Evaluating the Long-Term Cost-Effectiveness of the English NHS Diabetes Prevention Programme using a Markov Model.

BACKGROUND: In 2016, England launched the largest nationwide diabetes mellitus prevention programme, the NHS Diabetes Prevention Programme (NHS DPP). This paper seeks to evaluate the long-term cost-effectiveness of this programme. METHODS: A Markov cohort state transition model was developed with a 35-year time horizon and yearly cycles to compare referral to the NHS DPP to usual care for individuals with non-diabetic hyperglycaemia. The modelled cohort of individuals mirrored the age profile of referrals received by the programme by April 2020. A health system perspective was taken, with costs in UK £ Sterling (price year 2020) and outcomes in terms of quality-adjusted life-years (QALYs). Probabilistic analysis with 10,000 Monte Carlo simulations was used. Several sensitivity analyses were conducted to explore the uncertainty surrounding the base case results, particularly varying the length of time for which the effectiveness of the programme was expected to last. RESULTS: In the base case, using only the observed effectiveness of the NHS DPP at 3 years, it was found that the programme is likely to dominate usual care, by generating on average 40.8 incremental QALYs whilst saving £135,755 in costs for a cohort of 1000. At a willingness to pay of £20,000 per QALY, 98.1% of simulations were on or under the willingness-to-pay threshold. Scaling this up to the number of referrals actually received by the NHS DPP prior to April 2020, cost savings of £71.4 million were estimated over the 35-year time horizon and an additional 21,472 QALYs generated. These results are robust to several sensitivity analyses. CONCLUSION: The NHS DPP is likely to be cost-effective. Indeed, in the majority of the simulations, the NHS DPP was cost-saving and generated greater QALYs, dominating usual care. This research should serve as evidence to support the continued investment or recommissioning of diabetes prevention programmes.

Cardiac rehabilitation for children and young people (CardioActive): protocol for a single-blind randomised feasibility and acceptability study of a centre-based cardiac rehabilitation programme versus usual care in 11-16 years with heart conditions.

BACKGROUND: Congenital heart conditions are among the most common non-communicable diseases in children and young people (CYP), affecting 13.9 million CYP globally. While survival rates are increasing, support for young people adjusting to life with a heart condition is lacking. Furthermore, one in three CYP with heart conditions also experiences anxiety, depression or adjustment disorder, for which little support is offered. While adults are offered cardiac rehabilitation (CR) to support their mental and physical health, this is not offered for CYP.One way to overcome this is to evaluate a CR programme comprising exercise with mental health support (CardioActive; CA) for CYP with heart conditions. The exercise and mental health components are informed by the metacognitive model, which has been shown to be effective in treating anxiety and depression in CYP and associated with improving psychological outcomes in adult CR. METHOD AND ANALYSIS: The study is a single-blind parallel randomised feasibility trial comparing a CR programme (CA) plus usual care against usual care alone with 100 CYP (50 per arm) aged 11-16 diagnosed with a heart condition. CA will include six group exercise, lifestyle and mental health modules. Usual care consists of routine outpatient management. Participants will be assessed at three time points: baseline, 3-month (post-treatment) and 6-month follow-up. Primary outcomes are feasibility and acceptability (ie, referral rates, recruitment and retention rates, attendance at the intervention, rate of return and level of completion of follow-up data). Coprimary symptom outcomes (Strength and Difficulties Questionnaire and Paediatric Quality of Life) and a range of secondary outcomes will be administered at each time point. A nested qualitative study will investigate CYP, parents and healthcare staff views of CR and its components, and staff's experience of delivering CA. Preliminary health economic data will be collected to inform future cost-effectiveness analyses. Descriptive data on study processes and clinical outcomes will be reported. Data analysis will follow intention to treat. Qualitative data will be analysed using thematic analysis and the theoretical framework of acceptability. ETHICS AND DISSEMINATION: Ethical approval was granted on 14 February 2023 by the Greater Manchester East Research Ethics Committee (22/NW/0367). The results will be disseminated through peer-reviewed journals, conference presentations and local dissemination. TRIAL REGISTRATION NUMBER: ISRCTN50031147; NCT05968521.

