Search Results (166)

Background and aim: Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, tissues, or cells that target rare and severe diseases. ATMPs have shown promising clinical outcomes but are associated with high costs, raising questions about cost-effectiveness. Hence, this systematic review aims to analyze the cost-effectiveness and cost-utility profiles of the European Medicines Agency-authorized ATMPs for treating rare diseases. Methods: A systematic review was conducted following PRISMA guidelines. Studies were identified by searching PubMed, Embase, Web of Science, and ProQuest scientific databases. Economic evaluations reporting incremental cost-effectiveness/utility ratios (ICERs/ICURs) for ATMPs were included. Costs were standardized to 2023 Euros, and a cost-effectiveness plane was constructed to evaluate the results against willingness-to-pay (WTP) thresholds of EUR 50,000, EUR 100,000, and EUR 150,000 per QALY, as part of a sensitivity analysis. Results: A total of 61 studies met the inclusion criteria. ATMPs for rare blood diseases, such as tisagenlecleucel and axicabtagene ciloleucel, were found to be cost-effective in a majority of studies, with incremental QALYs ranging from 1.5 to 10 per patient over lifetime horizon. Tisagenlecleucel demonstrated a positive cost-effectiveness profile in the treatment of acute lymphoblastic leukemia (58%), while axicabtagene ciloleucel showed a positive profile in the treatment of diffuse large B-cell lymphoma (85%). Onasemnogene abeparvovec for spinal muscular atrophy (SMA) showed uncertain cost-effectiveness results, and voretigene neparvovec for retinal diseases was not cost-effective in 40% of studies, with incremental QALYs around 1.3 and high costs exceeding the WTP threshold set. Conclusions: ATMPs in treating rare diseases show promising economic potential, but cost-effectiveness varies across indications. Policymakers must balance innovation with system sustainability, using refined models and the long-term impact on patient outcomes. Full article

Background: Adolescents in Ethiopia, particularly in rural areas, face significant barriers to accessing comprehensive sexual and reproductive health (SRH) services, resulting in poor health outcomes. The youth-friendly health services (YFHS) initiative addresses these challenges by training Health Extension Workers (HEWs) to deliver tailored, age-appropriate care at the primary care level. This study evaluates the cost-effectiveness of YFHS implementation in rural health posts in the Jimma Zone, Ethiopia. Methods: Using an ingredient-based costing approach, costs were analyzed across six health posts, three implementing YFHS and three offering routine services. Health outcomes were modeled using disability-adjusted life years (DALYs) averted, and incremental cost-effectiveness ratios (ICERs) were calculated. Results: Results showed that YFHS reached 9854 adolescents annually at a cost of USD 29,680, compared to 2012.5 adolescents and USD 7519 in control sites. The study showed the ICER of USD 25.50 per DALY averted. The intervention improved health outcomes, including a 27% increase in antenatal care uptake, a 34% rise in contraceptive use, and a 0.065% reduction in abortion-related mortality, averting 52.11 DALYs versus 26.42 in controls. Conclusions: The ICER was USD 25.50 per DALY averted, well below Ethiopia’s GDP per capita, making it highly cost-effective by WHO standards. Scaling YFHS through HEWs offers a transformative, cost-effective strategy to advance adolescent SRH equity and achieve universal health coverage in Ethiopia. Full article

