Cost-effectiveness of multigene sequencing test and treatment for metastatic non-small cell lung cancer: A unique setting in the initial adoption phase in Japan allowing testing only after standard treatment.

Objective: To clarify the cost-effectiveness of comprehensive diagnosis and treatment of metastatic non-small cell lung cancer in Japan, from initial diagnosis to post-standard treatment, using three different strategies. Methods: A decision tree was created using three diagnostic and treatment strategies, assuming that Foundation One CDx (F1CDx), a comprehensive genome panel, was introduced in Japan in June 2019. This comprehensive decision tree includes Markov models, cost-effectiveness analyses (CEA), and cost-utility analyses (CUA) of the three strategies from the perspective of Japanese payers. Specifically, Strategy1 involves single-gene testing at the initial diagnosis and F1CDx after standard treatment; Strategy2 involves only single-gene testing at the initial diagnosis; Strategy3 involves F1CDx at the initial diagnosis. The incremental cost-effectiveness ratios (ICERs) of the three strategies are estimated. Sensitivity analyses were performed to assess the uncertainty of the parameter settings. Results: Strategy3 was dominated for both CUA and CEA. The ICER/quality-adjusted life year (QALY) for Strategy2 versus Strategy1 was USD 13,734 (JPY 2,080,923, USD 1 = JPY 151.39 on April 1st, 2024), which is less than the willingness to pay of USD 45,900 (JPY 7,500,000), and Strategy2 was more cost-effective than Strategy1. F1CDx was not cost-effective compared to multiple simultaneous single tests at the initial diagnosis, either after standard treatment or at the initial diagnosis. Sensitivity analysis also showed that the most influential factor on the ICER for both CEA and CUA was treatment cost. Conclusions: From both patient benefit and health economic perspectives, introducing F1CDx after standard treatment in June 2019 was not as cost-effective as multiple simultaneous single tests at the initial diagnosis but was more realistic from a health economic perspective than introducing F1CDx at the time of initial diagnosis. Therefore, the policy at the time of F1CDx introduction in Japan was appropriate from a short-term health-economic perspective.

Effect of CYP3A5*3, ABCC2 C-24T, and ABCC2 C3972T Genetic Polymorphisms on Direct Cost of Kidney Transplant Recipients.

Introduction Genetic variations can influence how kidney transplant recipients (KTRs) respond to immunosuppressive drugs. However, limited resources necessitate a cost-benefit analysis of pharmacogenetic testing to determine its role in routine practice. This study investigated the cost-effectiveness of three genetic polymorphisms (CYP3A5*3, ABCC2 -24C>T, and ABCC2 3972C>T) in KTRs. Methods This was a multicenter, prospective observational cohort study that included patients on tacrolimus-mycophenolate-prednisolone treatment. Ethnically diverse adult KTRs who had undergone kidney transplantation between 2020 and 2021 and consented were enrolled in the study. Deoxyribonucleic acid (DNA) was extracted from the collected blood samples using a commercially available kit. CYP3A5*3, ABCC2 -24C>T, and ABCC2 3972C>T single nucleotide polymorphisms (SNPs) were determined by polymerase chain reaction (PCR). Results Data was analyzed from 39 KTRs with an average age of 32.2 ± 7.0 years. The median annual healthcare cost per patient was MYR 52,700 (laboratory tests and immunosuppressants being the highest expenses). Notably, the annual cost was significantly higher in patients with the CYP3A5*3 variant compared to the wildtype (p < 0.001). Furthermore, an incremental cost-effectiveness analysis revealed that carriers of the CYP3A5*1 wildtype allele, the ABCC2 -24C>T T variant allele, and the ABCC2 3972C>T T variant allele were associated with a more cost-effective approach to kidney transplantation management, potentially reducing the risk of graft rejection and acute tubular necrosis (ATN). Conclusion While these findings suggest potential cost benefits for specific genotypes, further research with larger and more diverse patient populations is necessary to definitively establish the role of pharmacogenetic testing in optimizing cost-effectiveness for KTRs.

Challenges in contemporary spinal robotics: encouraging spine surgeons to drive transformative changes in the development of future robotic platforms.

