Previous Cost-effectiveness Analyses Research Articles

Determining the cost-effectiveness of follitropin alfa biosimilar compared to follitropin alfa originator in women undergoing fertility treatment in France

ObjectiveThe study assessed cost-effectiveness of follitropin alfa biosimilar versus the originator in terms of cost per cumulative live-birth (CLB) for the French healthcare system based on real-world evidence. Follitropin alfa biosimilars have been shown to have comparable clinical outcomes to the originator, in both clinical studies and real-world settings, in terms of oocyte retrieval and cumulative live-birth rate (CLBR). Previous health economic studies comparing the cost-effectiveness of follitropin alfa biosimilars against the originator utilised clinical trial data, leaving ambiguity over cost-effectiveness in real-world settings. Additionally, previous cost-effectiveness analysis has been performed for live-births following only fresh embryo transfers, whereas, fresh and frozen transfers are common in clinical practice. This study investigates the cost per CLB, which more closely models clinical practice. Study designA decision-tree cost-effectiveness model was developed based on the total costs and CLBR per ovarian stimulation (OS) for a follitropin alfa biosimilar (Bemfola®, Gedeon Richter Plc, Budapest, Hungary) and the originator (Gonal-f®, Merck KGaA, Darmstadt, Germany). A time horizon of one year from oocyte retrieval to embryo transfer was used but costs from resulting transfers were also included. Clinical inputs were taken from the REOLA real-world study or clinician insights, while acquisition costs were taken from French public databases. The output was cost per CLB following one OS. One-way sensitivity analysis was performed to determine the largest model drivers. ResultsCost per CLB was €18,147 with follitropin alfa biosimilar and €18,834 with the originator, saving €687 per CLB following OS with the biosimilar. When wastage estimates were considered the biosimilar cost saving is estimated to be between €796 and €1,155 per CLB further increasing cost savings. Irrespective of wastage, if used ubiquitously throughout France for ART, the biosimilar could save the French health system €13,994,190 or lead to 771 more births when compared to its higher-cost originator. Sensitivity analysis showed that the originator’s relative CLBR had the greatest impact on the model. ConclusionThis analysis demonstrates that the follitropin alfa biosimilar, Bemfola®, is a more cost-effective option for OS compared with the originator from a French healthcare payer perspective, in terms of cost per CLB.

Abstract WP104: Updating Cost-Effectiveness of Stroke Thrombectomy in Patients With Large Core Ischemic Stroke Using 2023 Clinical Trial Data

Introduction: Stroke thrombectomy (ST) is cost-effective for acute ischemic stroke (AIS) patients with large-vessel occlusion and low Alberta Stroke Program Early CT Score (ASPECTS <6) compared with non-endovascular standard care (SC). However, previous cost-effectiveness analyses (CA) were based on data that were not specific to low ASPECTS or had limited generalizability (e.g., data on Japanese patients with ASPECTS 3-5). As new results from two 2023 clinical trials that included patients with ASPECTS <6 emerged from Western countries and China, we aim to update the cost-effectiveness of ST using new data from different populations and assess how ST treatment delay might affect the comparative effectiveness of ST and SC. Methods: We updated the cost-effectiveness of ST compared with SC in AIS patients aged 67 years with ASPECTS <6 based on the data from two trials conducted in Western countries and China using a previously published Markov model that simulates the lifetime cost and quality-adjusted life years (QALYs) with annual cycles. The difference between ST and SC was characterized by the patient distribution of modified Rankin Scales at 90 days after hospital discharge (90-day mRS) as reported from the two trials. We conducted two separate CA at a willingness-to-pay threshold of $100,000/QALY using the 90-day mRS distribution from each trial with the ST treatment delay as observed in the trials (base case). In addition, we assessed how the effectiveness of ST varied with ST treatment delay up to 6 hours in addition to the observed delay in trials. Results: In the base case, irrespective of two trial datasets, ST is a dominant strategy that saved $8,431-$32,083 and nearly 1 QALY with a positive net monetary benefit (NMB) compared to SC, which had a negative NMB. Regarding ST treatment delay, the QALYs generated from ST decreased as additional treatment delay increased. If the additional ST treatment delay was within 2-3.5 hours, ST could generate more QALYs and was still cost-effective compared to SC. Conclusions: Our findings suggest ST is the preferred strategy over SC in AIS patients with low ASPECTS. ST remains effective and cost-effective even when ST is delayed beyond the median transfer time of 174 minutes in the U.S. reported in Stamm et al. JAMA study.

