Discounting the cost and effect for health intervention is a controversial topic over the last two decades. In particular, the cost-effectiveness of gene therapies is especially sensitive to the discount rate because of the substantial delay between the upfront cost incurred and long-lasing clinical benefits received. This study aims to investigate the influence of employing alternative discount rates on the incremental cost-effectiveness ratio (ICER) of gene therapies.

A systematic review was conducted to include health economic evaluations of gene therapies that were published until April 2023.

Sensitivity or scenario analysis indicated that discount rate represented one of the most influential factors for the ICERs of gene therapies. Discount rate for cost and benefit was positively correlated with the cost-effectiveness of gene therapies, that is, a lower discount rate significantly improves the ICERs. The alternative discount rate employed in some cases could be powerful to alter the conclusion on whether gene therapies are cost-effective and acceptable for reimbursement.

Although discount rate will have substantial influence on the ICERs of gene therapies, there lacks solid evidence to justify a different discounting rule for gene therapies. However, it is proposed that the discount rate in the reference case should be updated to reflect the real-time preference, which in turn will affect the ICERs and reimbursement of gene therapies more profoundly than conventional therapies.

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.

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.

Preventing the occurrence of depression/anxiety and suicide during adolescence can lead to substantive health gains over the course of an individual person’s life. This study set out to identify the expected population-level costs and health impacts of implementing universal and indicated school-based socio-emotional learning (SEL) programs in different country contexts.

A Markov model was developed to examine the effectiveness of delivering universal and indicated school-based SEL programs to prevent the onset of depression/anxiety and suicide deaths among adolescents. Intervention health impacts were measured in healthy life years gained (HLYGs) over a 100-year time horizon. Country-specific intervention costs were calculated and denominated in 2017 international dollars (2017 I$) under a health systems perspective. Cost-effectiveness findings were subsequently expressed in terms of I$ per HLYG. Analyses were conducted on a group of 20 countries from different regions and income levels, with final results aggregated and presented by country income group – that is, low and lower middle income countries (LLMICs) and upper middle and high-income countries (UMHICs). Uncertainty and sensitivity analyses were conducted to test model assumptions.

Implementation costs ranged from an annual per capita investment of I$0.10 in LLMICs to I$0.16 in UMHICs for the universal SEL program and I$0.06 in LLMICs to I$0.09 in UMHICs for the indicated SEL program. The universal SEL program generated 100 HLYGs per 1 million population compared to 5 for the indicated SEL program in LLMICs. The cost per HLYG was I$958 in LLMICS and I$2,006 in UMHICs for the universal SEL program and I$11,123 in LLMICs and I$18,473 in UMHICs for the indicated SEL program. Cost-effectiveness findings were highly sensitive to variations around input parameter values involving the intervention effect sizes and the disability weight used to estimate HLYGs.

The results of this analysis suggest that universal and indicated SEL programs require a low level of investment (in the range of I$0.05 to I$0.20 per head of population) but that universal SEL programs produce significantly greater health benefits at a population level and therefore better value for money (e.g., less than I$1,000 per HLYG in LLMICs). Despite producing fewer population-level health benefits, the implementation of indicated SEL programs may be justified as a means of reducing population inequalities that affect high-risk populations who would benefit from a more tailored intervention approach.

Health technology assessment (HTA) organizations vary in terms of how they conduct assessments. We assess whether and to what extent HTA bodies have adopted societal and novel elements of value in their economic evaluations.

After categorizing “societal” and “novel” elements of value, we reviewed fifty-three HTA guidelines. We collected data on whether each guideline mentioned each societal or novel element of value, and if so, whether the guideline recommended the element’s inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA.

The HTA guidelines mention on average 5.9 of the twenty-one societal and novel value elements we identified (range 0–16), including 2.3 of the ten societal elements and 3.3 of the eleven novel value elements. Only four value elements (productivity, family spillover, equity, and transportation) appear in over half of the HTA guidelines, whereas thirteen value elements are mentioned in fewer than one-sixth of the guidelines, and two elements receive no mention. Most guidelines do not recommend value element inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA.

Ideally, more HTA organizations will adopt guidelines for measuring societal and novel value elements, including analytic considerations. Importantly, simply recommending in guidelines that HTA bodies consider novel elements may not lead to their incorporation into assessments or ultimate decision making.

