Economic Evaluation Of Health Care Interventions Research Articles

The Short Form 6 Dimensions (SF-6D): Development and Evolution.

This paper considers the development and evolution of the short-form 6 dimensions (SF-6D), a generic preference-weighted measure consisting of a health classification with accompanying value set that was developed from one of the widest used health related quality of life measures, the SF-36 health survey. This enabled health state utility values to be directly generated from SF-36 and SF-12 data for a range of purposes, including to produce quality adjusted life years for use in economic evaluation of healthcare interventions across a range of different conditions and treatments. This paper considers the rationale for the development of the measure, the development process, performance and how the SF-6D has evolved since its conception. This includes the development of an updated version, SF-6D version 2 (SF-6Dv2), which was generated to deal with some criticisms of the first version, and now includes a standalone version for inclusion in studies without relying on use of SF-36 or SF-12. Valuation methods have also evolved, from standard gamble in-person interviews to online discrete choice experiment surveys. International work related to the SF-6Dv1 and SF-6Dv2 is considered. We also consider recommendations for use, highlighting key psychometric evidence and reimbursement agency recommendations.

Interpreting economic evaluations of healthcare interventions: A simple guide.

This guide aims to help healthcare professionals of all backgrounds make sense of economic evaluations to determine whether interventions represent value for money.

Recommendations for Emerging Good Practice and Future Research in Relation to Family and Caregiver Health Spillovers in Health Economic Evaluations: A Report of the SHEER Task Force.

Omission of family and caregiver health spillovers from the economic evaluation of healthcare interventions remains common practice. When reported, a high degree of methodological inconsistency in incorporating spillovers has been observed. To promote emerging good practice, this paper from the Spillovers in Health Economic Evaluation and Research (SHEER) task force aims to provide guidance on the incorporation of family and caregiver health spillovers in cost-effectiveness and cost-utility analysis. SHEER also seeks to inform the basis for a spillover research agenda and future practice. A modified nominal group technique was used to reach consensus on a set of recommendations, representative of the views of participating subject-matter experts. Through the structured discussions of the group, as well as on the basis of evidence identified during a review process, recommendations were proposed and voted upon, with voting being held over two rounds. This report describes 11 consensus recommendations for emerging good practice. SHEER advocates for the incorporation of health spillovers into analyses conducted from a healthcare/health payer perspective, and more generally inclusive perspectives such as a societal perspective. Where possible, spillovers related to displaced/foregone activities should be considered, as should the distributional consequences of inclusion. Time horizons ought to be sufficient to capture all relevant impacts. Currently, the collection of primary spillover data is preferred and clear justification should be provided when using secondary data. Transparency and consistency when reporting on the incorporation of health spillovers are crucial. In addition, given that the evidence base relating to health spillovers remains limited and requires much development, 12 avenues for future research are proposed. Consideration of health spillovers in economic evaluations has been called for by researchers and policymakers alike. Accordingly, it is hoped that the consensus recommendations of SHEER will motivate more widespread incorporation of health spillovers into analyses. The developing nature of spillover research necessitates that this guidance be viewed as an initial roadmap, rather than a strict checklist. Moreover, there is a need for balance between consistency in approach, where valuable in a decisionmaking context, and variation in application, to reflect differing decisionmaker perspectives and to support innovation.

Transparency, openness, and reproducible research practices are frequently underused in health economic evaluations

ObjectivesTo investigate the extent to which articles of economic evaluations of healthcare interventions indexed in MEDLINE incorporate research practices that promote transparency, openness, and reproducibility. Study Design and SettingWe evaluated a random sample of health economic evaluations indexed in MEDLINE during 2019. We included articles written in English reporting an incremental cost-effectiveness ratio in terms of costs per life years gained, quality-adjusted life years, and/or disability-adjusted life years. Reproducible research practices, openness, and transparency in each article were extracted in duplicate. We explored whether reproducible research practices were associated with self-report use of a guideline. ResultsWe included 200 studies published in 147 journals. Almost half were published as open access articles (n = 93; 47%). Most studies (n = 150; 75%) were model-based economic evaluations. In 109 (55%) studies, authors self-reported use a guideline (e.g., for study conduct or reporting). Few studies (n = 31; 16%) reported working from a protocol. In 112 (56%) studies, authors reported the data needed to recreate the incremental cost-effectiveness ratio for the base case analysis. This percentage was higher in studies using a guideline than studies not using a guideline (72/109 [66%] with guideline vs. 40/91 [44%] without guideline; risk ratio 1.50, 95% confidence interval 1.15–1.97). Only 10 (5%) studies mentioned access to raw data and analytic code for reanalyses. ConclusionTransparency, openness, and reproducible research practices are frequently underused in health economic evaluations. This study provides baseline data to compare future progress in the field.

