Economic Evaluations of Pathology Tests, 2010-2015: A Scoping Review

Background

Concerns about pathology testing such as the value provided by new tests and the potential for inappropriate utilization have led to a greater need to assess costs and benefits. Economic evaluations are a formal method of analyzing costs and benefits, yet for pathology tests, questions remain about the scope and quality of the economic evidence.

Investigating the Generalizability of Economic Evaluations Conducted in Italy: A Critical Review

ObjectivesTo assess the methodological quality of Italian health economic evaluations and their generalizability or transferability to different settings.MethodsA literature search was performed on the PubMed search engine to identify trial-based, nonexperimental prospective studies or model-based full economic evaluations carried out in Italy from 1995 to 2013. The studies were randomly assigned to four reviewers who applied a detailed checklist to assess the generalizability and quality of reporting. The review process followed a three-step blinded procedure. The reviewers who carried out the data extraction were blind as to the name of the author(s) of each study. Second, after the first review, articles were reassigned through a second blind randomization to a second reviewer. Finally, any disagreement between the first two reviewers was solved by a senior researcher.ResultsOne hundred fifty-one economic evaluations eventually met the inclusion criteria. Over time, we observed an increasing transparency in methods and a greater generalizability of results, along with a wider and more representative sample in trials and a larger adoption of transition-Markov models. However, often context-specific economic evaluations are carried out and not enough effort is made to ensure the transferability of their results to other contexts. In recent studies, cost-effectiveness analyses and the use of incremental cost-effectiveness ratio were preferred.ConclusionsDespite a quite positive temporal trend, generalizability of results still appears as an unsolved question, even if some indication of improvement within Italian studies has been observed.     

Simulation Modeling for the Economic Evaluation of Population-Based Dietary Policies: A Systematic Scoping Review

Simulation modeling can be useful to estimate the long-term health and economic impacts of population-based dietary policies. We conducted a systematic scoping review following the PRISMA-ScR (Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews) guideline to map and critically appraise economic evaluations of population-based dietary policies using simulation models. We searched Medline, Embase, and EconLit for studies published in English after 2005. Modeling studies were mapped based on model type, dietary policy, and nutritional target, and modeled risk factor–outcome pathways were analyzed. We included 56 studies comprising 136 model applications evaluating dietary policies in 21 countries. The policies most often assessed were reformulation (34/136), taxation (27/136), and labeling (20/136); the most common targets were salt/sodium (60/136), sugar-sweetened beverages (31/136), and fruit and vegetables (15/136). Model types included Markov-type (35/56), microsimulation (11/56), and comparative risk assessment (7/56) models. Overall, the key diet-related risk factors and health outcomes were modeled, but only 1 study included overall diet quality as a risk factor. Information about validation was only reported in 19 of 56 studies and few studies (14/56) analyzed the equity impacts of policies. Commonly included cost components were health sector (52/56) and public sector implementation costs (35/56), as opposed to private sector (18/56), lost productivity (11/56), and informal care costs (3/56). Most dietary policies (103/136) were evaluated as cost-saving independent of the applied costing perspective. An analysis of the main limitations reported by authors revealed that model validity, uncertainty of dietary effect estimates, and long-term intervention assumptions necessitate a careful interpretation of results. In conclusion, simulation modeling is widely applied in the economic evaluation of population-based dietary policies but rarely takes dietary complexity and the equity dimensions of policies into account. To increase relevance for policymakers and support diet-related disease prevention, economic effects beyond the health sector should be considered, and transparent conduct and reporting of model validation should be improved.     

Economic Evaluations Relating to Diabetes: A Descriptive Review and Their Compliance with Guidance