Background: Endoscopic therapies for Crohn’s disease (CD) strictures, including endoscopic balloon dilation (EBD) and endoscopic stricturotomy (ESt), are less invasive interventions compared to surgery. ESt is advantageous for strictures that are longer, more fibrotic, or adjacent to anatomic structures requiring precision, and it has shown a high rate of surgery-free survival. Methods: We designed a microsimulation state-transition model comparing ESt to surgical resection for CD strictures. We calculated quality-adjusted life years (QALYs) over a 10-year time horizon; secondary outcomes included costs (in 2022 USD) and incremental cost-effectiveness ratios (ICERs). We used a societal perspective to compare our strategies at a willingness-to-pay (WTP) threshold of 100,000 USD/QALY. Sensitivity analyses, both deterministic and probabilistic, were performed. Results: The surgery strategy cost more than 2.5 times the ESt strategy, but resulted in nine more QALYs per 100 persons. The ICER for the surgery strategy was 308,787 USD/QALY; thus, the ESt strategy was determined more cost-effective. One-way sensitivity analyses showed that quality of life after ESt as compared to that after surgery, the likelihood of repeat intervention, and surgical mortality and cost were the most influential parameters shifting cost-effectiveness. Probabilistic sensitivity analyses favored ESt in most (65.5%) iterations. Conclusions: Our study finds endoscopic stricturotomy to be a cost-effective strategy to manage primary or anastomotic Crohn’s disease strictures. Post-intervention quality of life and probabilities of requiring repeated interventions exert most influence on cost-effectiveness. The decision between ESt and surgery should be made considering patient and stricture characteristics, preferences, and cost-effectiveness. Full article

Background: This study aims to evaluate the cost-effectiveness of providing the 23-valent pneumococcal polysaccharide vaccine (PPV23) free of charge versus self-paying vaccination among adults aged 60 years and older in Nanning, Guangxi, China. Methods: A decision tree–Markov model was developed to compare three strategies (government-funded free vaccination, self-funded vaccination, and no vaccination) over a 5-year time horizon. The model incorporated local epidemiological data and cost parameters, applying a 3% discount rate. Sensitivity analyses were conducted on key parameters, including vaccine effectiveness against pneumonia and pneumonia treatment costs. Results: The benefit–cost ratios for free and self-funded vaccination were 0.075 and 0.015, respectively, both below the cost-effectiveness threshold of 1. However, the free vaccination strategy resulted in a higher net benefit (USD 399,651.32) compared to the self-funded strategy (USD 222,594.14), along with a lower Incremental Cost-Effectiveness Ratio (ICER) (USD 1.47 per USD 0.14 of avoided disease cost). Although both strategies yielded benefit–cost ratios far below the conventional threshold of 1, the free strategy demonstrated relatively greater economic efficiency. Sensitivity analyses confirmed that vaccine effectiveness against pneumonia and treatment costs were key drivers of economic outcomes. Conclusions: While neither vaccination strategy achieved conventional cost-effectiveness benchmarks in this setting, the free PPV23 vaccination program demonstrated relatively greater economic efficiency compared to the self-funded approach; although neither strategy met the conventional cost-effectiveness thresholds, they should be considered for inclusion in regional health policy for older adults. Full article

X-linked adrenoleukodystrophy (ALD) is an inherited metabolic disorder that can cause adrenal insufficiency and cerebral ALD (cALD) in childhood. Early detection prevents adverse health outcomes and can be achieved by newborn screening (NBS) followed by monitoring disease progression. However, monitoring is associated with high costs. This study evaluates the cost–effectiveness of NBS for ALD in The Netherlands compared to no screening using a health economic model. A decision tree combined with a Markov model was developed to estimate societal costs, including screening costs, healthcare costs, and productivity losses of parents, and health outcomes over an 18-year time horizon. Model parameters were derived from the literature and expert opinion. A probabilistic sensitivity analysis (PSA) was performed to assess uncertainty. The screening costs of detecting one ALD case by NBS was EUR 40,630. Until the age of 18 years, the total societal cost per ALD case was EUR 120,779 for screening and EUR 62,914 for no screening. Screening gained an average of 1.7 QALYs compared with no screening. This resulted in an incremental cost–effectiveness ratio (ICER) of EUR 34,084 per QALY gained for screening compared to no screening. Although the results are sensitive to uncertainty surrounding costs and effectiveness due to limited data, NBS for ALD is likely to be cost–effective using a willingness-to-pay (WTP) threshold of EUR 50,000– EUR 80,000 per QALY gained. Full article

