Trial-based Economic Evaluations Research Articles

Poor sample size reporting quality and insufficient sample size in economic evaluations conducted alongside pragmatic trials: a cross-sectional survey

ObjectivesEconomic evaluations based on well-designed and -conducted pragmatic randomized controlled trials (pRCTs) can provide valuable evidence on the cost-effectiveness of interventions, enhancing the relevance and applicability of findings to healthcare decision-making. However, economic evaluation outcomes are seldom taken into consideration during the process of sample size calculation in pragmatic trials. The reporting quality of sample size and information on its calculation in economic evaluations that are well-suited to pRCTs remain unknown. This study aims to assess the reporting quality of sample size and estimate the power values of economic evaluations in pRCTs. Study Design and SettingWe conducted a cross-sectional survey using data of pRCTs available from PubMed and OVID from 1 January 2010 to 24 April 2022. Two groups of independent reviewers identified articles; three groups of reviewers each extracted the data. Descriptive statistics presented the general characteristics of included studies. Statistical power analyses were performed on clinical and economic outcomes with sufficient data. ResultsThe electronic search identified 715 studies and 152 met the inclusion criteria. Of these, 26 were available for power analysis. Only 9 out of 152 trials (5.9%) considered economic outcomes when estimating sample size, and only one adjusted the sample size accordingly. Power values for trial-based economic evaluations and clinical trials ranged from 2.56% to 100% and 3.21%–100%, respectively. Regardless of the perspectives, in 14 out of the 26 studies (53.8%), the power values of economic evaluations for quality-adjusted life years (QALYs) were lower than those of clinical trials for primary endpoints (PEs). In 11 out of the 24 (45.8%) and in 8 out of the 13 (61.5%) studies, power values of economic evaluations for QALYs were lower than those of clinical trials for PEs from the healthcare and societal perspectives, respectively. Power values of economic evaluations for non-QALYs from the healthcare and societal perspectives were potentially higher than those of clinical trials in 3 out of the 4 studies (75%). The power values for economic outcomes in Q1 were not higher than those for other journal impact factor quartile categories. ConclusionTheoretically, pragmatic trials with concurrent economic evaluations can provide real-world evidence for healthcare decision makers. However, in pRCT-based economic evaluations, limited consideration, and inadequate reporting of sample-size calculations for economic outcomes could negatively affect the results’ reliability and generalisability. We thus recommend that future pragmatic trials with economic evaluations should report how sample sizes are determined or adjusted based on the economic outcomes in their protocols to enhance their transparency and evidence quality.

Rationale, Design and Baseline Characteristics of a Randomized Controlled Trial of a Cardiovascular Quality Improvement Strategy in India: The C-QIP Trial

BackgroundQuality of chronic care for cardiovascular disease (CVD) remains suboptimal worldwide. The Collaborative Quality ImProvement (C-QIP) trial aims to develop and test the feasibility and clinical effect of a multicomponent strategy among patients with prevalent CVD in India. MethodsThe C-QIP is a clinic-based, open randomized trial of a multicomponent intervention versus usual care that was locally developed and adapted for use in Indian settings through rigorous formative research guided by Consolidated Framework for Implementation Research (CFIR). The C-QIP intervention consisted of 5 components: 1) electronic health records and decision support system for clinicians, 2) trained non-physician health workers (NPHW), 3) text-message based lifestyle reminders, 4) patient education materials, 5) quarterly audit and feedback reports. Patients with CVD (ischemic heart disease, ischemic stroke, or heart failure) attending outpatient CVD clinics were recruited from September 2022 to September 2023 and were randomized to the intervention or usual care arm for at least 12 months follow-up. The co-primary outcomes are implementation feasibility, fidelity (i.e., dose delivered and dose received), acceptability, adoption and appropriateness, measured at multiple levels: patient, provider and clinic site-level, The secondary outcomes include prescription of guideline directed medical therapy (GDMT) (provider-level), and adherence to prescribed therapy, change in mean blood pressure (BP) and LDL-cholesterol between the intervention and control groups (patient-level). In addition, a trial-based process and economic evaluations will be performed using standard guidelines. ResultsWe recruited 410 socio-demographically diverse patients with CVD from four hospitals in India. Mean (SD) age was 57.5 (11.7) years, and 73.0% were males. Self-reported history of hypertension (48.5%) and diabetes (41.5%) was common. At baseline, mean (SD) BP was 127.9 (18.2) /76.2 (11.6) mm Hg, mean (SD) LDLc: 80.3 (37.3) mg/dl and mean (SD) HbA1c: 6.8% (1.6%). At baseline, the GDMT varied from 62.4% for patients with ischemic heart disease, 48.6% for ischemic stroke and 36.1% for heart failure. ConclusionThis study will establish the feasibility of delivering contextually relevant, and evidence-based C-QIP strategy and assess whether it is acceptable to the target populations. The study results will inform a larger scale confirmatory trial of a comprehensive CVD care model in low-resource settings. Trial registrationClinical Trials Registry India: CTRI/2022/04/041847; Clinicaltrials.gov number: NCT05196659