Child-Parent Agreement in the Assessment of Health-Related Quality of Life Using the CHU9D and the PedsQLTM.

This study examined the inter-rater agreement between child-self and parental proxy health-related quality of life (HRQoL) ratings (overall and domain level) using two different generic child-specific measures, the Child Health Utility 9D (CHU9D) and the Pediatric Quality of Life Inventory (PedsQLTM), in a community-based sample of Australian children. A secondary objective was to investigate the impact of age on child-parent agreement across the dimensions of the two measures. A total of 85 child-parent dyads (children aged 6-12 years) recruited from the community completed the self and proxy versions of the CHU9D and the PedsQLTM, respectively. The inter-rater agreement was estimated using Concordance Correlation Coefficients (CCC) and Gwet's Agreement Coefficient (AC1) for the overall sample and across age-groups. Agreement was low for overall HRQoL for both the CHU9D (CCC = 0.28) and the PedsQLTM (CCC = 0.39). Across the CHU9D dimensions, agreement was the highest for 'sad' (AC1 = 0.83) and lowest for 'tired' (AC1 = 0.31). The PedsQLTM demonstrated stronger agreement (AC1 = 0.41-0.6) for the physical health dimension but weaker for the psychosocial dimensions (AC1 < 0.4). Except for the 'tired' dimension, agreement was consistent across age-groups with the CHU9D, whilst the PedsQLTM showed poor agreement for most of the psychosocial health items among the older age-groups only (8-10 and 11-12 years). This study highlights that the agreement between child and parentproxy reported HRQoL may be influenced by both the measure used and the age of the child. These findings may have implications for the economic evaluation of healthcare interventions and services in child populations when both child and proxy perspectives are considered in the assessment of child HRQoL.

Economic evaluation of a community health worker model for tuberculosis care in Ho Chi Minh City, Viet Nam: a mixed-methods Social Return on Investment Analysis

BackgroundThere is extensive evidence for the cost-effectiveness of programmatic and additional tuberculosis (TB) interventions, but no studies have employed the social return on investment (SROI) methodology. We conducted a SROI analysis to measure the benefits of a community health worker (CHW) model for active TB case finding and patient-centered care.MethodsThis mixed-method study took place alongside a TB intervention implemented in Ho Chi Minh City, Viet Nam, between October-2017 – September-2019. The valuation encompassed beneficiary, health system and societal perspectives over a 5-year time-horizon. We conducted a rapid literature review, two focus group discussions and 14 in-depth interviews to identify and validate pertinent stakeholders and material value drivers. We compiled quantitative data from the TB program’s and the intervention’s surveillance systems, ecological databases, scientific publications, project accounts and 11 beneficiary surveys. We mapped, quantified and monetized value drivers to derive a crude financial benefit, which was adjusted for four counterfactuals. We calculated a SROI based on the net present value (NPV) of benefits and investments using a discounted cash flow model with a discount rate of 3.5%. A scenario analysis assessed SROI at varying discount rates of 0-10%.ResultsThe mathematical model yielded NPVs of US$235,511 in investments and US$8,497,183 in benefits. This suggested a return of US$36.08 for each dollar invested, ranging from US$31.66-US39.00 for varying discount rate scenarios.ConclusionsThe evaluated CHW-based TB intervention generated substantial individual and societal benefits. The SROI methodology may be an alternative for the economic evaluation of healthcare interventions.

A Value Set for the EQ-5D-Y-3L in the Netherlands.