In this paper, we develop an HIV/AIDS epidemic model that incorporates a saturated incidence rate to reflect the limited transmission capacity and the impact of behavioral saturation in contact patterns. The model is formulated as a system of seven non-linear ordinary differential equations representing key population compartments. In addition to model formulation, we introduce an optimal control problem involving three control measures: educational campaigns, screening of unaware infected individuals, and antiretroviral treatment for aware infected individuals. We begin by establishing the positivity and boundedness of the model solutions under constant control inputs. The existence and local and global stability of both the disease-free and endemic equilibrium points are analyzed, depending on the effective reproduction number (${\mathcal{R}}_e$). Bifurcation analysis reveals that the model undergoes a forward bifurcation at ${\mathcal{R}}_e=1$. A local sensitivity analysis of ${\mathcal{R}}_e$ identifies the disease transmission rate as the most sensitive parameter. The optimal control problem is then formulated by incorporating the dynamics of infected subpopulations, control costs, and time-dependent controls. The existence of optimal control solutions is proven, and the necessary conditions for optimality are derived using Pontryagin’s Maximum Principle. Numerical simulations support the theoretical analysis and confirm the stability of the equilibrium points. The optimal control strategies, evaluated using the Incremental Cost-Effectiveness Ratio (ICER), indicate that implementing both screening and treatment (Strategy D) is the most cost-effective intervention. These results provide important insights for designing effective and economically sustainable HIV/AIDS intervention policies. Full article

This study investigates optimal intervention strategies for controlling the spread of two co-circulating strains of SARS-CoV-2 within the Nigerian population. A newly formulated epidemiological model captures the transmission dynamics of the dual-strain system and incorporates three key control mechanisms: vaccination, non-pharmaceutical interventions (NPIs), and therapeutic treatment. To identify the most effective approach, Pontryagin’s Maximum Principle is employed, enabling the derivation of an optimal control function that minimizes both infection rates and associated implementation costs. Through numerical simulations, this study evaluates the performance of individual, paired, and combined intervention strategies. Additionally, a cost-effectiveness assessment based on the Incremental Cost-Effectiveness Ratio (ICER) framework highlights the most economically viable option, while results suggest that the combined application of vaccination and treatment strategies offers superior control over dual-strain transmission and implementing all three strategies together ensures the most robust suppression of the outbreak. Full article

Background: The aim of this study was to assess the cost effectiveness of a screening and treatment intervention approach for chronic HCV infection in Zimbabwe. Methods: Using a decision tree and a validated Markov model, we estimated the lifetime costs and health effects of screening for and treating HCV infections from a healthcare perspective. We evaluated three screening strategies, namely the following: i. no screening; ii. screening among the general population; and iii. screening among high-risk groups. Incremental cost effectiveness ratios were calculated for the strategies that were not dominated. We used deterministic and probabilistic sensitivity analyses to explore the impacts of parameter uncertainty on cost effectiveness outcomes. Results: The strategy of screening among high-risk groups and treating with sofosbuvir/velpatasvir had an incremental cost of USD 1201 and incremental quality-adjusted life years (QALY) of 2.01, yielding an incremental cost effectiveness ratio (ICER) of USD 604 per QALY gained as compared to no screening. The ICER was below the 0.5 times the gross domestic product per capita parameter (USD 796), making the intervention potentially cost effective. The strategy to screen among the general population was dominated, because it costed more and resulted in fewer QALYs than its comparators. Conclusions: Screening for HCV among high-risk populations followed by treatment using sofosbuvir/velpatasvir is cost effective under the assumptions made in this study. Full article