Evaluating the Short-Term Costs and Benefits of a Nationwide Diabetes Prevention Programme in England: Retrospective Observational Study.

BACKGROUND: Prevention programmes typically incur short-term costs and uncertain long-term benefits. We use the National Health Service (NHS) England Diabetes Prevention Programme (NHS-DPP) to investigate whether behaviour change programmes may be cost-effective even within the short-term participation period. METHODS: We analysed 384,611 referrals between June 2016 and March 2019. We estimated NHS costs using implementation costs and provider payments. We used linear regressions to relate utility changes to the number of sessions attended, based on responses to the five-level EQ-5D (EQ-5D-5L) at baseline and final session for 18,959 participants. We then calculated the corresponding quality-adjusted life year (QALY) change for all 384,611 referrals by combining the estimated regression coefficients with the observed level of attendance, with individuals that did not attend any programme sessions being assumed to experience zero benefit. In secondary analysis, we added weight change, recorded for 18,105 participants to the regression and applied predicted values to all referrals with missing weight change values estimated using multiple imputation with chained equations. We then estimated the cost-per-QALY generated. RESULTS: Average cost per referral was £119 (standard deviation: £118; 2020 price year, UK £ Sterling). Each session attended was associated with a 0.0042 increase in utility (95% confidence interval (CI): 0.0025-0.0059). This generated 1,773 QALYs across all referrals (95% CI: 889-2,656). Cost-per-QALY was £24,929 (95% CI: £16,635-49,720) when implementation costs were excluded. Secondary analysis showed each session attended and kilogram of weight lost were associated with 0.0034 (95% CI: 0.0016-0.0051) and 0.0025 (95% CI: 0.0020-0.0031) increases in utility, respectively. These generated 1,542 QALYs, at a cost-per-QALY of £28,661 when implementation costs were excluded. CONCLUSION: Participants experienced small utility gains from session attendance and weight loss during their programme participation. These benefits alone made this low-cost behaviour change programme potentially cost-effective in the short-term.

Does recruiting patients to diabetes prevention programmes via primary care reinforce existing inequalities in care provision between general practices? A retrospective observational study.

BACKGROUND: Primary care plays a crucial role in identifying patients' needs and referring at-risk individuals to preventive services. However, well-established variations in care delivery may be replicated in this prevention activity. OBJECTIVE: To examine whether recruiting patients to the English NHS Diabetes Prevention Programme via primary care reinforces existing inequalities in care provision between practices, in terms of clinical quality, accessibility and resources. METHODS: We generated annual practice-level counts of referrals across the first 4 years of the programme (June 2016 to March 2020). These were linked to 15 indicators of practice clinical quality, access and resources measured during 2018/19. We used random effects Poisson regressions to examine associations between referrals and these indicators, controlling for practice and population characteristics, for 6871 practices in England. RESULTS: On average, practices made 3.72 referrals per 1000 population annually and rates varied substantially between practices. Referral rates were positively associated with the quality of clinical care provided. A 1 SD higher level of achievement on Quality and Outcomes Framework diabetes indicators was associated with an 11% (95% CI: 8% to 14%) higher referral rate. This positive association was consistent across all five clinical quality indicators. There was no association between referral rates and accessibility, overall payments or staffing. Associations between referrals and receiving different supplementary payments over the core contract were mixed, with 8%-11% lower referral rates for some payments but not for others. CONCLUSION: Recruiting patients to diabetes prevention programmes via primary care reinforces existing inequalities between general practices in the clinical quality of care they provide. This leaves patients registered with practices providing lower quality clinical care even more disadvantaged. Providing additional support to lower quality practices or using alternative recruitment methods may be necessary to avoid differential engagement in prevention programmes from widening these variations and potential health inequalities further.

The effects of structure, process and outcome incentives on primary care referrals to a national prevention programme.