The field of spinal robotics has witnessed considerable advances, which have primarily focused on enhancing pedicle screw placement. This article critically evaluates the current direction of spinal robotics development, raising concerns about the disproportionate emphasis on pedicle screw placement when existing techniques already yield commendable results. Discussions on various parameters, including quality, cost-effectiveness, and accessibility, highlight the need for a broader perspective in the development of robotics for spinal surgery. Comparative analyses reveal that navigation systems offer cost-effective and time-efficient alternatives to robotics, with similar accuracy levels. Patient demand for robotic interventions is influenced by perceived superiority, warranting careful consideration of public sentiment. This article also underscores the need for future spine surgeons to maintain proficiency in traditional techniques. The influence of industry and key opinion leaders in steering the focus toward pedicle screw placement is discussed, emphasizing the need for a more holistic approach. Accessibility issues and legal considerations in the evolving field of spinal robotics are addressed, and the potential for robotics to enhance various aspects of surgical procedures beyond pedicle screw placement is explored. In conclusion, we advocate for a shift in focus in spinal robotics, emphasizing the untapped potential to streamline common surgical procedures (such as discectomy, laminectomy, and endoscopy), enhance precision, and improve patient outcomes in areas beyond pedicle screw placement. Future advances in spinal robotics have the potential to transform the surgical landscape, benefitting all stakeholders, including patients, surgeons, and hospitals.

Economic evaluations of robot-assisted radical prostatectomy and the factors affecting its cost-effectiveness: a systematic review.

This systematic review aims to summarize the progress made in the study of the cost-effectiveness of robot-assisted radical prostatectomy (RARP) worldwide and to analyze the economic factors influencing this, in an attempt to provide methodological guidance for conducting economic evaluation studies in a domestic context, and to put forward suggestions for improving the cost-effectiveness of RARP in emerging markets. We conducted a systematic literature review and analysis of studies published worldwide from January 2000 to July 2024 concerning the economic evaluation of RARP compared with laparoscopic radical prostatectomy (LRP) or open radical prostatectomy (ORP). A total of 16 papers were included. The literature was generally of good quality. Methodological approaches. varied among studies, leading to inconsistent economic findings. The choice of research settings, including the perspective of the study and time horizon, as well as differences in parameters such as surgical volumes and cost of equipment purchases, were the main factors that affected the cost-effectiveness of RARP. Based on the methodology used in the included studies, we suggest that short-term, localized economic evaluations should be carried out first, based on follow-up studies in emerging markets, whereas long-term economic evaluations can be performed when sufficient data are available. Referring to the analysis of the economic factors influencing cost-effectiveness in the included studies, we suggest that different research settings should be chosen according to the purpose for which policymakers allocate public funds, and that the cost-effectiveness of RARP can be enhanced through technical improvements and resource optimization.

Cost-Effectiveness Analysis of Pneumococcal Vaccines in the Pediatric Population: A Systematic Review.

Objectives: Pneumococcal disease, caused by Streptococcus pneumoniae, is the leading cause of mortality in children worldwide. The tremendous direct cost of hospital admissions and significant indirect costs from productivity loss contribute considerably to its economic burden, with vaccination being the only efficient protection against the illness. Our study aims to summarize the cost-effectiveness of the pneumococcal conjugate vaccine (PCV) implemented in the pediatric population. Methods: Employing the online databases PubMed, Embase, and Medline, we looked for economic evaluations from 2018 until March 2024. The Incremental Cost-Effectiveness Ratios (ICER) and Quality-Adjusted Life Years (QALY) were the primary outcomes for measuring the cost-effectiveness of PCVs. A 28-item CHEERS 2022 checklist was applied to assess the quality of the collected studies. Results: Of the 16 papers found, 9/16 discussed the lower-valent vaccines (PCV13, PCV10) and 7/16 examined the higher-valent vaccines (PCV20, PCV15). PCV13 and PCV10 involved greater costs and generated more QALY compared to no vaccination. Both PCV15 and PCV20 averted substantial healthcare costs and yielded greater quality of life than PCV13. Additionally, PCV20 was a dominant strategy compared to PCV15. Conclusions: Utilizing PCV13 is a very cost-effective option compared to not getting vaccinated. Transitioning from PCV13 to PCV20 would result in higher QALY gain and more cost-saving than switching to PCV15.

Diagnostic accuracy and treatment benefit of PET/CT in staging of colorectal cancer compared to conventional imaging.