Identification and prioritisation of items for a draft participant-reported questionnaire to measure use of social care, informal care, aids and adaptations.

Resource-use measurement is integral for assessing cost-effectiveness within trial-based economic evaluations. Methods for gathering resource-use data from participants are not well developed, with questionnaires typically produced for each trial and rarely validated. The healthcare module of a generic, modular resource-use measure, designed for collecting self-report resource-utilisation data, has recently been developed in the UK. The objective of this research is to identify and prioritise items for new, bolt-on modules, covering informal care, social care and personal expenses incurred due to health and care needs. Identification and prioritisation, conducted between April and December 2021, involved a rapid review of questionnaires included in the Database of Instruments for Resource Use Measurement and economic evaluations published from 2011 to 2021 to identify candidate items, an online survey of UK-based social care professionals to identify omitted social care items and focus groups with UK-based health economists and UK-based people who access social care services either for themselves or as carers to prioritise items. The review identified 203 items. Over half of the 24 survey respondents reported no missing items. Five academic health economists and four people who access social care services participated in focus groups. Feedback shaped the social and informal care modules and indicated that no specific personal expenses were essential to collect in all trials. Aids/adaptations were highlighted as costly personal expenses when relevant; therefore, the personal expenses module was narrowed to aids/adaptations only. Draft informal care, social care and aids/adaptations modules were developed, ready for further testing.

Conducting Trial-Based Economic Evaluations Using R: A Tutorial.

Trial-based economic evaluations are increasingly being conducted to support healthcare decision-making. When analysing trial-based economic evaluation data, different methodological challenges may be encountered, including (i) missing data, (ii) correlated costs and effects, (iii) baseline imbalances and (iv) skewness of costs and/or effects. Despite the broad range of methods available to account for these methodological challenges in effectiveness studies, they may not always be directly applicable in trial-based economic evaluations where costs and effects are analysed jointly, and more than one methodological challenge typically needs to be addressed simultaneously. The use of inappropriate methods can bias results and conclusions regarding the cost-effectiveness of healthcare interventions. Eventually, such low-quality evidence can hamper healthcare decision-making, which may in turn result in a waste of already scarce healthcare resources. Therefore, this tutorial aims to provide step-by-step guidance on how to combine appropriate statistical methods for handling the abovementioned methodological challenges using a ready-to-use R script. The theoretical background of the described methods is provided, and their application is illustrated using a simulated trial-based economic evaluation.

Advocating uniformity in spine surgery: a practical disease-specific guideline for trial-based economic evaluations

ObjectivesDespite the availability of general and national guidelines for the conduct and reporting of economic evaluations, there is heterogeneity in economic evolutions concerning spine surgery. This is partly the result...

Advocating uniformity in spine surgery: A practical disease-specific guideline for trial-based economic evaluations

Advocating uniformity in spine surgery: A practical disease-specific guideline for trial-based economic evaluations

PP92 Should Missing Data Be Multiply Imputed Prior To Longitudinal Linear Mixed-Model Analyses In Trial-Based Economic Evaluations?