Background and ObjectiveThere is increasing interest in preference-accompanied measures of health for paediatric populations. The child-friendly EQ-5D version, EQ-5D-Y-3L, is one such instrument, but the lack of a Dutch value set prevents its use in economic evaluations of healthcare interventions in the Netherlands. This study aims at covering this gap by collecting preferences using a standardised protocol for deriving EQ-5D-Y-3L value sets.MethodsComposite time trade-off data were collected using videoconferencing interviews, with each respondent completing ten composite time trade-off tasks. Discrete choice experiment data were collected using an online survey, with respondents each completing 15 paired comparisons. Respondents completed these tasks considering what they prefer for a hypothetical 10-year-old child. Discrete choice experiment data were analysed using a ten-parameter mixed-logit model and anchored to the quality-adjusted life-year scale using the mean observed composite time trade-off values.ResultsThe study collected preferences for 197 respondents using composite time trade-off and for 959 respondents using a discrete choice experiment. The discrete choice experiment sample was representative for the Dutch population in terms of age and sex. The level 3 weight for pain/discomfort was the largest, followed by feeling worried, sad or unhappy, usual activities, mobility and self-care. Health state values ranged between −0.218 and 1.ConclusionsThis study generated a Dutch value set for the EQ-5D-Y-3L, which can be used for the computation of quality-adjusted life-years for economic evaluations of healthcare interventions in paediatric populations.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40273-022-01192-0.

Comment on "The Unbearable Lightness of Difference Between Statistical and Clinical Significance".

We are glad to see that the short-term results of our RVlob trial comparing robotic-assisted lobectomy and video-assisted lobectomy recently published on Annals of Surgery1 have aroused wide discussion. We deeply appreciate the comment from Bertolaccini et al, which reminds us to prudently interpret the statistical significance. Regarding one of our primary endpoints, the extent of lymph nodes (LNs) dissection, Bertolaccini et al pointed out that the reported difference of one more LN dissected by robotic-assisted lobectomy was not clinically significant. Indeed, we are fully aware of the gap between statistical and clinical significance and regard the survival benefit as the most solid evidence. As mentioned in the Discussion section of our original article, the correlation between an improved number of LNs and long-term survival is insignificant according to some published retrospective studies.2,3 However, beneficial effects of more LNs dissected on proper staging and prognosis may be shielded by the limited sample size. Furthermore, consensus on the optimal number of resected LNs in lung cancer surgery is still lacking. The threshold values vary from 8 to 12 in different studies.4–6 Ultimately, since no surrogate endpoints can perform as well as the overall survival, we expect the further follow-up of our RVlob trial to demonstrate a convincing argument for the controversy. When it comes to the comparison of direct and indirect costs, heterogeneity between countries, and medical systems should never be neglected. According to a review written by Jacobs and Fassbender,7 there is a wide variation among the measurements and analyses of indirect costs. In our study, we have defined the indirect cost as the overhead costs and amortization of surgical equipment,1 which is inconsistent with the definition provided by Bertolaccini et al. We will appreciate if they could specify the reference for the “widely used guidelines for the economic evaluation of healthcare interventions” so that we could reflect on the suitability of our definition. Despite the different reimbursement conditions of various medical systems, the current high cost of robotic surgery remains a common issue worldwide. No matter how much of the robotic surgery fee is reimbursed by the public health service, breakthrough in technical barriers, and innovative inventions will undoubtedly improve the feasibility of the robot-assisted surgery. As we have mentioned in a previous letter8 discussing a propensity score-matched study of robotic segmentectomy,9 the continued debate on robotic surgery will urge us to provide more convincing evidence. Fortunately, we have already stepped out to provide the perioperative results of a prospective randomized-controlled trial rather than a retrospective comparative study. Meanwhile, prudent reflection on methodology and statistical analysis should always be promoted when evaluating new treatments, just as the letter from Bertolaccini et al.

Comment on The Unbearable Lightness of Difference Between Statistical and Clinical Significance.

Comment on The Unbearable Lightness of Difference Between Statistical and Clinical Significance.

The ICEpop Capability Measure for Adults Instrument for Capabilities: Development of a Tariff for the Dutch General Population

ObjectivesThe ICEpop Capability Measure for Adults (ICECAP-A) assesses 5 capabilities (stability, attachment, autonomy, achievement, and enjoyment) that are important to one’s quality of life and might be an important addition to generic health questionnaires currently used in economic evaluations. This study aimed to develop a Dutch tariff of the Dutch translation of the ICECAP-A. MethodsThe methods used are similar to those used in the development of the UK tariff. A profile case best–worst scaling task was presented to 1002 participants from the general Dutch population. A scale-adjusted latent class analysis was performed to test for preferences of ICECAP-A capabilities and scale heterogeneity. ResultsA 3-preference class 2-scale class model with worst choice as scale predictor was considered optimal and was used to calculate the resulting tariff. Results indicated that the capabilities stability, attachment, and enjoyment were considered more important aspects of quality of life than autonomy and achievement. Additionally, improving capabilities from low to moderate levels had a larger effect on quality of life than improving capabilities that were already at a higher level. ConclusionsThe ICECAP-A tariffs found in this study could be used in economic evaluations of healthcare interventions in The Netherlands.

