[Cost-effectiveness analysis in the treatment of hematologic malignancies].

Recent advancements in treatment have improved the prognosis of hematologic malignancies. However, the increasing cost of therapeutic drugs has become an urgent issue. Cost-effectiveness analysis is performed using the incremental cost-effective ratio (ICER), a value calculated by dividing the incremental cost by the incremental quality-adjusted life year (QALY). The ICER must be compared with the willingness-to-pay (WTP) threshold, which differs between countries. Since the analysis should be made over a long time horizon, it is necessary to model and extrapolate the long-term outcomes of clinical trials to calculate cumulative costs and QALYs. This article discusses several approaches to cost-effectiveness analysis for chronic myelogenous leukemia, multiple myeloma, and CAR-T therapy. As even expensive treatments could be cost-effective if they provide long treatment-free survival, it is essential to judge cost-effectiveness by an appropriate method, rather than price alone.

Cost effectiveness of a vascular access education and training program for hospitalized emergency department patients.

OBJECTIVE: Education and training in vascular access is a critical component to delivering quality vascular access care. Given that organizations must invest resources to implement and sustain high-quality vascular access programming, we aimed to demonstrate the cost effectiveness of a program (Operation STICK (OSTICK)) in the emergency department (ED). METHODS: This was an observational cohort study conducted at a tertiary care academic center with 120,000 ED visits. Consecutive hospitalized adults with ultrasound-guided (DIVA) and traditionally-placed (non-DIVA) peripheral intravenous catheters (PIVC) in the ED were included in the analysis. Two groups (OSTICK and non-OSTICK) were compared in the analysis: OSTICK PIVCs were inserted by clinicians with formal, standardized training in peripheral venous access while non-OSTICK PIVCs were inserted by staff with basic departmental training in PIVC care. Cost factors included number of procedures, wait time to establish a PIVC, complications, and training. Effect was complication-free PIVC functionality. Multiple linear regressions were used to estimate incremental cost (ΔC), incremental effect (ΔE), and incremental net benefit (INB) of the OSTICK program. RESULTS: From 10/1/2022 thru 3/31/2023, 21,259 PIVCs including 1681 OSTICK and 19,578 non-OSTICK PIVCs were included in the analysis. Average age was 64.8 and 53.7% were female. The estimate of incremental cost (ΔC) for each patient was -$83.175 (95% CI: -$103.953 to -$62.398; p<0.001), indicating that the OSTICK group saves money compared to the non-OSTICK group. The OSTICK group is also more effective at increasing the proportion of catheter dwell time relative to hospital length of stay (ΔE), with an estimate of 0.037 (95% CI: 0.016 to 0.059; p<0.001), compared to those in the non-OSTICK group. The estimated incremental cost-effectiveness ratio (ICER) for the OSTICK group compared with the non-OSTICK group was -$221.964 (95% CI: -$177.400 to -$381.716) per ten percentage points of PIVC dwell time to hospital length of stay increase. CONCLUSIONS: Strategic investment in vascular access education and training can yield impressive financial returns while simultaneously enhancing vascular access outcomes. It is imperative for organizations to recognize the significant impact of such initiatives and prioritize the implementation of comprehensive programs.

MRI software and cognitive fusion biopsies in people with suspected prostate cancer: a systematic review, network meta-analysis and cost-effectiveness analysis.

Background: Magnetic resonance imaging localises cancer in the prostate, allowing for a targeted biopsy with or without transrectal ultrasound-guided systematic biopsy. Targeted biopsy methods include cognitive fusion, where prostate lesions suspicious on magnetic resonance imaging are targeted visually during live ultrasound, and software fusion, where computer software overlays the magnetic resonance imaging image onto the ultrasound in real time. The effectiveness and cost-effectiveness of software fusion technologies compared with cognitive fusion biopsy are uncertain. Objectives: To assess the clinical and cost-effectiveness of software fusion biopsy technologies in people with suspected localised and locally advanced prostate cancer. A systematic review was conducted to evaluate the diagnostic accuracy, clinical efficacy and practical implementation of nine software fusion devices compared to cognitive fusion biopsies, and with each other, in people with suspected prostate cancer. Comprehensive searches including MEDLINE, and Embase were conducted up to August 2022 to identify studies which compared software fusion and cognitive fusion biopsies in people with suspected prostate cancer. Risk of bias was assessed with quality assessment of diagnostic accuracy studies-comparative tool. A network meta-analysis comparing software and cognitive fusion with or without concomitant systematic biopsy, and systematic biopsy alone was conducted. Additional outcomes, including safety and usability, were synthesised narratively. A de novo decision model was developed to estimate the cost-effectiveness of targeted software fusion biopsy relative to cognitive fusion biopsy with or without concomitant systematic biopsy for prostate cancer identification in biopsy-naive people. Scenario analyses were undertaken to explore the robustness of the results to variation in the model data sources and alternative assumptions. Results: Twenty-three studies (3773 patients with software fusion, 2154 cognitive fusion) were included, of which 13 informed the main meta-analyses. Evidence was available for seven of the nine fusion devices specified in the protocol and at high risk of bias. The meta-analyses show that patients undergoing software fusion biopsy may have: (1) a lower probability of being classified as not having cancer, (2) similar probability of being classified as having non-clinically significant cancer (International Society of Urological Pathology grade 1) and (3) higher probability of being classified at higher International Society of Urological Pathology grades, particularly International Society of Urological Pathology 2. Similar results were obtained when comparing between same biopsy methods where both were combined with systematic biopsy. Evidence was insufficient to conclude whether any individual devices were superior to cognitive fusion, or whether some software fusion technologies were superior to others. Uncertainty in the relative diagnostic accuracy of software fusion versus cognitive fusion reduce the strength of any statements on its cost-effectiveness. The economic analysis suggests incremental cost-effectiveness ratios for software fusion biopsy versus cognitive fusion are within the bounds of cost-effectiveness (£1826 and £5623 per additional quality-adjusted life-year with or with concomitant systematic biopsy, respectively), but this finding needs cautious interpretation. Limitations: There was insufficient evidence to explore the impact of effect modifiers. Conclusions: Software fusion biopsies may be associated with increased cancer detection in relation to cognitive fusion biopsies, but the evidence is at high risk of bias. Sufficiently powered, high-quality studies are required. Cost-effectiveness results should be interpreted with caution given the limitations of the diagnostic accuracy evidence. Study registration: This trial is registered as PROSPERO CRD42022329259. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis programme (NIHR award ref: 135477) and is published in full in Health Technology Assessment; Vol. 28, No. 61. See the NIHR Funding and Awards website for further information.