From a search of the Health Economics Evaluations Database (HEED), 301 studies relating to diabetes-related interventions were identified. Of these, 223 studies contained some original data, i.e., were applied studies. Over the 1990s, the number of studies undertaken in this area appears to be increasing year on year, and more of the studies are applicable to the USA than to all other countries combined. Most studies are peer reviewed, the main type of analysis is cost-consequence, and the interventions most often assessed are pharmaceutical and care services. Both the public and the private sectors sponsor many of these studies. However, only a fraction of these studies appear to accord with guidance for good economic evaluation. For example, when the search criteria were tightened to capture only those studies that consider both outcomes and costs, 173 studies were identified. Narrowing the search criteria to capture only those studies that were based on randomized controlled trial (RCT) or modeling data—the methods recommended by the US Panel and the National Institute of Clinical Excellence (NICE)—reduced the sample to 56 studies. With specific focus on those studies that evaluated pharmaceuticals, only the modeling approach appeared to show any compliance at all with another key recommendation of the existing guidance on methods; that of using outcome measures that are applicable to a long-term time horizon.
Though modeling does offer certain advantages over RCTs (for example, a larger range of comparators can be more readily incorporated, long-term outcome measures can be more easily and inexpensively estimated), modeling results have less internal validity. Also, modeling in itself relies on good RCT data. Therefore, the results of economic evaluations should be interpreted with care, irrespective of whether they are principally based on RCT or model-based methods.     

Genetic-Guided Pharmacotherapy for Atrial Fibrillation: A Systematic and Critical Review of Economic Evaluations

ObjectivesThis study aimed to examine the extent and quality of evidence from economic evaluations (EEs) of genetic-guided pharmacotherapy (PGx) for atrial fibrillation (AF) and to identify variables influential in changing base-case conclusions.MethodsFrom systematic searches, we included EEs of existing PGx testing to guide pharmacotherapy for AF, without restrictions on population characteristics or language. Articles excluded were genetic tests used to guide device-based therapy or focused on animals.ResultsWe found 18 EEs (46 comparisons), all model-based cost-utility analysis with or without cost-effectiveness analysis mostly from health system’s perspectives, of PGx testing to determine coumadin/direct-acting anticoagulant (DOAC) dosing (14 of 18), to stratify patients into coumadin/DOACs (3 of 18), or to increase patients’ adherence to coumadin (1 of 18) versus non-PGx. Most PGx to determine coumadin dosing found PGx more costly and more effective than standard or clinical coumadin dosing (19 of 24 comparisons) but less costly and less effective than standard DOAC dosing (14 of 14 comparisons). The remaining comparisons were too few to observe any trend. Of 61 variables influential in changing base-case conclusions, effectiveness of PGx testing was the most common (37%), accounted for in the models using time-based or medication-based approaches or relative risk. The cost of PGx testing has decreased and plateaued over time.ConclusionsEEs to date only partially inform decisions on selecting optimal PGx testing for AF, because most evidence focuses on PGx testing to determine coumadin dosing, but less on other purposes. Future EE may refer to the list of influential variables and the approaches used to account for the effect of PGx testing to inform data collection and study design.     

The Methodological Quality of Economic Evaluations of Guideline Implementation into Clinical Practice: A Systematic Review of Empiric Studies

OBJECTIVES: Despite the emphasis on efficiency of health-care services delivery, there is an imperfect evidence base to inform decisions about whether and how to develop and implement guidelines into clinical practice. In general, studies evaluating the economics of guideline implementation lack methodological rigor. We conducted a systematic review of empiric studies to assess advances in the economic evaluations of guideline implementation. METHODS: The Cochrane Effective Professional and Organisational Change Group specialized register and the MEDLINE database were searched for English publications between January 1998 and July 2004 that reported objective effect measures and implementation costs. We extracted data on study characteristics, quality of study design, and economic methodology. It was assessed whether the economic evaluations followed methodological guidance. RESULTS: We included 24 economic evaluations, involving 21 controlled trials and three interrupted time series designs. The studies involved varying settings, targeted professionals, targeted behaviors, clinical guidelines, and implementation strategies. Overall, it was difficult to determine the quality of study designs owing to poor reporting. In addition, most economic evaluations were methodologically flawed: studies did not follow guidelines for evaluation design, data collection, and data analysis. CONCLUSIONS: The increasing importance of the value for money of providing health care seems to be reflected by an increase in empiric economic evaluations of guideline implementation. Because of the heterogeneity and poor methodological quality of these studies, however, the resulting evidence is still of limited use in decision-making. There seems to be a need for more methodological guidance, especially in terms of data collection and data synthesis, to appropriately evaluate the economics of developing and implementing guidelines into clinical practice.     