Background: The prevalence of chronic conditions that increase the risk of influenza complications is high among individuals aged ≥50 years, and evidence suggests age-related changes in immune responses to vaccines begin to decline at this age. Persons aged 50–59 years have high rates of influenza infections and are also the most likely age group to be employed. Thus, the burden of influenza is high in this age group. Methods: We investigated the cost-effectiveness of vaccination with an adjuvanted quadrivalent influenza vaccine (aQIV) in a Spanish population aged ≥50 years at high risk of influenza complications. Using a static decision-tree model specifically designed to analyze Spanish data, we calculated incremental cost-effectiveness ratios (ICERs) for aQIV vs. egg-based QIV (QIVe; indicated for any age) and aQIV vs. high-dose QIV (HD-QIV; indicated for persons aged ≥60 years) from payer and societal perspectives. We compared ICERs against a willingness-to-pay threshold of EUR 25,000 per quality-adjusted life year (QALY) gained. The impact of input uncertainty on ICER was evaluated through a probabilistic sensitivity analysis (PSA) and a one-way deterministic sensitivity analysis (DSA). Results: The total incremental cost of vaccination with aQIV was EUR –86,591,967.67, which was associated with gains of 241.02 in QALY (EUR –359,268.05 per QALY gained) and 318.04 in life years (EUR −272,271.37 per life year gain). Compared with the willingness-to-pay threshold of EUR 25,000 per QALY gained, aQIV was the most cost-effective influenza vaccine relative to the combination of QIVe or HD-QIV. These findings were supported by PSA and DSA analyses. Conclusions: In the model, aQIV dominated QIVe and HD-QIV, demonstrating that aQIV use would be cost-saving for persons aged ≥50 years who are at high risk of influenza complications. Full article

Background/Objectives: This study was conducted to assess the cost-effectiveness of chlorhexidine-coated (AGBA) peripheral insertion central catheters (PICCs) versus standard PICCs for managing catheter-related complications among patients with hematologic disease. Methods: A decision tree health economic model was developed, incorporating quality-adjusted life years (QALYs) derived from the literature, as well as complication rates and per-patient costs from a randomized controlled trial. The base case incremental cost-effectiveness ratio (ICER) was assessed against established willingness to pay (WTP) thresholds. One-way sensitivity analyses were conducted to address assumptions and uncertainties. Results: The mean healthcare cost per patient of standard PICCs was RMB 21,987.32 (USD 3242.82, at an average exchange rate of RMB 678.03 = USD 100), affecting 0.68 QALYs in 90 days. The mean healthcare cost per patient of AGBA PICCs was RMB 19,696.23 (USD 2904.92), affecting 0.73 QALYs in 90 days, thus resulting in a saving of RMB 2291.10 (USD 428.44). After the model simulation, standard PICCs resulted in a reduction of 0.05 QALYs. The ICER for AGBA PICCs compared with standard PICCs was consistently centered at RMB 4271.31 (USD 629.96). Conclusions: one-way sensitivity analyses of cost-effectiveness versus WTP confirmed the robustness of the model across various parameter changes, indicating that AGBA PICCs could provide significant healthcare savings over a 1-year period when adopted in routine chemotherapy treatment for patients with hematologic disease. Full article

Background/Objectives: We aimed to compare non-pharmacological (non-PHM) and pharmacological (PHM) treatment for patients with non-acute lumbar disc herniation (LDH) and determine the feasibility of a large-scale study. Methods: This was a two-armed, parallel, multicenter, pragmatic controlled trial performed in South Korea. All patients underwent magnetic resonance imaging (MRI) scans both at the screening stage and the last follow-up. Patients with LDH findings on MRI were randomly assigned to non-PHM and PHM groups. Treatment was administered twice a week for a total of 8 weeks, and follow-up assessments were performed at weeks 9, 13, and 27 post-randomization. The primary outcome was the Oswestry Disability Index (ODI) score. A linear mixed model was used for primary analysis from intention-to-treat perspectives. The incremental cost-effectiveness ratio (ICER) was calculated for economic evaluation. Results: Thirty-six patients were enrolled, and thirty-five were included in the final analysis. At Week 9, the difference in ODI scores between the two groups was 5.17 (95% CI: −4.00 to 14.35, p = 0.262), and the numeric rating scale scores for lower back and leg pains were 1.89 (95% CI: 0.68 to 3.10, p = 0.003) and 1.52 (95% CI: 0.27 to 2.77, p = 0.018), respectively, confirming greater improvement in the non-PHM group than in the PHM group. The non-PHM group showed lower costs and higher quality-adjusted life years than the PHM group. The ICER calculated using the EuroQoL-5 Dimension (EQ-5D) was USD 20,926. Conclusions: We confirm the possibility that a non-PHM strategy could be a more effective and cost-effective treatment option than PHM for patients with non-acute lumbar disc herniation. Furthermore, this pilot study confirmed the feasibility of the main study in terms of design and patient compliance. Full article