BACKGROUND: Until recently the use of positron emission tomography (PET) CT for staging in colorectal cancer (CRC) has been limited to the detection of distant metastasis in advanced disease. But with the introduction of neoadjuvant treatments in CRC, accurate pre-treatment staging has become more relevant. AIMS: The aim of the study was to assess the staging accuracy for nodal and distant metastasis of PET/CT compared to computed tomography (CT) alone in CRC. Secondary endpoints were overall survival (OS) and cost of CT compared to PET/CT. METHODS: A retrospective analysis of 539 cases with CRC staged with PET/CT and or CT between 2015 and 2021 in a Swiss tertiary referral center was performed. In 471 patients for nodal staging and 479 for staging of distant metastasis the clinical stage of both modalities was compared with pathological stage. RESULTS: The distribution of UICC stages (n = 479) was as follows: Stage I 62 cases (12.9 %), Stage II 127 cases (26.5 %), Stage III 199 cases (41.5 %), Stage IV 91 cases (19.0 %). CT alone compared to PET was able to predict nodal involvement with a sensitivity of 55.2 % (95%CI 5.7-59.7 %) and 66.7 % (95%CI 62.4-70.9 %), respectively. The specificity was 67.0 % (95%CI 62.8-71.3 %) for CT and 63.6 % (95%CI 59.3-68.0 %) for PET. The positive predictive value was 49.5 % for CT vs. 51.8 % for PET. The sensitivity of metastasis detection was 53.6 % (95%CI 49.1-58.1 %) for CT and 82.5 % (95%CI 79.1-85.9 %) for PET. CONCLUSIONS: PET/CT showed higher sensitivity in the detection of lymph node involvement and metastases in CRC patients compared to CT alone.

Global Cost-Effectiveness of Transcatheter vs. Surgical Aortic Valve Replacement in Severe Aortic Stenosis: A Systematic Review and Meta-Analysis.

BACKGROUD: Transcatheter aortic valve replacement (TAVR) is a less invasive treatment option for patients with severe aortic stenosis (AS); however, its economic benefits in patients with low to intermediate surgical risk remain controversial and vary by country. We conducted a systematic review to compare the economic benefits of TAVR versus surgical aortic valve replacement (SAVR). METHODS: We searched six databases, including PubMed, Medline, Scopus, Web of Science, Embase, and Clinical Trials for randomized controlled trials on the economic benefits of TAVR with different valve types and SAVR in symptomatic AS patients with low to intermediate surgical risk, from inception to October 2023. We extracted data on quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER), with ICER converted to 2023 United States dollars (USD) exchange rates. RESULTS: Fifteen studies met the inclusion criteria, with the overall quality ranging from intermediate to high. Among these, TAVR was found to be cost-effective in 14 studies, while in one study conducted in a developing country, TAVR was not cost-effective. When adjusted to 2023 USD, the ICER values ranged from $3,669 to $340,038 per QALY gained. CONCLUSION: TAVR appears to be a cost-effective alternative to SAVR in patients with low to intermediate AS. In all studies, TAVR was associated with a significant increase in QALYs compared to SAVR. As it is an expensive procedure, the cost-effectiveness of TAVR depends on each country's ICER and willingness-to-pay threshold.

Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Moderate or Severe Hemophilia A in China.

BACKGROUND: It is still being determined if prophylaxis (PR) has superior cost effectiveness compared with on-demand (OD) treatment for moderate or severe hemophilia A (HA) children in China. OBJECTIVE/PURPOSE: To evaluate the cost-effectiveness of PR and OD treatment for children with moderate or severe HA without inhibitors in China. METHODS: A retrospective cost-effectiveness study was conducted on 640 HA children (373 and 267 children were on the PR and OD treatment, respectively) from January 2021 to November 2022. The Markov model was used to estimate the economic and clinical outcomes and would run for 17 yearly cycles with the initial age at 2 years. The transfer probabilities were extracted from the data of "Hemophilia Home Care Center" and the literature published. All patients' drug costs were collected from the data of "Hemophilia Home Care Center". One-way and probabilistic sensitivity analyses were conducted on the data to evaluate the robustness of the results. RESULTS/FINDINGS: PR was consistently associated with higher overall quality-adjusted life years (QALYs) compared with OD treatment (9.59 QALYs vs. 6.85 QALYs). The incremental cost-effectiveness ratio (ICER) of PR compared with the OD treatment was calculated to be approximately US$12,151.35 (RMB¥81,778.55) per QALY gained. This amount was lower than the willingness-to-pay (WTP) threshold of US$38,212.74 (RMB¥257,171.71). One-way sensitivity analysis found that the results were sensitive to the cost of OD and PR treatments. CONCLUSIONS/IMPLICATIONS: This study indicated that PR is cost-effective compared with OD treatment for children with moderate or severe HA without inhibitors in China.

