Healthcare Payer Perspective Research Articles

Cost-effectiveness of a gene sequencing test for Alzheimer's disease in Ontario.

Alzheimer's f disease (AD) affects approximately 250,000 Ontarians, a number that is expected to double by 2040. The Ontario Neurodegenerative Disease Research Initiative has developed an in-province genetic test (ONDRISeq), which currently runs in Ontario in an experimental capacity. The aim of this study is to estimate the costs and health outcomes associated with ONDRISeq to diagnose AD relative to out-of-country (OOC) testing (status quo). A cost-utility analysis was developed for a hypothetical cohort of 65-year-olds at risk of AD in Ontario over a 25-year time horizon. Costs and health outcomes (quality-adjusted life years (QALYs)) were assessed from a healthcare payer perspective. Cost-effectiveness was assessed with a $50,000 cost-effectiveness threshold. Probabilistic sensitivity analyses were conducted to evaluate parameter uncertainty. ONDRISeq saved $54 per patient relative to OOC testing and led to a small QALY gain in the base case (0.0014 per patient). Results were most sensitive to testing costs, uptake rates, and treatment efficacy. ONDRISeq represented better value for money relative to OOC testing throughout 75% of 10,000 probabilistic iterations. Using ONDRISeq is expected to provide health system cost savings. Switching to ONDRISeq for AD genetic testing in Ontario would be dependent on the ability to accommodate the expected testing volumes.

Economic impact of self-administered subcutaneous versus clinic-administered intravenous immunoglobulin G therapy in Alberta, Canada: a population-based cohort study

BackgroundSelf-administered subcutaneous immunoglobulin G (SCIg) reduces nursing time and eliminates the need for treatment at ambulatory care clinics, as compared with clinic-based intravenously administered IgG (IVIg), and are therapeutically equivalent. Estimating the economic impact of self-administered SCIg versus clinic-administered IVIg therapy may guide treatment recommendations.MethodsA retrospective population-based cohort study using administrative data from Alberta was performed; those treated with IgG between April 1, 2012 and March 31, 2019 were included. Costs for medical laboratory staff and nursing time, as well as ambulatory care visits were considered. Univariate generalized linear model regression with gamma distribution and log link was used to compare cost ($CDN 2020) between SCIg and IVIg administration. Stratified analysis by age (≥ 18-years; < 18-years) was performed.ResultsAmong 7,890 (6,148 adults; 1,742 children) individuals who received IgG, the average administration cost per patient-year of self-administered SCIg was $5,386 (95% confidence interval [CI] $5,039, $5,734) lower than clinic-administered IVIg; per patient-year cost of self-administered SCIg was $817 (95% CI $723, $912) versus $6,204 (95% CI $6,100, $6,308) for clinic-administered IVIg. The per patient-year cost of self-administered SCIg was $5,931 (95% CI $5,543, $6,319) lower among adults and $3,177 (95% CI $2,473, $3,882) lower among children compared with clinic-administered IVIg. An estimated $31.0 million (95% CI $29.0, $33.0) in cost savings to the health system would be realised if 80% of individuals switched from clinic-administered IVIg to self-administered SCIg.ConclusionsSelf-administered SCIg is substantially less costly from a health care payer perspective in Canada. Within this type of health system, switching to self-administered SCIg has the potential to reduce overall health care costs, lessen nursing burden, and may increase clinic-based capacity for others.

Cost-Effectiveness Analysis of Motixafortide Versus Plerixafor in Stem Cell Mobilization for Autologous Transplantation in Patients with Multiple Myeloma