EM4 Recommendations for Handling Uncertainty in Economic Evaluation: A Targeted Review of Pharmacoeconomic Guidelines

The appropriate handling of uncertainty is an essential element of economic evaluation of healthcare interventions. Accepted methods include deterministic and probabilistic analyses to characterise parametric uncertainty, and scenario analyses to test uncertainty propagated by methodological and structural assumptions. This targeted review examined which methods for handling uncertainty are recommended in pharmacoeconomic guidelines around the world.

Discounting in economic evaluation of healthcare interventions: what about the risk term?

Results from economic evaluations of long-term outcomes are strongly dependent on the chosen discount rate. A recent review of national guidelines for evaluation of healthcare interventions finds that “the level of currently used discount rates seems relatively high in many countries”. However, this conclusion comes from a comparison to rates derived or observed for investments in safe assets, while rate of return requirements are typically considerably higher when investment involves risk. This paper reviews recent literature on how to account for project-specific risk in determination of the social rate of discount and discusses implications for economic evaluation of healthcare interventions. It concludes that the available empirical evidence strongly suggests that the demand for and consumer value of health and healthcare is co-variant with income, which therefore implies that there is a non-diversifiable risk component of health-related investment.

Democracy, Culture and Cancer Patients' Willingness to Pay for Healthcare Services: A Meta-analysis.

Cancer is one of the leading causes of death globally and expenditure associated with cancer is quite high. Efficient resource allocation needs information on economic evaluations of healthcare interventions. The purposes of the study are to provide a comprehensive overview of cancer patients’ willingness to pay (WTP) for healthcare services and to investigate the influence of democracies, culture dimensions, and other factors on WTP. A meta-analysis was conducted using PubMed, Scopus, and Google Scholar to identify all studies of stated preference approach to estimate cancer patients’ WTP healthcare services. A set of criteria was constructed for selecting relevant studies. A total of 79 studies were related to selection criteria and held sufficient information for the purposes of meta-analysis. A total of 393 estimates of WTP from 79 healthcare valuation studies were pooled to identify the links between WTP and influential factors. The findings suggest that values of benefits in healthcare services are higher in more democratic nations, but they are lower in cultural traits that are stronger indulgence and uncertainty avoidance. Further, the types of cancer matter. Compared to breast cancer, WTP is higher for skin cancer, yet lower for liver cancer and lymphoma. A higher national income and public health expenditure increase WTP for healthcare services, while a higher death rate by cancer leads to lower values of healthcare benefits.

Reproducible research practices, openness and transparency in health economic evaluations: study protocol for a cross-sectional comparative analysis

IntroductionThere has been a growing awareness of the need for rigorously and transparent reported health research, to ensure the reproducibility of studies by future researchers. Health economic evaluations, the comparative...

Performance of the UKPDS Outcomes Model2 for Predicting Death and Cardiovascular Events in Patients with Type 2 Diabetes Mellitus from a German Population-Based Cohort.

Accurate prediction of relevant outcomes is important for targeting therapies and to support health economic evaluations of healthcare interventions in patients with diabetes. The United Kingdom Prospective Diabetes Study (UKPDS) risk equations are some of the most frequently used risk equations. This study aims to analyze the calibration and discrimination of the updated UKPDS risk equations as implemented in the UKPDS Outcomes Model2 (UKPDS-OM2) for predicting cardiovascular (CV) events and death in patients with type2 diabetes mellitus (T2DM) from population-based German samples. Analyses are based on data of 456 individuals diagnosed with T2DM who participated in two population-based studies in southern Germany (KORA (Cooperative Health Research in the Region of Augsburg)-A: 1997/1998, n = 178; KORA-S4: 1999-2001, n = 278). We compared the participants' 10-year observed incidence of mortality, CV mortality, myocardial infarction (MI), and stroke with the predicted event rate of the UKPDS-OM2. The model's calibration was evaluated by Greenwood-Nam-D'Agostino tests and discrimination was evaluated by C-statistics. Of the 456 participants with T2DM (mean age 65years, mean diabetes duration 8years, 56% male), over the 10-year follow-up time 129 died (61 due to CV events), 64 experienced an MI, and 46 a stroke. The UKPDS-OM2 significantly over-predicted mortality and CV mortality by 25% and 28%, respectively (Greenwood-Nam-D'Agostino tests: p < 0.01), but there was no significant difference between predicted and observed MI and stroke risk. The model poorly discriminated for death (C-statistic [95% confidence interval] = 0.64 [0.60-0.69]), CV death (0.64 [0.58-0.71]), and MI (0.58 [0.52-0.66]), and failed to discriminate for stroke (0.57 [0.47-0.66]). The study results demonstrate acceptable calibration and poor discrimination of the UKPDS-OM2 for predicting death and CV events in this population-based German sample. Those limitations should be considered when using the UKPDS-OM2 for economic evaluations of healthcare strategies or using the risk equations for clinical decision-making.