IntroductionFor the analysis of clinical effects, multiple imputation (MI) of missing data was shown to be unnecessary when using longitudinal linear mixed-models (LLM). It remains unclear whether this also applies to cost estimates from trial-based economic evaluations, that are generally right-skewed. Therefore, this study aimed to assess whether MI is required prior to LLM when analyzing longitudinal cost-effectiveness data.MethodsTwo-thousand complete datasets were simulated containing five time points. Incomplete datasets were generated with 10 percent, 25 percent, and 50 percent missing data in costs and effects, assuming a Missing At Random (MAR) mechanism. Statistical performance of six different methodological strategies was compared in terms of empirical bias (EB), root-mean-squared error (RMSE), and coverage rate (CR). Six strategies were compared: (i) LLM (LLM), (ii) MI prior to LLM (MI-LLM), (iii) mean imputation prior to LLM (M-LLM), (iv) complete-case analysis prior to seemingly unrelated regression (CCA-SUR), (v) MI prior to SUR (MI-SUR), and (vi) mean imputation prior to SUR (M-SUR). To evaluate the impact on the probability of cost-effectiveness at different willingness-to-pay [WTPs] thresholds, cost-effectiveness analyses were performed by applying the six strategies to two empirical datasets with 9% and 50% of missing data, respectively.ResultsFor costs and effects, LLM, MI-LLM, and MI-SUR performed better than M-LLM, CCA-SUR, and M-SUR, as indicated by smaller EBs and RMSEs, as well as CRs closer to the nominal levels of 0.95. However, even though LLM, MI-LLM, and MI-SUR performed equally well for effects, MI-LLM and MI-SUR were found to perform better than LLM for costs at 10 percent and 25 percent missing data. At 50 percent missing data, all strategies resulted in relatively high EBs and RMSEs for costs. In both empirical datasets, LLM, MI-LLM, and MI-SUR all resulted in similar probabilities of cost-effectiveness at different WTPs.ConclusionsWhen opting for using LLM for analyzing trial-based economic evaluation data, researchers are advised to multiply impute missing values first. Otherwise, MI-SUR may also be used.

MSR127 Generalizability of Trial-Based Economic Evaluations. Methods to Account for Covariates and Impact on Cost-Effectiveness and Value of Information

Economic evaluations based on clinical trials may lack generalizability if heterogeneity is not accounted for. We explore different methods to account for covariates to obtain cost-effectiveness results in different populations and assess the expected value of perfect information (EVPI).

Systematic review of economic evaluations for internet- and mobile-based interventions for mental health problems

In view of the staggering disease and economic burden of mental disorders, internet and mobile-based interventions (IMIs) targeting mental disorders have often been touted to be cost-effective; however, available evidence is inconclusive and outdated. This review aimed to provide an overview of the cost-effectiveness of IMIs for mental disorders and symptoms. A systematic search was conducted for trial-based economic evaluations published before 10th May 2021. Electronic databases (including MEDLINE, PsycINFO, CENTRAL, PSYNDEX, and NHS Economic Evaluations Database) were searched for randomized controlled trials examining IMIs targeting mental disorders and symptoms and conducting a full health economic evaluation. Methodological quality and risk of bias were assessed. Cost-effectiveness was assumed at or below £30,000 per quality-adjusted life year gained. Of the 4044 studies, 36 economic evaluations were reviewed. Guided IMIs were likely to be cost-effective in depression and anxiety. The quality of most evaluations was good, albeit with some risks of bias. Heterogeneity across studies was high because of factors such as different costing methods, design, comparison groups, and outcomes used. IMIs for anxiety and depression have potential to be cost-effective. However, more research is needed into unguided (preventive) IMIs with active control conditions (e.g., treatment as usual) and longer time horizon across a wider range of disorders.Trial registration: PROSPERO Registration No. CRD42018093808.

The handling of missing data in trial-based economic evaluations: should data be multiply imputed prior to longitudinal linear mixed-model analyses?