A prospective study of lumbar facet arthroplasty in the treatment of degenerative spondylolisthesis and stenosis: cost-effective assessment from the Total Posterior Spine system (TOPSTM) IDE Study: 2-year model revision and sensitivity analyses based on 305 subjects

BACKGROUND CONTEXTA previous cost-effectiveness analysis published in 2022 found that the Total Posterior Spine (TOPSTM) system was dominant over transforaminal lumbar interbody fusion (TLIF). This analysis required updating to reflect a more complete dataset and pricing considerations. PURPOSETo evaluate the cost-effectiveness of TOPSTM system as compared with TLIF based on an updated and complete FDA investigational device exemption (IDE) data set. STUDY DESIGN/SETTINGCost-utility analysis of the TOPSTM system compared to TLIF. PATIENT SAMPLEA multicenter, FDA IDE, randomized control trial (RCT) investigated the efficacy of TOPSTM compared to TLIF with a current population of n=305 enrolled and n=168 with complete 2-year follow-up. OUTCOME MEASURESCost and quality adjusted life years (QALYs) were calculated to determine our primary outcome measure, the incremental cost-effectiveness ratio. Secondary outcome measures included: net monetary benefit as well at willingness-to-pay (WTP) thresholds. METHODSThe primary outcome of cost-effectiveness is determined by incremental cost-effectiveness ratio. A Markov model was used to simulate the health outcomes and costs of patients undergoing TOPSTM or TLIF over a 2-year period. alternative scenario sensitivity analysis, one-way sensitivity analysis, and probabilistic sensitivity analysis were conducted to assess the robustness of the model results. RESULTSThe updated base case result demonstrated that TOPSTM was immediately and longitudinally dominant compared with the control with an incremental cost-effectiveness ratio of −9,637.37 $/QALY. The net monetary benefit was correspondingly $2,237, both from the health system's perspective and at a WTP threshold of 50,000 $/QALY at the 2-year time point. This remained true in all scenarios tested. The Alternative Scenario Sensitivity Analysis suggested cost-effectiveness irrespective of payer type and surgical setting. To remain cost-effective, the cost difference between TOPSTM and TLIF should be no greater than $1,875 and $3,750 at WTP thresholds of $50,000 and 100,000 $/QALY, respectively. CONCLUSIONSThis updated analysis confirms that the TOPSTM device is a cost-effective and economically dominant surgical treatment option for patients with lumbar stenosis and degenerative spondylolisthesis compared to TLIF in all scenarios examined.

Real-time pulmonary artery pressure monitoring in heart failure patients: an updated cost-effectiveness analysis.

Previous cost-effectiveness analysis suggests that CardioMEMS is cost-effective compared with usual care for patients with persistent New York Heart Association class III symptoms and at least one heart failure (HF) hospitalization within 12months. The aim of the paper is to perform an update of the cost-effectiveness analysis of CardioMEMS using the most recent data from the published literature. A Microsoft Excel Markov model from a previous UK cost-effectiveness study of CardioMEMS was updated using the clinical effectiveness of pulmonary artery pressure (PAP)-guided treatment derived from the pivotal trials. The model included the device costs (and the implantation procedure and related complications), costs of remote monitoring, costs of HF-related hospitalizations, and costs of usual care. Quality-adjusted life years (QALYs) were estimated based on utilities from pivotal trials and published literature. Cost-effectiveness results were estimated as incremental cost per QALY gained of CardioMEMS compared with usual care. Scenario analyses were also performed using data from real-world studies that showed a significant decrease in HF-related hospitalizations. In the base case analysis over a time horizon of 10years, PAP-guided HF therapy increased cost compared with usual care by £6337 (i.e. from £22770 in usual care to £29107 in PAP-guided HF therapy) and the QALYs per patient for usual care and PAP-guided patients were 2.62 and 2.94, respectively, reflecting an increase of 0.32 QALYs with PAP-guided treatment. The resultant incremental cost-effectiveness ratio (ICER), the ratio between incremental costs and the QALYs, is estimated at £19761/QALY. Scenario analyses suggest that the ICER for CardioMEMS can range from being dominant to £27910/QALY. Probabilistic sensitivity analyses suggested that PAP-guided HF therapy has 81.9% probability of being cost-effective at a threshold of £30000/QALY. Our model suggests that CardioMEMS is likely to be cost-effective in the United Kingdom, at the currently considered thresholds of £20000-30000/QALY.

