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Background: Guidelines in healthcare should be evidence-based, satisfy patient needs and improve patient outcome.

Methods: We performed a cost–utility analysis in Graves' disease (GD) and estimated incremental costs after the introduction of a national guideline adding the Graves' Recurrent Events After Therapy (GREAT) score with genetic determinants (GREAT+) to predict recurrence, a thyroid nurse, preoperative calcium/vitamin D treatment and thyroid-stimulating immunoglobulins.

Findings: Antithyroid drugs (ATDs) were less costly, achieved 0.88 quality-adjusted life years (QALYs) over 8 years and dominated over radioactive iodine (RAI) treatment. The relevant incremental cost-effectiveness ratio was ATD versus thyroid surgery (Tx). Tx was more costly than ATD but was also more effective. The incremental cost-effectiveness ratio was equal to 40,488 Euro per QALY gained. In recurrent GD, the QALY weight for surgery after ATD was 0.76 compared with 0.79 when surgery was the initial treatment. If individuals requiring surgery could be identified at start of first treatment, QALYs would be higher (6.32) and the cost lower (13,945 Euro). The net cost increase after the new guideline was 17.6%, which was partially an effect from more time being spent with the thyroid nurse. If the GREAT+ score was also applied, the total increased net cost was 14.8% if 24% of the tested patients changed treatment to Tx.

Interpretation: Tx was more cost-effective than RAI when ablative treatment is advocated. Prediction score for recurrence directing patients to earlier Tx is cost-effective and enables the introduction of a specialist thyroid nurse. Health economic evaluations should accompany future guidelines.

Introduction: If a person is in diabetes remission, even if only for a short time, this reduces the risk of later diabetes complications and lowers healthcare costs. A recent study shows that long-term remission of type 2 diabetes can be achieved through calorie restriction using total diet replacement. However, this intervention involves support through face-to-face meetings every 2 to 4 weeks over a 2-year period, which is not feasible in routine care with limited resources. Therefore, we have developed an eHealth programme to help patients achieve diabetes remission through calorie restriction in a cost-effective manner. Our primary hypothesis is that an eHealth programme will be non-inferior to face-to-face meetings in helping patients with type 2 diabetes achieve remission through caloric restriction. Our second hypothesis is that eHealth support will be more cost-effective than face-to-face support.

Methods and analysis: The eHealth DIabetes remission Trial is a multicentre, two-arm, non-inferiority, open-label, randomised controlled parallel group trial with blinded endpoint assessment conducted at two centres in Sweden. The study duration is 2 years. People with type 2 diabetes (≤6 years duration) use total diet replacement (approximately 900 kcal/day) with the aim of losing 15 kg and achieving diabetes remission. Participants are randomly assigned to either the eHealth support group or the face-to-face support group. The treatment programme to achieve and maintain weight loss is the same in both groups, but the method of support differs between the groups. The primary outcome is haemoglobin A1c (HbA1c) after 1 year. The secondary outcome is HbA1c at 6 months and 2 years. Other important secondary outcomes are diabetes remission rate, body weight and cost-effectiveness. The latter is assessed using the incremental cost per quality-adjusted life-years gained.

Ethics and dissemination: The study was approved by the Swedish Ethical Review Authority (Dnr 2022-02242-01, 2023-03707-02). The results will be published in peer-reviewed scientific journals and discussed at national and international conferences and with patient organisations.

INTRODUCTION: The prevalence of type 2 diabetes (T2D) within sub-Saharan Africa (SSA) is increasing. Despite the pathophysiology of T2D differing by ethnicity and sex, risk stratification and guidelines for the prevention of T2D are generic, relying on evidence from studies including predominantly Europeans. Accordingly, this study aims to develop ethnic-specific and sex-specific risk prediction models for the early detection of dysglycaemia (impaired glucose tolerance and T2D) to inform clinically feasible, culturally acceptable and cost-effective risk management and prevention strategies using dietary modification in SSA and European populations.

METHODS AND ANALYSIS: This multinational collaboration will include the prospective cohort data from two African cohorts, the Middle-Aged Soweto Cohort from South Africa and the Research on Obesity and Diabetes among African Migrants Prospective cohort from Ghana and migrants living in Europe, and a Swedish cohort, the Pre-Swedish CArdioPulmonary bioImage Study. Targeted proteomics, as well as targeted and untargeted metabolomics, will be performed at baseline to discover known and novel ethnic-specific and sex-specific biomarkers that predict incident dysglycaemia in the different longitudinal cohorts. Dietary patterns that explain maximum variation in the biomarker profiles and that associate with dysglycaemia will be identified in the SSA and European cohorts and used to build the prototypes for dietary interventions to prevent T2D. A comparative cost-effectiveness analysis of the dietary interventions will be estimated in the different populations. Finally, the perceptions of at-risk participants and healthcare providers regarding ethnic-specific and sex-specific dietary recommendations for the prevention of T2D will be assessed using focus group discussions and in-depth interviews in South Africa, Ghana, Germany (Ghanaian migrants) and Sweden.