Men with an magnetic resonance imaging scan that shows possible prostate cancer (PCa) are offered prostate biopsies, where samples of the prostate tissue are collected with a needle, to confirm the presence and severity of cancer. Different biopsy methods exist. In a cognitive fusion biopsy, clinicians will target abnormal looking parts of the prostate by looking at the magnetic resonance imaging scan alongside 'live' ultrasound images. During a software fusion (SF) biopsy, a computer software is used to overlay the magnetic resonance imaging scan onto the ultrasound image. This study evaluated whether SF is better at detecting cancer compared with cognitive fusion biopsy, and whether it represents value for money for the National Health Service. We did a comprehensive review of the literature. We combined and re-analysed the evidence, and assessed its quality. We investigated whether SF biopsies are sufficient value for money. Compared with cognitive fusion, patients receiving a SF biopsy may have: (1) a lower probability of having a 'no cancer' result, (2) similar probability of having a benign, non-clinically significant (CS) cancer result and (3) higher probability of detecting CS cancer. However, it is uncertain to what extent SF is more accurate than cognitive fusion, because of concerns about the quality of the evidence. We found no evidence that any SF devices were superior to others. Using additional, random biopsies alongside software or cognitive fusion would increase the detection of PCa. We also looked for evidence on the value for money of the SF biopsies to detect PCa and found no relevant studies. We weighed the costs and the benefits of SF biopsy compared to cognitive fusion to determine whether it could be a good use of National Health Service money. The poor quality of information makes the value of the technologies largely unknown.

Cost-effectiveness analysis of polymyxin B versus colistin for treating patients with carbapenem-resistant gram-negative bacterial infections.

The prevalence of carbapenem-resistant gram-negative bacterial (CRGNB) infection is continuously increasing, and polymyxin B and colistin are considered last-resort drugs. This study compared the cost-effectiveness of polymyxin B with that of colistin for the treatment of intensive care unit patients with CRGNB infection from the Chinese healthcare perspective. A decision-analytic Markov model was constructed to assess the cost-effectiveness of polymyxin B compared with colistin over a period of 5 years using evidence from phase trials and other publicly available studies. The model was developed in Treeage Pro 2022 and comprises a decision tree depicting initial hospitalization and a Markov model with four states projecting long-term health and economic impacts following discharge. Uncertainty was tested with one-way sensitivity analyses and probabilistic sensitivity analyses. The quality-adjusted life years (QALYs), direct medical costs, and incremental cost-effectiveness ratio (ICER) were estimated at willingness-to-pay (WTP) thresholds of $12,674 to $38,024 per QALY. According to the base analyses, the cost incurred by patients receiving colistin treatment was $12,244.77, leading to a gain of 1.35 QALYs. In contrast, patients treated with polymyxin B had a lower cost of $7,306.71 but yielded 1.07 QALYs. The ICRE of colistin was $18032.25/QALY. At a $12,674/QALY threshold, the results were sensitive to several variables, including the probability of cure with polymyxin B, the cost of drugs, the utility of discharge to home, the utility of discharge to long-term care, and the cost of nephrotoxicity with renal replacement therapy. After all model inputs varied across a wide range of reasonable values, only the probability of being cured with polymyxin B resulted in an ICER above the $38,024/QALY threshold. According to the probabilistic sensitivity analyses, colistin was the optimal strategy in 38.2% and 62.8% of the simulations, at $12,674/QALY and $38,024/QALY, respectively. Our study findings suggest that, when considering the Chinese healthcare perspective, colistin is likely to be more cost-effective than polymyxin B for patients with CRGNB infection, especially when the WTP threshold is set at one-time the per capita GDP. However, as the WTP threshold increases from one to three times the per capita GDP, the cost-effectiveness acceptability of colistin improves, increasing from 38.2 to 62.8%.