Will a government subsidy increase couples' further fertility intentions? A real-world study from a large-scale online survey in Eastern China.

STUDY QUESTION: How many couples with at least one child under 3 years would like to have another one or more child(ren) in Eastern China and will an in-cash subsidy be conducive to couple's fertility intentions? SUMMARY ANSWER: In sum, only 15.1% of respondents had further fertility intentions (FFI) before learning about the subsidy, and the planned in-cash subsidy policy increased respondents' overall FFI by 8.5%. WHAT IS KNOWN ALREADY: Fertility has been declining globally and has reached a new low in China. The reasons why the Chinese three-child policy was under-realized, and how couples will react to a planned monthly ¥1000 (€141.2) subsidy policy, are not fully understood. STUDY DESIGN SIZE DURATION: During January and February 2022, a cross-sectional online survey aiming to understand families' expenses of raising a child under 3 years old, and couples' FFI, was conducted. During the survey period, 272 510 respondents scanned the QR code. This study reports the findings pertaining to questions on respondents' sociodemographic characteristics, household factors, FFI, and changes in intention from negative to positive after learning about the planned in-cash subsidy. After exclusion, 144 893 eligible responses were included. PARTICIPANTS/MATERIALS SETTING METHODS: Respondents' FFI, the effect of a planned ¥1000/month*36 months' in-cash subsidy (€5083.2 in total) on people with a negative FFI before the subsidy, and potential reasons for persistent negative FFI after learning about the subsidy were collected through an anonymous online survey. Stepwise binary logistic regression models were used to select associated factors. The potential fertility rate change and government costs were estimated. A stratified analysis by current child number and sensitivity analysis were also conducted. MAIN RESULTS AND THE ROLE OF CHANCE: In sum, 15.7% (22 804/144 893) of respondents were male, 15.1% of respondents reported a positive FFI, and 10.0% (12 288/123 051) without an FFI at first changed their intention after learning about the planned in-cash subsidy policy. For those who still said 'no FFI', 46.5%, 20.6%, and 14.7% chose pressure on housing status, expenses on children's education, and lack of time or energy for caring for another child as their first reasons. FFI was strongest in participants receiving the most financial support from their parents, i.e. grandparents (OR = 1.73, 95% CI = 1.63-1.84 for the >¥100 000/year group), and weakest in those already having two children (OR = 0.23, 95% CI = 0.22-0.24). For those with no FFI before learning about the subsidy policy, respondents with the highest house loan/rent (>¥120 000/year, OR = 1.27, 95% CI = 1.18-1.36) were more likely to change their FFI from 'No' to 'Yes', and those with the highest household income (>¥300 000/year, OR = 0.65, 95% CI = 0.60-0.71) were least susceptible to the policy. In our study population, about 1843 more births every year and an additional 0.3 children per woman were projected under a conservative estimation. Annual estimated cost at the provincial scale would be ¥817.7 (€115.5) million, about 1.02‰ of the total General Public Budget Revenue in 2022. The findings were generally robust in the stratified analysis and sensitivity analysis. LIMITATIONS REASONS FOR CAUTION: Selection bias and information errors may exist in the online survey responses. The large sample size and detailed further analysis were used to minimize such biases. WIDER IMPLICATIONS OF THE FINDINGS: Fertility intentions in Eastern China are rather low. Policymakers should focus more on financial and childcare burdens for a better realization of the three-child policy, including housing, education and childcare services. An in-cash subsidy, which has never been used in China previously, shows promising potential for increasing FFI. However, the application of such policy should be in line with local conditions for better cost-effectiveness regarding fertility-boosting and fiscal sustainability for the government in the long run. STUDY FUNDING/COMPETING INTERESTS: This work was supported by the National Key Research and Development Plan of China (2019YFC0840702). The authors declare no conflict of interests. TRIAL REGISTRATION NUMBER: N/A.

Cost-effectiveness of home care compared to hospital care in patients with chronic obstructive pulmonary disease (COPD): a systematic review.

