Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature.

Cost-effectiveness analyses commonly use population or sample averages, which can mask key differences across subgroups and may lead to suboptimal resource allocation. Despite there being several new methods developed over the last decade, there is no recent summary of what methods are available to researchers. This review sought to identify advances in methods for addressing patient heterogeneity in economic evaluations and to provide an overview of these methods. A literature search was conducted using the Econlit, Embase and MEDLINE databases to identify studies published after 2011 (date of a previous review on this topic). Eligible studies needed to have an explicit methodological focus, related to how patient heterogeneity can be accounted for within a full economic evaluation. Sixteen studies were included in the review. Methodologies were varied and included regression techniques, model design and value of information analysis. Recent publications have applied methodologies more commonly used in other fields, such as machine learning and causal forests. Commonly noted challenges associated with considering patient heterogeneity included data availability (e.g., sample size), statistical issues (e.g., risk of false positives) and practical factors (e.g., computation time). A range of methods are available to address patient heterogeneity in economic evaluation, with relevant methods differing according to research question, scope of the economic evaluation and data availability. Researchers need to be aware of the challenges associated with addressing patient heterogeneity (e.g., data availability) to ensure findings are meaningful and robust. Future research is needed to assess whether and how methods are being applied in practice.

Longitudinal realist evaluation of the Dementia PersonAlised Care Team (D-PACT) intervention: protocol.

BACKGROUND: Different dementia support roles exist but evidence is lacking on which aspects are best, for whom, and in what circumstances, and on their associated costs and benefits. Phase 1 of the Dementia PersonAlised Care Team programme (D-PACT) developed a post-diagnostic primary care-based intervention for people with dementia and their carers and assessed the feasibility of a trial. AIM: Phase 2 of the programme aims to 1) refine the programme theory on how, when, and for whom the intervention works; and 2) evaluate its value and impact. DESIGN & SETTING: A realist longitudinal mixed-methods evaluation will be conducted in urban, rural, and coastal areas across South West and North West England where low-income or ethnic minority populations (for example, South Asian) are represented. Design was informed by patient, public, and professional stakeholder input and phase 1 findings. METHOD: High-volume qualitative and quantitative data will be collected longitudinally from people with dementia, carers, and practitioners. Analyses will comprise the following: 1) realist longitudinal case studies; 2) conversation analysis of recorded interactions; 3) statistical analyses of outcome and experience questionnaires; 4a) health economic analysis examining costs of delivery; and 4b) realist economic analysis of high-cost events and 'near misses'. All findings will be synthesised using a joint display table, evidence appraisal tool, triangulation, and stakeholder co-analysis. CONCLUSION: The realist evaluation will describe how, why, and for whom the intervention does or does not lead to change over time. It will also demonstrate how a non-randomised design can be more appropriate for complex interventions with similar questions or populations.

Acknowledging Patient Heterogeneity in Economic Evaluations in Schizophrenia: A Systematic Review.

OBJECTIVES: Schizophrenia is a severe mental illness with heterogeneous etiology, range of symptoms, and course of illness. Cost-effectiveness analysis often applies averages from populations, which disregards patient heterogeneity even though there are a range of methods available to acknowledge patient heterogeneity. This review evaluates existing economic evaluations of interventions in schizophrenia to understand how patient heterogeneity is currently reflected in economic evaluation. METHODS: Electronic searches of MEDLINE, Embase, and PsycINFO via Ovid and the Health Technology Assessment database were run to identify full economic evaluations of interventions aiming to reduce the symptoms associated with schizophrenia. Two levels of screening were used, and explicit inclusion criteria were applied. Prespecified data extraction and critical appraisal were performed. RESULTS: Seventy-six relevant studies were identified. More than half (41 of 76) of the articles acknowledged patient heterogeneity in some way through discussion or methods. There was a range of patient characteristics considered, including demographics and socioeconomic factors (eg, age, educational level, ethnicity), clinical characteristics (eg, symptom severity, comorbidities), and preferences (eg, preferences related to outcomes or symptoms). Subgroup analyses were rarely reported (8 of 76). CONCLUSIONS: Patient heterogeneity was frequently mentioned in studies but was rarely thoroughly investigated in the identified economic evaluations. When investigated, included patient characteristics and methods were found to be heterogeneous. Understanding and acknowledging patient heterogeneity may alter the conclusions of cost-effectiveness evaluations; subsequently, we would encourage further research in this area.

Factors Limiting Subgroup Analysis in Cost-Effectiveness Analysis and a Call for Transparency.