Deprescribing of proton pump inhibitors in older patients: A cost-effectiveness analysis.

Over-prescribing of proton-pump inhibitors (PPIs) is widely observed in older patients. Clinical findings have showed that deprescribing service significantly decreased inappropriate PPIs utilization. We aimed to examine the cost-effectiveness of PPI deprescribing service from the perspective of Hong Kong public healthcare provider. A decision-analytic model was constructed to examine the clinical and economic outcomes of PPI deprescribing service (deprescribing group) and usual care (UC group) in a hypothetical cohort of older PPI-users aged ≥65 years in the ambulatory care setting. The model inputs were retrieved from literature and public data. The model time-frame was one-year. Base-case analysis and sensitivity analysis were performed. Primary model outcomes were direct medical cost and quality-adjusted life-years (QALYs) loss. In base-case analysis, the deprescribing service (versus UC) reduced total direct medical cost by USD235 and saved 0.0249 QALY per PPI user evaluated. The base-case results were robust to variation of all model inputs in one-way sensitivity analysis. In probabilistic sensitivity analysis, the deprescribing group was accepted as cost-effective (versus the UC group) in 100% of the 10,000 Monte Carlo simulations. In conclusion, the PPI deprescribing service saved QALYs and reduced total direct medical cost in older PPIs users, and showed a high probability to be accepted as the cost-effective option from the perspective of public healthcare provider in Hong Kong.

Lenvatinib plus pembrolizumab for untreated advanced renal cell carcinoma: a systematic review and cost-effectiveness analysis.

Background: Renal cell carcinoma is the most common type of kidney cancer, comprising approximately 85% of all renal malignancies. Patients with advanced renal cell carcinoma are the focus of this National Institute for Health and Care Excellence multiple technology appraisal. A patient's risk of disease progression depends on a number of prognostic risk factors; patients are categorised as having intermediate/poor risk or favourable risk of disease progression. Objectives: The objectives of this multiple technology appraisal were to appraise the clinical effectiveness and cost-effectiveness of lenvatinib plus pembrolizumab versus relevant comparators listed in the final scope issued by the National Institute for Health and Care Excellence: sunitinib, pazopanib, tivozanib, cabozantinib and nivolumab plus ipilimumab. Methods: The assessment group carried out clinical and economic systematic reviews and assessed the clinical and cost-effectiveness evidence submitted by Eisai, Hatfield, Hertfordshire, UK (the manufacturer of lenvatinib) and Merck Sharp & Dohme, Whitehouse Station, NJ, USA (the manufacturer of pembrolizumab). The assessment group carried out fixed-effects network meta-analyses using a Bayesian framework to generate evidence for clinical effectiveness. As convergence issues occurred due to sparse data, random-effects network meta-analysis results were unusable. The assessment group did not develop a de novo economic model, but instead modified the partitioned survival model provided by Merck Sharp & Dohme. Results: The assessment group clinical systematic review identified one relevant randomised controlled trial (CLEAR trial). The CLEAR trial is a good-quality, phase III, multicentre, open-label trial that provided evidence for the efficacy and safety of lenvatinib plus pembrolizumab compared with sunitinib. The assessment group progression-free survival network meta-analysis results for all three risk groups should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons owing to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. The assessment group overall survival network meta-analysis results for the intermediate-/poor-risk subgroup suggested that there was a numerical, but not statistically significant, improvement in the overall survival for patients treated with lenvatinib plus pembrolizumab compared with patients treated with cabozantinib or nivolumab plus ipilimumab. Because of within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption, the assessment group overall survival network meta-analysis results for the favourable-risk subgroup and the all-risk population should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons. Only one cost-effectiveness study was included in the assessment group review of cost-effectiveness evidence. The study was limited to the all-risk population, undertaken from the perspective of the US healthcare system and included comparators that are not recommended by the National Institute for Health and Care Excellence for patients with untreated advanced renal cell carcinoma. Therefore, the extent to which resource use and results are generalisable to the NHS is unclear. The assessment group cost-effectiveness results from the modified partitioned survival model focused on the intermediate-/poor-risk and favourable-risk subgroups. The assessment group cost-effectiveness results, generated using list prices for all drugs, showed that, for all comparisons in the favourable-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated fewer benefits than all other treatments available to NHS patients. For the intermediate-/poor-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated more benefits than treatment with cabozantinib and nivolumab plus ipilimumab. Conclusions: Good-quality clinical effectiveness evidence for the comparison of lenvatinib plus pembrolizumab with sunitinib is available from the CLEAR trial. For most of the assessment group Bayesian hazard ratio network meta-analysis comparisons, it is difficult to reach conclusions due to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. However, the data (clinical effectiveness and cost-effectiveness) used to populate the economic model are relevant to NHS clinical practice and can be used to inform National Institute for Health and Care Excellence decision-making. The assessment group cost-effectiveness results, generated using list prices for all drugs, show that lenvatinib plus pembrolizumab is less cost-effective than all other treatment options. Study registration: This study is registered as PROSPERO CRD4202128587. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis Programme (NIHR award ref: NIHR134985) and is published in full in Health Technology Assessment; Vol. 28, No. 49. See the NIHR Funding and Awards website for further award information.