EE248 Patient-Informed Value Elements for Economic Evaluation of Treatment for Major Depressive Disorder: A Systematic Review and Synthesis

Our recent study in individuals with major depressive disorder (MDD) elicited six prioritized attributes in selecting treatments to manage their condition: Treatment Mode, Time to Treatment Effect, MDD Relief, Quality of Work, Interaction with Others, and Affordability. This study aims to identify whether previous cost-effectiveness analyses (CEAs) in MDD addressed these patient-important attributes, whether patients were engaged in the modeling process, and whether patient preferences for treatment attributes were incorporated into the economic evaluations.

EE197 Cost-Effectiveness of Monoclonal Antibodies (MABs) for Rheumatoid Arthritis (RA) in the United States

In the last 2 decades, biologics have changed the treatment of RA. The 9 approved MABs on the market have shown improved outcomes. However, these agents created a massive economic burden among U.S. payers. Current methods of cost-containment relegate costs to patients while obstructing clinicians. Pharmacoeconomic analysis is commonly used by payers in decision-making. However, previous cost-effectiveness analyses (CEAs) present varying results with the use of Quality–Adjusted Life Years (QALYs). Therefore, there is a common necessity for a CEA using clinical endpoints in RA.

EE40 Cost-Effectiveness of GLP-1 Receptor Agonists Versus Long-Acting Insulins in Type 2 Diabetes From the Healthcare Sector Perspective: A Model-Based Analysis Using Real-World Data

Most previous cost-effectiveness analyses (CEAs) of glucagon-like peptide-1 receptor agonists (GLP-1RAs) versus long-acting insulins (LAIs) utilized the effectiveness parameters which were derived from clinical trials and not country/population-specific, and it may thus have limited study generalizability to real-world settings. We conducted a model-based CEA of GLP-1RAs versus LAIs from the Taiwan healthcare sector perspective using data from a real-world Taiwanese cohort of type 2 diabetes (T2D) to inform real-world decision-making.

Economic impact of the first pass effect in mechanical thrombectomy for acute ischaemic stroke treatment in Spain: a cost-effectiveness analysis from the national health system perspective

ObjectiveThe mechanical thrombectomy (MT) benefit is related to the degree of reperfusion achieved. First pass effect (FPE) is defined as complete/near revascularisation of the large-vessel occlusion (modified Thrombolysis in Cerebral...

Usage and cost-effectiveness of elective oocyte freezing: a retrospective observational study

BackgroundThe previous model-based cost-effectiveness analyses regarding elective oocyte cryopreservation remained debatable, while the usage rate may influence the cost per live birth. The aim of this study is to disclose the usage and cost-effectiveness of the planned cryopreserved oocytes after oocyte thawing in real-world situations.MethodsThis was a retrospective single-center observational study. Women who electively cryopreserved oocytes and returned to thaw the oocytes were categorized as thawed group. The oocytes were fertilized at our center and the sperm samples for each individual was retrieved from their respective husbands. Clinical outcomes were traced and the cumulative live birth rate per thawed case was calculated. The costs from oocyte freezing cycles to oocyte thawing, and embryo transfer cycles were accordingly estimated. The cumulative cost per live birth was defined by the cumulative cost divided by the live births per thawed case.ResultsWe recruited 645 women with 840 oocyte retrieval cycles for elective oocyte freezing from November 2002 to December 2020. The overall usage rate was 8.4% (54/645). After the storage duration exceeded ten years, the probabilities of thawing oocytes were 10.6%, 26.6%, and 12.7% from women who cryopreserved their oocytes at the age ≤ 35 years, 36–39 years, and ≥ 40 years, respectively (P = 0.304). Among women who thawed their oocytes, 31.5% (17/54) of women achieved at least one live birth. For the age groups of ≤ 35 years, 36–39 years, and ≥ 40 years, the cumulative live birth rates per thawed case were 63.6%, 42.3%, and 17.6%, respectively (P = 0.045), and the cumulative costs for one live birth were $11,704, $17,189, and $35,642, respectively (P < 0.001).ConclusionsThe overall usage rate was 8.4% in our cohort. The cumulative live birth rate was greatest in the youngest group and the cumulative cost per live birth was highest in the oldest group, which was threefold greater than that in the group aged ≤ 35 years. The findings added to the limited evidence of the usage rate in real-world situations, which could hopefully aid future analysis and decision-making in public health policy and for women willing to preserve fertility.Trial registrationNone.

Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease.

Sickle cell disease (SCD) is a clinically heterogeneous disease with many acute and chronic complications driven by ongoing vaso-occlusion and hemolysis. It causes a disproportionate burden on Black and Hispanic communities. Our objective was to follow the SMDM/ISPOR Task Force recommendations for good practices and create a conceptual model of the progression of SCD under current clinical practice to inform cost-effectiveness analyses (CEA) of promising curative therapies in the pipeline over a lifetime horizon. We used consultations with experts, providers, and patients to identify acute events and chronic conditions in the conceptual model. We compared our model structure to previous CEA models of interventions for SCD, assessed the prevalence of the identified disease attributes in Medicaid and Medicare claims databases, and identified relevant outcomes following the 2nd Panel in CEA. We determined an appropriate modeling technique and relevant data sources for parameterizing the model. The conceptual model structure included four dimensions of disease: chronic pain, acute events, chronic conditions, and treatment complications, spanning 26 disease attributes with significant impacts on health-related quality of life and resource. We modeled chronic pain separately to reflect its importance to patients and interaction with all other disease attributes. We identified additional data sources for health state utilities and non-medical costs and benefits of SCD. We will use a microsimulation model with age- and sex-specific transitions between health states predicted by patient demographic characteristics and disease history. Developing the model structure through an explicit process of model conceptualization can increase the transparency and accuracy of results. We will populate the conceptual model with the data sources described and evaluate the cost-effectiveness of curative therapies.

Real-world cost-effectiveness of first-line gemcitabine + nab-paclitaxel versus FOLFIRINOX in patients with advanced pancreatic cancer: A population-based retrospective cohort study in Ontario, Canada.

529 Background: Currently, there are no direct randomized control trials (RCTs) comparing gemcitabine and nab-paclitaxel (Gem-Nab) and FOLFIRINOX for advanced pancreatic cancer (APC). Thus, previous model-based cost-effectiveness analyses were based on indirect comparisons of RCT data. While it is well known that RCT-based efficacy does not often translate to real-world effectiveness, there is limited literature investigating the comparative cost-effectiveness of Gem-Nab versus FOLFIRINOX for APC in the real-world. The objective of this study is to examine the real-world cost-effectiveness of Gem-Nab versus FOLFIRINOX in patients with APC in Ontario, Canada. Methods: This population-based retrospective cohort study compared all patients treated with first-line Gem-Nab or FOLFIRINOX for APC with ECOG performance status 0-1 in Ontario from April 2015 to March 2019. Patients were linked to administrative databases to identify key characteristics and costing data. Using propensity scores and a stabilizing weights method, an inverse probability of treatment weighted cohort was developed. Mean survival and total costs were calculated over a 5-year time horizon, adjusted for censoring and discounted at 1.5% (per Canadian guidelines). Incremental cost-effectiveness ratio and net monetary benefit were computed (measured in life-years and quality-adjusted life years) to estimate cost-effectiveness from the public healthcare payer’s perspective. A sensitivity analysis was conducted using the propensity score matching method. Results: 1,988 patients were identified (Gem-Nab: 928, FOLFIRINOX: 1,060). Mean survival was lower for patients in the Gem-Nab group than the FOLFIRINOX group (0.98 versus 1.26 life-years, incremental -0.28 (95% confidence interval -0.47, -0.13)). Patients in the Gem-Nab group also incurred greater mean 5-year total costs (Gem-Nab: $103,884, FOLFIRINOX: $101,518). Key cost contributors include ambulatory cancer care, acute in-patient hospitalization, and systemic therapy drug acquisition. Gem-Nab was dominated by FOLFIRINOX, as it is less effective and more costly. Results from the sensitivity analysis were similar. Conclusions: In routinely treated unselected patients, Gem-Nab is likely more costly and less effective than FOLFIRINOX and therefore, not considered cost-effective at any commonly accepted willingness-to-pay threshold.