Background: To compare, through a systematic literature review, the cost-effectiveness ratio of home care compared to hospital care for following up patients with chronic obstructive pulmonary disease (COPD). Methods: This review was registered in PROSPERO, and the bibliographic search was performed in six primary databases [MedLine (via PubMed), Scopus, LILACS, SciELO, Web of Science, and Embase], two dedicated databases for economic studies (NHS Economic Evaluation Database (NHS EED) and Cost-Effectiveness Analysis (CEA) Registry), and two databases for partially searching the "gray literature" (DansEasy and ProQuest). This review only included studies that compared home and hospital care for patients diagnosed with COPD, regardless of publication year or language. Two reviewers selected the studies, extracted the data, and assessed the risk of bias independently. A JBI tool was used for risk of bias assessment. Results and discussion: 7,279 studies were found, of which 14 met the eligibility criteria. Only one study adequately met all items of the risk of bias assessment. Thirteen studies found lower costs and higher effectiveness for home care. Home care showed a better cost-effectiveness ratio than hospital care for COPD patients. Regarding effectiveness, there is no possibility of choosing a more effective care for COPD patients, given the incipience of the data presented on eligible studies. However, considering the analyzed data refer only to high-income countries, caution is required when extrapolating this conclusion to low- and low-middle-income countries. Systematic review registration: https://www.crd.york.ac.uk/prospero/, identifier CRD42022319488.

Self-care towards the end of life: A systematic review and narrative synthesis on access, quality and cost.

BACKGROUND: Policy and practice encourages patients to engage in self-care, with individual patient management and remote monitoring of disease. Much is known of the moderate stage of chronic disease, without a clear understanding of how self-care applies towards the end of life. AIM: To review the current evidence on practices of self-care in life-limiting conditions and its impacts on healthcare utilisation, quality of life and associated costs. DESIGN: We systematically searched 10 scientific databases (MEDLINE, CINAHL, Embase, PsycINFO, Cochrane Central, Cochrane Database of Systematic Reviews, Scopus, Sociological Abstracts, Social Work Abstracts and Health Management Information Consortium) from inception to October 2023 with citation and hand searching. A narrative synthesis was undertaken, with quality and relevance assessed using Gough's Weight of Evidence framework. Titles and abstracts were independently screened by three researchers. RESULTS: Findings from 33 studies revealed self-care as increasingly burdensome or unfeasible towards the end of life, with patients delaying use of professional care. Self-care became increasingly burdensome for patients, carers and professionals as illness progressed. Self-monitoring may exacerbate hospitalisations as patients delayed seeking professional help until crises arose. Findings regarding quality were inconclusive, with some evidence suggesting that self-care can decrease care costs. CONCLUSIONS: This review has shown that research on self-care is an evolving area of study, with a current focus on acute care and hospitalisations. Future research should seek to provide a more complete account of the relation between self-care and non-acute care use, and quality, with further efforts to study self-care costs incurred through self-funding.

Prevalence of Chronic Disease and Cost Effectiveness of a Free Clinic.

Free clinics are a widespread, yet limited research has examined the health status of people served and the cost effectiveness of free clinics. The purpose of this study was to describe the sociodemographic characteristics and prevalence of chronic conditions in patients seen at a free clinic and estimate the clinic's cost-effectiveness. The study was a retrospective chart review of 350 randomly selected paper medical charts (2009-2020) and 1,676 electronic medical records (2020-2022) at the Noor free clinic in California supported by private donors, non-profits, and foundations. Cost-effectiveness was calculated from annual clinic operating costs, services provided, quality-adjusted life years (QALYs) saved, and projected savings from preventing emergency department visits. Of the 350 paper charts reviewed, 336 met inclusion criteria. Patients had an average age of 46.6 years, with most identifying as female (60%), Hispanic (69%), Spanish-speaking (62%), and employed (59%). About 64% had incomes below the federal poverty line. Chronic conditions included overweight/obesity (75%), chronic pain (46%), hypertension (28%), hyperlipidemia (24%), type 2 diabetes (24%), and mood disorders (23%). A quarter of patients had at least one chronic condition, and over half had multiple conditions. The clinic's services in 2022 resulted in an estimated 146 QALYs saved, $11.5 million in cost savings, and a return on investment of $13.18 per $1 invested. The free clinic provided medical care to low-income, Hispanic populations without insurance who historically have been disenfranchised from healthcare access. Cost-effectiveness analysis suggested that it provided significant health benefits and cost savings.