Despite widespread use, evidence is sparse on whether financial incentives in healthcare should be linked to structure, process or outcome. We examine the impact of different incentive types on the quantity and effectiveness of referrals made by general practices to a new national prevention programme in England. We measured effectiveness by the number of referrals resulting in programme attendance. We surveyed local commissioners about their use of financial incentives and linked this information to numbers of programme referrals and attendances from 5170 general practices between April 2016 and March 2018. We used multivariate probit regressions to identify commissioner characteristics associated with the use of different incentive types and negative binomial regressions to estimate their effect on practice rates of referral and attendance. Financial incentives were offered by commissioners in the majority of areas (89%), with 38% using structure incentives, 69% using process incentives and 22% using outcome incentives. Compared to practices without financial incentives, neither structure nor process incentives were associated with statistically significant increases in referrals or attendances, but outcome incentives were associated with 84% more referrals and 93% more attendances. Outcome incentives were the only form of pay-for-performance to stimulate more participation in this national disease prevention programme.

A Systematic Review of Model-Based Economic Evaluations of Treatments for Venous Leg Ulcers.

OBJECTIVE: The aim of this review was to identify, and assess the quality of, published model-based economic evaluations relating to treatments for patients with venous leg ulcers to help inform future decision-analytic models in this clinical area. METHODS: A systematic literature search was performed on six electronic databases, from database inception until 21 May 2018. Search results were screened against predefined criteria by two independent reviewers. Data was then extracted from the included studies using a standardised form, whilst the decision-analytic model-specific Philips Checklist was used to assess quality and to inform model critique. RESULTS: A total of 23 models were identified, 12 studies used a Markov modelling approach, five used decision trees and six studies did not detail the model type. Studies were predominantly from the National Health Service (NHS)/payer perspective, with only two taking a societal perspective. Interventions were wide ranging, but dressing technologies (11/23) were most common. The intervention studied was found to be dominant in 22/23 studies. The reporting quality of papers was mostly low, with evidence behind model structures, time horizons and data selection consistently underreported across the included papers. CONCLUSIONS: This review has identified a sizeable literature of model-based economic evaluations, evaluating treatments for venous leg ulcers. However, the methods used to conduct such studies were generally poorly reported. In particular, the reporting of evidence surrounding the model structure, justification of the time horizon used and the rationale for selecting data inputs should be focused on in any future models developed.

Can You Repeat That? Exploring the Definition of a Successful Model Replication in Health Economics.

The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) modelling taskforce suggests decision models should be thoroughly reported and transparent. However, the level of transparency and indeed how transparency should be assessed are yet to be defined. One way may be to attempt to replicate the model and its outputs. The ability to replicate a decision model could demonstrate adequate reporting transparency. This review aims to explore published definitions of replication success across all scientific disciplines and to consider how such a definition should be tailored for use in health economic models. A literature review was conducted to identify published definitions of a 'successful replication'. Using these as a foundation, several definitions of replication success were constructed, to be applicable to replications of economic decision models, with the associated strengths and weaknesses of such definitions discussed. A substantial body of literature discussing replicability was found; however, relatively few studies, ten, explicitly defined a successful replication. These definitions varied from subjective assessments to expecting exactly the same results to be reproduced. Whilst the definitions that have been found may help to construct a definition specific to health economics, no definition was found that completely encompassed the unique requirements for decision models. Replication is widely discussed in other scientific disciplines; however, as of yet, there is no consensus on how replicable models should be within health economics or what constitutes a successful replication. Replication studies can demonstrate how transparently a model is reported, identify potential calculation errors and inform future reporting practices. It may therefore be a useful adjunct to other transparency or quality measures.

Barriers and Facilitators to Model Replication Within Health Economics.

