Economic evaluation supports public funding decisions about the use of health technologies within the Portuguese National Health System (NHS). The methods guide for economic evaluation in Portugal serves both companies preparing economic evaluation submissions and the independent commission appraising the evidence submitted.

This article presents the revised methods guide for economic evaluation in Portugal. The revisions reflect advances in economic evaluation, updates to regulatory policies, and responses to the evolving economic context. The paper highlights the most significant changes to the guidance, comparing the new Portuguese guidelines to those from the United Kingdom and Canada. The discussion is framed around key comments received during public consultation.

The updated guidelines recommend cost-effectiveness analyses based on quality-adjusted life years and advocate for long-term modelling, a 4 percent discount rate, and a focus on NHS costs. New features include guidance on the identification and management of uncertainty within a dynamic appraisal process with regular contract negotiations (which can trigger reappraisals). The guide also covers how cost-effectiveness models, typically centrally developed, should be adapted to the Portuguese context. It highlights the key role of structured expert elicitation to address uncertainties in evidence, including those related to model adaptation.

The revision was developed through stakeholder consultations and aligns with international best practices, offering more explicit and transparent methods to support health resource allocation decisions.

Mental health issues are prevalent among children and young people (CYP) with chronic conditions like epilepsy, yet few access evidence-based psychological therapies. Evidence from the Mental Health Intervention for Children with Epilepsy (MICE) trial supports the effectiveness of a personalised modular psychological intervention, but cost-effectiveness is unknown.

To assess the cost-effectiveness of the MICE intervention compared with assessment-enhanced usual care at 12-months follow-up, taking a health and social care perspective.

We conducted a within-trial economic evaluation. Outcomes were the Strengths and Difficulties Questionnaire (SDQ; primary) and quality-adjusted life years (QALYs; secondary) for CYP, caregivers, and CYP and caregivers combined. Sensitivity analyses examined missing data and intervention-costing assumptions.

Cost-effectiveness results for the SDQ indicated that MICE had a higher probability of being cost-effective compared with control at a willingness to pay ≥£368 per unit improvement. For QALYs, MICE had a lower probability of being cost-effective for CYP compared with control (35 to 42%) across the £20 000–£30 000 per QALY threshold range. However, at the upper threshold this finding was reversed in sensitivity analyses with missing data imputed (45 to 58%) and with MICE costed at 75%, assuming the intervention partly substituted standard services (46 to 55%). Furthermore, MICE had a higher probability of being cost-effective for caregiver QALYs (52 to 63%) and combined CYP and caregiver QALYs (62 to 75%).

MICE appears to be cost-effective compared with assessment-enhanced usual care when considering QALYs for CYP and caregivers combined, though uncertainty exists across willingness-to-pay thresholds.

The National Institute for Health and Care Excellence (NICE) in England introduced early value assessments (EVAs) as an evidence-based method of accelerating access to promising health technologies that could address unmet needs and contribute to the National Health Service’s Long Term Plan. However, there are currently no published works considering differences and commonalities in methods used between Assessment Reports for EVAs.

This rapid scoping review included all completed EVAs published on the NICE website up to 23 July 2024. One reviewer screened potentially relevant records for eligibility, checked by a second reviewer. Pairs of independent reviewers extracted information on the methods used in included EVAs using a prepiloted form; these were checked for accuracy. Data were described in graphical or tabular format with an accompanying narrative summary.

In total, seventeen EVA Reports of sixteen EVAs were included in this scoping review. Five Reports did not specify how many reviewers undertook screening, whereas five did not report data extraction methods. Five EVAs planned to conduct meta-analyses, nine planned narrative syntheses, and seven planned narrative summaries. Eleven conceptual decision models were presented, with available evidence used to construct cost-utility analyses (N = 5); cost-effectiveness analyses (CEAs; N = 4); a mix of CEAs and cost-consequence analyses (CCA; N = 2); one CCA; and one cost-comparison.