Three Therapy Regimens and Their Cost-effectiveness for Girls With Central Precocious Puberty: A Real-World Clinical Study.

The objective of this study was to evaluate the effectiveness and cost-effectiveness of different therapy regimens for girls with central precocious puberty (CPP). This study retrospectively analyzed CPP girls from 2013 to 2021 and grouped them into GnRHa, Mixed, and GnRHa+GH based on therapy regimen. While comparing the differences among these groups, initial age and bone age of GnRHa group girls were significantly lower than Mixed and GnRHa+GH groups, but their growth level was significantly higher (P < .05). In the Mixed group, starting with GnRHa alone, the predicted adult height improvement (ΔPAH) decreased to -1.0 cm in the second year, prompting addition of growth hormone (GH) therapy in the third year, resulting in a ΔPAH increase to 3.0 cm. At therapy completion, final predicted adult heights (PAHs) were similar among the groups at 155.6 to 156.7 cm, with ΔPAH between 5.8 and 6.5 cm and no significant intergroup differences (P > .05). Younger CPP girls with greater height can begin therapy with GnRHa alone. Consideration of combining GH therapy depends on growth velocity and PAH during treatment.

Cost-effectiveness of an extended-role general practitioner clinic for persistent physical symptoms: results from the Multiple Symptoms Study 3 (MSS3) pragmatic randomised controlled trial.

OBJECTIVES: To evaluate the cost-effectiveness of an extended-role general practitioner (GP) symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as "medically unexplained symptoms"). METHODS: A 52-week within-trial cost-utility analysis of a pragmatic multicentre randomised controlled trial comparing SC+UC (n=178) against UC alone (n=176), conducted from the primary perspective of the UK National Health Service (NHS) and personal and social services (PSS). Base-case quality-adjusted life-years (QALYs) were measured using EQ-5D-5L. Missing data were imputed using multiple imputation (MI). Cost-effectiveness results were presented as incremental cost-effectiveness ratios (ICERs) and incremental net monetary benefits (INMBs). Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 non-parametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and capability ICECAP-A outcomes to estimate QALYs and years of full capability (YFC) respectively, varying intervention costs, missing data mechanism assumptions). RESULTS: Multiple imputation analysis showed that, compared to UC alone, SC+UC was more expensive [(adjusted mean cost difference: 704; 95% CI:£605, £807)] and more effective [(adjusted mean QALY difference: 0.0447 (95% CI:0.0067, 0.0826)] yielding an ICER of £15,765/QALY, INMB of £189.22 (95% CI:-£573.62, £948.28) and a 69% probability of the SC+UC intervention arm being cost-effective at a threshold of £20000 per QALY. Results were robust to most sensitivity analyses, but sensitive to missing data assumptions (2 of the 8 scenarios investigated), SF-6D and ICECAP-A quality of life outcomes. CONCLUSIONS: A Symptoms Clinic is likely to be a potentially cost-effective treatment for patients with persistent physical symptoms.

Advances and Challenges Associated with Low-Cost Pulse Oximeters in Home Care Programs: A Review.

Oximeters have significantly evolved since their invention and are essential for monitoring chronic diseases in home care. However, commercial models can present an economic barrier. Therefore, we conducted a review of the use of low-cost pulse oximeters in the home care of patients with respiratory diseases. Our review included studies addressing oxygen saturation and heart rate monitoring in adults, focusing on the use of portable devices. Our search identified advances in vital signs monitoring that could provide accessible solutions for non-clinical settings. Although there are challenges related to clinical validation and accuracy, these oximeters may improve medical care, particularly in resource-limited areas. As a result, the accessibility of these devices opens up new possibilities for patients with chronic respiratory diseases in home care, enabling regular self-monitoring and increasing control over their health.

Cost-utility of nelarabine for the first-line treatment of newly diagnosed pediatric T-cell acute lymphoblastic leukemia in Canada.

BACKGROUND: The Children's Oncology Group (COG)-AALL0434 trial investigated the addition of nelarabine to the augmented Berlin-Frankfurt-Münster (aBFM) protocol in patients (1.0-30.99 years) with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost-effectiveness data. We estimated the cost-utility of nelarabine for this indication from a Canadian public healthcare payer perspective. METHODS: We developed a microsimulation model that followed hypothetical patients with newly diagnosed T-ALL from post-induction therapy to death. Three health states were modeled: relapse-free, post-relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long-term mortality risks were sourced from literature. Total healthcare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted. RESULTS: Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11 years post diagnosis) and while patients were in the relapse-free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold. CONCLUSION: Incorporating nelarabine into aBFM was cost-effective across different scenarios and assumptions. These results support its funding by public and private payers.