BACKGROUND: Model replication is important because it enables researchers to check research integrity and transparency and, potentially, to inform the model conceptualization process when developing a new or updated model. OBJECTIVE: The aim of this study was to evaluate the replicability of published decision analytic models and to identify the barriers and facilitators to replication. METHODS: Replication attempts of 5 published economic modeling studies were made. The replications were conducted using only publicly available information within the manuscripts and supplementary materials. The replicator attempted to reproduce the key results detailed in the paper, for example, the total cost, total outcomes, and if applicable, incremental cost-effectiveness ratio reported. Although a replication attempt was not explicitly defined as a success or failure, the replicated results were compared for percentage difference to the original results. RESULTS: In conducting the replication attempts, common barriers and facilitators emerged. For most case studies, the replicator needed to make additional assumptions when recreating the model. This was often exacerbated by conflicting information being presented in the text and the tables. Across the case studies, the variation between original and replicated results ranged from -4.54% to 108.00% for costs and -3.81% to 0.40% for outcomes. CONCLUSION: This study demonstrates that although models may appear to be comprehensively reported, it is often not enough to facilitate a precise replication. Further work is needed to understand how to improve model transparency and in turn increase the chances of replication, thus ensuring future usability.

Early, specialist vocational rehabilitation to facilitate return to work after traumatic brain injury: the FRESH feasibility RCT.

BACKGROUND: Up to 160,000 people incur traumatic brain injury (TBI) each year in the UK. TBI can have profound effects on many areas of human functioning, including participation in work. There is limited evidence of the clinical effectiveness and cost-effectiveness of vocational rehabilitation (VR) after injury to promote early return to work (RTW) following TBI. OBJECTIVE: To assess the feasibility of a definitive, multicentre, randomised controlled trial (RCT) of the clinical effectiveness and cost-effectiveness of early, specialist VR plus usual care (UC) compared with UC alone on work retention 12 months post TBI. DESIGN: A multicentre, feasibility, parallel-group RCT with a feasibility economic evaluation and an embedded mixed-methods process evaluation. Randomisation was by remote computer-generated allocation. SETTING: Three NHS major trauma centres (MTCs) in England. PARTICIPANTS: Adults with TBI admitted for > 48 hours and working or studying prior to injury. INTERVENTIONS: Early specialist TBI VR delivered by occupational therapists (OTs) in the community using a case co-ordination model. MAIN OUTCOME MEASURES: Self-reported RTW 12 months post randomisation, mood, functional ability, participation, work self-efficacy, quality of life and work ability. Feasibility outcomes included recruitment and retention rates. Follow-up was by postal questionnaires in two centres and face to face in one centre. Those collecting data were blind to treatment allocation. RESULTS: Out of 102 target participants, 78 were recruited (39 randomised to each arm), representing 39% of those eligible and 5% of those screened. Approximately 2.2 patients were recruited per site per month. Of those, 56% had mild injuries, 18% had moderate injuries and 26% had severe injuries. A total of 32 out of 45 nominated carers were recruited. A total of 52 out of 78 (67%) TBI participants responded at 12 months (UC, n = 23; intervention, n = 29), completing 90% of the work questions; 21 out of 23 (91%) UC respondents and 20 out of 29 (69%) intervention participants returned to work at 12 months. Two participants disengaged from the intervention. Face-to-face follow-up was no more effective than postal follow-up. RTW was most strongly related to social participation and work self-efficacy. It is feasible to assess the cost-effectiveness of VR. Intervention was delivered as intended and valued by participants. Factors likely to affect a definitive trial include deploying experienced OTs, no clear TBI definition or TBI registers, and repatriation of more severe TBI from MTCs, affecting recruitment of those most likely to benefit/least likely to drop out. LIMITATIONS: Target recruitment was not reached, but mechanisms to achieve this in future studies were identified. Retention was lower than expected, particularly in UC, potentially biasing estimates of the 12-month RTW rate. CONCLUSIONS: This study met most feasibility objectives. The intervention was delivered with high fidelity. When objectives were not met, strategies to ensure feasibility of a full trial were identified. Future work should test two-stage recruitment and include resources to recruit from 'spokes'. A broader measure covering work ability, self-efficacy and participation may be a more sensitive outcome. TRIAL REGISTRATION: Current Controlled Trials ISRCTN38581822. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 22, No. 33. See the NIHR Journals Library website for further project information.

