Adaptation and content validation of measure yourself medical outcomes profile (MYMOP) for 7-11 year-old children.

BACKGROUND: The Measure Yourself Medical Outcome Profile (MYMOP) is an individualised tool designed for adults but used with children without any evidence of validation in this population. Individualised instruments are patient-specific rather than disease-specific and therefore can be applied across various health conditions. This study sought to adapt, and content validate the MYMOP for application in 7-11 year old children. METHODS: There were two main phases of the four iterations: expert consultation (three rounds) and interviews with child-parent pairs at the Outpatient clinics of a Children's Hospital. Thematic analysis was undertaken using an inductive, interpretative approach. RESULTS: Four paediatricians completed the first survey, five paediatricians participated in the focus group, and four paediatric health-related quality of life (HRQOL) research experts completed the second survey. Several changes were recommended to the MYMOP by the expert groups. Twenty-five children (17 general medicine, and 8 diabetes/endocrine clinic) aged 7-11 years completed the draft paediatric MYMOP (P-MYMOP) and were interviewed. Results demonstrated that the majority of participants were able to identify their own problems and activity limitations, and all participants understood the 7-point faces scale. Most parents and children perceived that the P-MYMOP would be useful to complete before clinic appointments. CONCLUSIONS: The P-MYMOP is the first content-validated generic individualised HRQOL measure for children 7-11 years old. Given that validation is an iterative process, further research to assess its feasibility, reliability, and construct validity is required.

Findings from three methods to identify falls in hospitals: Results from the Ambient Intelligent Geriatric Management system fall prevention trial.

OBJECTIVE: To (a) compare characteristics of patients who fall with those of patients who did not fall; and (b) characterise falls (time, injury severity and location) through three fall reporting methods (incident system reports, medical notes and clinician reports). METHODS: A substudy design within a stepped-wedge clinical trial was used: 3239 trial participants were recruited from two inpatient Geriatric Evaluation and Management Units and one general medicine ward in two Australian states. To compare the characteristics of patients who had fallen with those who had not, descriptive tests were used. To characterise falls through three reporting methods, bivariate logistic regressions were used. RESULTS: Patients who had fallen were more likely than patients who had not fallen to be cognitively impaired (51% vs. 29%, p < 0.01), admitted with falls (38% vs. 28%, p = 0.01) and have poor health outcomes such as prolonged length of stay (24 [16-34] vs. 12 [8-19] days [IQR], p < 0.01) and less likely to be discharged directly to the community (62% vs. 47%, p < 0.01). Most falls were captured from medical notes (93%), with clinician (71%) and incident reports (68%) missing 21%-25% of falls. The proportion of injurious falls identified through incident reports was higher than medical records or clinician reports (40% vs. 34% vs. 37%). CONCLUSIONS: This study reaffirms the need to improve reporting falls in incident systems and at clinical handover to the team leader. Research should continue to use more than one method of identifying falls, but include data from medical records. Many falls cause injury, resulting in poor health outcomes.

Pragmatic review of interventions to prevent catheter-associated urinary tract infections (CAUTIs) in adult inpatients.

BACKGROUND: Urinary tract infections (UTIs) are one of the most common hospital-acquired complications. Insertion of a urinary catheter and the duration of catheterization are the main risk factors, with catheter-associated UTIs (CAUTIs) accounting for 70-80% of hospital-acquired UTIs. Guidance is available regarding the prevention of hospital-acquired CAUTIs; however, how best to operationalize this guidance remains a challenge. AIM: To map and summarize the peer-reviewed literature on model-of-care interventions for the prevention of CAUTIs in adult inpatients. METHODS: PubMed, CINAHL and SCOPUS were searched for articles that reported UTI, CAUTI or urinary catheter outcomes. Articles were screened systematically, data were extracted systematically, and interventions were classified by intervention type. FINDINGS: This review included 70 articles. Interventions were classified as single component (N=19) or multi-component (N=51). Single component interventions included: daily rounds or activities (N=4), protocols and procedure changes (N=6), reminders and order sets (N=5), audit and feedback interventions (N=3), and education with simulation (N=1). Overall, daily catheter reviews and protocol and procedure changes demonstrated the most consistent effects on catheter and CAUTI outcomes. The components of multi-component interventions were categorized to map common elements and identify novel ideas. CONCLUSION: A range of potential intervention options with evidence of a positive effect on catheter and CAUTI outcomes was identified. This is intended to provide a 'menu' of intervention options for local decision makers, enabling them to identify interventions that are relevant and feasible in their local setting.

A systematic review of randomised controlled trials evaluating the use of patient-reported outcome measures (PROMs).

