Real world evaluation of kidney failure risk equations in predicting progression from chronic kidney disease to kidney failure in an Australian cohort.

BACKGROUND: Chronic kidney disease progression to kidney failure is diverse, and progression may be different according to genetic aspects and settings of care. We aimed to describe kidney failure risk equation prognostic accuracy in an Australian population. METHODS: A retrospective cohort study was undertaken in a public hospital community-based chronic kidney disease service in Brisbane, Australia, which included a cohort of 406 adult patients with chronic kidney disease Stages 3-4 followed up over 5 years (1/1/13-1/1/18). Risk of progression to kidney failure at baseline using Kidney Failure Risk Equation models with three (eGFR/age/sex), four (add urinary-ACR) and eight variables (add serum-albumin/phosphate/bicarbonate/calcium) at 5 and 2 years were compared to actual patient outcomes. RESULTS: Of 406 patients followed up over 5 years, 71 (17.5%) developed kidney failure, while 112 died before reaching kidney failure. The overall mean difference between observed and predicted risk was 0.51% (p = 0.659), 0.93% (p = 0.602), and - 0.03% (p = 0.967) for the three-, four- and eight-variable models, respectively. There was small improvement in the receiver operating characteristic-area under the curve from three-variable to four-variable models: 0.888 (95%CI = 0.819-0.957) versus 0.916 (95%CI = 0.847-0.985). The eight-variable model showed marginal receiver operating characteristic-area under the curve improvement: 0.916 (95%CI = 0.847-0.985) versus 0.922 (95%CI = 0.853-0.991). The results were similar in predicting 2 year risk of kidney failure. CONCLUSIONS: The kidney failure risk equation accurately predicted progression to kidney failure in an Australian chronic kidney disease population. Younger age, male sex, lower estimated glomerular filtration rate, higher albuminuria, diabetes mellitus, tobacco smoking and non-Caucasian ethnicity were associated with increased risk of kidney failure. Cause-specific cumulative incidence function for progression to kidney failure or death, stratified by chronic kidney disease stage, demonstrated differences within different chronic kidney disease stages, highlighting the interaction between comorbidity and outcome.

Estimating cost-effectiveness in public health: a summary of modelling and valuation methods.

It is acknowledged that economic evaluation methods as they have been developed for Health Technology Assessment do not capture all the costs and benefits relevant to the assessment of public health interventions. This paper reviews methods that could be employed to measure and value the broader set of benefits generated by public health interventions. It is proposed that two key developments are required if this vision is to be achieved. First, there is a trend to modelling approaches that better capture the effects of public health interventions. This trend needs to continue, and economists need to consider a broader range of modelling techniques than are currently employed to assess public health interventions. The selection and implementation of alternative modelling techniques should be facilitated by the production of better data on the behavioural outcomes generated by public health interventions. Second, economists are currently exploring a number of valuation paradigms that hold the promise of more appropriate valuation of public health interventions outcomes. These include the capabilities approach and the subjective well-being approach, both of which offer the possibility of broader measures of value than the approaches currently employed by health economists. These developments should not, however, be made by economists alone. These questions, in particular what method should be used to value public health outcomes, require social value judgements that are beyond the capacity of economists. This choice will require consultation with policy makers, and perhaps even the general public. Such collaboration would have the benefit of ensuring that the methods developed are useful for decision makers.

Making the economic case for prevention--a view from Wales.