The use of population averages in cost-effectiveness analysis may hide important differences across subgroups, potentially resulting in suboptimal resource allocation, reduced population health and/or increased health inequalities. We discuss the factors that limit subgroup analysis in cost-effectiveness analysis and propose more thorough and transparent reporting. There are many issues that may limit whether subgroup analysis can be robustly included in cost-effectiveness analysis, including challenges with prespecifying and justifying subgroup analysis, identifying subgroups that can be implemented (identified and targeted) in practice, resource and data requirements, and statistical and ethical concerns. These affect every stage of the design, development and reporting of cost-effectiveness analyses. It may not always be possible to include and report relevant subgroups in cost effectiveness, e.g. due to data limitations. Reasons for not conducting subgroup analysis may be heterogeneous, and the consequences of not acknowledging patient heterogeneity can be substantial. We recommend that when potentially relevant subgroups have not been included in a cost-effectiveness analysis, authors report this and discuss their rationale and the limitations of this. Greater transparency of subgroup reporting should provide a starting point to overcoming these challenges in future research.

Carer preferences for home support services in later stage dementia.

OBJECTIVES: To examine the relative importance of different home support attributes from the perspective of carers of people with later-stage dementia. METHOD: Preferences from 100 carers, recruited through carers' organisations, were assessed with a Discrete Choice Experiment (DCE) survey, administered online and by paper questionnaire. Attributes were informed by an evidence synthesis and lay consultations. A conditional logit model was used to estimate preference weights for the attributes within a home support 'package'. RESULTS: The most preferred attributes were 'respite care, available regularly to fit your needs' (coefficient 1.29, p = < 0.001) and 'home care provided regularly for as long as needed' (coefficient 0.93, p = < 0.001). Cost had a significant effect with lower cost packages preferred. Findings were similar regardless of the method of administration, with respite care considered to be the most important attribute for all carers. Carers reported that completing the DCE had been a positive experience; however, feedback was mixed overall. CONCLUSIONS: These carer preferences concur with emerging evidence on home support interventions for dementia. Respite care, home care and training on managing difficulties provided at home are important components. Carers' preferences revealed the daily challenges of caring for individuals with later stage dementia and the need for tailored and specialised home support.

Costs of formal and informal care at home for people with dementia: 'Expert panel' opinions from staff and informal carers.

Effective home support in dementia is key in delaying nursing home admission. However, home support is frequently not tailored to the individual needs of people with dementia. Staff allocating home support services may not identify important care needs, which only be recognised by informal carers. The purpose of this study was to explore the balance of informal and formal home support and their associated costs from the perspectives of both informal carers and paid staff. Five case vignettes of people with dementia were designed based on an existing English data set from a European study into transition into long-term care (the RightTimePlaceCare programme), representing 42 per cent of the English sample. In total, 14 informal carers and 14 paid staff were consulted in separate groups, as expert panels, regarding their recommendations for home care services for each vignette. Care recommendations of carers and staff were costed based on nationally available unit costs and compared. Informal carers allocated fewer hours of care than staff. Personal and domestic home care and day care centres were the most frequently recommended formal services by both groups, and some vignettes of people with dementia were recommended for care home admission. The ratio of costs of informal versus formal support was relatively equal for paid staff, yet unbalanced from the perspectives of informal carers with a greater proportion of formal care costs. Recommendations from this study can help shape dementia care to be more tailored to the individual needs of people with dementia and their carers.

A Systematic Review of the Economic Evidence for Home Support Interventions in Dementia.

BACKGROUND: Recent evidence signals the need for effective forms of home support to people with dementia and their carers. The cost-effectiveness evidence of different approaches to support is scant. OBJECTIVES: To appraise economic evidence on the cost-effectiveness of home support interventions for dementia to inform future evaluation. METHODS: A systematic literature review of full and partial economic evaluations was performed using the British National Health Service Economic Evaluation Database supplemented by additional references. Study characteristics and findings, including incremental cost-effectiveness ratios, when available, were summarized narratively. Study quality was appraised using the National Health Service Economic Evaluation Database critical appraisal criteria and independent ratings, agreed by two reviewers. Studies were located on a permutation matrix describing their mix of incremental costs/effects to aid decision making. RESULTS: Of the 151 articles retrieved, 14 studies met the inclusion criteria: 8 concerning support to people with dementia and 6 to carers. Five studies were incremental cost-utility analyses, seven were cost-effectiveness analyses, and two were cost consequences analyses. Five studies expressed incremental cost-effectiveness ratios as cost per quality-adjusted life-year (£6,696-£207,942 per quality-adjusted life-year). In four studies, interventions were dominant over usual care. Two interventions were more costly but more beneficial and were favorable against current acceptability thresholds. CONCLUSIONS: Occupational therapy, home-based exercise, and a carers' coping intervention emerged as cost-effective approaches for which there was better evidence. These interventions used environmental modifications, behavior management, physical activity, and emotional support as active components. More robust evidence is needed to judge the value of these and other interventions across the dementia care pathway.