BACKGROUND: Patient-reported outcome measures (PROMs) could play an important role in identifying patients' needs and goals in clinical encounters, improving communication and decision-making with clinicians, while making care more patient-centred. Comprehensive evidence that PROMS are an effective intervention is lacking in single randomised controlled trials (RCTs). METHODS: A systematic search was performed using controlled vocabulary related to the terms: clinical care setting and patient-reported outcome. English language studies were included if they were a RCT with a PROM as an intervention in a patient population. Included studies were analysed and their methodologic quality was appraised using the Cochrane Risk of Bias tool. The protocol was registered with PROSPERO (CRD42016034182). RESULTS: Of 4302 articles initially identified, 115 underwent full-text review resulting in 22 studies reporting on 25 comparisons. The majority of included studies were conducted in USA (11), among cancer patients (11), with adult participants only (20). Statistically significant and robust improvements were reported in the pre-specified outcomes of the process of care (2) and health care (3). Additionally, five, eight and three statistically significant but possibly non-robust findings were reported in the process of care, health and patient satisfaction outcomes, respectively. CONCLUSIONS: Overall, studies that compared PROM to standard care either reported a positive effect or were not powered to find pre-specified differences. There is justification for the use of a PROM as part of standard care, but further adequately powered studies on their use in different contexts are necessary for a more comprehensive evidence base.

Older People Who Are Frequent Users of Acute Care: A Symptom of Fragmented Care? A Case Series Report on Patients' Pathways of Care.

Older frequent users of acute care can experience fragmented care. There is a need to understand the issues in a local context before attempting to address fragmented care. 0.5% (n=61) of the population in a defined local government area were identified as having ≥4 unplanned emergency department (ED) presentations/ admissions to an acute-care hospital over 13 months. A retrospective case-series study was conducted to examine detailed pathways of care for 17 patients within the identified population. The two dominant presentation reasons were clinical symptoms associated with a declining/significant loss of capacity in fundamental self-care activities and chronic cardiac/respiratory conditions. Of patients discharged home, 21% of discharge letters were delayed >7 days and only 19% received a written discharge plan. Half of community dwelling patients received home nursing and/or assistance. Frequent users of acute care can experience untimely hospital communication and may require more coordinated care provided in the community to assist self-care and manage chronic conditions.

Mortality reductions from marginal increases in public spending on health.

There is limited empirical evidence of the nature of any relationship between health spending and health outcomes in Australia. We address this by estimating the elasticity of health outcomes with respect to public healthcare spending using an instrumental variable (IV) approach to account for endogeneity of healthcare spending to health outcomes. Results suggest that, based on the conditional mean, a 1% increase in public health spending was associated with a 2.2% (p < 0.05) reduction in the number of standardised Years of Life Lost (YLL). Sensitivity analyses and robustness checks supported this conclusion. Further exploration using IV quantile regression indicated that marginal returns on public health spending were significantly greater for areas with poorer health outcomes compared to areas with better health outcomes. On average, marginal increases in public health spending reduce YLL, but areas with poorer health outcomes have the greatest potential to benefit from the same marginal increase in public health spending compared to areas with better health outcomes. Understanding the relationship between health spending and outcomes and how this differs according to baseline health outcomes can help meet dual policy objectives to improve the productivity of the healthcare system and reduce inequity.

Management of Musculoskeletal Conditions in Rural South Australia: A Randomised Controlled Trial.

With age, the prevalence of musculoskeletal conditions increases markedly. This rural-based study determined the benefits of two approaches for managing musculoskeletal conditions: a multiple-component 'Self-management Plus' intervention, and usual care. The intervention combined self-management education with physical activity and health professional support. 6-month outcomes included: Clinical Global Impression-Improvement Scale (CGI-IS) and Quality of Life (QoL). A total of 145 people were recruited; mean (SD) age was 66.1 (11.1) and 63.3 (10.9) years for intervention and control groups respectively. The intervention resulted in greater improvements in global functioning (CGI-IS mean (SD) = 3.2 (1.3)) than usual care (CGI-IS mean (SD) = 4.2 (1.5)). There was no difference in QoL improvement between study groups. A multiple-component 'Self-management Plus' intervention had a positive effect on physical functioning for older adults with musculoskeletal conditions. However, recruitment and retention of participants was problematic, which raises questions about the intervention's feasibility in its current form.

Building multidisciplinary health workforce capacity to support the implementation of integrated, people-centred Models of Care for musculoskeletal health.