ETHICS AND DISSEMINATION: Ethical clearance has been obtained from all participating sites. The study results will be disseminated at scientific conferences and in journal publications, and through community engagement events and diabetes organisations in the respective countries.

Several literature review studies have been conducted on cost-effectiveness threshold values. However, only a few are systematic literature reviews, and most did not investigate the different methods, especially in-depth reviews of directly eliciting WTP per QALY. Our study aimed to 1) describe the different direct approach methods to elicit WTP/QALY; 2) investigate factors that contribute the most to the level of WTP/QALY value; and 3) investigate the relation between the value of WTP/QALY and GDP per capita and give some recommendations on feasible methods for eliciting WTP/QALY in low- and middle-income countries (LMICs). A systematic review concerning select studies estimating WTP/QALY from a direct approach was carried out in seven databases, with a cut off date of 03/2022. The conversion of monetary values into 2021 international dollars (i$) was performed via CPI and PPP indexes. The influential factors were evaluated with Bayesian model averaging. Criteria for recommendation for feasible methods in LMICs are made based on empirical evidence from the systematic review and given the resource limitation in LMICs. A total of 12,196 records were identified; 64 articles were included for full-text review. The WTP/QALY method and values varied widely across countries with a median WTP/QALY value of i$16,647.6 and WTP/QALY per GDP per capita of 0.53. A total of 11 factors were most influential, in which the discrete-choice experiment method had a posterior probability of 100%. Methods for deriving WTP/QALY vary largely across studies. Eleven influential factors contribute most to the level of values of WTP/QALY, in which the discrete-choice experiment method was the greatest affected. We also found that in most countries, values for WTP/QALY were below 1 x GDP per capita. Some important principles are addressed related to what LMICs may be concerned with when conducting studies to estimate WTP/QALY.

OBJECTIVE: This study was conducted with the objective of exploring the usage of health-related quality of life (HRQOL) outcomes and willingness of health technology assessment (HTA) and public health stakeholders to use the EQ-5D-5L instrument in healthcare decision-making processes in Vietnam.

METHOD: In this qualitative study, 11 interviews were held with key stakeholders involved in healthcare decision-making for HTA between June 2021 and June 2022. The interviewees included members of the Vietnamese pharmacoeconomic council and public-health professionals from a diverse array of regions of Vietnam. The data collection involved obtaining verbal consent, warm-up discussions and interviews conducted via Zoom, with subsequent verification by interviewees. The analysis employed a theoretical thematic approach, adopting a deductive methodology to identify and analyse underlying ideas and meanings within the empirical data.

RESULTS: This study highlights the general importance and viability of HRQOL measures, and more particularly the EQ-5D-5L instrument, in healthcare decision-making in Vietnam. Challenges have been identified, including insufficient recognition, interpretation, standardisation and educational initiatives relating to HRQOL measurements. This study advocates for official training programmes on HRQOL measurements, guidelines for the application of the EQ-5D-5L and an open HRQOL database in Vietnam. Concerns regarding validity and outcome variation in HRQOL measurements underline the necessity for continuous psychometric properties assessments and regular updates to national HRQOL data in the Vietnamese context.

CONCLUSION: HRQOL outcomes are important, and Vietnamese stakeholders express a readiness to employ the EQ-5D-5L in healthcare decision-making, especially HTA. Nevertheless, HRQOL measurements, including the EQ-5D-5L, are currently inadequately used in Vietnam, and further efforts are required to improve utilisation.

A better understanding of population distributions of capability and inequalities is relevant for public health and social welfare. We surveyed 3639 representatively sampled individuals on background characteristics and on self-reported capability using the capability-adjusted life years Sweden (CALY-SWE) questionnaire, which enables aggregating attribute-level answers into a quality weight score.

We analysed the resulting capability distribution by comparing subgroups. Subgroups were formed along axes of inequality that we selected based on theoretical reasoning and relevance. We stratified into groupings of plurality and disadvantage, based on the human dignity principle and the needs and solidarity principle featured in the Swedish platform for healthcare priority-setting. While disadvantage factors come with inherent need-related disadvantages that explain inequality, such as unemployment, no such normatively acceptable reason exist for plurality factors, such as gender.

The results averaged per individual showed marked inequalities for the disadvantage groups of individuals with poor self-rated health, long-term sick leave unemployment, and payment difficulties. For plurality groups, the largest inequalities occurred in groups with lower education and groups residing in urban areas, but generally inequalities were lower than disadvantage groups.

