Optimising equity of access: how should we allocate slots to the most competitive trials in Cystic Fibrosis (CF)?

BACKGROUND: Trial participation can allow people with CF early access to CFTR modulator therapies, with high potential for clinical benefit. Therefore, the number of people wishing to participate can substantially exceed the number of slots available. We aimed to understand how the CF community thinks slots to competitive trials should be allocated across the UK and whether this should be driven by clinical need, patients' engagement/adherence or be random. For the latter, we explored site-level versus registry-based, national randomisation processes. METHODS: We developed an online survey, recruiting UK-based stakeholders through social media, newsletters and personal contacts. Closed questions were analysed for frequencies and percentages of responses. Free-text questions were analysed using thematic analysis. RESULTS: We received 203 eligible responses. Overall, 75% of stakeholders favoured allocation of slots to individual sites based on patient population size, although pharma favoured allocation based on previous metrics. Currently, few centres have defined strategies for allocating slots locally. At face-value, stakeholders believe all eligible participants should have an equal chance of getting a slot. However, further questioning reveals preference for prioritisation strategies, primarily perceived treatment adherence, although healthcare professionals were less likely to favour this strategy than other stakeholder groups. The majority of stakeholders would prefer to allocate slots and participate in trials locally but 80% said if necessary, they would engage in a system of national allocation. CONCLUSIONS: Fair allocation to highly competitive trials does not appear to have a universally acceptable solution. Therefore, transparency and empathy remain critical to negotiate this uncertain territory.

Local Health Jurisdiction Staff Deliver Health Promotion to Small Worksites, Washington.

CONTEXT: Worksites can serve as community sites for local health jurisdictions (LHJs) to assist with implementation of evidence-based interventions (EBIs) to prevent and control chronic diseases. OBJECTIVE: To assess the feasibility and effectiveness of using LHJ staff to disseminate Connect to Wellness (CtW), an effective dissemination package for increasing implementation of EBIs for chronic disease control by small worksites. DESIGN: Single-arm, multisite intervention trial, with measurement at baseline, after 6 months of intervention, and after a maintenance period of 6 months. SETTING: Six geographically dispersed counties in Washington State. Target worksites had 20 to 250 employees. PARTICIPANTS: Nine staff members from 6 LHJs delivered CtW to 35 worksites. INTERVENTION: Connect to Wellness seeks to increase worksites' implementation of 14 EBIs classified as communication, policy, or program approaches to increasing 4 behaviors: cancer screening, healthy eating, physical activity, and tobacco cessation. MAIN OUTCOME MEASURE: Evidence-based intervention implementation measured on a scale from 0% to 100%. RESULTS: Participating worksites showed a significant increase (P < .001, t test) in total mean implementation scores from baseline (33%) to 6-month follow-up (47%). Increases in implementation for communications, policy, healthy eating, and tobacco EBIs were statistically significant at 6 months and maintained at 12 months. Increased implementation at 6 months of a group physical activity program was not sustained after the program became unavailable, and total implementation scores at 12 months (38%) showed little change from baseline. CONCLUSIONS: Local health jurisdiction-delivered CtW increased worksites' implementation of EBIs at 6 months, and increased implementation in communication, policy, healthy eating, and tobacco was maintained at 12 months. This package, delivered by LHJ staff working part-time on CtW, was nearly as successful as prior delivery by staff working full-time on CtW.

Feasibility of WHO mhGAP-intervention guide in reducing experienced discrimination in people with mental disorders: a pilot study in a rural Kenyan setting.