To address the burden of musculoskeletal (MSK) conditions, a competent health workforce is required to support the implementation of MSK models of care. Funding is required to create employment positions with resources for service delivery and training a fit-for-purpose workforce. Training should be aligned to define "entrustable professional activities", and include collaborative skills appropriate to integrated and people-centred care and supported by shared education resources. Greater emphasis on educating MSK healthcare workers as effective trainers of peers, students and patients is required. For quality, efficiency and sustainability of service delivery, education and research capabilities must be integrated across disciplines and within the workforce, with funding models developed based on measured performance indicators from all three domains. Greater awareness of the societal and economic burden of MSK conditions is required to ensure that solutions are prioritised and integrated within healthcare policies from local to regional to international levels. These healthcare policies require consumer engagement and alignment to social, economic, educational and infrastructure policies to optimise effectiveness and efficiency of implementation.

Stepped-wedge cluster randomised controlled trial to assess the effectiveness of an electronic medication management system to reduce medication errors, adverse drug events and average length of stay at two paediatric hospitals: a study protocol.

INTRODUCTION: Medication errors are the most frequent cause of preventable harm in hospitals. Medication management in paediatric patients is particularly complex and consequently potential for harms are greater than in adults. Electronic medication management (eMM) systems are heralded as a highly effective intervention to reduce adverse drug events (ADEs), yet internationally evidence of their effectiveness in paediatric populations is limited. This study will assess the effectiveness of an eMM system to reduce medication errors, ADEs and length of stay (LOS). The study will also investigate system impact on clinical work processes. METHODS AND ANALYSIS: A stepped-wedge cluster randomised controlled trial (SWCRCT) will measure changes pre-eMM and post-eMM system implementation in prescribing and medication administration error (MAE) rates, potential and actual ADEs, and average LOS. In stage 1, 8 wards within the first paediatric hospital will be randomised to receive the eMM system 1 week apart. In stage 2, the second paediatric hospital will randomise implementation of a modified eMM and outcomes will be assessed. Prescribing errors will be identified through record reviews, and MAEs through direct observation of nurses and record reviews. Actual and potential severity will be assigned. Outcomes will be assessed at the patient-level using mixed models, taking into account correlation of admissions within wards and multiple admissions for the same patient, with adjustment for potential confounders. Interviews and direct observation of clinicians will investigate the effects of the system on workflow. Data from site 1 will be used to develop improvements in the eMM and implemented at site 2, where the SWCRCT design will be repeated (stage 2). ETHICS AND DISSEMINATION: The research has been approved by the Human Research Ethics Committee of the Sydney Children's Hospitals Network and Macquarie University. Results will be reported through academic journals and seminar and conference presentations. TRIAL REGISTRATION NUMBER: Australian New Zealand Clinical Trials Registry (ANZCTR) 370325.

What sort of follow-up services would Australian breast cancer survivors prefer if we could no longer offer long-term specialist-based care? A discrete choice experiment.

BACKGROUND: Early diagnosis and improved treatment outcomes have increased breast cancer survival rates that, in turn, have led to increased numbers of women undergoing follow-up after completion of primary treatment. The current workload growth is unsustainable for breast cancer specialists who also provide care for women newly diagnosed or with a recurrence. Appropriate and acceptable follow-up care is important; yet, currently we know little about patient preferences. The aim of this study was to explore the preferences of Australian breast cancer survivors for alternative modes of delivery of follow-up services. METHODS: A self-administered questionnaire (online or paper) was developed. The questionnaire contained a discrete choice experiment (DCE) designed to explore patient preferences with respect to provider, location, frequency and method of delivery of routine follow-up care in years 3, 4 and 5 after diagnosis, as well as the perceived value of 'drop-in' clinics providing additional support. Participants were recruited throughout Australia over a 6-month period from May to October 2012. Preference scores and choice probabilities were used to rank the top 10 most preferred follow-up scenarios for respondents. RESULTS: A total of 836 women participated in the study, of whom 722 (86.4%) completed the DCE. In the absence of specialist follow-up, the 10 most valued surveillance scenarios all included a Breast Physician as the provider of follow-up care. The most preferred scenario is a face-to-face local breast cancer follow-up clinic held every 6 months and led by a Breast Physician, where additional clinics focused on the side effects of treatment are also provided. CONCLUSION: Beyond the first 2 years from diagnosis, in the absence of a specialist led follow-up, women prefer to have their routine breast cancer follow-up by a Breast Physician (or a Breast Cancer Nurse) in a dedicated local breast cancer clinic, rather than with their local General Practitioner. Drop-in clinics for the management of treatment related side effects and to provide advice to both develop and maintain good health are also highly valued by breast cancer survivors.

A risk-adjusted economic evaluation of alternative models of involvement of practice nurses in management of type 2 diabetes.