Evaluating the effectiveness of different approaches to home support for people in later stage dementia: a protocol for an observational study.

BACKGROUND: Dementia is a major health problem with a growing number of people affected by the condition, both directly and indirectly through caring for someone with dementia. Many live at home but little is known about the range and intensity of the support they receive. Previous studies have mainly reported on discrete services within a single geographical area. This paper presents a protocol for study of different services across several sites in England. The aim is to explore the presence, effects, and cost-effectiveness of approaches to home support for people in later stage dementia and their carers. METHODS: This is a prospective observational study employing mixed methods. At least 300 participants (people with dementia and their carers) from geographical areas with demonstrably different ranges of services available for people with dementia will be selected. Within each area, participants will be recruited from a range of services. Participants will be interviewed on two occasions and data will be collected on their characteristics and circumstances, quality of life, carer health and burden, and informal and formal support for the person with dementia. The structured interviews will also collect qualitative data to explore the perceptions of older people and carers. CONCLUSIONS: This national study will explore the components of appropriate and effective home support for people with late stage dementia and their carers. It aims to inform commissioners and service providers across health and social care.

Approaches to Information Sharing and Assessment: Evidence From a Demonstration Program.

This article categorizes and delineates approaches to information sharing and assessment in a demonstration program established by central government in England. Its purpose was to develop and test a set of principles relating to a general assessment framework for adults in demonstrator sites, maximizing the use of information technology where feasible. The method employed comprised the systematic analysis of documents associated with the funding application and a telephone interview with personnel in each site. Data were collected from 17 initiatives, 13 of which provided detailed information on information sharing within the assessment process. A taxonomy of approaches was produced and information sharing in the assessment process reported in terms of setting and personnel; approaches to data collection, storage, and transfer; and changes to the process. A classification of the initiatives within a demonstration program was a useful means of describing them. Measures of intermediate outcomes captured changes in information sharing between agencies. Local initiatives were successful in promoting electronic information sharing between health and social care agencies.

An examination of assessment arrangements and service use for older people in receipt of care management.

With anticipated greater demand for formal care services globally, this article examines the sociodemographic and health characteristics of frail older people in receipt of community support. Data were collected from audits of case files of older people receiving care management at two time points during which two government policy initiatives were implemented to promote greater standardization in health and social care provision for older people in England. Findings at Time 2 revealed that there were higher levels of physical and mental impairment and more health care assessments undertaken. There was a slight decrease in home care receipt but a marginal increase of more intensive home care provision. Service users living with a carer were less likely to receive home care but more likely to receive respite care or day care than those living alone. The policy goal of widening access to specialist health and social care services for older people with mental health problems was achieved. Guidance that focused eligibility criteria on the identification of older people with complex needs required the availability of appropriate support and services. Irrespective of policy initiatives, the sociodemographic characteristics of older people and the availability of informal support are principal determinants of service provision.

Cost effectiveness of pilot self-assessment sites in community care services in England.

OBJECTIVE: Self-assessment has been advocated in community care but little is known of its cost effectiveness in practice. We evaluated cost effectiveness of pilot self-assessment approaches. METHODS: Data were collected from 13 pilot projects in England, selected by central government, between October 2006 and November 2007. These were located within preventative services for people with low-level needs, occupational therapy, or assessment and care management. Cost effectiveness, over usual care, was assessed by incremental cost-effectiveness ratios (ICERs), in British pounds per unit gain in assessment satisfaction. A public-sector perspective was adopted; the provider costs of the agencies taking part. RESULTS: At 2006-07 prices, including start-up and on-going costs, only three pilots demonstrated cost effectiveness. Two pilots in assessment and care management had ICERs of £3810 and £755 per satisfaction gained, well below a benchmark from a trial of usual assessment of £18296 per satisfaction gained. When extrapolating uptake to numbers accessing assessments over 1 year, one occupational therapy pilot, of £123/satisfaction gained, also fell below this benchmark in sensitivity analysis. There was less evidence for preventative services. CONCLUSIONS AND IMPLICATIONS: Most pilot projects were not cost effective. However, self assessment is potentially cost effective in assessment and care management and occupational therapy services. Better quality cost data from pilot sites would have permitted more detailed analysis. Measuring downstream effects in terms of users' well being from receipt of self-assessment would also be beneficial.