IntroductionFor the analysis of clinical effects, multiple imputation (MI) of missing data were shown to be unnecessary when using longitudinal linear mixed-models (LLM). It remains unclear whether this also applies to trial-based economic evaluations. Therefore, this study aimed to assess whether MI is required prior to LLM when analyzing longitudinal cost and effect data.MethodsTwo-thousand complete datasets were simulated containing five time points. Incomplete datasets were generated with 10, 25, and 50% missing data in follow-up costs and effects, assuming a Missing At Random (MAR) mechanism. Six different strategies were compared using empirical bias (EB), root-mean-squared error (RMSE), and coverage rate (CR). These strategies were: LLM alone (LLM) and MI with LLM (MI-LLM), and, as reference strategies, mean imputation with LLM (M-LLM), seemingly unrelated regression alone (SUR-CCA), MI with SUR (MI-SUR), and mean imputation with SUR (M-SUR).ResultsFor costs and effects, LLM, MI-LLM, and MI-SUR performed better than M-LLM, SUR-CCA, and M-SUR, with smaller EBs and RMSEs as well as CRs closers to nominal levels. However, even though LLM, MI-LLM and MI-SUR performed equally well for effects, MI-LLM and MI-SUR were found to perform better than LLM for costs at 10 and 25% missing data. At 50% missing data, all strategies resulted in relatively high EBs and RMSEs for costs.ConclusionLLM should be combined with MI when analyzing trial-based economic evaluation data. MI-SUR is more efficient and can also be used, but then an average intervention effect over time cannot be estimated.

How cost-effective is nutrition care delivered in primary healthcare settings? A systematic review of trial-based economic evaluations.

Nutrition care is an effective lifestyle intervention for the treatment and prevention of many noncommunicable diseases. Primary care is a high-value setting in which to provide nutrition care. The objective of this review was to evaluate the cost-effectiveness of nutrition care interventions provided in primary care settings. Medline, Embase, the Cumulative Index to Nursing and Allied Health Literature (CINAHL), the Cochrane Central Register of Controlled Trials, EconLit, and the National Health Service Economic Evaluation Database (NHS EED) were searched from inception to May 2021. Data extraction was guided by the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) reporting guidelines. Randomized trials of nutrition interventions in primary care settings were included in the analysis if incremental cost-effectiveness ratios were reported. The main outcome variable incremental cost-effectiveness ratios (ICERs) and reported interpretations were used to categorize interventions by the cost-effectiveness plane quadrant. Of 6837 articles identified, 10 were included (representing 9 studies). Eight of the 9 included studies found nutrition care in primary care settings to be more costly and more effective than usual care. High study heterogeneity limited further conclusions. Nutrition care in primary care settings is effective, though it requires investment; it should, therefore, be considered in primary care planning. Further studies are needed to evaluate the long-term cost-effectiveness of providing nutrition care in primary care settings. PROSPERO registration no. CRD42020201146.

Immediate sequential bilateral surgery versus delayed sequential bilateral surgery for cataracts.