AIMS: To determine the cost-effectiveness of alternative models of practice nurse involvement in the management of type 2 diabetes within the primary care setting. METHODS: Linked routinely collected clinical data and resource use (general practitioner visits, hospital services and pharmaceuticals) were used to undertake a risk-adjusted cost-effectiveness analysis of alternative models of care for the management of diabetes patients. These models were based on the reported level of involvement of practice nurses in the provision of clinical-based activities. Potential confounders were controlled for by using propensity score-weighted regression analyses. The impact of alternative models of care on outcomes and costs was measured and incremental cost-effectiveness estimated. The uncertainty around the estimates of cost-effectiveness was illustrated through bootstrapping. RESULTS: Although the difference in total cost between two models of care was not statistically significant, the high-level model was associated with better outcomes (larger mean reductions in HbA(1c)). The upper 95% confidence intervals showed that the incremental cost per 1% decrease in HbA(1c) is only $454, and per one additional patient to achieve an HbA(1c) value of less than 53 mmol/mol (7.0%) is $323. Further analyses showed little uncertainty surrounding the decision to adopt the high-level model. CONCLUSIONS: The results provide a strong indication that the high-level model is a cost-effective way of managing diabetes patients. Our findings highlight the need for effective incentives to encourage general practices to better integrate practice nurses in the provision of clinical services.

A risk adjusted cost-effectiveness analysis of alternative models of nurse involvement in obesity management in primary care.

OBJECTIVE: Controlled evaluations are subject to uncertainty regarding their replication in the real world, particularly around systems of service provision. Using routinely collected data, we undertook a risk adjusted cost-effectiveness (RAC-E) analysis of alternative applied models of primary health care for the management of obese adult patients. Models were based on the reported level of involvement of practice nurses (registered or enrolled nurses working in general practice) in the provision of clinical-based activities. DESIGN AND METHODS: Linked, routinely collected clinical data describing clinical outcomes (weight, BMI, and obesity-related complications) and resource use (primary care, pharmaceutical, and hospital resource use) were collected. Potential confounders were controlled for using propensity weighted regression analyses. RESULTS: Relative to low level involvement of practice nurses in the provision of clinical-based activities to obese patients, high level involvement was associated with lower costs and better outcomes (more patients losing weight, and larger mean reductions in BMI). Excluding hospital costs, high level practice nurse involvement was associated with slightly higher costs. Incrementally, the high level model gets one additional obese patient to lose weight at an additional cost of $6,741, and reduces mean BMI by an additional one point at an additional cost of $563 (upper 95% confidence interval $1,547). CONCLUSION: Converted to quality adjusted life year (QALY) gains, the results provide a strong indication that increased involvement of practice nurses in clinical activities is associated with additional health benefits that are achieved at reasonable additional cost. Dissemination activities and incentives are required to encourage general practices to better integrate practice nurses in the active provision of clinical services.

The importance of high-quality evidence of the long-term impact of nonfatal events used in randomized controlled trials: a case study of prasugrel.

Randomized controlled trials (RCTs) are the centerpiece of evidence-based medicine. However, because of the limited follow-up, additional evidence on the long-term consequences of the outcomes used in trials is commonly required for clinical and policy decision making. This article provides insights into the importance and challenges of using such evidence through the case study of nonfatal myocardial infarction (MI) and nonfatal bleeding with prasugrel.

Cost-effectiveness of screening high-risk HIV-positive men who have sex with men (MSM) and HIV-positive women for anal cancer.