A cost-effectiveness analysis of RefluxStop against relevant therapeutic alternatives for chronic gastroesophageal reflux disease in Sweden.

INTRODUCTION: The standard treatment for gastroesophageal reflux disease (GERD) is proton pump inhibitors (PPIs). In selected cases, Nissen fundoplication is offered as a surgical treatment option, but alternative endoscopic and minimally invasive surgical alternatives are emerging. RefluxStop is a new technology for the treatment of GERD. RESEARCH DESIGN AND METHODS: A cost-effectiveness analysis of RefluxStop in comparison to PPI therapy and Nissen fundoplication in the Swedish healthcare setting was conducted using a Markov model and available comprehensive population and clinical trial-based long-term data. Benefits were measured in quality-adjusted life-years (QALYs). Uncertainty was determined by deterministic and probabilistic sensitivity analyses. RESULTS: The base case incremental cost-effectiveness ratios (ICERs) for RefluxStop in comparison to PPIs and Nissen fundoplications were SEK 48,152 (€ 4,531) and SEK 62,966 (€ 5,925) per QALY gained, respectively. At a cost-effectiveness threshold of SEK 500,000 per QALY gained, RefluxStop has a high likelihood of being cost-effective, with probabilities of 96% and 100% against Nissen fundoplication and PPIs, respectively. The results of the model remained robust with sensitivity analysis. CONCLUSIONS: RefluxStop may offer a highly cost-effective long-term treatment alternative for chronic GERD patients over lifelong PPI therapy, but also in comparison with laparoscopic Nissen fundoplication.

Gastroesophageal reflux disease (GERD) is typically managed by proton pump inhibitor (PPI)-based medical management or antireflux surgery (i.e. Nissen fundoplication) in selected cases. However, alternative endoscopic and minimally invasive surgical alternatives have emerged. RefluxStop is a novel implantable device that aims to treat chronic GERD. We performed a cost-effectiveness analysis of RefluxStop from the perspective of the Swedish healthcare system including available treatment options in Sweden (i.e. PPI therapy and Nissen fundoplication). Benefits were measured using quality-adjusted life-years (QALYs). At the cost-effectiveness threshold of SEK 500,000 per QALY gained for Sweden, RefluxStop demonstrated a high likelihood of cost-effectiveness, with probabilities of 96% and 100% against Nissen fundoplication and PPI therapy, respectively. RefluxStop is likely to be a highly cost-effective long-term treatment alternative for chronic GERD patients as compared to lifelong PPI therapy and laparoscopic Nissen fundoplication.

Cost-effectiveness of Procalcitonin (PCT) guidance for antibiotics management of adult sepsis patients in the Egyptian context.

BACKGROUND: Sepsis, which is described as a life-threatening organ malfunction brought on by an unbalanced host response to infection, continues to be a significant healthcare issue that affects millions of individuals each year. It is well-known that sepsis can affect anyone around the world, but the employed survey results showed that there are significant regional variations in sepsis incidence as well as mortality rates. Although there are no definite estimates for Egypt, the highest rates were in Low-Middle-Income Countries (LMICs). Procalcitonin (PCT) is a host response marker with high specificity for bacterial infections, unlike C-reactive protein (CRP) or white blood cell count (WBC), which represent the traditional methods of detecting inflammation and infection. Its dynamic profile and superior prognostic prediction make it invaluable for assessing response to antibiotic treatment and improving clinical care for sepsis patients. Our main purpose was to evaluate the cost-effectiveness of PCT guidance compared to no PCT guidance in the antibiotic management of adult sepsis patients according to the Egyptian context. METHODS: We developed a decision tree model to compare the PCT-guided antibiotic management duration endpoint versus the conventional laboratory culture-based antibiotic management in adult sepsis patients. We employed the"Delphi technique" to reach a satisfactory consensus regarding the resources attributed to each compared alternative. The primary measure of the study was the additional cost associated with each Quality-Adjusted Life Year (QALY) gained by sepsis survivors over a one-year time horizon. Base-case, deterministic and probabilistic sensitivity analyses were conducted using TreeAge, Software. RESULTS: Base-case analysis showed no dominance for either alternative and resulted in an Incremental Cost-Effectiveness Ratio (ICER) value of 297,783.57 Egyptian Pounds per Quality Adjusted Life Year (L.E/QALY) in favor of the PCT guidance alternative, Deterministic sensitivity analysis revealed that the highest impact magnitudes on ICER reside with seven input parameters, the top two parameters that had the most significant influence were the costs of ICU stay with and without PCT guidance. The CEAC showed a slightly higher probability in terms of acceptability in favor of the no PCT guidance choice along the WTP scale till reaching equal probabilities at the willingness-to-pay (WTP) value point of 390,000 (state currency) after which the - probability supports the PCT guidance choice. CONCLUSIONS: In the Egyptian context, PCT guidance has no cost-effectiveness domination over no PCT guidance in Antibiotics management for adult sepsis patients. This may be attributed to the high cost of PCT investigation that shall be resolved by standardization of its cost when applying the approach of DRG cost packages.