Renal cell carcinoma is the most common type of kidney cancer. Several drug treatment options are available for NHS patients with advanced or metastatic disease, and the choice of treatment varies depending on a patient's risk of disease progression. A new drug combination, lenvatinib plus pembrolizumab, may soon become available to treat NHS patients. This review explored whether treatment with lenvatinib plus pembrolizumab offered value for money to the NHS. We reviewed the effectiveness of treatment with lenvatinib plus pembrolizumab versus other NHS treatment options. We also estimated the costs and benefits of treatment with lenvatinib plus pembrolizumab versus current NHS treatments for patients with higher and lower risks of disease progression. Compared with current NHS treatments, treatment with lenvatinib plus pembrolizumab may increase the time that people with a higher risk of disease progression (i.e. worsening disease) were alive. However, for patients with a lower risk of disease progression, the available evidence is limited and only shows that treatment with lenvatinib plus pembrolizumab may prolong the time that patients have a stable level of disease. For all patients, compared to all current NHS treatments, treatment with lenvatinib plus pembrolizumab is very expensive. Compared with current NHS treatments for untreated renal cell carcinoma, using published prices (which do not include any discounts that are offered to the NHS), treatment with lenvatinib plus pembrolizumab may not provide good value for money to the NHS.

Cost-effectiveness analysis of a lung cancer screening program in the netherlands: a simulation based on NELSON and NLST study outcomes.

BACKGROUND: In the Netherlands, lung cancer is the leading cause of cancer-related death, accounting for more than 10,000 annual deaths. Lung cancer screening (LCS) studies using low-dose computed tomography (LDCT) have demonstrated that early detection reduces lung cancer mortality. However, no LCS program has been implemented yet in the Netherlands. A national LCS program has the potential to enhance the health outcomes for lung cancer patients in the Netherlands. OBJECTIVE AND METHODS: This study evaluates the cost-effectiveness of LCS compared to no-screening in the Netherlands, by simulating the screening outcomes based on data from NEderlands-Leuvens Longkanker Screenings ONderzoek (NELSON) and National Lung Screening Trial (NLST). We simulated annual screening up to 74 years of age, using inclusion criteria from the respective studies. A decision tree and Markov model was used to predict the incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICERs) for the screening population. The analysis used a lifetime horizon and a societal perspective. RESULTS: Compared to no-screening, LCS resulted in an ICER of €5,169 per QALY for the NELSON simulation, and an ICER of €17,119 per QALY for the NLST simulation. The screening costs were highly impactful for the cost-effectiveness. The most influential parameter was the CT scan cost. Cost reduction for CT from €201 to €101 per scan would reduce the ICER to €2,335 using NELSON criteria. Additionally, LCS could prevent 15,115 and 12,611 premature lung cancer deaths, accompanied by 1.66 and 1.31 QALYs gained per lung cancer case for the NELSON and NLST simulations, respectively. CONCLUSION: LCS was estimated to be cost-effective in the Netherlands for both simulations at a willingness-to-pay threshold of €20,000 per QALY. Using the NELSON criteria, less than €5,500 per QALY had to be spent. Lowering the cost per CT exam would lead to a further reduction of this amount.

The effects and safety of testosterone replacement therapy for men with hypogonadism: the TestES evidence synthesis and economic evaluation.