BACKGROUND: Anal cancer is uncommon and predominantly a disease of the elderly. The human papillomavirus (HPV) has been implicated as a causal agent, and HPV infection is usually transmitted sexually. Individuals who are human immunodeficiency virus (HIV)-positive are particularly vulnerable to HPV infections, and increasing numbers from this population present with anal cancer. OBJECTIVE: To estimate the cost-effectiveness of screening for anal cancer in the high-risk HIV-positive population [in particular, men who have sex with men (MSM), who have been identified as being at greater risk of the disease] by developing a model that incorporates the national screening guidelines criteria. DATA SOURCES: A comprehensive literature search was undertaken in January 2006 (updated in November 2006). The following electronic bibliographic databases were searched: Applied Social Sciences Index and Abstracts (ASSIA), BIOSIS previews (Biological Abstracts), British Nursing Index (BNI), Cumulative Index to Nursing and Allied Health Literature (CINAHL), Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), EMBASE, MEDLINE, MEDLINE In-Process & Other Non-Indexed Citations, NHS Database of Abstracts of Reviews of Effects (DARE), NHS Health Technology Assessment (HTA) Database, PsycINFO, Science Citation Index (SCI), and Social Sciences Citation Index (SSCI). STUDY SELECTION: Published literature identified by the search strategy was assessed by four reviewers. Papers that met the inclusion criteria contained the following: data on population incidence, effectiveness of screening, health outcomes or screening and/or treatment costs; defined suitable screening technologies; prospectively evaluated tests to detect anal cancer. Foreign-language papers were excluded. Searches identified 2102 potential papers; 1403 were rejected at title and a further 493 at abstract. From 206 papers retrieved, 81 met the inclusion criteria. A further treatment paper was added, giving a total of 82 papers included. DATA EXTRACTION: Data from included studies were extracted into data extraction forms by the clinical effectiveness reviewer. To analyse the cost-effectiveness of screening, two decision-analytical models were developed and populated. RESULTS: The reference case cost-effectiveness model for MSM found that screening for anal cancer is very unlikely to be cost-effective. The negative aspects of screening included utility decrements associated with false-positive results and with treatment for high-grade anal intraepithelial neoplasia (HG-AIN). Sensitivity analyses showed that removing these utility decrements improved the cost-effectiveness of screening. However, combined with higher regression rates from low-grade anal intraepithelial neoplasia (LG-AIN), the lowest expected incremental cost-effectiveness ratio remained at over 44,000 pounds per quality-adjusted life-year (QALY) gained. Probabilistic sensitivity analysis showed that no screening retained over 50% probability of cost-effectiveness to a QALY value of 50,000 pounds. The screening model for HIV-positive women showed an even lower likelihood of cost-effectiveness, with the most favourable sensitivity analyses reporting an incremental cost per QALY of 88,000 pounds. LIMITATIONS: Limited knowledge is available about the epidemiology and natural history of anal cancer, along with a paucity of good-quality evidence concerning the effectiveness of screening. CONCLUSIONS: Many of the criteria for assessing the need for a screening programme were not met and the cost-effectiveness analyses showed little likelihood that screening any of the identified high-risk groups would generate health improvements at a reasonable cost. Further studies could assess whether the screening model has underestimated the impact of anal cancer, the results of which may justify an evaluative study of the effects of treatment for HG-AIN.

Antenatal screening for haemoglobinopathies in primary care: a cohort study and cluster randomised trial to inform a simulation model. The Screening for Haemoglobinopathies in First Trimester (SHIFT) trial.

OBJECTIVES: To assess the effectiveness, cost-effectiveness, acceptability and feasibility of offering universal antenatal sickle cell and thalassaemia (SCT) screening in primary care when pregnancy is first confirmed and to model the cost-effectiveness of early screening in primary care versus standard care. DESIGN: A population-based cohort study, cluster randomised trial and refinement of a published decision model. SETTING: Twenty-five general practices from two UK primary care trusts (PCTs) in two inner city boroughs with a high proportion of residents from minority ethnic groups. PARTICIPANTS: Practices were considered eligible if they agreed to be randomised and they were able to provide anonymous data on all eligible pregnant women. Participants were at least 18 years old and consented to take part in the evaluation. INTERVENTIONS: Practices were allocated to intervention, using minimisation and stratifying for PCT and number of partners at the practice, as follows: screening in primary care with parallel father testing (test offered to mother and father simultaneously; n = 8 clusters, 1010 participants); screening in primary care with sequential father testing (test offered to father only if mother identified as carrier; n = 9 clusters, 792 participants); and screening in secondary care with sequential father testing (standard care; n = 8 clusters, 619 participants). MAIN OUTCOME MEASURES: Data on gestational age at pregnancy confirmation and screening date were collected from trial practices for 6 months before randomisation in the cohort phase. The primary outcome measure was timing of SCT screening, measured as the proportion of women screened before 70 days' (10 weeks') gestation. Other outcomes included: offer of screening, rates of informed choice and proportion of women who knew the carrier status of their baby's father by 77 days (11 weeks). RESULTS: For 1441 eligible women in the cohort phase, the median [interquartile range (IQR)] gestational age at pregnancy confirmation was 7.6 weeks (6.0 to 10.7 weeks) and 74% presented in primary care before 10 weeks. The median gestational age at screening was 15.3 weeks (IQR 12.6 to 18.0 weeks). Only 4.4% were screened before 10 weeks. The median delay between pregnancy confirmation and screening was 6.9 weeks (4.7 to 9.3 weeks). In the intervention phase, 1708 pregnancies from 25 practices were assessed for the primary outcome measure. Completed questionnaires were obtained from 464 women who met eligibility criteria for the main analysis. The proportion of women screened by 10 weeks (70 days) was 9/441 (2%) in standard care, compared with 161/677 (24%) in primary care with parallel testing, and 167/590 (28%) in primary care with sequential testing. The proportion of women offered screening by 10 weeks (70 days) was 3/90 (3%) in standard care (note offer of test ascertained for questionnaire respondents only), compared with 321/677 (47%) in primary care with parallel testing, and 281/590 (48%) in primary care with sequential testing. The proportion of women screened by 26 weeks (182 days) was similar across the three groups: 324/441 (73%) in standard care, 571/677 (84%, 0.09) in primary care with parallel testing, and 481/590 (82%, 0.148) in primary care with sequential testing. The screening uptake of fathers was 51/677 (8%) in primary care with parallel testing, and 16/590 (3%) in primary care with sequential testing, and 13/441 (3%) in standard care. The predicted average total cost per pregnancy of offering antenatal SCT screening was estimated to be 13 pounds in standard care, 18.50 pounds in primary care with parallel testing, and 16.40 pounds in primary care with sequential testing. The incremental cost-effectiveness ratio (ICER) was 23 pounds in primary care with parallel testing and 12 pounds in primary care with sequential testing when compared with standard care. Women offered testing in primary care were as likely to make an informed choice as those offered screening by midwives later in pregnancy, but less than one-third of women overall made an informed choice about screening. CONCLUSIONS: Offering antenatal SCT screening as part of pregnancy-confirmation consultations significantly increased the proportion of women screened before 10 weeks (70 days), from 2% in standard care to between 16% and 27% in primary care, but additional resources may be required to implement this. There was no evidence to support offering fathers screening at the same time as women. TRIAL REGISTRATION: Current Controlled Trials ISRCTN00677850.