Future EVA Reports should enhance the transparency of the methods used. Furthermore, EVAs could provide opportunities for the adoption of innovative methodological approaches and more flexible communication between EVA authors and key stakeholders, including patients and clinicians, companies, and NICE.

Health economic evaluations are important for healthcare resource allocation. Reviews of health economic evaluations for medical devices have highlighted concerns about the quality of these studies. The complexity of medical devices, including learning curve effects, organizational impact, dynamic pricing, low evidence, and incremental innovation presents unique challenges compared with pharmaceuticals. To support developing a methodological quality assessment instrument for medical device economic evaluations, we conducted a systematic review to identify and evaluate existing economic evaluation quality assessment instruments for suitability in medical device evaluations.

A comprehensive search of databases (MEDLINE, EMBASE, EconLit, CINAHL, and Web of Science) and grey literature was conducted. Two reviewers screened titles and abstracts. Full-text, peer-reviewed primary studies introducing original instruments were included. Only methodological quality assessment instruments were considered for data extraction. Each item was assessed for its suitability in evaluating medical device economic evaluations and inclusion of medical device-specific features.

The search identified 4203 citations and 77 grey literature sources. Fifteen results underwent full-text assessment, with five relevant instruments identified. A previous systematic review identified 10 additional instruments, which we also considered. Of these 25 articles, 13 were included in the review. These instruments lack specificity for medical devices, particularly in addressing features like learning curve effects, organizational impact, and incremental innovation. Instruments should include items specific to these unique characteristics.

Existing instruments contain general items related to health economic evaluation studies, highlighting the need for an instrument specifically tailored to evaluate the methodological quality of medical device economic evaluation studies.

Life-cycle health technology assessment (HTA) requires an index economic model to establish how estimated cost-effectiveness evolves with emerging evidence. We developed an open-source index economic evaluation of entrectinib, a tumor-agnostic therapy with conditional market authorization. Our objective was to replicate the initial HTA report from publicly available information, aiming to identify key operational and methodological aspects for operationalizing life-cycle decision-making.

We used partitioned survival analysis to determine tumor-agnostic and tumor-specific cost-effectiveness, using publicly available HTA reviews for parameterization. We estimated incremental costs in 2021 Canadian and US dollars (CAD and USD) from a public-payer healthcare perspective, quality-adjusted life years (QALYs), and incremental net monetary benefit (INMB). We assessed the impact of treatment effectiveness, extrapolation assumptions, and next-generation sequencing (NGS) costs.

One-third of the parameters (n = 30) were unavailable in the Canadian reimbursement review and were sourced from international reviews. Tumor-agnostic incremental costs were CAD 68,451 (95 percent confidence interval: 35,466, 92,155) and USD 54,608 (28,294, 73,518), and QALYs were 0.13 (−0.42, 0.42), yielding INMB CAD −55,803 at 100,000/QALY (USD −44,518). Full extrapolation of treatment effectiveness also yielded negative INMB (CAD −66,664). Inclusion of NGS costs diminished the expected value. Heterogeneity was considerable across tumor indications.

We developed an open-source index economic evaluation to operationalize life-cycle HTA for a conditionally authorized tumor-agnostic therapy. Our findings outline key operational and methodological considerations necessary for the development of index economic models that support life-cycle HTA, offering insights into their potential integration into regular HTA and policy decision-making processes.

The methods of economic evaluation and HTA should be based on best practices and standards, tailored to unique country contexts that can be systematically applied to inform decisions. This paper outlines standards for the conduct of economic evaluations for HTA in Ghana.

A five-step process was followed to develop the HTA reference case as a methodological and reporting benchmark. These include (a) a review of literature and evidence synthesis, (b) a review of country policies, (c) a review and adaption of international frameworks, (d) expert/stakeholder consultations, and (e) the development of a methodological framework. A series of stakeholder consultations were done to refine, finalize, and validate the outcomes of the processes to generate a finalized reference case.