Background: Low levels of testosterone cause male hypogonadism, which is associated with sexual dysfunction, tiredness and reduced muscle strength and quality of life. Testosterone replacement therapy is commonly used for ameliorating symptoms of male hypogonadism, but there is uncertainty about the magnitude of its effects and its cardiovascular and cerebrovascular safety. Aims of the research: The primary aim was to evaluate the safety of testosterone replacement therapy. We also assessed the clinical and cost-effectiveness of testosterone replacement therapy for men with male hypogonadism, and the existing qualitative evidence on men's experience and acceptability of testosterone replacement therapy. Design: Evidence synthesis and individual participant data meta-analysis of effectiveness and safety, qualitative evidence synthesis and model-based cost-utility analysis. Data sources: Major electronic databases were searched from 1992 to February 2021 and were restricted to English-language publications. Methods: We conducted a systematic review with meta-analysis of individual participant data according to current methodological standards. Evidence was considered from placebo-controlled randomised controlled trials assessing the effects of any formulation of testosterone replacement therapy in men with male hypogonadism. Primary outcomes were mortality and cardiovascular and cerebrovascular events. Data were extracted by one reviewer and cross-checked by a second reviewer. The risk of bias was assessed using the Cochrane Risk of Bias tool. We performed one-stage meta-analyses using the acquired individual participant data and two-stage meta-analyses to integrate the individual participant data with data extracted from eligible studies that did not provide individual participant data. A decision-analytic Markov model was developed to evaluate the cost per quality-adjusted life-years of the use of testosterone replacement therapy in cohorts of patients of different starting ages. Results: We identified 35 trials (5601 randomised participants). Of these, 17 trials (3431 participants) provided individual participant data. There were too few deaths to assess mortality. There was no difference between the testosterone replacement therapy group (120/1601, 7.5%) and placebo group (110/1519, 7.2%) in the incidence of cardiovascular and/or cerebrovascular events (13 studies, odds ratio 1.07, 95% confidence interval 0.81 to 1.42; p = 0.62). Testosterone replacement therapy improved quality of life and sexual function in almost all patient subgroups. In the testosterone replacement therapy group, serum testosterone was higher while serum cholesterol, triglycerides, haemoglobin and haematocrit were all lower. We identified several themes from five qualitative studies showing how symptoms of low testosterone affect men's lives and their experience of treatment. The cost-effectiveness of testosterone replacement therapy was dependent on whether uncertain effects on all-cause mortality were included in the model, and on the approach used to estimate the health state utility increment associated with testosterone replacement therapy, which might have been driven by improvements in symptoms such as sexual dysfunction and low mood. Limitations: A meaningful evaluation of mortality was hampered by the limited number of defined events. Definition and reporting of cardiovascular and cerebrovascular events and methods for testosterone measurement varied across trials. Conclusions: Our findings do not support a relationship between testosterone replacement therapy and cardiovascular/cerebrovascular events in the short-to-medium term. Testosterone replacement therapy improves sexual function and quality of life without adverse effects on blood pressure, serum lipids or glycaemic markers. Future work: Rigorous long-term evidence assessing the safety of testosterone replacement therapy and subgroups most benefiting from treatment is needed. Study registration: The study is registered as PROSPERO CRD42018111005. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/68/01) and is published in full in Health Technology Assessment; Vol. 28, No. 43. See the NIHR Funding and Awards website for further award information.

Testosterone is a hormone which is vital for sexual activity, bone growth and muscle development in men. Men with low testosterone levels may experience problems with erections and may suffer from brittle bones (osteoporosis), weakness, feeling down (low mood) and tiredness. The manifestations of low testosterone can be treated with testosterone replacement therapy. However, there is current uncertainty about the positive effects of testosterone replacement therapy and its safety. We brought together results from all available medical studies that looked at the use of testosterone replacement therapy in men with low testosterone and contacted the doctors who led these studies to gather further information on their participants. We found 35 studies (5601 participants) conducted in different countries, 17 of which provided additional information on their participants. We did not find any evidence to show that testosterone replacement therapy increases the risk of heart problems, or any evidence to show that some men who take testosterone replacement therapy benefit more than others. Men with low testosterone reported having low mood, poor concentration and lack of energy; however, medical studies often failed to prove that these manifestations improved with testosterone replacement therapy. Most medical studies were conducted among white men in North America using questionnaires designed specifically for them; therefore, the results may not reflect the experiences of men in other countries and from more diverse ethnic backgrounds. There is too much uncertainty about the benefits of testosterone replacement therapy to accurately estimate its value for money for the NHS. We think our findings offer some reassurance to doctors and patients that testosterone replacement therapy does not increase the risk of heart problems. New studies are needed to find out whether some groups of men (such as older or younger men) are more likely to benefit from testosterone replacement therapy more than others. It is also important to develop tools which better reflect the experience of men from a diverse range of social and ethnic backgrounds. To inform men with low testosterone about our findings, we are creating a website with dedicated YouTube video clips.

AI for BPH Surgical Decision-Making: Cost Effectiveness and Outcomes.

PURPOSE OF REVIEW: Benign prostatic hyperplasia (BPH) is prevalent in nearly 70% of men over the age of 60, leading to significant clinical challenges due to varying symptom presentations and treatment responses. The decision to undergo surgical intervention is not straightforward; the American Urological Association recommends consideration of surgical treatment after inadequate or failed response to medical therapy. This review explores the role of artificial intelligence (AI), including machine learning and deep learning models, in enhancing the decision-making processes for BPH management. RECENT FINDINGS: AI applications in this space include analysis of non-invasive imaging modalities, such as multiparametric Magnetic Resonance Imaging (MRI) and Ultrasound, which enhance diagnostic precision. AI models also concatenate serum biomarkers and histopathological analysis to distinguish BPH from prostate cancer (PC), offering high accuracy rates. Furthermore, AI aids in predicting patient outcomes post-treatment, supporting personalized medicine, and optimizing therapeutic strategies. AI has demonstrated potential in differentiating BPH from PC through advanced imaging and predictive models, improving diagnostic accuracy, and reducing the need for invasive procedures. Despite promising advancements, challenges remain in integrating AI into clinical workflows, establishing standard evaluation metrics, and achieving cost-effectiveness. Here, we underscore the potential of AI to improve patient outcomes, streamline BPH management, and reduce healthcare costs, especially with continued research and development in this transformative field.

Cost-effectiveness analysis of biologic sequential treatments for moderate-to-severe psoriasis: A Malaysian healthcare system perspective.