On the population level, the largest aggregated disability inequalities occurred for self-reported ever NEET (persons who were ever involuntarily not in employment, education or training from age 16 to 29) and payment difficulties groups. Additionally, we assessed the contribution of covariates to these inequalities by counterfactually altering one covariate at a time in a multinomial logistic model of the CALY-SWE attribute-level answers. The most relevant covariates were employment situation, financial difficulties, income, and age group.

For policy-making, this indicates the importance of both plurality and disadvantage inequality axes. Prioritization should adopt nuanced contextualizations of inequality when broadening the perspective beyond healthcare to wider public health and social welfare, for example, elderly care and education.

Background: This study aimed to assess the cost-effectiveness of durvalumab as a treatment option for patients with inoperable stage III non-small cell lung cancer (NSCLC) from healthcare and partial societal perspectives in Vietnam.

Method: A lifetime partitioned survival model was used to evaluate the costs and quality-adjusted life years (QALYs) associated with consolidation durvalumab in comparison with the standard of care alone. Local costs and utilities were incorporated into the model. In the base-case analysis, no discount was applied to the acquisition cost of durvalumab. Scenario-based, one-way and probabilistic-sensitivity analyses were conducted.

Results: The base-case analysis revealed that the intervention resulted in an increase of 1.38 life years or 1.08 QALYs for patients, but the intervention was not deemed cost-effective from either perspective in the base-case analysis. However, with a 70% reduction in the durvalumab acquisition cost, the intervention was observed to be cost-effective when evaluated from a healthcare perspective and when examining the undiscounted results from a partial societal standpoint.

Conclusion: This study provides evidence regarding the cost-effectiveness of durvalumab for the treatment of inoperable stage III NSCLC in Vietnam for various scenarios. The intervention was not cost-effective at full acquisition cost, but it is important to acknowledge that cost-effectiveness arguments alone cannot solely guide decision-makers in Vietnam; other criteria, such as budget impact and ethical concerns, are crucial factors to consider in decision-making processes.

Purpose: Our aim was to elicit a value set for Capability-Adjusted Life Years Sweden (CALY-SWE); a capability-grounded quality of life instrument intended for use in economic evaluations of social interventions with broad consequences beyond health.

Methods: Building on methods commonly used in the quality-adjusted life years EQ-5D context, we collected time-trade off (TTO) and discrete choice experiment (DCE) data through an online survey from a general population sample of 1697 Swedish participants. We assessed data quality using a score based on the severity of inconsistencies. For generating the value set, we compared different model features, including hybrid modeling of DCE and TTO versus TTO data only, censoring of TTO answers, varying intercept, and accommodating for heteroskedasticity. We also assessed the models’ DCE logit fidelity to measure agreement with potentially less-biased DCE data. To anchor the best capability state to 1 on the 0 to 1 scale, we included a multiplicative scaling factor.

Results: We excluded 20% of the TTO answers of participants with the largest inconsistencies to improve data quality. A hybrid model with an anchor scale and censoring was chosen to generate the value set; models with heteroskedasticity considerations or individually varying intercepts did not offer substantial improvement. The lowest capability weight was 0.114. Health, social relations, and finance and housing attributes contributed the largest capability gains, followed by occupation, security, and political and civil rights.

Conclusion: We elicited a value set for CALY-SWE for use in economic evaluations of interventions with broad social consequences.

BACKGROUND: Rheumatic heart disease (RHD) is a devastating yet preventable condition that disproportionately affects low-middle-income countries and indigenous populations in some high-income countries. Various preventive interventions have been implemented across the globe, but evidence for the effectiveness of these measures in reducing the incidence or prevalence of acute rheumatic fever and RHD is scattered. This systematic review aims to assess the effectiveness of preventive interventions and identify the strategies used to reduce the burden of RHD.

METHODS AND RESULTS: A comprehensive search was conducted to identify relevant studies on RHD prevention interventions including interventions for primordial, primary, and secondary prevention. Effectiveness measures for the interventions were gathered when available. The findings indicate that school-based primary prevention services targeting the early detection and treatment of Group A Streptococcus pharyngitis infection with penicillin have the potential to reduce the incidence of Group A Streptococcus pharyngitis and acute rheumatic fever. Community-based programs using various prevention strategies also reduced the burden of RHD. However, there is limited evidence from low-middle-income countries and a lack of rigorous evaluations reporting the true impact of the interventions. Narrative synthesis was performed, and the methodological quality appraisal was done using the Joanna Briggs Institute critical appraisal tools.

CONCLUSIONS: This systematic review underscores the importance of various preventive interventions in reducing the incidence and burden of Group A Streptococcus pharyngitis, acute rheumatic fever, and RHD. Rigorous evaluations and comprehensive analyses of interventions are necessary for guiding effective strategies and informing public health policies to prevent and reduce the burden of these diseases in diverse populations.

REGISTRATION: URL: https://www.crd.york.ac.uk/prospero/; Unique identifier: CRD42020170503.