Integrating services for depression into primary care is key to reducing the treatment gap in low- and middle-income countries. We examined the value of providing the Healthy Activity Programme (HAP), a behavioral activation psychological intervention, within services for depression delivered by primary care workers in Chitwan, Nepal using data from the Programme for Improving Mental Health Care.

People diagnosed with depression were randomized to receive either standard treatment (ST), comprised of psychoeducation, antidepressant medication, and home-based follow up, or standard treatment plus psychological intervention (T + P). We estimated incremental costs and health effects of T + P compared to ST, with quality adjusted life years (QALYs) and depression symptom scores over 12 months as health effects. Nonparametric uncertainty analysis provided confidence intervals around each incremental effectiveness ratio (ICER); results are presented in 2020 international dollars.

Sixty participants received ST and 60 received T + P. Implementation costs (ST = $329, T + P = $617) were substantially higher than service delivery costs (ST = $18.7, T + P = $22.4) per participant. ST and T + P participants accrued 46.5 and 49.4 QALYs, respectively. The ICERs for T + P relative to ST were $4422 per QALY gained (95% confidence interval: $2484 to $9550) – slightly above the highly cost-effective threshold – and −$53.21 (95% confidence interval: −$105.8 to −$30.2) per unit change on the Patient Health Questionnaire.

Providing HAP within integrated depression services in Chitwan was cost-effective, if not highly cost-effective. Efforts to scale up integrated services in Nepal and similar contexts should consider including evidence-based psychological interventions as a part of cost-effective mental healthcare for depression.

To evaluate the cost-effectiveness of the nonsurgical periodontal treatment (NSPT) compared with supragingival therapy in type II diabetics with periodontitis.

A decision tree analysis was used to estimate the costs and health outcomes of two periodontal therapies in a hypothetical cohort of type II diabetics with periodontitis. The analysis was developed from the perspective of a third-party payer at 1 year and 5 years. Probabilities were derived from two systematic reviews. The costs and resource use were validated by a Delphi expert panel. All costs were expressed in USD, using the 25 May 2021 Colombian pesos market exchange rate (USD 1 = COP 3,350).

NSPT was a dominant alternative compared with subsidized supragingival therapy in type II diabetics with periodontitis, generating savings of USD 87 and 400, during the first year or up to 5 years, respectively, and improving dental survival from 32 to 69 percent.

NSPT can generate savings by reducing the complications derived from uncontrolled periodontitis and tooth loss.

Health technology assessment (HTA) plays a central role in the coverage and reimbursement decision-making process for public health expenditure in many countries, including Thailand. However, there have been few attempts to quantitatively understand the benefits of using HTA to inform resource allocation decisions. The objective of this research was to simulate the expected net monetary benefit (NMB) from using HTA-based decision criteria compared to a first-come, first-served (FCFS) approach using data from Thailand.

A previously published simulation model was adapted to the Thai context which aimed to simulate the impact of using different decision-making criteria to adopt or reject health technologies for public reimbursement. Specifically, the simulation model provides a quantitative comparison between an HTA-based funding rule and a counterfactual (FCFS) funding rule to make decisions on which health technologies should be funded. The primary output of the model was the NMB of using HTA-based decision criteria compared to the counterfactual approach. The HTA-based decision rule in the model involved measuring incremental cost-effectiveness ratios against a cost-effectiveness threshold. The counterfactual decision rule was a FCFS (random) selection of health technologies.

The HTA-based decision rule was associated with a greater NMB compared to the counterfactual. In the investigated analyses, the NMB ranged from THB24,238 million (USD725 million) to THB759,328 million (USD22,719 million). HTA-based decisions led to fewer costs, superior health outcomes (more quality-adjusted life-years).

The results support the hypothesis that HTA can provide health and economic benefits by improving the efficiency of resource allocation decision making.

Extrapolation is often required to inform cost-effectiveness (CE) evaluations of immune-checkpoint inhibitors (ICIs) since survival data from pivotal clinical trials are seldom complete. The objectives of this study were to evaluate the accuracy of estimates of long-term overall survival (OS) predicted in French CE assessment reports of ICIs, and to identify models presenting the best fit to the observed long-term survival data.

A systematic review of French assessment reports of ICIs in the metastatic setting since inception until May 2020 was performed. A targeted literature review was conducted to collect associated extended follow-up of randomized controlled trials (RCTs) used in the CE assessment reports. Difference between projected and observed OS was calculated. A range of standard parametric and spline-based models were applied to the extended follow-up data from the RCT to determine the best-fitting survival models.