Trends in healthcare incident reporting and relationship to safety and quality data in acute hospitals: results from the National Reporting and Learning System.

BACKGROUND: Internationally, there is increasing recognition of the need to collect and analyse data on patient safety incidents, to facilitate learning and develop solutions. The National Patient Safety Agency (NPSA) for England and Wales has been capturing incident data from acute hospitals since November 2003. OBJECTIVES: This study analyses patterns in reporting of patient safety incidents from all acute hospitals in England to the NPSA National Reporting and Learning System, and explores the link between reporting rates, hospital characteristics, and other safety and quality datasets. METHODS: Reporting rates to the NPSA National Reporting and Learning System were analysed as trends over time, from the point at which each hospital became connected to the system. The relationships between reporting rates and other safety and quality datasets were assessed using correlation and regression analyses. RESULTS: Reporting rates increased steadily over the 18 months analysed. Higher reporting rates correlated with positive data on safety culture and incident reporting from the NHS Staff Survey, and with better risk-management ratings from the NHS Litigation Authority. Hospitals with higher overall reporting rates had a lower proportion of their reports in the "slips, trips and falls" category, suggesting that these hospitals were reporting higher numbers of other types of incident. There was no apparent association between reporting rates and the following data: standardised mortality ratios, data from other safety-related reporting systems, hospital size, average patient age or length of stay. CONCLUSIONS: Incident reporting rates from acute hospitals increase with time from connection to the national reporting system, and are positively correlated with independently defined measures of safety culture, higher reporting rates being associated with a more positive safety culture.

A preliminary model-based assessment of the cost-utility of a screening programme for early age-related macular degeneration.