Economic evaluation of Zika Contraception Access Network in Puerto Rico during the 2016–17 Zika virus outbreak

During the 2016-2017 Zika virus (ZIKV) outbreak, the prevention of unintended pregnancies was identified as a primary strategy to prevent birth defects. This study estimated the cost-effectiveness of the Zika Contraception Access Network (Z-CAN), an emergency response intervention that provided women in Puerto Rico with access to the full range of reversible contraception at no cost and compared results with a preimplementation hypothetical cost-effectiveness analysis (CEA). We evaluated costs and outcomes of Z-CAN from a health sector perspective compared to no intervention using a decision tree model. Number of people served, contraception methods mix, and costs under Z-CAN were from actual program data and other input parameters were from the literature. Health outcome measures included the number of Zika-associated microcephaly (ZAM) cases and unintended pregnancies. The economic benefits of the Z-CAN intervention were ZIKV-associated direct costs avoided, including lifetime medical and supportive costs associated with ZAM cases, costs of monitoring ZIKV-exposed pregnancies and infants born from Zika-virus infected mothers, and the costs of unintended pregnancies prevented during the outbreak as a result of increased contraception use through the Z-CAN intervention. The Z-CAN intervention cost a total of $26.1 million, including costs for the full range of reversible contraceptive methods, contraception related services, and programmatic activities. The program is estimated to have prevented 85% of cases of estimated ZAM cases and unintended pregnancies in the absence of Z-CAN. The intervention cost was projected to have been more than offset by $79.9 million in ZIKV-associated costs avoided, 96% of which were lifetime ZAM-associated costs, as well as $137.0 million from avoided unintended pregnancies, with total net savings in one year of $216.9 million. The results were consistent with the previous CEA study. Z-CAN was likely cost-saving in the context of a public health emergency response setting.

Cost-Effectiveness of Open vs. Endoscopic Repair of Zenker's Diverticulum.

To evaluate the cost-effectiveness of open versus endoscopic surgical repair of Zenker's diverticulum. In this study, an economic decision tree was utilized to compare the cost-effectiveness of open surgery compared to endoscopic surgery. The primary outcome in this analysis was the incremental cost-effectiveness ratio (ICER) that was calculated based on the economic decision tree. The probability of post-operative esophageal perforation complications, revision rates, and effectiveness of each procedure along with associated costs were extracted to construct the decision tree. Univariate sensitivity analysis was then utilized to determine how changes in esophageal perforation rate affect the cost-effectiveness of each surgical approach. The ICER of open surgery for Zenker's diverticulum was $67 877, above most acceptable willingness to pay (WTP) thresholds. Additionally, if the probability of esophageal perforation with endoscopic surgery is above 5%, then open surgery becomes a more cost-effective option. Probabilistic sensitivity analysis using Monte Carlo simulations also showed that at the WTP thresholds of $30 000 and $50 000, endoscopic surgery is the most cost-effective method with 83.9% and 67.6% certainty, respectively. Open surgery and endoscopic surgery are 2 treatment strategies for Zenker's diverticulum that each have their own advantages and disadvantages that can complicate the decision-making process. With no previous cost-effectiveness analysis of open versus endoscopic surgery for Zenker's diverticulum, our results support the endoscopic approach at most common WTP thresholds. Particularly with the current focus on rising healthcare costs, our results can serve as an important adjunct to medical decision-making for patients undergoing treatment for Zenker's diverticulum.

MO682TAILORING AI-BASED REFERRAL TO INTENSIFIED INTERVENTION PROGRAMS FOR PERITONITIS PREVENTION WITH COST-EFFECTIVENESS SIMULATION

