico border. Interventions should continue to target this population with the aim of changing dietary patterns as one method of improving the health of the community and preventing and controlling diabetes.

A 3-stage model for assessing the probable economic effects of direct-to-consumer advertising of pharmaceuticals.

BACKGROUND: The pharmaceutical industry employs a variety of marketing strategies that have previously been directed primarily toward physicians. However, mass media direct-to-consumer (DTC) advertising of prescription drugs has emerged as a ubiquitous promotional strategy. OBJECTIVE: This article explores the economics of DTC advertising in greater depth than has been done in the past by using a 3-stage economic model to assess the pertinent literature and to show the probable effects of DTC advertising in the United States. METHODS: Economics literature on the subject was searched using the Journal of Economic Literature. Health services literature was searched using computer callback devices. RESULTS: Spending on DTC advertising in the United States increased from $17 million in 1985 to $2.5 billion in 2000. Proponents of DTC advertising claim that it provides valuable product-related information to health care professionals and patients, may contribute to better use of medications, and helps patients take charge of their own health care. Opponents argue that DTC advertising provides misleading messages rather than well-balanced, evidence-based information. The literature is replete with opinions about the effects of prescription drug advertising on pharmaceutical drug prices and physician-prescribing patterns, but few studies have addressed the issues beyond opinion surveys. The economic literature on advertising effects in other markets, however, may provide insight. CONCLUSION: DTC advertising indirectly affects the price and the quantity of production of pharmaceuticals via its effect on changes in consumer demand.

Evaluation of patient preference and willingness to pay for attributes of maintenance medication for chronic obstructive pulmonary disease (COPD).

BACKGROUND: Chronic obstructive pulmonary disease (COPD) is a leading cause of morbidity and mortality worldwide. COPD is characterized by poor treatment adherence, and patient medication preferences may contribute to adherence. METHODS: A discrete choice experiment with an internet panel drawn from the USA was used to evaluate preference and willingness to pay (WTP) of COPD patients for long-acting maintenance medications. Key attributes derived from earlier qualitative research (brief literature review and focus groups) with COPD patients on maintenance therapy included symptom relief, speed of feeling medication start to work, inhaler ease of use, rescue medication use, side effects, and monthly out-of-pocket co-pay. Patients were presented with hypothetical medications with different profiles and asked which they preferred. Utilities and marginal WTP in monthly co-pay dollars were estimated for all patients and by severity. RESULTS: Utilities for 515 participants were in the expected direction and highest for the most favorable attribute levels. Each attribute evaluated was important, and participants were willing to pay a premium to obtain each benefit. On average, WTP was as high as $US64 for complete symptom relief, $US59 for no side effects, $US32 to rarely use rescue medication, $US16 for a quick and easy to use inhaler, and $US13 for feeling medication work quickly (within 5 min; average WTP $US18/month for patients with severe/very severe COPD). CONCLUSION: As expected, efficacy and safety were most valued by patients; however, this study showed that other COPD medication attributes, such as rescue medication, ease of use, and feeling medication work quickly, are also important in patient preferences.