BACKGROUND: It is widely acknowledged that adverse lifestyle behaviours in the population now will place an unsustainable burden on health service resources in the future. It has been estimated that the combined cost to the NHS in Wales of overweight and obesity, alcohol and tobacco is in excess of £540 million.In the current climate of financial austerity, there can be a tendency for the case for prevention efforts to be judged on the basis of their scope for cost savings. This paper was prompted by discussion in Wales about the evidence for the cost savings from prevention and early intervention and a resulting concern that these programmes were thus being evaluated in policy terms using an incorrect metric. Following a review of the literature, this paper contributes to the discussion of the potential role that economics can play in informing decisions in this area. DISCUSSION: This paper argues that whilst studies of the economic burden of diseases provide information about the magnitude of the problem faced, they should not be used as a means of priority setting. Similarly, studies discussing the likelihood of savings as a result of prevention programmes may be distorting the arguments for public health.Prevention spend needs to be considered purposefully, resulting in a strategic commitment to spending. The role of economics in this process is to provide evidence demonstrating that information and support can be provided cost effectively to individuals to change their lifestyles thus avoiding lifestyle related morbidity and mortality. There is growing evidence that prevention programmes represent value for money using the currently accepted techniques and decision making metrics such as those advocated by NICE. SUMMARY: The issue here is not one of arguing that the economic evaluation of prevention and early intervention should be treated differently, although in some instances that may be appropriate, rather it is about making the case for these interventions to be treated and evaluated to the same standard. The difficulty arises when a higher standard of cost saving may be expected from prevention and public health programmes.The paper concludes that it is of vital importance that during times of budget constraints, as currently faced, the public health budgets are not eroded to fund secondary care budget shortfalls, which are more easily identifiable. To do so would diminish any possibility of reducing the future burden faced by the NHS of lifestyle-related illnesses.

The cost effectiveness of NHS physiotherapy support for occupational health (OH) services.

BACKGROUND: Musculoskeletal pain is detrimental to quality of life (QOL) and disruptive to activities of daily living. It also places a major economic burden on healthcare systems and wider society. In 2006, the Welsh Assembly Government (WAG) established a three tiered self-referral Occupational Health Physiotherapy Pilot Project (OHPPP) comprising: 1.) telephone advice and triage, 2.) face-to-face physiotherapy assessment and treatment if required, and 3.) workplace assessment and a return-to-work facilitation package as appropriate. This study aimed to evaluate the feasibility and cost-effectiveness of the pilot service. METHODS: A pragmatic cohort study was undertaken, with all OHPPP service users between September 2008 and February 2009 being invited to participate. Participants were assessed on clinical status, yellow flags, sickness absence and work performance at baseline, after treatment and at 3 month follow up. Cost-effectiveness was evaluated from both top-down and bottom-up perspectives and cost per Quality Adjusted Life Year (cost/QALY) was calculated. The cost-effectiveness analysis assessed the increase in service cost that would be necessary before the cost-effectiveness of the service was compromised. RESULTS: A total of 515 patients completed questionnaires at baseline. Of these, 486 were referred for face to face assessment with a physiotherapist and were included in the analysis for the current study. 264 (54.3%) and 199 (40.9%) were retained at end of treatment and 3 month follow up respectively. An improvement was observed at follow up in all the clinical outcomes assessed, as well as a reduction in healthcare resource usage and sickness absence, and improvement in self-reported work performance. Multivariate regression indicated that baseline and current physical health were associated with work-related outcomes at follow up. The costs of the service were £194-£360 per service user depending on the method used, and the health gains contributed to a cost/QALY of £1386-£7760, which would represent value for money according to current UK thresholds. Sensitivity analyses demonstrated that the service would remain cost effective until the service costs were increased to 160% per user. CONCLUSIONS: This pragmatic evaluation of the OHPPP indicated that it was likely to be feasible in terms of service usage and could potentially be cost effective in terms of QALYs. Further, the study confirmed that improving physical health status for musculoskeletal pain patients is important in reducing problems with work capacity and related costs. This study suggests that this type of service could be potentially be useful in reducing the burden of pain and should be further investigated, ideally via randomised controlled trials assessing effectiveness and cost-effectiveness.

Exploring the role of economics in prioritization in public health: what do stakeholders think?