Of the 121 CE assessment reports published, 11 reports met the inclusion criteria. OS was underestimated in 73 percent of the CE assessment reports. The mean relative difference between each source was −13 percent (median: −15 percent; IQR: −0.4 to 26 percent). Models providing the best fit were those that could reflect nonmonotonic hazards.

Based on the available data at the time of submission, longer-term survival of ICIs was not fully captured by the extrapolation models used in CE assessments. Standard and flexible parametric models which can capture nonmonotonic hazard functions provided the best fit to the extended follow-up data. However, these models may still have performed poorly if fitted to survival data available at the time of submission to the French National Authority for Health.

Depression and anxiety are among the most common mental health conditions treated in primary care. They frequently co-occur and involve recommended treatments that overlap. Evidence from randomised controlled trials (RCTs) shows specific stepped care interventions to be cost-effective in improving symptom remission. However, most RCTs have focused on either depression or anxiety, which limits their generalisability to routine primary care settings. This study aimed to evaluate the cost-effectiveness of a collaborative stepped care (CSC) intervention to treat depression and/or anxiety among adults in Australian primary care settings.

A quasi-decision tree model was developed to evaluate the cost-effectiveness of a CSC intervention relative to care-as-usual (CAU). The model adapted a CSC intervention described in a previous Dutch RCT to the Australian context. This 8-month, cluster RCT recruited patients with depression and/or anxiety (n = 158) from 30 primary care clinics in the Netherlands. The CSC intervention involved two steps: (1) guided self-help with a nurse at a primary care clinic; and (2) referral to specialised mental healthcare. The cost-effectiveness model adopted a health sector perspective and synthesised data from two main sources: RCT data on intervention pathways, remission probabilities and healthcare service utilisation; and Australia-specific data on demography, epidemiology and unit costs from external sources. Incremental costs and incremental health outcomes were estimated across a 1-year time horizon. Health outcomes were measured as disability-adjusted life years (DALYs) due to remitted cases of depression and/or anxiety. Incremental cost-effectiveness ratios (ICERs) were measured in 2019 Australian dollars (A$) per DALY averted. Uncertainty and sensitivity analyses were performed to test the robustness of cost-effectiveness findings.

The CSC intervention had a high probability (99.6%) of being cost-effective relative to CAU. The resulting ICER (A$5207/DALY; 95% uncertainty interval: dominant to 25 345) fell below the willingness-to-pay threshold of A$50 000/DALY. ICERs were robust to changes in model parameters and assumptions.

This study found that a Dutch CSC intervention, with nurse-delivered guided self-help treatment as a first step, could potentially be cost-effective in treating depression and/or anxiety if transferred to the Australian primary care context. However, adaptations may be required to ensure feasibility and acceptability in the Australian healthcare context. In addition, further evidence is needed to verify the real-world cost-effectiveness of the CSC intervention when implemented in routine practice and to evaluate its effectiveness/cost-effectiveness when compared to other viable stepped care interventions for the treatment of depression and/or anxiety.

Nowadays, many relevant gene-drug associations have been discovered, but pharmacogenomics (PGx)-guided treatment needs to be cost-effective as well as clinically beneficial to be incorporated into standard health-care.

To address current challenges, this systematic review provides an update regarding previously published studies, which assessed the cost-effectiveness of pharmacogenomics testing for the prescription of antidepressants and antipsychotics.

Our initial screening revealed 1159 articles, which was subsequently reduced to 32 articles, deducted by analysis of their abstract. Full-text analysis performed by all authors resulted in 18 papers that were further included in the analysis.

Of the 18 studies evaluations, 16 studies (88.89%) drew conclusions in favor of PGx testing, of which 9 (50%) were cost-effective and 7 (38.9%) were less costly based on cost analysis. In brief, we found sufficient evidence on the cost-effectiveness of PGx in psychiatric disease care. More precisely, supportive evidence exists for CYP2D6 and CYP2C19 gene-drug associations and for combinatorial PGx panels, but evidence is limited for many other drug–gene combinations. Amongst the limitations of the field are the unclear explanation of perspective and cost inputs in many economic studies, as well as the underreporting of study design elements, which can influence significantly the economic evaluations.

Overall, this systematic review highlights the need for additional research on economic evaluations of PGx implementation with an emphasis on psychiatric pharmacogenomics.

We estimated the cost-effectiveness of home fortification with micronutrient powder delivered in a sales-based programme in reducing the prevalence of Fe deficiency anaemia among children 6–59 months in Bangladesh.