Cost and Cost-Effectiveness of Distributing HIV Self-Tests within Assisted Partner Services in Western Kenya.

Background: Assisted partner services (APS) is a recommended public health approach to promote HIV testing for sexual partners of individuals diagnosed with HIV. We evaluated the cost and cost-effectiveness of integrating oral HIV self-testing (HIVST) into existing APS programs. Methods: Within the APS-HIVST study conducted in western Kenya (2021-2022), we conducted micro-costing, time-and-motion, and provider surveys to determine incremental HIVST distribution cost (2022 USD). Using a decision tree model, we estimated the incremental cost per new diagnosis (ICND) for HIVST incorporated into APS, compared to APS with provider-delivered testing only. Scenario, parameter and probabilistic sensitivity analyses were conducted to explore influential assumptions. Results: The cost per HIVST distributed within APS was USD 8.97, largest component costs were testing supplies (38%) and personnel (30%). Under conditions of a facility-based testing uptake of <91%, or HIVST utilization rates of <27%, HIVST integration into APS is potentially cost-effective. At a willing-to-pay threshold of USD 1000, the net monetary benefit was sensitive to the effectiveness of HIVST in increasing testing rates, phone call rates, HIVST sensitivity, HIV prevalence, cost of HIVST, space allocation at facilities, and personnel time during facility-based testing. In a best-case scenario, the HIVST option was cheaper by USD 3037 and diagnosed 11 more cases (ICND = 265.82). Conclusions: Implementers and policy makers should ensure that HIVST programs are implemented under conditions that guarantee efficiency by focusing on facilities with low uptake for provider-delivered facility-based testing, while deliberately targeting HIVST utilization among the few likely to benefit from remote testing. Additional measures should focus on minimizing costs relating to personnel and testing supplies.

A Randomized Controlled Trial Comparing Loss versus Gain Incentives to Improve Adherence to an Obesity Treatment Intervention in Adolescents.

BACKGROUND/OBJECTIVES: Adherence to pediatric obesity treatment can be challenging. Monetary incentives improve adherence to lifestyle interventions, with incentives framed as loss often more effective than those framed as gain. The objectives of this study were to determine if monetary incentives in the form of gift cards would improve adherence to an obesity treatment intervention and whether framing the incentive as either loss or gain affected adherence. METHODS: Sixty adolescents with obesity (body mass index of ≥95th percentile for age and sex) were recruited from our pediatric obesity treatment program. They were randomized into one of three groups and given a monthly adherence score (AS) of up to 100 points. These points were based on completing a medical visit, reporting on diet intake, and measuring daily steps on a wearable tracker. The Gain Group (GG), N = 20, started each month with USD 0 in a virtual account and increased their monetary reward up to USD 100 depending on AS. The Loss Group (LG), N = 21, began each month with USD 100 in their virtual account, which decreased based on adherence. The Control Group (CG), N = 19, received USD 10 monthly. RESULTS: Adherence was highest in the GG, with 66.0 points, compared to the LG, with 54.9 points, and CG, with 40.6 points, with p < 0.01. The GG had greater adherence to their step goal (14.6) and dietary reporting (18.7) compared to the LG (10.0 and 13.9) and the CG (3.9 and 8.1), p < 0.005. CONCLUSIONS: Gain-framed incentives are superior to loss-framed ones in improving adherence to pediatric obesity treatments.