BACKGROUND: Debates surrounding the use of conventional approaches in public health and the existence of perceived barriers to using the results of economic evaluations have led to questions posed as to how to establish priorities within public health schemes. The aims of this study were therefore to explore the feasibility and validity of economic evaluation techniques in developing priorities within public health programmes and consider the extent to which different presentational approaches are likely to be incorporated into decision-making, from perspectives of relevant stakeholders. METHODS: An advisory board, representative of potential users of economic evaluations, was set up to identify preferences for how findings from economic evaluations might be presented to decision makers and to test the impact of different approaches, different outputs and different presentational styles. The board was divided into two groups, each of which was given three hypothetical 'scenarios' to consider. The scenarios comprised descriptions of methods and outputs, with costs, effects, target population and context of intervention constant across all scenarios. RESULTS: The perceived validity of estimates of effectiveness was vitally important, along with sufficient information to gauge whether designs were appropriate and to assess implementation practicalities. Cost-benefit analysis and cost-utility analysis were the preferred approaches despite their complexity, although participants required benchmarks to place net-benefit estimates from cost-benefit analyses into context. CONCLUSION: Further research is required to substantiate and build on these preliminary findings and collaborations between economists and policy makers are needed to develop clear, rigorous and standard guidance relating to economic evaluation, recognizing the diversity of public health strategies.

A pragmatic randomised controlled trial of the Welsh National Exercise Referral Scheme: protocol for trial and integrated economic and process evaluation.

BACKGROUND: The benefits to health of a physically active lifestyle are well established and there is evidence that a sedentary lifestyle plays a significant role in the onset and progression of chronic disease. Despite a recognised need for effective public health interventions encouraging sedentary people with a medical condition to become more active, there are few rigorous evaluations of their effectiveness. Following NICE guidance, the Welsh national exercise referral scheme was implemented within the context of a pragmatic randomised controlled trial. METHODS/DESIGN: The randomised controlled trial, with nested economic and process evaluations, recruited 2,104 inactive men and women aged 16+ with coronary heart disease (CHD) risk factors and/or mild to moderate depression, anxiety or stress. Participants were recruited from 12 local health boards in Wales and referred directly by health professionals working in a range of health care settings. Consenting participants were randomised to either a 16 week tailored exercise programme run by qualified exercise professionals at community sports centres (intervention), or received an information booklet on physical activity (control). A range of validated measures assessing physical activity, mental health, psycho-social processes and health economics were administered at 6 and 12 months, with the primary 12 month outcome measure being 7 day Physical Activity Recall. The process evaluation explored factors determining the effectiveness or otherwise of the scheme, whilst the economic evaluation determined the relative cost-effectiveness of the scheme in terms of public spending. DISCUSSION: Evaluation of such a large scale national public health intervention presents methodological challenges in terms of trial design and implementation. This study was facilitated by early collaboration with social research and policy colleagues to develop a rigorous design which included an innovative approach to patient referral and trial recruitment, a comprehensive process evaluation examining intervention delivery and an integrated economic evaluation. This will allow a unique insight into the feasibility, effectiveness and cost effectiveness of a national exercise referral scheme for participants with CHD risk factors or mild to moderate anxiety, depression, or stress and provides a potential model for future policy evaluations. TRIAL REGISTRATION: Current Controlled Trials ISRCTN47680448.

Free breakfasts in schools: design and conduct of a cluster randomised controlled trial of the Primary School Free Breakfast Initiative in Wales [ISRCTN18336527].