Cross-sectional interviews with local and central-level programme staff and document reviews were conducted. Using an activity-based costing approach, we estimated start-up and implementation costs of the programme. The incremental cost per anaemia case averted and disability-adjusted life years (DALY) averted were estimated by comparing the home fortification programme and no intervention scenarios.

The home fortification programme was implemented in 164 upazilas (sub-districts) in Bangladesh.

Caregivers of child 6–59 months and BRAC staff members including community health workers were the participants for this study.

The home fortification programme had an estimated total start-up cost of 35·46 million BDT (456 thousand USD) and implementation cost of 1111·63 million BDT (14·12 million USD). The incremental cost per Fe deficiency anaemia case averted and per DALY averted was estimated to be 1749 BDT (22·2 USD) and 12 558 BDT (159·3 USD), respectively. Considering per capita gross domestic product (1516·5 USD) as the cost-effectiveness threshold, the home fortification programme was highly cost-effective. The programme coverage and costs for nutritional counselling of the beneficiary were influential parameters for cost per DALY averted in the one-way sensitivity analysis.

The market-based home fortification programme was a highly cost-effective mechanism for delivering micronutrients to a large number of children in Bangladesh. The policymakers should consider funding and sustaining large-scale sales-based micronutrient home fortification efforts assuming the clear population-level need and potential to benefit persists.

The newer cancer treatment technologies hold the potential of providing improved health outcomes at an additional cost. So it becomes obligatory to assess the costs and benefits of a new technology, before defining its clinical value. We assessed the cost-effectiveness of intensity-modulated radiotherapy (IMRT) as compared to 2-dimensional radiotherapy (2-DRT) and 3-dimensional radiotherapy (3D-CRT) for treating head and neck cancers (HNC) in India. The cost-effectiveness of 3-DCRT as compared to 2-DRT was also estimated.

A probabilistic Markov model was designed. Using a disaggregated societal perspective, lifetime study horizon and 3 percent discount rate, future costs and health outcomes were compared for a cohort of 1000 patients treated with any of the three radiation techniques. Data on health system cost, out of pocket expenditure, and quality of life was assessed through primary data collected from a large tertiary care public sector hospital in India. Data on xerostomia rates following each of the radiation techniques was extracted from the existing randomized controlled trials.

IMRT incurs an incremental cost of $7,072 (2,932–13,258) and $5,164 (463–10,954) per quality-adjusted life year (QALY) gained compared to 2-DRT and 3D-CRT, respectively. Further, 3D-CRT as compared to 2-DRT requires an incremental cost of $8,946 (1,996–19,313) per QALY gained.

Both IMRT and 3D-CRT are not cost-effective at 1 times GDP per capita for treating HNC in India. The costs and benefits of using IMRT for other potential indications (e.g. prostate, lung) require to be assessed before considering its introduction in India.

The purpose of this study was to evaluate the current state of health economic evaluations (HEEs) submitted by pharmaceutical companies to the Italian Medicines Agency (AIFA) as part of their pricing and reimbursement (P&R) dossiers, and to explore potential future actions in order to enhance their quality.

All company dossiers submitted from October 2016 to December 2018 were reviewed to select those containing pharmacoeconomic studies. The general characteristics of HEEs were described and their quality assessed based on a checklist adapted from Philips et al. (Review of guidelines for good practice in decision-analytic modelling in health technology assessment. Health Technol Assess. 2004;8: 1–158).

Of the 299 dossiers submitted to AIFA, 105 included one or more pharmacoeconomic studies, of which fifty-three were cost-effectiveness analyses. Overall, the compliance of the HEEs with the quality checklist was highly variable: some studies reached high methodological standards whereas others had serious flaws (mean 59.22 percent, range 19.35–90.32 percent). The main weaknesses were the unjustified exclusion of relevant alternatives, poor description and justification of model data and assumptions, and insufficient exploration of uncertainty and study validity. Non-homogeneity across studies was found in study perspectives, discount rates, methods for costing, estimating quality-adjusted life-years and conducting sensitivity analyses.

Based on the results of this study, the recommended actions for increasing the quality of HEEs within reimbursement submissions in Italy are twofold: first, to set methodological standards for conducting and reporting HEEs; second, to strengthen the internal assessment process, also through the acquisition of companies' models and re-evaluation of results. These actions will hopefully provide greater contribution to the evidence-based P&R decision making.