OBJECTIVE: In Malaysia, there is now a dearth of recommendations pertaining to the priority of biologic treatments for the effective management of psoriasis, given the multitude of available therapeutic alternatives. Present analysis reports results of a cost-effectiveness model that determines the most optimal arrangement of biologic treatments, with a particular focus of adding biosimilars to the existing treatment pathway for psoriasis in Malaysia. METHODS: A Markov model was developed to compare the cost effectiveness of various biologic sequential treatments in a hypothetical cohort of moderate to severe psoriasis patient in Malaysia over a lifetime horizon. The model simulated the progression of patients through three lines of active biologic therapy, before transitioning to best supportive care. Costs and effects were discounted annually at a rate of 3%. RESULTS: First line secukinumab has produced lowest incremental cost effectiveness ratios (ICERs) when compared to first line systemic [ICERs value; US$152,474 (first set analysis) and US$110,572 (second set analysis)] and first line phototherapy [ICERs value; US$147,057 (first set analysis) and US$107,616 (second set analysis)]. However, these values were slightly higher than the Malaysian based threshold of three times gross domestic product per capita, US$104,337. A 40% reduction in the unit costs of reference biologics renders most of the evaluated treatment sequences cost-effective. CONCLUSION: Adding biosimilar to the current treatment sequence could achieve cost savings ranging from 4.3% to 10.8% without significant loss of effectiveness. Given the significant impact of comorbidities and the resulting decline in quality of life among individuals with psoriasis, it may be justifiable to establish a threshold of up to US$184,000 per quality-adjusted life year (QALY) for the provision of therapies in the context of Malaysia.

Cost-effectiveness analysis of immune checkpoint inhibitors as first-line therapy in advanced biliary tract cancer.

Aim: To assess the cost-effectiveness of immune checkpoint inhibitors as first-line treatments for advanced biliary tract cancer (BTC).Methods: This pharmacoeconomic evaluation employed the fractional polynomial network meta-analysis and partitioned survival model. Costs and utilities were collected from the literature and databases. Sensitivity analyses were used to examine uncertainties.Results: The incremental cost-effectiveness ratios (ICERs) of first-line treatment strategies were $761,371.37 per quality-adjusted life-year (QALY) or $206,222.53/QALY in the US and $354,678.79 /QALY or $213,874.22/QALY in China, respectively. The sensitivity analysis results were largely consistent with the base case.Conclusion: From the US and Chinese payer perspectives, adding durvalumab or pembrolizumab to chemotherapy is unlikely to be cost effective in the first-line setting for advanced BTC.

[Box: see text].

Extended Postoperative Antibiotic Prophylaxis Is Associated with No Clinical Value and Higher Projected Cost Following Adult Spinal Surgery: A Stratified Meta-Analysis and Probability-Based Cost Projections.

¼ We aimed to determine the cost-effectiveness of different protocols of extended postoperative antibiotic prophylaxis (E-PAP) following adult spinal surgery.¼ Both stratified (randomized controlled trials only) and nonstratified (all studies) analyses demonstrated that E-PAP has no significant value in reducing the rate of surgical site infection (SSI), deep SSI, or superficial SSI.¼ Notably, the E-PAP protocols were associated with a significant increase in the length of hospital stay, resulting in an additional expenditure of $244.4 per episode for the E-PAP 72 hours protocol compared with PAP 24 hours and $309.8 per episode for the E-PAP >48 hours protocol compared with PAP <48 hours.¼ E-PAP does not demonstrate any significant reduction in the rate of SSIs following spine surgery. However, these extended protocols were significantly associated with an increase in the length of hospital stay and higher overall projected costs.

Cost-effectiveness analysis of app-delivered self-management support (selfBACK) in addition to usual care for people with low back pain in Denmark.

OBJECTIVES: This study aims to investigate the cost-effectiveness of individually tailored self-management support, delivered via the artificial intelligence-based selfBACK app, as an add-on to usual care for people with low back pain (LBP). DESIGN: Secondary health-economic analysis of the selfBACK randomised controlled trial (RCT) with a 9-month follow-up conducted from a Danish national healthcare perspective (primary scenario) and a societal perspective limited to long-term productivity in the form of long-term absenteeism (secondary scenario). SETTING: Primary care and an outpatient spine clinic in Denmark. PARTICIPANTS: A subset of Danish participants in the selfBACK RCT, including 297 adults with LBP randomised to the intervention (n=148) or the control group (n=149). INTERVENTIONS: App-delivered evidence-based, individually tailored self-management support as an add-on to usual care compared with usual care alone among people with LBP. OUTCOME MEASURES: Costs of healthcare usage and productivity loss, quality-adjusted life-years (QALYs) based on the EuroQol-5L Dimension Questionnaire, meaningful changes in LBP-related disability measured by the Roland-Morris Disability Questionnaire (RMDQ) and the Pain Self-Efficacy Questionnaire (PSEQ), costs (healthcare and productivity loss measured in Euro) and incremental cost-effectiveness ratios (ICERs). RESULTS: The incremental costs were higher for the selfBACK intervention (mean difference €230 (95% CI -136 to 595)), where ICERs showed an increase in costs of €7336 per QALY gained in the intervention group, and €1302 and €1634 for an additional person with minimal important change on the PSEQ and RMDQ score, respectively. At a cost-effectiveness threshold value of €23250, the selfBACK intervention has a 98% probability of being cost-effective. Analysis of productivity loss was very sensitive, which creates uncertainty about the results from a societal perspective limited to long-term productivity. CONCLUSIONS: From a healthcare perspective, the selfBACK intervention is likely to represent a cost-effective treatment for people with LBP. However, including productivity loss introduces uncertainty to the results. TRIAL REGISTRATION NUMBER: NCT03798288.