Background/Objectives: Functional dyspepsia is associated with abdominal pain and nausea, which leads to reduced quality of life, loss of productivity, and economic loss for patients. Itopride hydrochloride (itopride) stimulates the gastrointestinal smooth muscles, thereby promoting gastric emptying. It has been shown to significantly improve symptoms in patients with functional dyspepsia without severe side effects. Itopride has been available in Vietnam for many years; however, the cost-effectiveness of the drug has not been established. The aim of this study is to estimate the cost-effectiveness of itopride for the treatment of functional dyspepsia in Vietnam. Methods: A 3-stage Markov model with the following health states—controlled functional dyspepsia, uncontrolled functional dyspepsia, and dead—was developed. Functional dyspepsia was used to assess itopride over 10 years using 8-week cycles. A broader Vietnamese societal perspective was assumed for the analysis. Input was retrieved from the literature and through local clinical input from physicians in Vietnam. Output was reported as an incremental cost-effectiveness ratio (ICER) per quality-adjusted life years (QALY). A GDP/capita threshold (very cost-effective: 1 × GDP = Vietnamese Dong (VND) 64.1 M, cost-effective: 3 × GDP = VND 192.2 M) was used as recommended by the WHO in Vietnam. One-way and probabilistic sensitivity analyses were performed. Results: Itopride use resulted in an additional 0.28 QALYs at an extra cost of VND 11.2 M. This resulted in an ICER of VND 39.7 M per QALY, which is lower than the threshold of VND 192.2 M. One-way sensitivity analyses showed that the ICER was sensitive to varying the efficacy VND 31.8 M to VND 88.3 M), cost of itopride (ICER: VND 43.1 M to VND 56.5 M), and the health utility values (ICER: VND 45.2 M to VND 55.3 M). More than 80% of the simulations in the probabilistic sensitivity analysis were cost-effective at the 1 × GDP (VND 64.1 M) threshold, and 91.3% were cost-effective at the 3 × GDP (VND 192.2 M) threshold. Conclusion: This study shows that itopride hydrochloride is a very cost-effective treatment for functional dyspepsia in Vietnam, with the ICER (VND 39.7 M/QALY) being even lower than the 1 × GDP (VND 64.1 M) threshold. Full article

Objectives: Pneumococcal disease (PD), caused by S. pneumoniae, is a serious global health issue, primarily for adults over 65, due to its high mortality and morbidity rates. Recently, broader-serotype vaccines have been introduced to cope with tremendous hospital costs and decreasing quality of life. Our study aims to systematically review the cost-effectiveness of current PCVs (pneumococcal conjugate vaccines) and PPVs (pneumococcal polysaccharide vaccine) from 2018 to April 2024. Methods: Articles were identified through PubMed, Embase, and Cochrane. Key outcomes include an improved incremental cost-effectiveness ratio (ICER) and quality-adjusted life-years (QALY), with the article’s quality assessed via the Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 2022). In total, 23 studies were included, with 22 studies of high quality and 1 of moderate quality. Results: These articles showed that PCV20 was the most cost-effective option compared with other vaccines, including PPV23, PCV13, PCV15, and PCV15/PPV23, for both young and older adults, regardless of risk factors. PCV20, when used alone, saved greater costs than PCV20, followed by PPV23. Conclusions: For countries applying lower-valency vaccines, switching to PCV20 as a single regimen would be the most beneficial for averting pneumococcal cases and reducing costs in adults aged 18–64 and over 65. Full article