Metastatic disease of the long bones: a review of the health care burden in a major trauma centre.

BACKGROUND: More than 140,000 new cases of cancer are diagnosed annually in Canada, nearly half of which metastasize to bone. The implications for orthopedic oncology services are potentially huge. We reviewed the experience in a major Canadian orthopedic trauma centre treating long bone metastases. The primary aim was to quantify the caseload, and the secondary aim was to report on the methods of fixation. METHODS: We conducted a retrospective review of all patients treated for pathologic lesions or fracture secondary to metastatic disease over a 20-year period from July 1987 to March 2007. RESULTS: The mean number of cases treated annually was 13. Most patients came from the local oncology centre. The median length of stay in hospital was 11 days. In-hospital mortality was 14%. The fatal pulmonary embolus rate was 5% for femoral lesions. The revision rate for the operative intervention was 3%. CONCLUSION: The caseload was much lower than anticipated, likely owing to under-referring from oncology services. The high mortality rate may reflect delay in seeking orthopedic opinion, but overall the fixation methods appeared durable.

Identification and recognition of depression in community care assessments: impact of a national policy in England.

BACKGROUND: Depression continues to be under-recognized in older people. Most policies addressing this issue focus on the primary health care team. However, recognition may be improved by use of assessment tools and collaboration between secondary health and social care, particularly at the assessment stage. This study aimed to evaluate whether the Single Assessment Process (SAP), introduced in England from April 2004, promoting such processes, improved the identification and correct recognition of depression by enhancing the content of statutory community care assessments by social services care managers. METHODS: An observational study compared depression identification and its accuracy ("correct recognition") in samples of older people before and after SAP introduction. Participants were interviewed using standardized measures including the Geriatric Depression Scale (GDS). Depression elicited from the GDS was compared with that recorded in community care assessments with calculation of inter-rater reliabilities (kappa statistic) pre- and post-SAP. Logistic regression examined the associations between the policy's introduction, potential confounding factors (depression, cognitive impairment, function, behavior and characteristics) and the identification and correct recognition of depression. RESULTS: Whilst the identification of depression was more likely after SAP, its correct recognition did not improve after the policy, with only slight agreement between GDS and community care assessments. The existence of depression and cognitive impairment made identification, but not correct recognition, more likely. CONCLUSIONS: Correct recognition of depression was not improved in these statutory care assessments following the policy. Recognizing and thus responding to depression in a coordinated and appropriate way in the community requires further action.

Developing multidisciplinary assessment--exploring the evidence from a social care perspective.

BACKGROUND: This paper provides an initial evaluation of the impact of the Single Assessment Process (SAP) in England upon practice regarding multidisciplinary assessment. OBJECTIVES: To investigate changes in recorded health needs of older people and in the number of multidisciplinary assessments undertaken using social care agencies' case files. To examine differences in approaches to the use of multidisciplinary assessment for older people with different health needs. METHODS: An audit of case files of older people living in the community in receipt of social care in three areas carried out at two time periods before and after implementation of the SAP in 2004. Information extracted from files covered a number of domains including evidence of multidisciplinary assessments, and presence of mental and physical health problems. RESULTS: Data from 144 files at Time 1 were compared with 145 files from Time 2. Little evidence emerged that older people at Time 2 were more dependent. However, significantly more multidisciplinary assessments were undertaken following the introduction of the SAP, in particular by occupational therapists and secondary health care teams. Cognitive impairment was a significant predictor of multidisciplinary assessment at both time periods. CONCLUSIONS: The impact of the introduction of the SAP has been mediated by the influence of other policies in England: intermediate care and integrated health and social care provision. Nevertheless, the data suggest that consideration be given to more effective targeting of multidisciplinary assessments on the grounds of both cost and more accurate identification of those who will benefit from the process.

Integrated specialist assessment of older people and predictors of care-home admission.