AimsStigma can have a negative impact on help-seeking behaviour, treatment adherence and recovery of people with mental disorders. This study aimed to determine the feasibility of the WHO Mental Health Treatment Gap Interventions Guidelines (mhGAP-IG) to reduce stigma in face-to-face contacts during interventions for specific DSM-IV/ICD 10 diagnoses over a 6-month period. METHODS: This study was conducted in 20 health facilities across Makueni County in southeast Kenya which has one of the poorest economies in the country and has no psychiatrist or clinical psychologist. We recruited 2305 participants from the health facilities catchment areas that had already been exposed to community mental health services. We measured stigma using DISC-12 at baseline, followed by training to the health professionals on intervention using the WHO mhGAP-IG and then conducted a follow-up DISC-12 assessment after 6 months. Proper management of the patients by the trained professionals would contribute to the reduction of stigma in the patients. RESULTS: There was 59.5% follow-up at 6 months. Overall, there was a significant decline in 'reported/experienced discrimination' following the interventions. A multivariate linear mixed model regression indicated that better outcomes of 'unfair treatment' scores were associated with: being married, low education, being young, being self-employed, higher wealth index and being diagnosed with depression. For 'stopping self' domain, better outcomes were associated with being female, married, employed, young, lower wealth index and a depression diagnosis. In regards to 'overcoming stigma' domain; being male, being educated, employed, higher wealth index and being diagnosed with depression was associated with better outcomes. CONCLUSIONS: The statistically significant (p < 0.05) reduction of discrimination following the interventions by trained health professionals suggest that the mhGAP-IG may be a useful tool for reduction of discrimination in rural settings in low-income countries.

Are lower levels of red blood cell transfusion more cost-effective than liberal levels after cardiac surgery? Findings from the TITRe2 randomised controlled trial.

OBJECTIVE: To assess the incremental cost and cost-effectiveness of a restrictive versus a liberal red blood cell transfusion threshold after cardiac surgery. DESIGN: A within-trial cost-effectiveness analysis with a 3-month time horizon, based on a multicentre superiority randomised controlled trial from the perspective of the National Health Service (NHS) and personal social services in the UK. SETTING: 17 specialist cardiac surgery centres in UK NHS hospitals. PARTICIPANTS: 2003 patients aged >16 years undergoing non-emergency cardiac surgery with a postoperative haemoglobin of <9 g/dL. INTERVENTIONS: Restrictive (transfuse if haemoglobin <7.5 g/dL) or liberal (transfuse if haemoglobin <9 g/dL) threshold during hospitalisation after surgery. MAIN OUTCOME MEASURES: Health-related quality of life measured using the EQ-5D-3L to calculate quality-adjusted life years (QALYs). RESULTS: The total costs from surgery up to 3 months were £17 945 and £18 127 in the restrictive and liberal groups (mean difference is -£182, 95% CI -£1108 to £744). The cost difference was largely attributable to the difference in the cost of red blood cells. Mean QALYs to 3 months were 0.18 in both groups (restrictive minus liberal difference is 0.0004, 95% CI -0.0037 to 0.0045). The point estimate for the base-case cost-effectiveness analysis suggested that the restrictive group was slightly more effective and slightly less costly than the liberal group and, therefore, cost-effective. However, there is great uncertainty around these results partly due to the negligible differences in QALYs gained. CONCLUSIONS: We conclude that there is no clear difference in the cost-effectiveness of restrictive and liberal thresholds for red blood cell transfusion after cardiac surgery. TRIAL REGISTRATION NUMBER: ISRCTN70923932; Results.

Treating severe and enduring anorexia nervosa: a randomized controlled trial.