BACKGROUND: School-based breakfast provision is increasingly being seen as a means of improving educational performance and dietary behaviour amongst children. Furthermore, recognition is growing that breakfast provision offers potential as a means of addressing social inequalities in these outcomes. At present however, the evidence base on the effectiveness of breakfast provision in bringing about these improvements is limited. METHODS/DESIGN: This paper describes the research design of a large scale evaluation of the effectiveness of the Welsh Assembly Government's Primary School Free Breakfast Initiative. A cluster randomised trial, with school as the unit of randomisation was used for the outcome evaluation, with a nested qualitative process evaluation. Quantitative outcome measures included dietary habits, attitudes, cognitive function, classroom behaviour, and school attendance. The study recruited 111 primary schools in Wales, of which 56 were randomly assigned to control condition and 55 to intervention. Participants were Year 5 and 6 students (aged 9-11 years) in these schools. Data were collected for all 111 schools at each of three time points: baseline, 4 month and 12 month follow-up. This was achieved through a repeated cross-sectional survey of approximately 4350 students on each of these occasions. Of those students in Year 5 at baseline, 1975 provided data at one or both of the follow-ups, forming a nested cohort. The evaluation also included a nested process evaluation, using questionnaires, semi-structured interviews and case studies with students, school staff, and local authority scheme coordinators as key informants. DISCUSSION: An overview of the methods used for the evaluation is presented, providing an example of the feasibility of conducting robust evaluations of policy initiatives using a randomised trial design with nested process evaluation. Details are provided of response rates and the flow of participants. Reflection is offered on methodological issues encountered at various stages through the course of the study, focusing upon issues associated with conducting a randomised trial of a government policy initiative, and with conducting research in school settings.

Cluster-randomised trial of risk communication to enhance informed uptake of cervical screening.

BACKGROUND: Women overestimate both population and individual risk of cervical cancer. This may contribute to the recognised excess screening frequency for low-risk women. AIM: To investigate whether an individualized risk communication package could affect stated preferences for screening interval and actual screening behaviour. DESIGN: Pragmatic, practice-based cluster randomised controlled trial. SETTING: Twenty-nine practices (15 intervention, 14 control) in North Wales recruited 1890 women attending for cervical smears. METHOD: A risk communication package containing visual material was compared with normal practice. Practice nurses received training in its delivery. The short-term primary outcome was stated preference for screening interval; the long-term primary outcome was actual screening behaviour. RESULTS: In the short term, intervention arm women were significantly less likely to prefer a shorter than recommended interval (odds ratio [OR] = 0.51, 95% confidence interval [CI] = 0.41 to 0.64; P < 0.0001). At the five-year follow-up, fewer women in the intervention arm had attended for screening sooner than their recommended recall. The magnitude of difference in excess screening interval preference and behaviour was similar, but behaviour had a wider confidence interval and a marginally non-significant P-value (OR = 0.61, 95% CI = 0.36 to 1.03; P = 0.063). Better knowledge and more accurate risk perceptions were demonstrated, with an improvement in measures of anxiety. The extra cost per woman receiving the intervention was 6 Pounds. CONCLUSIONS: Women's perception of risk contributes to determining screening intervals in addition to practice factors. Simple risk information delivered in primary care affected women's stated preferences for tests. The impact on actual screening behaviour was more equivocal. Overall, the intervention showed a substantial benefit and any disbenefit can be ruled out. This approach to providing risk information could, at low cost, benefit other screening programmes and may relieve anxiety.

General practitioners' use of magnetic resonance imaging: an open randomized trial comparing telephone and written requests and an open randomized controlled trial of different methods of local guideline dissemination.

AIM: To determine the impact and cost-effectiveness of telephone versus written access to magnetic resonance imaging (MRI), and of different strategies for disseminating locally produced guidelines, upon requests by general practitioners (GPs) for knee and lumbar spine investigation. MATERIAL AND METHODS: Two sequential pragmatic open cluster-randomized trials were conducted within 39 general practices. The outcome measure in each trial was concordance of request with local guidelines. Trial 1: practices requested MRI by telephone or in writing. Trial 2: all practices received guidelines, plus either: a practice-based seminar, practice-specific audit feedback, both seminar and feedback, or neither. RESULTS: A total of 414 requests were assessed in the two trials. Trial 1: telephone access cost pound4.86 more per request but rates of concordant requests were equivalent (65%/64%: telephone/written). Trial 2: compared to the control group, costs per practice were pound1911 higher in seminar group, pound1543 higher in feedback group and pound3578 higher for those receiving both. Concordance was greater following the intervention (74% vs 65%; P < 0.05), but there was no difference between the four study groups. CONCLUSIONS: Method of access did not affect concordance. Written access was more cost-effective. Seminars and feedback were no more effective in modifying practice than guidelines alone, which was thus the most cost-effective option.