OBJECTIVES: To estimate the cost-effectiveness of screening for age-related macular degeneration (AMD) by developing a decision analytic model that incorporated and assessed all of the National Screening Committee criteria. A further objective was to identify the major areas of uncertainty in the model, and so inform future research priorities in this disease area. DATA SOURCES: Major databases were searched in March 2004 and updated in January 2005. REVIEW METHODS: Systematic literature reviews covered the epidemiology and natural history of AMD, the screening and treatment effectiveness and health-related quality of life relating to AMD. A hybrid cohort-individual sampling model was implemented to describe the range of pathways between the incidence of age-related maculopathy (ARM) and death via clinical presentation and treatment at different stages of the disease. As significant shortfalls in the data available from the literature were apparent, so a range of primary data sources were also used to populate the model. To obtain estimates for the value of parameters deemed to be within an expert's remit, data describing some parameters were elicited from relevant experts. The data identified informed probability distributions describing the uncertainty around the model parameters. To incorporate joint parameter uncertainty (i.e. correlations between parameters), the AMD natural history model was calibrated probabilistically. Randomly sampled sets of input parameters were assigned weights representing the accuracy of their predictions of a set of observed model outputs. The analysis of the AMD screening model estimated the costs, numbers of quality-adjusted life-years (QALYs) and cases of blindness in a general population sample of 50-year-olds over the remainder of their lifetime, for 16 alternative screening options (including no screening). The reference case analysis incorporated current treatment options of laser photocoagulation and photodynamic therapy. Sensitivity analyses describing six alternative sets of intervention strategies, based on horizon scanning of potential future treatments for AMD, were also undertaken. RESULTS: There remains significant uncertainty about whether any form of screening for AMD is cost-effective. However, annual screening from age 60 years seems to provide the highest mean net benefits, but this is based on a cost-effectiveness estimate that has very poor precision (high levels of uncertainty). The probabilistic sensitivity analysis shows that the 95% credible interval for annual screening from age 60 years ranges from this option dominating the previous option to an incremental cost per QALY of over 0.5 million pounds sterling. Plotting a cost-effectiveness acceptability frontier shows that although annual screening from age 60 years has the highest net benefits at a value of QALY of 30,000 pounds sterling, the associated probability of this option being the most cost-effective option is only around 20%. The sensitivity analyses around potential future treatment options indicate that screening may become more cost-effective with the new treatments. CONCLUSIONS: The conclusions focus on the interpretation of the results from the perspective of defining the major areas of uncertainty, which were defined as disease progression, rates of clinical presentation, screening test and optician effectiveness, treatment effectiveness, and costs of blindness. Future research may be best targeted at assessing how routine data may be used to describe clinical presentation rates of ARM. Other potential studies include a pilot study of the effectiveness of screening and opticians' referral patterns for AMD and a costing study of blindness as a continuum of association with deterioration in vision.

The clinical effectiveness and cost-effectiveness of screening programmes for amblyopia and strabismus in children up to the age of 4-5 years: a systematic review and economic evaluation.

OBJECTIVES: To estimate the cost-effectiveness of screening for amblyopia and strabismus in children aged up to 4-5 years, also identifying the major areas of uncertainty and so inform future research priorities in this disease area. DATA SOURCES: Major electronic databases were searched in January 2006. REVIEW METHODS: Systematic literature reviews were undertaken to determine the prevalence and natural history, the screening methods, the effectiveness of treatment options and health-related quality of life issues relating to amblyopia and strabismus. The review of treatment interventions was restricted to high-quality reviews, meta-analyses and guidelines. The data derived from the review informed the structure and implementation of the decision-analytic model. RESULTS: The amblyopia screening model was analysed in detail to estimate the cost and effects of six alternative screening options comprising screening at different ages (3, 4 and 5 years) and using alternative sets of tests (visual acuity testing and the cover tests, with and without autorefraction). The reference case results showed that screening programmes that included autorefraction dominated screening programmes without autorefraction. Analyses based on the cost per case of amblyopia prevented showed screening at either 3 or 4 years prevented additional cases at a low absolute cost (3000-6000 pounds sterling). However, when these results were extrapolated to estimate the cost per quality-adjusted life-year (QALY) gained, the reference case analysis found that no form of screening is likely to be cost-effective at currently accepted values of a QALY. The wide-ranging sensitivity analyses found that the results were robust to most parameter changes. The only parameter that radically affected the results was the utility effect of loss of vision in one eye. No direct evidence of a utility effect was identified and the reference case assumed no effect. When a small effect is assumed (a reduction in utility of 2%), the incremental cost per QALY gained becomes extremely attractive for screening at both 3 and at 4 years. The expected value of perfect information was shown to be large when the unilateral vision loss utility parameter was allowed to vary, but not when it was kept constant at zero. CONCLUSIONS: The results show that the cost-effectiveness of screening for amblyopia is dependent on the long-term utility effects of unilateral vision loss. There is limited evidence on any such effect, although our subjective interpretation of the available literature is that the utility effects are likely to be minimal. Any utility study investigating such effects would need to be careful to avoid introducing bias. The reference case model did not represent potential treatment-related utility effects, primarily due to an increased probability of treated children being bullied at school. The evidence indicates that this may be a problem, and additional sensitivity analyses show that small utility decrements from bullying would improve the cost-effectiveness of early screening significantly. A prospective study of the utility effects of bullying would usefully inform the analysis, although such a study would need to be carefully planned in order to distinguish whether the overall incidence of bullying decreases with reduced school-age treatment, or whether it is displaced to other children.

Cost-utility analysis of deferasirox compared to standard therapy with desferrioxamine for patients requiring iron chelation therapy in the United Kingdom.