The Ghana reference case is made up of 14 components comprising: evidence synthesis, evaluation type, perspectives on cost, perspectives of outcomes, choice of comparator, data sources, outcome measures, discount rate, uncertainty, equity considerations, time horizon, heterogeneity, transparency, and budget impact. These provide methodological considerations and reporting requirements for economic evaluations for HTA. It provides a framework to ensure the best research methods are adopted to harmonize the evidence-generation process with the expectations of policy and decision-makers and ensure that policy decisions are based on uniform evidence.

Recommendations set out in this reference case when followed can provide context-specific evidence to support a rigorous and transparent system for evaluating healthcare interventions and technologies. It will support decision-making, ultimately improving the quality and efficiency of healthcare delivery in the country.

Previous economic evidence about interventions for schizophrenia is outdated, non-transparent and/or limited to a specific clinical context.

We developed a de novo discrete event simulation (DES) model for estimating the cost-effectiveness of interventions in schizophrenia in the UK.

The DES model was developed based on the structure of previous models, populated with demographic, clinical and cost data from the UK, and antipsychotics' effects from recent network meta-analyses. We simulated treatment pathways for patients with first-episode schizophrenia including events such as relapse, remission, treatment discontinuation, cardiovascular disease and death and estimated costs (2020£) taking the National Health Service perspective and quality-adjusted life years (QALYs) over ten years. Using the model, we ranked ten first-line antipsychotics based on their QALYs and cost-effectiveness.

Amisulpride was associated with the highest QALYs, followed by risperidone long-acting injection (LAI), aripiprazole-LAI (6.121, 6.084, 6.070, respectively) and others (5.947–6.058). The most cost-effective antipsychotics were amisulpride, olanzapine and risperidone-LAI, with total probability of rankings of 1, ≤2, ≤3, that is, 95%, 89%, 80%, respectively; meanwhile, the least cost-effective were cariprazine, lurasidone and quetiapine, with total probability of rankings of 10, ≥9, ≥8, that is, 96%, 92%, 81%, respectively. Results were robust across sensitivity analyses and influenced primarily by relapse relevant parameters.

Our findings suggest amisulpride (or risperidone-LAI where oral treatment is inappropriate) as the best overall first-line option based on QALYs and cost-effectiveness. Our ranking may be used to guide decision-making between antipsychotics. Our model is open source and could be applied to the other settings.

International development agendas increasingly push for access to healthcare for all through universal healthcare coverage. Health economic evaluations and health technology assessment (HTA) could provide evidence to support this but do not routinely incorporate consideration of equitable access.

We undertook an international scoping review of health economic evaluation and HTA guidelines to examine how well issues of healthcare access and equity are represented, evidence recommendations, and gaps in current guidance to support evidence generation in this area. Guidelines were sourced from guideline repositories and websites of international agencies and organizations providing best practice methods guidance. Articles providing methods guidance for the conduct of HTA, or health economic evaluation, were included, except where they were not available in English and a suitable translation could not be obtained.

The search yielded forty-seven national, four international, and nine independent guidelines, along with eighty-six articles providing specific methods guidance. The inclusion of equity and access considerations in current guidance is extremely limited. Where they do feature, detail on specific methods for providing evidence on these issues is sparse.

Economic evaluation could be a valuable tool to provide evidence for the best healthcare strategies that not only maximize health but also ensure equitable access to care for all. Such evidence would be invaluable in supporting progress towards universal healthcare coverage. Clear guidance is required to ensure evaluations provide evidence on the best strategies to support equitable access to healthcare, but such guidance rarely exists in current best practice and guidance documents.

The effectiveness and cost-effectiveness of early intervention for psychosis (EIP) services are well established in high-income countries but not in low- and middle-income countries (LMICs). Despite the scarcity of local evidence, several EIP services have been implemented in LMICs. Local evaluations are warranted before adopting speciality models of care in LMICs. We aimed to estimate the cost-effectiveness of implementing EIP services in Brazil.