BACKGROUND: Effective community-based care for older people requires the integration of assessment approaches by old age psychiatry, geriatric medicine and social services care management. This study examines the value of such collaboration in the assessment of older people at risk of entering care homes. METHOD: A randomized controlled trial of integrated assessment (care management with additional clinical assessment by old age psychiatrist or geriatrician) versus care management assessment only, for older people at risk of care-home admission. Older people's characteristics, physical and cognitive functioning, depression, behaviour and quality of care measures were recorded at assessment and 6 months. Differences in clinicians' and care managers' recommendations and placement rates between experimental and control groups were analysed. Logistic regression, examining associations between different needs-related characteristics and the likelihood of care-home admission, was used to characterize placement decisions. RESULTS: Clinicians recommended fewer older people for placement than did care managers in their usual assessments (a reduction of 85%). This did not, however, translate into a reduction in actual numbers of placements, although placements were delayed in those receiving the integrated assessment. Placements of those receiving the integrated assessment were influenced more by clinical factors, most notably cognitive impairment, which clinicians detected more readily than care managers. CONCLUSIONS: In this at-risk group, integrated assessment offers a means of potentially both delaying care-home admissions and taking account of a wider range of factors in placement decisions. Such an approach is viewed as an important policy goal both in the UK and internationally.

The value of specialist clinical assessment of older people prior to entry to care homes.

OBJECTIVE: to ascertain the value of employing a specialist clinician's contribution to the assessment of older people prior to care home entry. DESIGN: randomised controlled trial. METHOD: 256 older people at risk of care home entry were randomly allocated to either a control group, who received the usual care management assessment, or to an experimental group who, in addition, received a clinical assessment by a geriatrician or old age psychiatrist. The value of the additional assessment was evaluated by an analysis of clinical recommendations, questionnaires eliciting the views of stakeholders and research interviews with older people and their carers at initial assessment and 6 months. Data on service use and costs over 6 months and on destination at 6 and 12 months were also collected. RESULTS: the clinical assessment uncovered covert morbidity previously unknown to care managers particularly in respect of cognitive impairment and was adjudged acceptable to the different stakeholders involved. Those receiving the clinical assessment experienced less deterioration in their physical functioning, had less contact with nursing homes and emergency services and their carers experienced reduced levels of distress. Overall, the costs of care for those receiving the assessment were no greater with NHS costs actually lower. CONCLUSIONS: the potential benefits in involving specialist clinicians in the assessment process include identifying previously undiagnosed conditions and enhancing care managers' decision making. Such an assessment could be provided at a modest marginal cost. The approach is fully compatible with proposals for the role of the community geriatrician and commensurate with current good clinical practice in old age psychiatry.

Public funding for residential and nursing home care: projection of the potential impact of proposals to change the residential allowance in services for older people.

BACKGROUND: This paper investigates the potential effects of a policy change in the funding of UK residential care. The White Paper Modernising Social Services (Cm 4169, 1998) outlined plans to change the distribution of the Residential Allowance (RA), payable in support of residents in independent residential or nursing home care, from a component of income support paid direct to establishments to a grant to local authorities. This change was intended to remove the perverse incentive in accessing independent residential care more favourably than local authority care. A further objective was to encourage local authorities to use the grant to support home-based alternatives to residential care. The policy rests on a model in which price signals dictate the choice of care for an older person. By, in effect, raising the price of independent residential and nursing home care, the policy provides an incentive for authorities to seek alternatives to institutional care. METHODS: Managers from 16 UK social services departments attended a focus group discussion, completed questionnaires and provided information to assist in calculating the potential diversionary effect of the policy. RESULTS: Managerial estimates indicated a small diversionary effect of the policy; A potential effect of 0.26 and 0.19 per 1000 older people diverted from residential and nursing care respectively. CONCLUSIONS: The study indicated that wider organisational factors other than price are likely to play a greater role in deciding whether an older person is admitted to care. Changes in public funding alone do not reflect the complexities involved in decision-making concerning the residential placement of older people.

Quality assurance practices in care management: a perspective from the United Kingdom.

Alongside the development of care management in many countries has emerged a need to monitor the quality of both care management practices, as well as the quality of services provided as a consequence of care planning by care managers. Designing and implementing quality assurance practices in care management present special challenges. This article examines current practices from the perspective of the United Kingdom (UK). First, current performance initiatives at the central government level are described, including an indication of their limitations in judging quality in care management; second, existing work on assuring quality in care management is reviewed along with its relevance for the UK; and third, a local study, identifying measures within an analytical framework and using them to monitor care management against set dimensions, is described. Parallel developments are now occurring at both national and local levels in the UK, with local work both influencing and being informed by national developments. Future issues to address include the problems in collecting relevant data and in extending methods of analysis to monitor care management more comprehensively.