BACKGROUND: There are no evidence-based treatments for severe and enduring anorexia nervosa (SE-AN). This study evaluated the relative efficacy of cognitive behavioral therapy (CBT-AN) and specialist supportive clinical management (SSCM) for adults with SE-AN. METHOD: Sixty-three participants with a diagnosis of AN, who had at least a 7-year illness history, were treated in a multi-site randomized controlled trial (RCT). During 30 out-patient visits spread over 8 months, they received either CBT-AN or SSCM, both modified for SE-AN. Participants were assessed at baseline, end of treatment (EOT), and at 6- and 12-month post-treatment follow-ups. The main outcome measures were quality of life, mood disorder symptoms and social adjustment. Weight, eating disorder (ED) psychopathology, motivation for change and health-care burden were secondary outcomes. RESULTS: Thirty-one participants were randomized to CBT-AN and 32 to SSCM with a retention rate of 85% achieved at the end of the study. At EOT and follow-up, both groups showed significant improvement. There were no differences between treatment groups at EOT. At the 6-month follow-up, CBT-AN participants had higher scores on the Weissman Social Adjustment Scale (WSAS; p = 0.038) and at 12 months they had lower Eating Disorder Examination (EDE) global scores (p = 0.004) and higher readiness for recovery (p = 0.013) compared to SSCM. CONCLUSIONS: Patients with SE-AN can make meaningful improvements with both therapies. Both treatments were acceptable and high retention rates at follow-up were achieved. Between-group differences at follow-up were consistent with the nature of the treatments given.

American Cancer Society's QuitLink: randomized trial of Internet assistance.

Eligible smokers (N = 6,451) visiting the American Cancer Society's Internet site offering cessation assistance were, with informed consent, randomized to receive access either to a static Internet site with quitting advice or to one of five interactive sites provided by cooperating research partners. Three-month follow-up surveys were conducted via online survey with E-mail prompts, or telephone calls, to assess quitting success; 54% of participants provided follow-up data. Results showed no significant overall difference in cessation rates among participants assigned to the interactive or static sites. We found large differences in the utilization of the five interactive sites. When sites were grouped by level of use, a significantly higher reported 3-month cessation rate was observed among participants assigned to the more highly utilized sites than among those assigned to the less utilized sites (12.2% vs. 10.2% of all randomized participants, 26.0% vs. 22.1% of followed participants). These findings show that interactive Internet sites yielding high levels of utilization can increase quitting success among smokers seeking assistance via the Internet.

An analysis of UK waste minimization clubs: key requirements for future cost effective developments.

The UK waste strategy is based upon use of the best practicable environmental option (BPEO), by those making waste management decisions. BPEO is supported by the use of the waste hierarchy, with its range of preferable options for dealing with waste, and the proximity principle, where waste is treated/disposed of as close to its point of origin as possible. The national waste strategy emphasizes the key role of waste minimization and encourages industry, commerce and the public to move towards sustainable waste management practice for economic and environmental reasons. Waste minimization clubs have been used, since the early 1990s, to demonstrate to industry/commerce that reducing waste production can lead to significant financial savings. There have been around 75 such clubs in the UK and they receive support from a wide range of agencies, including the Environmental Technology Best Practice Program. The early Demonstration Clubs had significant savings to cost ratios, e.g. Aire and Calder at 8.4, but had very high costs, e.g. Aire and Calder at 400,000 pounds. It is acknowledged that the number of clubs will have to be approximately doubled in the next few years so as to have an adequate coverage of the UK. There are at present, marked regional variations in club development and cognizance needs to be taken, by facilitators, of the need for extensive coverage of the UK. Future clubs will probably have to operate in a financially constrained climate and they need to be designed to deliver significant savings and waste reduction at low cost. To aid future club design, final reports of all projects should report in a standard manner so that cost benefit analysis can be used to inform facilitators about the most effective club type. rights reserved.

Assessment of diagnostic features of bulimia nervosa: interview versus self-report format.

In order to determine the clinical severity of bulimia nervosa and to measure symptomatic improvement during treatment, the behavioral and psychological features of the syndrome must be assessed. Focused semistructured interviews have recently been designed for this purpose, but such instruments can be time-consuming and costly. The present study compared the assessment of the key symptoms of bulimia nervosa using the Eating Disorder Examination with data obtained in self-report inventories and in patients' diaries of binge eating and purging. Results suggest that once the diagnosis has been established and patients have been instructed in the construct of a binge, the essential features of bulimia nervosa (frequency of binge eating and purging, and overconcern with body shape and weight) can be evaluated with self-report measures. These findings may be useful for the purposes of repeated assessment of progress and measurement of outcome in treatment studies.