Abstract Background and Aims Peritonitis is a common and potentially severe complication for peritoneal dialysis (PD) patients. It is associated with mortality and technique failure risk and contributes significantly to their healthcare cost. Despite several peritonitis prevention programs based on education and training have been implemented worldwide, it has been reported a large variability of efficacy across patients groups and healthcare settings. In order to avoid unnecessary treatment of low risk patients, healthcare prevention programs should be personalized based on accurate patients’ risk profiling, so that high risk patients may be addressed with intensified prevention programs. However, referral strategy (i.e. defining when risk is too much and deserves special attention) depends the availability, efficacy and cost of medical interventions. In this study, we demonstrate through a program implementation simulator, how different referral strategies to inform peritonitis prevention program among PD patients informed by AI-based risk stratification tools, produce different healthcare and health economics outcomes. In particular, the simulation considers a prevention program characterized by standard of care, which affects all patients as well as an intensive intervention for a subset of high-risk patients (e.g. special training or medical treatment). Method The Peritonitis Risk Score model was trained and validated among 9325 PD patients treated in FMC network (Model accuracy, AUC=0.86). The pharmaco-economic model simulation was performed considering a cohort of 22,900 adult PD patients, treated in Fresenius Medical Care dialysis network between January 1, 2011 and December 31, 2018, for which the Peritonitis Risk Score was computed at a given date. The occurrence of an acute peritonitis in the month after prediction has been registered. We simulated the program outcomes in terms of proportion of referrals to the intensified prevention program, false omission rate, peritonitis risk reduction, overall cost-savings, number needed to treat. We considered the following scenario based on previous cost-effectiveness analysis on peritonitis risk prevention: Results Given the action threshold selected, 5.3% of patients entered the intensified intervention program (PPV=9.5%); the false omission rate was 2.2%. Cost savings for the intensified healthcare where generated when the effect size of the intensified intervention exceeded 1.4 (figure 1A). For that effect size the number needed to treat for each prevented peritonitis was NNT=23.4. Overall, 162 peritonitis/month could be prevented in the whole network (peritonitis with no intervention=592; Peritonitis after intervention=430). When a less conservative threshold was selected, 12.2% of patients entered the intensified prevention program (PPV=7.3%), generating a false omission rate=1.9%. Cost savings were never generated (i.e. the intensified program needed investment to be sustained) but with the same effect size of 1.4 additional 24 peritonitis/months could be saved in the whole network (peritonitis with no intervention=592; Peritonitis after intervention=406). The number needed to treat for the intensified program was NTT=30.4 (figure 1B). Conclusion Cost-effectiveness simulating tool provides a rational evaluation framework for AI-based referral to peritonitis preventive programs. This tool can be easily adapted for any healthcare program based on patient risk score.

Cost-Effectiveness Analysis of Smoking Cessation Interventions in the United Kingdom Accounting for Major Neuropsychiatric Adverse Events

ObjectivesSmoking is a leading cause of death worldwide. Cessation aids include varenicline, bupropion, nicotine replacement therapy (NRT), and e-cigarettes at various doses (low, standard and high) and used alone or in combination with each other. Previous cost-effectiveness analyses have not fully accounted for adverse effects nor compared all cessation aids. The objective was to determine the relative cost-effectiveness of cessation aids in the United Kingdom. MethodsAn established Markov cohort model was adapted to incorporate health outcomes and costs due to depression and self-harm associated with cessation aids, alongside other health events. Relative efficacy in terms of abstinence and major adverse neuropsychiatric events was informed by a systematic review and network meta-analysis. Base case results are reported for UK-licensed interventions only. Two sensitivity analyses are reported, one including unlicensed interventions and another comparing all cessation aids but removing the impact of depression and self-harm. The sensitivity of conclusions to model inputs was assessed by calculating the expected value of partial perfect information. ResultsWhen limited to UK-licensed interventions, varenicline standard-dose and NRT standard-dose were most cost-effective. Including unlicensed interventions, e-cigarette low-dose appeared most cost-effective followed by varenicline standard-dose + bupropion standard-dose combined. When the impact of depression and self-harm was excluded, varenicline standard-dose + NRT standard-dose was most cost-effective, followed by varenicline low-dose + NRT standard-dose. ConclusionAlthough found to be most cost-effective, combined therapy is currently unlicensed in the United Kingdom and the safety of e-cigarettes remains uncertain. The value-of-information analysis suggested researchers should continue to investigate the long-term effectiveness and safety outcomes of e-cigarettes in studies with active comparators.