Value of Nonpharmacological Interventions for People With an Acquired Brain Injury: A Systematic Review of Economic Evaluations

ObjectivesAcquired brain injury (ABI) has long-lasting effects, and patients and their families require continued care and support, often for the rest of their lives. For many individuals living with an ABI disorder, nonpharmacological rehabilitation treatment care has become increasingly important care component and relevant for informed healthcare decision making. Our study aimed to appraise economic evidence on the cost-effectiveness of nonpharmacological interventions for individuals living with an ABI.MethodsThis systematic review was registered in PROSPERO (CRD42020187469), and a protocol article was subject to peer review. Searches were conducted across several databases for articles published from inception to 2021. Study quality was assessed according the Consolidated Health Economic Evaluation Reporting Standards checklist and Population, Intervention, Control, and Outcomes criteria.ResultsOf the 3772 articles reviewed 41 publications met the inclusion criteria. There was a considerable heterogeneity in methodological approaches, target populations, study time frames, and perspectives and comparators used. Keeping these issues in mind, we find that 4 multidisciplinary interventions studies concluded that fast-track specialized services were cheaper and more cost-effective than usual care, with cost savings ranging from £253 to £6063. In 3 neuropsychological studies, findings suggested that meditated therapy was more effective and saved money than usual care. In 4 early supported discharge studies, interventions were dominant over usual care, with cost savings ranging from £142 to £1760.ConclusionsThe cost-effectiveness evidence of different nonpharmacological rehabilitation treatments is scant. More robust evidence is needed to determine the value of these and other interventions across the ABI care pathway.     

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

ObjectivesSchizophrenia 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.MethodsElectronic 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.ResultsSeventy-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).ConclusionsPatient 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.     

Diagnostics and Treatments of COVID-19: A Living Systematic Review of Economic Evaluations

ObjectivesAs healthcare systems continue to respond to the COVID-19 pandemic, cost-effectiveness evidence will be needed to identify which tests and treatments for COVID-19 offer value for money. We sought to review economic evaluations of diagnostic tests and treatments for COVID-19, critically appraising the methodological approaches used and reporting cost-effectiveness estimates, using a “living” systematic review approach.MethodsKey databases (including MEDLINE, EconLit, Embase) were last searched on July 12, 2021. Gray literature and model repositories were also searched. Only full economic evaluations published in English were included. Studies were quality assessed and data were extracted into standard tables. Results were narratively summarized. The review was completed by 2 reviewers independently, with disagreements resolved through discussion with a senior reviewer.ResultsOverall, 3540 records were identified, with 13 meeting the inclusion criteria. After quality assessment, 6 were excluded because of very severe limitations. Of the 7 studies included, 5 were cost-utility analyses and 2 were cost-effectiveness analyses. All were model-based analyses. A total of 5 evaluated treatments (dexamethasone, remdesivir, hypothetical) and 2 evaluated hypothetical testing strategies. Cost-effectiveness estimates were sensitive to the treatment effect on survival and hospitalization, testing speed and accuracy, disease severity, and price.ConclusionsPresently, there are few economic evaluations for COVID-19 tests and treatments. They suggest treatments that confer a survival benefit and fast diagnostic tests may be cost effective. Nevertheless, studies are subject to major evidence gaps and take inconsistent analytical approaches. The evidence may improve for planned updates of this “living” review.     

Economic Evaluations in the Diagnosis and Management of Traumatic Brain Injury: A Systematic Review and Analysis of Quality

BackgroundEconomic evaluations provide a unique opportunity to identify the optimal strategies for the diagnosis and management of traumatic brain injury (TBI), for which uncertainty is common and the economic burden is substantial.ObjectiveThe objective of this study was to systematically review and examine the quality of contemporary economic evaluations in the diagnosis and management of TBI.MethodsTwo reviewers independently searched MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, NHS Economic Evaluation Database, Health Technology Assessment Database, EconLit, and the Tufts CEA Registry for comparative economic evaluations published from 2000 onward (last updated on August 30, 2013). Data on methods, results, and quality were abstracted in duplicate. The results were summarized quantitatively and qualitatively.ResultsOf 3539 citations, 24 economic evaluations met our inclusion criteria. Nine were cost-utility, five were cost-effectiveness, three were cost-minimization, and seven were cost-consequences analyses. Only six studies were of high quality. Current evidence from high-quality studies suggests the economic attractiveness of the following strategies: a low medical threshold for computed tomography (CT) scanning of asymptomatic infants with possible inflicted TBI, selective CT scanning of adults with mild TBI as per the Canadian CT Head Rule, management of severe TBI according to the Brain Trauma Foundation guidelines, management of TBI in dedicated neurocritical care units, and early transfer of patients with TBI with nonsurgical lesions to neuroscience centers.ConclusionsThreshold-guided CT scanning, adherence to Brain Trauma Foundation guidelines, and care for patients with TBI, including those with nonsurgical lesions, in specialized settings appear to be economically attractive strategies.     