The Use of Decision-Analytic Models in Atopic Eczema: A Systematic Review and Critical Appraisal.

OBJECTIVE: The objective of this systematic review was to identify and assess the quality of published economic decision-analytic models within atopic eczema against best practice guidelines, with the intention of informing future decision-analytic models within this condition. METHODS: A systematic search of the following online databases was performed: MEDLINE, EMBASE, Cumulative Index to Nursing and Allied Health Literature (CINAHL), Cochrane Central Register of Controlled Trials, Database of Abstracts of Reviews of Effects, Cochrane Database of Systematic Reviews, NHS Economic Evaluation Database, EconLit, Scopus, Health Technology Assessment, Cost-Effectiveness Analysis Registry and Web of Science. Papers were eligible for inclusion if they described a decision-analytic model evaluating both the costs and benefits associated with an intervention or prevention for atopic eczema. Data were extracted using a standardised form by two independent reviewers, whilst quality was assessed using the model-specific Philips criteria. RESULTS: Twenty-four models were identified, evaluating either preventions (n = 12) or interventions (n = 12): 14 reported using a Markov modelling approach, four utilised decision trees and one a discrete event simulation, whilst five did not specify the approach. The majority, 22 studies, reported that the intervention was dominant or cost effective, given the assumptions and analytical perspective taken. Notably, the models tended to be short-term (16 used a time horizon of ≤1 year), often providing little justification for the limited time horizon chosen. The methodological and reporting quality of the studies was generally weak, with only seven studies fulfilling more than 50% of their applicable Philips criteria. CONCLUSIONS: This is the first systematic review of decision models in eczema. Whilst the majority of models reported favourable outcomes in terms of the cost effectiveness of the new intervention, the usefulness of these findings for decision-making is questionable. In particular, there is considerable scope for increasing the range of interventions evaluated, for improving modelling structures and reporting quality.

Economic evidence for the prevention and treatment of atopic eczema: a protocol for a systematic review.

BACKGROUND: Eczema, synonymous with atopic eczema or atopic dermatitis, is a chronic skin disease that has a similar impact on health-related quality of life as other chronic diseases. The proposed research aims to provide a comprehensive systematic assessment of the economic evidence base available to inform economic modelling and decision making on interventions to prevent and treat eczema at any stage of the life course. Whilst the Global Resource of Eczema Trials (GREAT) database collects together the effectiveness evidence for eczema, there is currently no such systematic resource on the economics of eczema. It is important to gain an overview of the current state of the art of economic methods in the field of eczema in order to strengthen the economic evidence base further. METHODS/DESIGN: The proposed study is a systematic review of the economic evidence surrounding interventions for the prevention and treatment of eczema. Relevant search terms will be used to search MEDLINE, EMBASE, Database of Abstracts of Reviews of Effects, Cochrane Database of Systematic Reviews, Cochrane Central Register of Controlled Trials, National Health Service (NHS) Economic Evaluation Database, Health Technology Assessment, Cumulative Index to Nursing and Allied Health Literature, EconLit, Scopus, Cost-Effectiveness Analysis Registry and Web of Science in order to identify relevant evidence. To be eligible for inclusion studies will be primary empirical studies evaluating the cost, utility or full economic evaluation of interventions for preventing or treating eczema. Two reviewers will independently assess studies for eligibility and perform data abstraction. Evidence tables will be produced presenting details of study characteristics, costing methods, outcome methods and quality assessment. The methodological quality of studies will be assessed using accepted checklists. DISCUSSION: The systematic review is being undertaken to identify the type of economic evidence available, summarise the results of the available economic evidence and critically appraise the quality of economic evidence currently available to inform future economic modelling and resource allocation decisions about interventions to prevent or treat eczema. We aim to use the review to offer guidance about how to gather economic evidence in studies of eczema and/or what further research is necessary in order to inform this. SYSTEMATIC REVIEW REGISTRATION: PROSPERO CRD42015024633.