Age-related cataract affects both eyes in most cases. Most people undergo cataract surgery in both eyes on separate days, referred to as delayed sequential bilateral cataract surgery (DSBCS). An alternative procedure involves operating on both eyes on the same day, but as two separate procedures, known as immediate sequential bilateral cataract surgery (ISBCS). Potential advantages of ISBCS include fewer hospital visits for the patient, faster visual recovery, and lower healthcare costs. Nevertheless, concerns exist about possible bilateral, postoperative, sight-threatening adverse effects with ISBCS.Therefore, there is a clear need for evaluating evidence regarding the safety, effectiveness, and cost-effectiveness of ISBCS versus DSBCS. To assess the safety of ISBCS compared to DSBCS in people with bilateral age-related cataracts and to summarise current evidence for the incremental resource use, utilities, costs, and cost-effectiveness associated with the use of ISBCS compared to DSBCS in people with bilateral age-related cataracts (primary objectives). The secondary objective was to assess visual and patient-reported outcomes of ISBCS compared to DSBCS in people with bilateral age-related cataracts. We searched CENTRAL (which contains the Cochrane Eyes and Vision Trials Register; 2021, Issue 5); Ovid MEDLINE; Ovid Embase; the ISRCTN registry; ClinicalTrials.gov; the WHO ICTRP; and DARE and NHS EED on the CRD Database on 11 May 2021. There were no language restrictions. We limited the searches to a date range of 2007 onwards. We included randomised controlled trials (RCTs) to assess complications, refractive outcomes, best-corrected distance visual acuity (BCDVA) and patient-reported outcome measures (PROMs) with ISBCS compared to DSBCS. We included non-randomised (NRSs), prospective, and retrospective cohort studies comparing ISBCS and DSBCS for safety assessment, because of the rare incidence of important adverse events. To assess cost-effectiveness of ISBCS compared to DSBCS, we included both full and partial economic evaluations, and both trial-based and model-based economic evaluations. We used standard Cochrane methodological procedures and assessed risk of bias for NRSs using the ROBINS-I tool. For cost-evaluations, we used the CHEC-list, the CHEERS-checklist, and the NICE-checklist to investigate risk of bias. We assessed the certainty of evidence with the GRADE tool. We reported results for economic evaluations narratively. We included 14 studies in the review; two RCTs, seven NRSs, and six economic evaluations (one study was both an NRS and economic evaluation). The studies reported on 276,260 participants (7384 for ISBCS and 268,876 for DSBCS) and were conducted in Canada, the Czech Republic, Finland, Iran, (South) Korea, Spain (Canary Islands), Sweden, the UK, and the USA.Overall, we considered the included RCTs to be at'high to some concerns' risk of bias for complications, 'some concerns' risk of bias for refractive outcomes and visual acuity, and 'high' risk of bias for PROMs. The overall risk of bias for NRSs was graded 'serious' regarding complications and 'serious to critical' regarding refractive outcomes. With regard to endophthalmitis, we found thatrelative effects were estimated imprecisely and with low certainty, so that relative estimates were not reliable. Nonetheless, we found a very low risk of endophthalmitis in both ISBCS (1/14,076 participants) and DSBCS (55/556,246 participants) groups. Based on descriptive evidence and partially weak statistical evidence we found no evidence of an increased risk of endophthalmitis with ISBCS.Regarding refractive outcomes, we found moderate-certainty (RCTs) and low-certainty (NRSs) evidence there was no difference in the percentage of eyes that did not achieve refraction within 1.0 dioptre of target one to three months after surgery (RCTs: risk ratio (RR) 0.84, 95% confidence interval (CI) 0.57 to 1.26; NRSs: RR 1.02, 95% CI 0.60 to 1.75). Similarly, postoperative complications did not differ between groups (RCTs: RR 1.33, 95% CI 0.52 to 3.40; NRSs: 1.04, 95% CI 0.47 to 2.29), although the certainty of this evidence was very low for both RCTs and NRSs. Furthermore, we found low-certainty (RCTs) to very low-certainty (NRSs) evidence that total costs per participant were lower for ISBCS compared to DSBCS, although results of individual studies could not be pooled. Only one study reported on cost-effectiveness. This study found that ISBCS is cost-effective compared to DSBCS, butdid not measure quality-adjusted life years using preferred methods and calculated costs erroneously. Finally, regarding secondary outcomes, we found limited evidence on BCDVA (data of two RCTs could not be pooled, although both studies individually found no difference between groups (very low-certainty evidence)). Regarding PROMs, we found moderate-certainty evidence (RCTs only) that there was no difference between groups one to three months after surgery (standardised mean difference -0.08, 95% CI -0.19 to 0.03). Current evidence supports there are probably no clinically important differences in outcomes between ISBCS and DSBCS, but with lower costs for ISBCS. However, the amount of evidence is limited, and the certainty of the evidence was graded moderate to very low. In addition, there is a need for well-designed cost-effectiveness studies.

Trial-Based Economic Evaluations of Supported Employment for Adults with Severe Mental Illness: A Systematic Review.

To identify, appraise, and summarize outcomes reported in trial-based economic evaluations of Individual Placement and Support programs for adults with severe mental illness. Six databases were searched, including Medline, PsycINFO, CINAHL, Cochrane, Scopus, and EconLit. Inclusion criteria were trial-based, full economic evaluations comparing Individual Placement and Support programs to traditional vocational rehabilitation programs for adults 18years and older with severe mental illness. Study quality was appraised using the Consolidated Health Economic Evaluation Reporting Standards statement. Of the 476 articles identified in the database search, seven were included in the review. Studies conducted across Europe (n = 4) and Japan (n = 1) suggested that Individual Placement and Support may be a cost-effective alternative to traditional vocational rehabilitation programs. Two studies conducted in the United States demonstrated that Individual Placement and Support led to better vocational outcomes, but at neutral or higher costs than traditional vocational rehabilitation, depending on the benefit measure used. Trial-based economic evaluations of supported employment for adults with severe mental illness are limited and heterogeneous. The interpretation of economic outcomes warrants consideration of factors that may impact cost-effectiveness, such as geographical location. Future studies should evaluate whether the benefits of IPS outweigh additional costs for patients and other stakeholders.