Toward a uniform pathway in managing incidental lung nodules on computerised tomography [CT] coronary angiography; for a cost effective and safe outcome

Abstract Background National Institute for Health and Care Excellence guidelines (NICE CG95) recommends computed tomography coronary angiography [CTCA] as the first line investigation for all patients with suspected typical or atypical angina irrespective of pre-test probability due to its excellent performance and cost effectiveness. However previous cost effectiveness analyses have not factored in the burden of lung nodules [LN] or extracardiac incidentalomas, which are commonly identified on CTCA. Purpose As we increasingly detect LN on CTCA scans, it is prudent that a uniform pathway is followed for surveillance of LN. The British Thoracic Society (BTS) has enabled evidence-based development of an algorithm for the management of LN which defines a cut off LN size of ≥5mm requiring further follow up. We aimed to assess the compliance of LN follow up in our patients according to radiology recommendation on CTCA reports and also to assess how well these recommendations adhere to BTS guidelines. Method 117 patients who underwent CTCA as the initial diagnostic investigation were retrospectively identified from the rapid access chest pain clinic database. Data was collected during a 1 year period between January - December 2017. Data was analysed to determine whether appropriate surveillance pathway was followed for repeat imaging. According to BTS guidelines, nodules ≥5mm should have a systematic work up for surveillance and hence we have stratified our data based on the nodule size. In case of multiple nodules, the largest nodule size was considered. Results Out of 117 patients, 77% patients were female with average age 57.8±9.0 years. 40% patients were found to have incidental LN with an average size of 4.63±1.92mm; 44% had a history of cigarette smoking. Among patient with nodules, 22 (19%) had CT thorax to assess interval change and interestingly 16 (73%) had persisting LN; whereas in 4 (18%), the LN resolved and only 2 (9%) showed increase in LN size. Repeat CT chest was advised for these 2 patients; 1 was advised to undergo biopsy which eventually ruled out malignancy. 6 patients (5%) from group 1 were recommended for repeat CT despite having LN size &amp;lt;5mm. In the remaining 25 patients (21%) with LN, 15 (60%) did not have further surveillance imaging even though this was recommended by the reporting radiologist. Of these only 3 (12%) had LN ≥5mm which qualified for surveillance CT. Conclusion CTCA frequently detects incidentalomas especially LN which cannot be overlooked and require further imaging irrespective of the primary presentation. Our findings also suggest a discrepancy on surveillance CT recommendation against the current BTS guidelines, with potential significant impact on the overall cost effectiveness of CTCA. A systematic approach to LN surveillance could be best achieved with a multidisciplinary team approach (e.g. referral pathway to nodule MDT) and adherence to a standard guideline. Lung nodule surveillance following CTCA Funding Acknowledgement Type of funding source: None

Cost-Effectiveness of 4CMenB Infant Vaccination in England: A Comprehensive Valuation Considering the Broad Impact of Serogroup B Invasive Meningococcal Disease

ObjectivesThis cost-effectiveness analysis (CEA) of 4CMenB infant vaccination in England comprehensively considers the broad burden of serogroup B invasive meningococcal disease (MenB IMD), which has not been considered, or was only partially considered in previous CEAs. MethodsA review of previous MenB vaccination CEAs was conducted to identify aspects considered in the evaluation of costs and health outcomes of the disease burden of MenB IMD. To inform the model structure and comprehensive analysis, the aspects were grouped into 5 categories. A stepwise analysis was conducted to analyze the impact of each category, and the more comprehensive consideration of disease burden, on the incremental cost-effectiveness ratio (ICER). ResultsMenB IMD incidence decreased by 46.0% in infants and children 0-4 years old within 5 years after introduction of the program. Stepwise inclusion of the 5 disease burden categories to a conventional narrow CEA setting reduced the ICER from £360 595 to £18 645—that is, considering the impact of all 5 categories, 4CMenB infant vaccination is cost-effective at a threshold of £20 000 per QALY gained. ConclusionsWhen considering comprehensively the MenB IMD burden, 4CMenB infant vaccination can be cost-effective, a finding contrary to previous CEAs. This analysis allows policy decision-makers globally to infer the impact of current disease burden considerations on the cost-effectiveness and the comprehensive assessment necessary for MenB IMD. Although this comprehensive CEA can help inform decision making today, it may be limited in capturing the full disease burden and complex interactions of health and economics of MenB IMD.

Cost-Effectiveness of Hepatocellular Carcinoma Surveillance: An Assessment of Benefits and Harms.