Background: Human Papillomavirus (HPV) is the primary cause of cervical cancer. Single-dose HPV vaccination can effectively prevent high-risk HPV infection that causes cervical cancer and accelerate progress toward achieving cervical cancer elimination goals. We modelled the potential impact of adopting single-dose HPV vaccination strategies on health and economic outcomes in Kenya, where a two-dose schedule is the current standard. Methods: Using a validated compartmental transmission model of HPV and HIV in Kenya, we evaluated the costs from the payer’s perspective to vaccinate girls by age 10 with either one or two doses and increasing coverage levels (0%, 70%, 77%, 90%). Additionally, we modelled single-dose strategies supplemented with either catch-up vaccination of adolescent girls and young women or vaccination for all by age 10, funded with the first five-years of cost savings of switching from a two- to one-dose schedule. Costs and outcomes were discounted at 3% annually, and incremental cost-effectiveness ratios (ICERs) were calculated per disability-adjusted-life-year (DALY) averted. Results: All one-dose and the two-dose 90% coverage strategies were on the efficiency frontier, dominating the remaining two-dose strategies. The two-dose 90% coverage strategy had a substantially higher ICER (US$6508.80/DALY averted) than the one-dose 90% coverage (US$197.44/DALY averted). Transitioning from a two- to one-dose schedule could result in US$21.4 Million saved over the first five years, which could potentially fund 2.75 million supplemental HPV vaccinations. With this re-investment, all two-dose HPV vaccination scenarios would be dominated. The greatest DALYs were averted with the single-dose HPV vaccination schedule at 90% coverage supplemented with catch-up for 11–24-year-old girls, which had an ICER of US$78.73/DALYs averted. Conclusions: Considering the logistical and cost burdens of a two-dose schedule, a one-dose schedule for girls by age 10 would generate savings that could be leveraged for catch-up vaccination for older girls and accelerate cervical cancer elimination in Kenya. Full article

Background/Objectives: To evaluate the health benefits, costs, and cost-effectiveness of vaccination with bivalent respiratory syncytial virus stabilized prefusion F vaccine (RSVpreF) for the prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in Greek adults 60 years of age and older. Methods: A Markov model was adapted to simulate lifetime risk of health and economic outcomes from the public payer’s perspective over a lifetime horizon. Epidemiology, vaccine effectiveness, utilities, and direct medical costs (EUR, 2024) were obtained from published studies, official sources, and local experts. Model outcomes included the number of medically attended RSV cases, stratified by care setting (i.e., hospital, emergency department [ED], outpatient visits [OV]), and attributable RSV-related deaths, costs, life years (LY), quality-adjusted life-years (QALY), and incremental cost-effectiveness ratios (ICERs) of RSVpreF vaccination compared with no vaccination. Results: The model projected 258,170 hospitalizations, 112,248 ED encounters, 1,201,604 OV, and 25,463 deaths related to RSV in Greek older adults resulting in direct medical costs of EUR 1.6 billion over the lifetime horizon. Assuming RSV vaccination would reach the same coverage rates as pneumococcal and influenza programmes, 18,118 hospitalizations, 7874 ED encounters, 48,079 OV, and 1706 deaths could be prevented over the modelled time horizon. The health benefits associated with RSVpreF contributed to an incremental gain of 10,976 LYs and 7230 QALYs compared with no vaccination. The incremental analysis reported that vaccination with RSVpreF was estimated to be a cost-effective strategy resulting in ICERs of EUR 12,991 per LY gained, EUR 19,723 per QALY gained, and EUR 7870 per hospitalized RSV case avoided compared with no vaccination. Conclusions: Vaccination with RSVpreF was a cost-effective strategy for the prevention of RSV disease in Greek adults over 60 years of age. The introduction of RSV vaccination can improve public health by averting RSV cases and deaths and has the potential to fulfil an unmet medical need. Full article