The economic impact of two diagnostic strategies in the management of restorations in primary teeth: a health economic analysis plan for a trial-based economic evaluation

BackgroundDifferent approaches have been used by dentists to base their decision. Among them, there are the aesthetical issues that may lead to more interventionist approaches. Indeed, using a more interventionist strategy (the World Dental Federation - FDI), more replacements tend to be indicated than using a minimally invasive one (based on the Caries Around Restorations and Sealants—CARS). Since the resources related to the long-term health effects of these strategies have not been explored, the economic impact of using the less-invasive strategy is still uncertain. Thus, this health economic analysis plan aims to describe methodologic approaches for conducting a trial-based economic evaluation that aims to assess whether a minimally invasive strategy is more efficient in allocating resources than the conventional strategy for managing restorations in primary teeth and extrapolating these findings to a longer time horizon.MethodsA trial-based economic evaluation will be conducted, including three cost-effectiveness analyses (CEA) and one cost-utility analysis (CUA). These analyses will be based on the main trial (CARDEC-03/NCT03520309), in which children aged 3 to 10 were included and randomized to one of the diagnostic strategies (based on FDI or CARS). An examiner will assess children’s restorations using the randomized strategy, and treatment will be recommended according to the same criteria. The time horizon for this study is 2 years, and we will adopt the societal perspective. The average costs per child for 24 months will be calculated. Three different cost-effectiveness analyses (CEA) will be performed. For CEAs, the effects will be the number of operative interventions (primary CEA analysis), the time to these new interventions, the percentage of patients who did not need new interventions in the follow-up, and changes in children’s oral health-related quality of life (secondary analyses). For CUA, the effect will be tooth-related quality-adjusted life years (QALYs). Intention-to-treat analyses will be conducted. Finally, we will assess the difference when using the minimally invasive strategy for each health effect (∆effect) compared to the conventional strategy (based on FDI) as the reference strategy. The same will be calculated for related costs (∆cost). The discount rate of 5% will be applied for costs and effects. We will perform deterministic and probabilistic sensitivity analyses to handle uncertainties. The net benefit will be calculated, and acceptability curves plotted using different willingness-to-pay thresholds. Using Markov models, a longer-term economic evaluation will be carried out with trial results extrapolated over a primary tooth lifetime horizon.DiscussionThe main trial is ongoing, and data collection is still not finished. Therefore, economic evaluation has not commenced. We hypothesize that conventional strategy will be associated with more need for replacements of restorations in primary molars. These replacements may lead to more reinterventions, leading to higher costs after 2 years. The health effects will be a crucial aspect to take into account when deciding whether the minimally invasive strategy will be more efficient in allocating resources than the conventional strategy when considering the management of restorations in primary teeth. Finally, patients/parents preferences and consequent utility values may also influence this final conclusion about the economic aspects of implementing the minimally invasive approach for managing restorations in clinical practice. Therefore, these trial-based economic evaluations may bring actual evidence of the economic impact of such interventions.Trial registrationNCT03520309. Registered May 9, 2018. Economic evaluations (the focus of this plan) are not initiated at the moment.

Deprescribing for community-dwelling elderly: A systematic review of economic evaluations

Abstract Background Multimorbidity among the elderly represents a serious challenge for health systems. Older adults are often exposed to polypharmacy and inappropriate medication, which are associated with adverse events and increased healthcare use. This study aims to identify and synthetize the economic evidence of deprescribing interventions among community-dwelling elderly. Methods Literature was systematically reviewed on the cost and effectiveness of deprescribing interventions in adults aged ≥65 years living in the community. MEDLINE, EconLit, Scopus, Web of Science, CEA-TUFTS, CRD York and Google Scholar databases were searched from inception to February 2021. Grey literature was also explored. Titles and abstracts, and subsequently full-text articles were screened according to inclusion and exclusion criteria. Studies' quality was appraised using the extended Consensus on Health Economics Criteria list. Results A total of 6,154 articles were identified, of which 89 were retrieved for full-text review, yielding 14 studies. Most were conducted in Europe (n = 9), followed by North (n = 3) and South America (n = 1), and China (n = 1). Settings included community pharmacies, primary care/general practices and patientś homes. Most deprescribing interventions were delivered within a pharmacist-doctor collaboration. Twelve studies were trial-based economic evaluations with a time horizon varying from 2 to 12 months. Cost-effectiveness ranged from dominant to an incremental cost-effectiveness ratio of $112,932/QALY. Nine studies scored >80% (good) and two scored ≤50% (low) on critical quality appraisal. Conclusions There are few economic evaluations of deprescribing interventions focused on community-dwelling elderly. Although results varied across settings, time horizon and intervention complexity, most interventions were cost-effective according to the WHO threshold. Deprescribing interventions are promising from an economic viewpoint, but more studies are needed. Key messages There is a paucity of economic evaluation studies conducted on deprescribing interventions of community-dwelling elderly. Research suggests that most interventions are cost-effective or even dominant compared to usual care.