The value of hepatocellular carcinoma (HCC) surveillance is defined by the balance of benefits, i.e., early tumor detection, and potential harms, related to false positive and indeterminate results. Although physical harms can be observed in 15%-20% of patients with cirrhosis undergoing HCC surveillance, previous cost-effectiveness analyses have not incorporated costs of harms. We aimed to evaluate the cost-effectiveness of HCC surveillance including both benefits and harms. We constructed a Markov model to compare surveillance strategies of ultrasound (US) alone, US and alpha fetoprotein (AFP), and no surveillance in 1 million simulated patients with compensated cirrhosis. Harms included imaging and biopsy in patients undergoing surveillance for HCC. Model inputs were based on literature review, and costs were derived from the Medicare fee schedule, with all costs inflated to 2018 dollars. The primary outcome was the incremental cost-effectiveness ratio per incremental quality-adjusted life-year. In the base case analysis, US with AFP was the dominant strategy over both US alone and no surveillance. In a probabilistic sensitivity analysis, US with AFP was the most cost-effective strategy in 80.1% of simulations at a willingness-to-pay threshold of $100,000 per quality-adjusted life-year. In our threshold analyses, an HCC incidence >0.4% per year and surveillance adherence >19.5% biannually were necessary for US with AFP to be cost-effective compared with no surveillance. Accounting for both surveillance-related benefits and harms, US and AFP is more cost-effective for HCC surveillance than US alone or no surveillance in patients with compensated cirrhosis.

Cost-effectiveness analysis is a mandatory strategy for health systems: evidence from a study involving therapies for hepatitis C.

Cost-effectiveness analysis is essential in health decision making. Several countries use it as synthesis of evidence to incorporate health technologies. The protease inhibitors (PI) boceprevir (BOC) and telaprevir (TVR) are indicated for chronic hepatitis C treatment and were incorporated in guidelines worldwide. Pre-marketing clinical trials showed higher sustained virological response rates in relation to previous therapies, but the incorporation of PIs generated a significant financial impact. The aim of this study was to discuss the relevance of cost-effectiveness analysis through a study that involved the inclusion of PIs in a clinical protocol. The analysis was part of a real-life study that included patients infected with hepatitis C virus genotype 1 treated in a tertiary university hospital in Brazil. Triple therapies (TT) with ribavirin (RBV), peginterferon α-2a (Peg-INF α-2a) and BOC or TVR were compared to dual therapy with RBV and Peg-INF α-2a. Sensitivity analysis of the cost-effectiveness ratio indicated an 88.2% chance of TTs presenting a higher cost per cure. The incremental cost-effectiveness ratios (ICER) exceeded the Brazilian gross domestic product (GDP) per capita by three times in all proposed scenarios. The sensitivity of ICER showed an 88.4% chance of TT not being cost-effective. The impact of PI incorporation was negative and the conduct about this could have been different if a previous cost-effectiveness analysis had been conducted.

An economic analysis of different treatments for bleeding in patients with acquired haemophilia

Acquired haemophilia A (AHA), with potentially high risk of morbidity and mortality, occurs as a result of inhibitors against factor VIII. Bleeding due to AHA can be treated with activated prothrombin complex concentrate (aPCC), recombinant activated factor VII (rFVIIa) or recently, recombinant porcine-sequence factor VIII (rpFVIII). We extended our previous cost-effectiveness analysis (CEA) comparing rpFVIII against the available traditional options. For high-titred, haemorrhaging AHA patients treated with either aPCC, rFVIIa or rpFVIII, over the course of 6-days, a Markov simulation was conducted to evaluate the outcomes when these patients transitioned into any of the four following health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death, with states (2), (3) and (4) modelled as absorbing states. All model parameters were obtained from the medical literature, except the costs of aPCC, rFVIIa and the factor VIII assay, which came from our institutional data. Excluding the cost of the initial treatment on day 0,the total subsequenttreatment cost of rFVIIa was substantially more than the costs of aPCC and rpFVIII ($13925 vs. $1778 vs. $6957, respectively). The average quality-adjusted life days (QALDs) gained from rpFVIII was lowest (4·89 vs. 4·9 for rFVIIa and 4·91 for aPCC). Overall, aPCC dominated the other two treatments. The model was determined to be robust across the tested ranges for all input variables. Based on this economic model, for AHA patients with high titres who were bleeding, aPCC was the most cost-effective treatment option and may be considered for use if there is no clinical contraindication.