A model-based economic evaluation of EIP services was conducted from the Brazilian healthcare system perspective. A Markov model was developed using a cohort study conducted in São Paulo. Cost data were retrieved from local sources. The outcome of interest was the incremental cost-effectiveness ratio (ICER) measured as the incremental costs over the incremental quality-adjusted life-years (QALYs). Sensitivity analyses were performed to test the robustness of the results.

The study included 357 participants (38% female), with a mean (SD) age of 26 (7.38) years. According to the model, implementing EIP services in Brazil would result in a mean incremental cost of 4,478 Brazilian reals (R$) and a mean incremental benefit of 0.29 QALYs. The resulting ICER of R$ 15,495 (US dollar [USD] 7,640 adjusted for purchase power parity [PPP]) per QALY can be considered cost-effective at a willingness-to-pay threshold of 1 Gross domestic product (GDP) per capita (R$ 18,254; USD 9,000 PPP adjusted). The model results were robust to sensitivity analyses.

This study supports the economic advantages of implementing EIP services in Brazil. Although cultural adaptations are required, these data suggest EIP services might be cost-effective even in less-resourced countries.

Lewy body dementia (LBD) is a prevalent yet frequently underdiagnosed form of dementia, accounting for up to 15% of all dementia cases.

This study aims to increase awareness and advocacy for LBD by gathering and critically assessing the economic evidence, including the cost of illness and cost-effectiveness of interventions for managing LBD.

A systematic literature review was undertaken with EMBASE, Medline, CINAHL, PsycINFO, NHS Economic Evaluation Database and EconLit. This search was supplemented by grey literature on Google Scholar and reviewing the reference lists of identified studies. The papers included in the review were published between 2008 and 2023, and involved participants with LBD (dementia with Lewy bodies or Parkinson's disease dementia), which either addressed the cost of illness or conducted an economic evaluation.

Thirteen papers were included, comprising ten cost-of-illness studies and three economic evaluations. The cost of LBD tends to be higher than that of other forms of dementia, such as Alzheimer's disease, and these costs escalate more steeply as the disease progresses. These cost differences may not be solely influenced by the subtype of dementia, but possibly also by patient characteristics like physical and cognitive abilities. Cost-effectiveness of potential interventions for LBD is limited.

Despite numerous drug trials and other interventions for dementia, very few have targeted LBD, let alone explored the cost-effectiveness of such therapies for LBD. This disparity highlights the urgent need for cost-effective strategies and interventions targeting LBD. We propose the establishment of universally accepted standards for LBD research.

Patients with diabetes have a higher risk of developing chronic kidney disease (CKD). Early detection of CKD through microalbuminuria screening, followed by treatment, delays the progression of CKD. We evaluated the cost-effectiveness of population-based screening of microalbuminuria among normotensive type 2 diabetes mellitus patients aged >40 years compared with no screening scenario using a decision tree combined with the Markov model.

We considered two scenarios: Scenario I – dipstick microalbuminuria followed by spot-urine albumin–creatinine ratio (ACR) and serum creatinine in sequence; Scenario II – spot urine ACR plus serum creatinine. A mathematical cohort of the target population was simulated over a lifetime horizon with an annual cycle. Data for the model were obtained from secondary resources. The incremental cost-effectiveness ratios (ICERs) were estimated for screening scenarios compared to nonscreening scenario, along with sensitivity analyses.

The discounted ICER per quality-adjusted life years gained for annual microalbuminuria screening in the normotensive diabetic population in India were ₹ 24,114 (US$ 308) and ₹ 13,790 (US$ 176) for scenarios I and II, respectively. Annual screening by scenarios I and II resulted in a reduction of 180 and 193 end-stage renal disease (ESRD) cases per 100,000 population, respectively, resulting in a cost saving of ₹ 12.3 and 13.3 Crore spent on ESRD management over 10 years. Both scenarios were also cost-effective even at the screening frequencies of 5 and 10 yearly.

Microalbuminuria screening was cost-effective at the threshold of one-time GDP per capita in India.