A systematic review of the quality and scope of decision modelling studies in child oral health research

BackgroundDecision analytic models are often used in economic evaluations to estimate long-term costs and effects of treatment which span beyond the time-frame of a clinical trial, therefore providing a better understanding of the long-term implications of decisions that conventional trial-based economic evaluations fail to provide. This is particularly relevant for considering oral health interventions in children as treatments may affect adult oral health. However, in the field of child oral health there has not been an evaluation of the quality and scope of decision analytical models which extend into adulthood. The aim of this review is to examine the scope and quality of decision modelling studies, with horizons extending into adulthood, within the field of child oral health.MethodsThe following databases were searched: NHS Economic Evaluation Database (CRD York), MEDLINE, EMBASE, CINAHL, Web of Science, Scopus, the Cochrane Library and Econlit. Full economic evaluations, in the field of child oral health, published after 1997 which included a decision model with a horizon that extended beyond the age of 18 years old were included. Included studies were appraised against the Drummond checklist and the Consolidated Health Economic Evaluation Reporting Standards by calibrated reviewers.ResultsFour hundred studies were identified, of which nine met the inclusion criteria. Of the nine, eight were cost-effectiveness models. The majority focussed on the prevention or management of dental caries. The mean percentage of applicable Drummond checklist criteria met by the studies in this review was 82% (median = 85%, range = 54–100%). Discounting of costs and performing an incremental analysis were noted as key methodological weaknesses. The mean percentage of applicable CHEERS criteria met by each study was 82% (median = 87%, range = 32–96%). Justifying the type of model, analytical methods used, and sources of funding were most commonly unreported.ConclusionsThere is a paucity of decision analytical models in the field of child oral health. Most of those that are available are of high methodological and reporting quality.

The statistical approach in trial-based economic evaluations matters: get your statistics together!

BackgroundBaseline imbalances, skewed costs, the correlation between costs and effects, and missing data are statistical challenges that are often not adequately accounted for in the analysis of cost-effectiveness data. This study aims to illustrate the impact of accounting for these statistical challenges in trial-based economic evaluations.MethodsData from two trial-based economic evaluations, the REALISE and HypoAware studies, were used. In total, 14 full cost-effectiveness analyses were performed per study, in which the four statistical challenges in trial-based economic evaluations were taken into account step-by-step. Statistical approaches were compared in terms of the resulting cost and effect differences, ICERs, and probabilities of cost-effectiveness.ResultsIn the REALISE study and HypoAware study, the ICER ranged from 636,744€/QALY and 90,989€/QALY when ignoring all statistical challenges to − 7502€/QALY and 46,592€/QALY when accounting for all statistical challenges, respectively. The probabilities of the intervention being cost-effective at 0€/ QALY gained were 0.67 and 0.59 when ignoring all statistical challenges, and 0.54 and 0.27 when all of the statistical challenges were taken into account for the REALISE study and HypoAware study, respectively.ConclusionsNot accounting for baseline imbalances, skewed costs, correlated costs and effects, and missing data in trial-based economic evaluations may notably impact results. Therefore, when conducting trial-based economic evaluations, it is important to align the statistical approach with the identified statistical challenges in cost-effectiveness data. To facilitate researchers in handling statistical challenges in trial-based economic evaluations, software code is provided.

Multivariate Generalized Linear Mixed-Effects Models for the Analysis of Clinical Trial-Based Cost-Effectiveness Data.