Cost-effectiveness analysis of COVID-19 booster doses and oral antivirals: Case studies in the Indo-Pacific.

BACKGROUND: Decision-makers in middle-income countries need evidence on the cost-effectiveness of COVID-19 booster doses and oral antivirals to appropriately prioritise these healthcare interventions. METHODS: We used a dynamic transmission model to assess the cost-effectiveness of COVID-19 booster doses and oral antivirals in Fiji, Indonesia, Papua New Guinea, and Timor-Leste. We conducted cost-effectiveness analysis from both healthcare and societal perspectives using data collated from publicly available sources. We developed an interactive R Shiny which allows the user to vary key model assumptions, such as the choice of discounting rate, and view how these assumptions affect model results. FINDINGS: Booster doses were cost saving and therefore cost-effective in all four middle-income settings from both healthcare and societal perspectives using 3% discounting. Providing oral antivirals was cost-effective from a healthcare perspective if procured at a low generic price (US$25) or middle-income reference price (US$250); however, their cost-effectiveness was strongly influenced by rates of wastage or misuse, and the ongoing costs of care for patients hospitalised with COVID-19. The cost or wastage of rapid antigen tests did not appear strongly influential over the cost-effectiveness of oral antivirals in any of the four study settings. CONCLUSIONS: Our results support that COVID-19 booster programs are cost-effective in middle-income settings. Oral antivirals demonstrate the potential to be cost-effective if procured at or below a middle-income reference price of US$250 per schedule. Further research should quantify the rates of wastage or misuse of oral COVID-19 antivirals in middle-income settings.

Hyperthermic intraoperative peritoneal chemotherapy and cytoreductive surgery for people with peritoneal metastases: a systematic review and cost-effectiveness analysis.

Background: We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis. Methods: We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence. Results: The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal cancer but may be cost-effective for the remaining comparisons. Limitations: We were unable to obtain individual participant data as planned. The limited number of randomised controlled trials for each comparison and the paucity of data on health-related quality of life mean that the recommendations may change as new evidence (from trials with a low risk of bias) emerges. Conclusions: In people with peritoneal metastases from colorectal cancer with limited peritoneal metastases and who are likely to withstand major surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should not be used in routine clinical practice (strong recommendation). There is considerable uncertainty as to whether hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy or cytoreductive surgery + systemic chemotherapy should be offered to patients with gastric cancer and peritoneal metastases (no recommendation). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered routinely to women with stage III or greater epithelial ovarian cancer and metastases confined to the abdomen requiring and likely to withstand interval cytoreductive surgery after chemotherapy (strong recommendation). Future work: More randomised controlled trials are necessary. Study registration: This study is registered as PROSPERO CRD42019130504. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.

Cancers of the bowel, ovary or stomach can spread to the lining of the abdomen ('peritoneal metastases'). Chemotherapy (the use of drugs that aim to kill cancer cells) given by injection or tablets ('systemic chemotherapy') is one of the main treatment options. There is uncertainty about whether adding cytoreductive surgery (cytoreductive surgery; an operation to remove the cancer) and 'hyperthermic intraoperative peritoneal chemotherapy' (warm chemotherapy delivered into the lining of the abdomen during cytoreductive surgery) are beneficial. We reviewed all the information from medical literature published until 14 April 2022, to answer the above uncertainty. We found the following from eight trials, including about 1000 participants. In people with peritoneal metastases from bowel cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably does not provide any benefits and increases harm compared to cytoreductive surgery + systemic chemotherapy, while cytoreductive surgery + systemic chemotherapy appears to increase survival compared to systemic chemotherapy alone. There is uncertainty about the best treatment for people with peritoneal metastases from stomach cancer. In women with peritoneal metastases from ovarian cancer who require systemic chemotherapy before cytoreductive surgery to shrink the cancer to allow surgery ('advanced ovarian cancer'), hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably increases survival compared to cytoreductive surgery + systemic chemotherapy. In people who can withstand a major operation and in whom cancer can be removed, cytoreductive surgery + systemic chemotherapy should be offered to people with peritoneal metastases from bowel cancer, while hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered to women with peritoneal metastases from 'advanced ovarian cancer'. Uncertainty in treatment continues for gastric cancer. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.

Comparative study of safflower yellow adjuvant to conventional treatment regimen and conventional treatment regimen on diabetic nephropathy.