A Scoping Review of Item-Level Missing Data in Within-Trial Cost-Effectiveness Analysis

ObjectivesCost-effectiveness analysis (CEA) alongside randomized controlled trials often relies on self-reported multi-item questionnaires that are invariably prone to missing item-level data. The purpose of this study is to review how missing multi-item questionnaire data are handled in trial-based CEAs.MethodsWe searched the National Institute for Health Research journals to identify within-trial CEAs published between January 2016 and April 2021 using multi-item instruments to collect costs and quality of life (QOL) data. Information on missing data handling and methods, with a focus on the level and type of imputation, was extracted.ResultsA total of 87 trial-based CEAs were included in the review. Complete case analysis or available case analysis and multiple imputation (MI) were the most popular methods, selected by similar numbers of studies, to handle missing costs and QOL in base-case analysis. Nevertheless, complete case analysis or available case analysis dominated sensitivity analysis. Once imputation was chosen, missing costs were widely imputed at item-level via MI, whereas missing QOL was usually imputed at the more aggregated time point level during the follow-up via MI.ConclusionsMissing costs and QOL tend to be imputed at different levels of missingness in current CEAs alongside randomized controlled trials. Given the limited information provided by included studies, the impact of applying different imputation methods at different levels of aggregation on CEA decision making remains unclear.     

Use of Checklists in Reviews of Health Economic Evaluations, 2010 to 2018

Objective

It is useful for reviewers of economic evaluations to assess quality in a manner that is consistent and comprehensive. Checklists can allow this, but there are concerns about their reliability and how they are used in practice. We aimed to describe how checklists have been used in systematic reviews of health economic evaluations.

Measurements of Intrinsic Capacity in Older Adults: A Scoping Review and Quality Assessment

ObjectivesThis review summarizes the measurements of intrinsic capacity in 5 domains across different studies and evaluates the quality of research papers.DesignScoping review of papers written in English and Chinese published in peer-reviewed journals.Setting and ParticipantsThe intrinsic capacity of older adults was assessed using the multidomain structure (Cognition, Locomotion, Psychological, Sensory and Vitality) proposed by the World Health Organization.MethodsWe searched PubMed, MEDLINE, and Web of Science for papers in English, and CNKI, CBM for papers written in Chinese published until September 13, 2022. Both cross-sectional and cohort studies of multidomain measurements of intrinsic capacity were included. Three independent reviewers appraised the quality of studies, and Cohen’s kappa was calculated to determine interrater reliability. Data were listed by author, year, setting, country, age range and number of participants, measurement and calculation of intrinsic capacity, and data acquisition method.ResultsWe included 53 studies. Twenty-one studies were of high quality, 31 studies were of moderate quality, and 1 study was of low quality. Measurements of intrinsic capacity and derivation of the summative index score were heterogeneous. Intrinsic capacity was usually assessed in 4 or 5 domains. Sensory was the most frequently overlooked domain or subdivided into vision and hearing in some studies. Indicators of vitality were the most heterogeneous. We also found consistency in heterogeneous measurements. The most common measurements of cognition, locomotion, and psychological capacity were the Mini-Mental State Examination, Short Physical Performance Battery, and Geriatric Depression Scale respectively. Self-reported questionnaires were commonly adopted in sensory domain. The Mini-Nutritional Assessment and grip strength were the most measured indicators of vitality.Conclusions and ImplicationsThe focus on capacity and disease should be balanced to better promote healthy aging in older adults. Heterogeneity of intrinsic capacity measurements underscores the need for consensus about standardized measurements and calculation procedures.