Economic evaluations conducted alongside randomized controlled trials are a popular vehicle for generating high-quality evidence on the incremental cost-effectiveness of competing health care interventions. Typically, in these studies, resource use (and by extension, economic costs) and clinical (or preference-based health) outcomes data are collected prospectively for trial participants to estimate the joint distribution of incremental costs and incremental benefits associated with the intervention. In this article, we extend the generalized linear mixed-model framework to enable simultaneous modeling of multiple outcomes of mixed data types, such as those typically encountered in trial-based economic evaluations, taking into account correlation of outcomes due to repeated measurements on the same individual and other clustering effects. We provide new wrapper functions to estimate the models in Stata and R by maximum and restricted maximum quasi-likelihood and compare the performance of the new routines with alternative implementations across a range of statistical programming packages. Empirical applications using observed and simulated data from clinical trials suggest that the new methods produce broadly similar results as compared with Stata’s merlin and gsem commands and a Bayesian implementation in WinBUGS. We highlight that, although these empirical applications primarily focus on trial-based economic evaluations, the new methods presented can be generalized to other health economic investigations characterized by multivariate hierarchical data structures.

Joint Longitudinal Models for Dealing With Missing at Random Data in Trial-Based Economic Evaluations

ObjectivesIn trial-based economic evaluation, some individuals are typically associated with missing data at some time point, so that their corresponding aggregated outcomes (eg, quality-adjusted life-years) cannot be evaluated. Restricting the analysis to the complete cases is inefficient and can result in biased estimates, while imputation methods are often implemented under a missing at random (MAR) assumption. We propose the use of joint longitudinal models to extend standard approaches by taking into account the longitudinal structure to improve the estimation of the targeted quantities under MAR. MethodsWe compare the results from methods that handle missingness at an aggregated (case deletion, baseline imputation, and joint aggregated models) and disaggregated (joint longitudinal models) level under MAR. The methods are compared using a simulation study and applied to data from 2 real case studies. ResultsSimulations show that, according to which data affect the missingness process, aggregated methods may lead to biased results, while joint longitudinal models lead to valid inferences under MAR. The analysis of the 2 case studies support these results as both parameter estimates and cost-effectiveness results vary based on the amount of data incorporated into the model. ConclusionsOur analyses suggest that methods implemented at the aggregated level are potentially biased under MAR as they ignore the information from the partially observed follow-up data. This limitation can be overcome by extending the analysis to a longitudinal framework using joint models, which can incorporate all the available evidence.

Neuropsychological rehabilitation interventions for people with an acquired brain injury. A protocol for a systematic review of economic evaluation.

Background: New emerging evidence has demonstrated the need for effective interventions to help people living with an acquired brain injury (ABI). Evidence on cost-effectiveness, which can help inform use of limited resources, is scarce in this area and therefore the purpose of this systematic review is to critically appraise and consolidate the current evidence on economic evaluations of ABI rehabilitation interventions. Methods: Systematic review methodology will be applied to identify, select and extract data from published economic evaluation studies (trial-based, non-trial based, simulation-based, decision model and trial-based model economic evaluations) of ABI treatment interventions in adults. A systematic literature search will be conducted on the following electronic databases: EMBASE, Econlit, CINAHL, Medline, the National Health Service Economic Evaluation Database and PsyclNFO. This review will only include cost-effectiveness analysis studies (e.g., cost per life year gained), cost-benefit and cost minimisation analyses in which the designs were randomised controlled trials (RCTs), non-RCT studies, cost-utility analyses (e.g., cost per quality-adjusted life year (QALY) gained or cost per disability-adjusted life year averted), cohort studies, and modelling studies. Only studies that were published in the english language, associated with adults who have an ABI will be included. There will be no restrictions on perspective, sample size, country, follow-up duration or setting. The search strategy terms will include the following: acquired brain injury, brain*; cost*; or cost-benefit analysis*. Following data extraction, a narrative summary and tables will be used to summarize the characteristics and results of included studies. Discussion: The findings from this review will be beneficial to health policy decision makers when examining the evidence of economic evaluations in this field. In addition, it is anticipated that this review will identify gaps in the current economic literature to inform future-related research. Systematic review registration: PROSPERO CRD42020187469 (25 th June 2020).