Novel Approaches to Value Assessment Within the Cost-Effectiveness Framework

Novel Approaches to Value Assessment Within the Cost-Effectiveness Framework

Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy

The concept of a reference case, first proposed by the US Panel on Cost-Effectiveness in Health and Medicine, has been used to specify the required methodological features of economic evaluations of healthcare interventions. In the case of gene therapy, there is a difference of opinion on whether a specific methodological reference case is required. The aim of this article was to provide a more detailed analysis of the characteristics of gene therapy and the extent to which these characteristics warrant modifications to the methods suggested in general reference cases for economic evaluation. We argue that a completely new reference case is not required, but propose a tailored checklist that can be used by analysts and decision makers to determine which aspects of economic evaluation should be considered further, given the unique nature of gene therapy.

Valuation of Quality Weights for EuroQol 5-Dimensional Health States With the Time Trade-Off Method in the Capital of Iran

BackgroundThe EuroQol 5-dimension (EQ-5D) is a standard instrument that is widely used for measuring health-related quality of life and quality-adjusted life years in economic evaluation of healthcare interventions. ObjectiveTo estimate a preference valuation set for EQ-5D 3-level (3L) health states from the perspective of the general population in the capital of Iran. MethodsEight hundred seventy adults aged ≥18 years were interviewed in Tehran (Iran's capital) from July to November 2013. The participants were selected by a stratified random sampling method and were interviewed face-to-face at their usual residence. Forty-two health states were selected and valued from the 243 states derived from the EQ-5D-3L instrument. Each respondent valued 11 health states using the time trade-off method. Generalized least squares regression with random effect was used to predict values for health states. ResultsThe analysis was performed for 846 respondents. The final model yielded the best fit for the time trade-off value at the individual level with an overall R2 of 0.45 and a mean absolute error of 0.214. The mean values for the 42 health states ranged from 0.934 for state 11121 to -0.142 for state 33333. ConclusionsThis study provided for the first time a value set for calculating quality-adjusted life years from the EQ-5D instrument in Iran. The Iranian EQ-5D-3L value set slightly differs from the value sets of the UK and the United States.

Estimating health opportunity costs in low-income and middle-income countries: a novel approach and evidence from cross-country data

The economic evaluation of healthcare interventions requires an assessment of whether the improvement in health outcomes they offer exceeds the improvement in health that would have been possible if the...

Mapping the cancer-specific QLQ-C30 onto the generic EQ-5D-5L and SF-6D in colorectal cancer patients

ABSTRACTIntroduction: Economic evaluation of healthcare interventions usually needs accurate data on utility and health-related quality-of-life scores. The aim of this study is to map QLQ-C30 scale score onto EQ-5D-5L and SF-6D utility values in colorectal cancer (CRC) patients.Methods: EQ-5D-5L, SF-6D, and QLQ-C30 were completed by 252 patients with CRC who were referred to three cancer centers in Tehran between May and September 2017. Moreover, OLS, Tobit, and CLAD models were used to predict EQ-5D-5L and SF-6D values. The goodness of fit of models was evaluated using Pred R2 and Adj R2. In addition, their predictive performance was assessed by MAE, RMSE, ICC, MID, and Spearman’s correlation coefficients between observed and predicted EQ-5D-5L and SF-6D values. Models were validated using a 10-fold cross-validation method.Results: Considering the goodness of fit and predictive ability of models, the OLS Model 2 performed best for EQ-5D-5L (Adj R2 = 58.09%, Pred R2 = 58.93%, MAE = 0.0932, RMSE = 0.129) and the OLS Model 3 performed best for SF-6D (Adj R2 = 54.90%, Pred R2 = 55.62%, MAE = 0.0485, RMSE = 0.0634).Conclusion: Our results demonstrated that algorithms developed based on OLS Models 1 and 2 are the best for predicted EQ-5D-5L and SF-6D values, respectively.