Diabetic nephropathy (DN) is one of the most important comorbidities of diabetic patients, which places large physiological and economic burdens on patients. Safflower yellow, a natural pigment extracted from the petals of safflower, has been put into adjuvant therapy. Databases including Chinese National Knowledge Infrastructure (CNKI), Wanfang Database, MEDLINE and etc. will be searched for relevant articles. A meta-analysis was carried out to assess the efficacy and safety of safflower yellow adjuvant to conventional treatment regimen using mean differences (MD) and rate ratios (RR). A cost-effectiveness analysis was also conducted based on the result of meta-analysis. Finally, 28 articles involving 2251 patients were included in meta-analysis. The results showed that compared with conventional treatment, the fasting blood-glucose (FBG) [MD = 0.40], urinary albumin ejection rate (UAER) within 24 h [MD = 48.16], serum creatinine (Scr) [MD = 9.63], blood urea nitrogen (BUN) [MD = 1.73] were significantly lower and the clinical efficacy [RR = 1.28] was more remarkable in safflower yellow adjuvant to conventional treatment group. Our analysis suggested that safflower yellow adjuvant to conventional treatment regimen not only had better clinical efficacy but more cost-effective than conventional treatment regimen.

The Cost Effectiveness of Adjunctive Treatments for Proton Pump Inhibitor-Refractory Gastroesophageal Reflux Disease.

BACKGROUND AND OBJECTIVE: Half of patients with gastroesophageal reflux disease (GERD) experience persistent symptoms while on proton pump inhibitors (PPIs), thus driving efforts to develop novel adjunctive therapies for PPI-refractory GERD. An economic analysis was performed to establish at what cost and efficacy such potential medications are likely to become cost effective in clinical practice. METHODS: A Markov decision model was used to examine a hypothetical cohort of patients being evaluated for PPI-refractory GERD in the USA. The model compared 3 strategies: (1) usual care (i.e., upfront diagnostic testing with upper endoscopy ± ambulatory pH testing); (2) use of a PPI-adjunctive therapy after positive ambulatory pH testing; and (3) empiric use of a PPI-adjunctive therapy (i.e., diagnostic testing only after failing empiric treatment). The primary outcome was incremental cost per quality-adjusted life year (QALY) gained (third-party payer perspective) over a 10-year time horizon using a willingness to pay threshold of $100,000/QALY. RESULTS: In two-way sensitivity analyses varying the cost and effectiveness of the PPI-adjunctive therapy, most combinations revealed that use of the medication after positive pH testing was the most cost-effective approach. Empiric treatment was the preferred strategy only when the therapy was highly efficacious (≥ 87.5% response rate) and low cost (≤ $109/month). Use of PPI-adjunctive treatments were not cost effective when the cost exceeded $1150/month. CONCLUSION: Use of PPI-adjunctive therapies in those with persistent GERD symptoms may become cost effective when guided by ambulatory pH tests. These data can guide investigators, industry, and payers as they develop, validate, and price new treatments for PPI-refractory GERD.

[Priority setting in intensive care from an ethical perspective]. / Prioriteringar i intensivvården ur ett etiskt perspektiv.

Priority setting at intensive care units is legally regulated in accordance with the so-called ethical platform, which states that all priorities must be based on three lexically ranked principles: the principle of human dignity (a ban on discrimination, e.g. based on social standing), the principle of needs and solidarity, and the principle of cost-effectiveness. Prioritization for intensive care is particularly difficult as it requires comparisons between widely different patient categories, occurs in acute situations and is fraught with great uncertainty about the prognosis. Sometimes the degree of severity is maximal for several patients: without treatment, they die. Then treatment effect and cost-effectiveness become more decisive for prioritization decisions. Moreover, withholding and withdrawing intensive care are increasingly considered as morally equivalent. Difficult priority decisions risk moral stress among the intensive care staff.

The need for a broad perspective when assessing value-for-money for out-of-hours primary care.

BACKGROUND: Out-of-hours primary care (OOH-PC) has emerged as a promising solution to improve efficiency, accessibility, and quality of care and to reduce the strain on emergency departments. As this modality gains traction in diverse healthcare settings, it is increasingly important to fully assess its societal value-for-money and conduct thorough process evaluations. However, current economic evaluations mostly emphasise direct- and short-term effect measures, thus lacking a broader societal perspective. AIM: This study offers a comprehensive overview of current effect measures in OOH-PC evaluations and proposes additional measures from the evaluation of integrated care programmes. APPROACH AND DEVELOPMENT: First, we systematically identified the effect measures from published cost-effectiveness studies and classified them as process, outcome, and resource use measures. Second, we elaborate on the incorporation of 'productivity gains', 'health promotion and early intervention', and 'continuity of care' as additional effects into economic evaluations of OOH-PC. Seeking care affects personal and employee time, potentially resulting in decreased productivity. Challenges in taking time off work and limited access to convenient care are often cited as barriers to accessing primary care. As such, OOH-PC can potentially reduce opportunity costs for patients. Furthermore, improving access to healthcare is important in determining whether people receive promotional and preventive services. Health promotion involves empowering people to take control of their health and its determinants. Given the unscheduled nature and the fragmented or rotational care in OOH-PC, the degree to which interventions and modalities provide continuity should be monitored, assessed, and included in economic evaluations. Continuity of care in primary care improves patient satisfaction, promotes adherence to medical advice, reduces reliance on hospitals, and reduces mortality. CONCLUSION: Although it is essential to also address local settings and needs, the integration of broader scope measures into OOH-PC economic evaluations improves the comprehensive evaluation that aligns with welfare gains.