OBJECTIVE: The primary objective of the study was to evaluate the cost-utility of deferasirox (Exjade) compared to standard therapy using desferrioxamine (Desferal) for the control of iron overload in patients receiving frequent blood transfusions. The perspective adopted was that of the National Health Service in the UK. METHODS: Phase II/III clinical trials have shown deferasirox in the recommended doses of 20-30 mg/kg per day to have similar efficacy to desferrioxamine at equivalent doses in the control of chronic iron overload. The main difference between them is in the mode of administration. Desferrioxamine is administered parenterally as a slow subcutaneous infusion typically infused 8-12 hours a day for 5-7 days a week. In comparison, deferasirox provides 24 hour chelation via a once daily oral tablet dispersed in water or juice. An excel based economic model was developed to evaluate the annual healthcare costs and quality of life, or utility, benefits associated with differences in mode of administration, using beta-thalassaemia as the reference case. A community utility study using time trade-off methods was performed to determine utility outcomes associated with iron chelation therapy (ICT) mode of administration. RESULTS: In the reference case (patient mean weight 42 kg), deferasirox 'dominated' desferrioxamine, i.e. resulted in lower net costs and higher quality adjusted life years (QALYs). Drug dose and cost is patient weight related. Incremental cost per QALY gained was pound 7775 for patients with a mean weight of 62 kg. CONCLUSIONS: The cost-utility analysis did not take drug compliance into account. However, Deferasirox is cost-effective compared to standard iron chelation therapy with desferrioxamine, due to the cost and quality of life benefits derived from a simpler and more convenient oral mode of administration.

A review and critique of modelling in prioritising and designing screening programmes.

OBJECTIVES: To undertake a structured review and critical appraisal of methods for the model-based cost-utility analysis of screening programmes. Also to develop guidelines and an assessment checklist of good practice in the development of screening models. DATA SOURCES: Major electronic databases of healthcare and operational research literatures were searched up to June 2003. REVIEW METHODS: Searches of the literature were undertaken to identify applied and methodological studies of economic evaluations of healthcare screening programmes. All applied screening models were also reviewed in three broad disease areas (cancer, cardiovascular disease and diabetes), as well as antenatal screening. A second-level review focused on particular aspects of the modelling process through case study assessments of screening models for three specific disease areas (colorectal cancer, abdominal aortic aneurysms and antenatal screening for haemoglobinopathies). A separate literature review of studies reporting the utility effects of screening was also undertaken. Guidelines and an assessment checklist for good practice for screening modelling were developed. RESULTS: Few relevant methodological studies were identified, and no studies reporting direct empirical comparisons of alternative methodologies were retrieved. From the review of disease-based screening models, it was apparent that many alternative modelling methods had been applied, including some relatively new approaches that had not been widely disseminated. Natural history modelling is the preferred approach. Alternative modelling approaches were generally only used to extrapolate the observed effects of screening and were unsuitable for evaluating unobserved screening options. More complex model structures may incorporate important additional aspects of the disease natural history, although any benefits should outweigh the consequences of additional unobservable input parameters and increased complexity in implementing the model. No direct comparisons of more detailed and less detailed screening model structures informed areas in which more realistic representations of the disease process may be most beneficial, so only general aspects of good practice could be defined. Two structural aspects that were not well handled by existing screening models included post-diagnosis disease progression and screening uptake. Most models described the former using historical mortality rates, rather than treatment models that are representative of current treatment patterns for different stages of the disease. Constant screening uptake rates were applied to all screening programmes and attendance was not linked to disease incidence or progression. Evidence exists to inform a more detailed representation of screening uptake. The most commonly applied modelling techniques were cohort Markov models and individual sampling simulation models. Individual sampling simulation models may provide more flexibility in their representation of a screening decision problem, but any benefits should outweigh the consequences of the need to assess both variability and uncertainty. Complex mathematical models describing input parameters as continuous variables have analysed the cost-effectiveness of screening; these require further development to estimate the cost-utility of screening directly, or to inform a more detailed representation of the preclinical section of a natural history model (with a traditional state-based model describing pathways' post-clinical presentation). Calibration is a common aspect of screening models, whereby models are fitted to observed data describing outputs of the model in order to populate unobserved input parameters. The review concluded that the estimation of a reference case input parameter set is not recommended. CONCLUSIONS: The review of methods for the model-based cost-utility analysis of screening programmes identified the natural history modelling approach as the preferred general method of evaluation for screening programmes. State transition models have generally been used to represent disease natural histories, with individual sampling models more prevalent than in treatment intervention evaluations. No comparative methodological studies were identified, so no empirical data were available to inform the relative merits of alternative methodologies. The defined guidelines and assessment checklist are informed, therefore, by theoretical interpretations of the impact of alternative approaches to different components of the modelling process when applied to the cost-utility analysis of screening programmes. Further research is needed into methods with the potential to improve the accuracy of screening models, and to respond to the needs of model users.