Introduction: The Phase 3 GENESIS trial (NCT03246529) compared the efficacy and safety of motixafortide and granulocyte-colony stimulating factor (M+G) to placebo + G (PBO+G) for the mobilization of hematopoietic stem cells (HSCs) prior to autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM). Compared with PBO+G, M+G significantly increased the proportion of patients who were able to successfully mobilize ≥6x106 CD34+ cells/kg for ASCT within 2 apheresis days (92.5% vs. 26.2%; p<0.0001). In a cost-effectiveness model (CEM) based on the GENESIS trial, M+G yielded additional quality-adjusted life years (QALYs) and cost savings relative to G alone from a United States (US) healthcare payer perspective (Lamotte et al. (2022) Value Health 25(7): S346). Plerixafor + G (P+G) is also used for HSC mobilization prior to ASCT in patients with MM. To date, M+G and P+G have not been directly compared in head-to-head trials; therefore, the relative efficacy and cost effectiveness of these therapies is currently unknown. Methods: The Bucher method was used to assess the relative efficacy of M+G in the GENESIS trial and P+G in the Phase 3 AMD-3102 trial (NCT00103662), with the PBO+G arms of both trials used as a common comparator. Risk ratios (RRs) were derived for binary trial endpoints (i.e., proportion with mobilization of ≥6x106 CD34+ cells/kg within 1-4 apheresis days, successful engraftment, use of rescue therapy, proportion who underwent ASCT, incidence of serious adverse events [SAEs]) and mean differences were derived for continuous trial endpoints (i.e., number of study drug doses received, frequency and amount of G doses, and number of apheresis sessions). The RRs were then used to update the CEM to compare the cost effectiveness of M+G and P+G from a US healthcare payer perspective over a lifetime horizon; model assumptions and sources for unit costs and quality of life scores were unchanged from the previously published CEM. Results: The mean age at baseline was 59-60 years in GENESIS and 58 years in AMD-3102; heterogeneity was observed for the mean time since diagnosis (6-7 vs. 11-12 months), prior radiotherapy (10%-11% vs. 27%-31%), and the ethnic and sex distribution of the PBO+G arms (White: 95% vs. 83%; males: 57% vs. 70%). Notably, prior lenalidomide use was markedly lower in AMD-3102 (<10%) than in GENESIS (70%), which likely contributed to the higher mobilization rates in the AMD-3102 PBO+G arm than in the GENESIS PBO+G arm. In the Bucher analysis, healthcare resource utilization (HRU) outcomes significantly favored M+G during primary mobilization, including less frequent administration of G (mean difference [MD]: -0.97; 95% CI -1.43, -0.50; p=0.0001), lower G doses (MD: -920 mcg; 95% CI -1,368, -471; p=0.0001), and fewer apheresis sessions (MD: -0.98; 95% CI -1.34, -0.63; p<0.0001) (Table 1). The proportion of patients with successful mobilization of ≥6x106 CD34+ cells/kg within 1 apheresis day was significantly greater with M+G (88.8%) than with P+G (54.2%; RR 2.97; 95% CI 1.08, 8.17; p=0.0341). Similar results were observed between groups for other outcomes, including ASCT rates (RR 1.06; 95% CI 0.93, 1.21; p=0.4124), engraftment durability by 100 days (RR 0.98; 95% CI 0.87, 1.11; p=0.7894), and SAE rate (RR 2.36; 95% CI 0.26, 21.05; p=0.4521); analyses were limited to publicly available data for P+G, and were not feasible for the use of rescue therapy because of insufficient sample size. In the cost-effectiveness analysis, M+G was dominant to P+G over a lifetime horizon, resulting in additional QALYs (+0.0358) and lower total costs (-$17,378 USD; Table 2) as per base case deterministic results. The key cost drivers were the probability of successful transplantation, long-term maintenance costs, and the time to engraftment in poor mobilizers. In a probabilistic sensitivity analysis, M+G was dominant in 66% of simulations and 92% cost effective at a WTP threshold of $50,000 per QALY. Conclusions: The results of this analysis show that the use of M+G was associated with significantly lower HRU relative to P+G for the mobilization of HSCs prior to ASCT in patients with MM, as well as significantly greater successful mobilization of ≥6x106 CD34+ cells/kg within 1 apheresis day. Cost-effectiveness analysis results showed that M+G was dominant to P+G, yielding additional QALYs and net cost savings over a lifetime horizon from a US healthcare payer perspective. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Cost-effectiveness of minimally invasive partial nephrectomy and percutaneous cryoablation for cT1a renal cell carcinoma.

There is growing evidence that partial nephrectomy (PN) and percutaneous cryoablation (PCA) yield comparable outcomes for patients with cT1a renal cell carcinoma (RCC), although the cost-effectiveness of both treatments still needs to be assessed. To perform a cost-effectiveness analysis of PN and PCA for patients with cT1a RCC. A decision analysis was created over a 5-year span from a healthcare payer's perspective computing expected costs and outcomes of PN and PCA in terms of quality-adjusted life-years (QALYs) and incremental cost-effectiveness (ICER). After each treatment, the following states were modelled using data from the recent literature: procedural complications, no evidence of disease (NED), local recurrence, metastases, and death from RCC- or non-RCC-related causes. Probabilistic and deterministic sensitivity analyses were performed. PCA and PN yielded health benefits of 3.68 QALY and 3.67 QALY. Overall expected costs were $20,491 and $26,478 for PCA and PN. On probabilistic sensitivity analysis, PCA was more cost-effective than PN in 84.78% of Monte Carlo simulations. PCA was more cost-effective until its complication risk was at least 38% higher than PN. PCA was more cost-effective than PN when (i) PCAs annual local recurrence risk was < 3.5% higher than that of PN in absolute values; (ii) PCAs annual metastatic risk was < 1.0% higher than that of PN; or (iii) PCAs annual cancer-specific mortality risk < 0.65% higher than that of PN. PCA remained cost-effective until its procedural cost is above $13,875. PCA appears to be more cost-effective than PN for the treatment of cT1a RCC, although the currently available evidence is of limited quality. PCA may be the better treatment strategy in the majority of scenarios varying procedural complications, recurrence, metastatic risk, and RCC-mortality in clinically plausible ranges. • For patients with cT1a RCCs, PCA yields a comparable health benefit at lower costs compared to PN, making PCA the dominant and therefore more cost-effective treatment strategy over PN. • PCA was more cost-effective than PN when (i) PCAs annual local recurrence risk was < 3.5% higher than PN in absolute values; (ii) PCAs annual metastatic risk was < 1.0% higher than PN; or (iii) PCAs annual cancer-specific mortality risk < 0.65% higher than PN. • PCA is more cost-effective than PN for the treatment of cT1a RCC, and it remained so in the majority of scenarios varying procedural complications, recurrence, metastatic risk, and RCC mortality.

Survival modelling and cost-effectiveness analysis of treatments for newly diagnosed metastatic hormone-sensitive prostate cancer.

In metastatic hormone-sensitive prostate cancer (mHSPC) treatment, survival benefits have been shown by adding docetaxel or recent androgen receptor axis-targeted therapies (ARATs) abiraterone, apalutamide, or enzalutamide to androgen deprivation therapy (ADT). However, the optimal treatment strategy in terms of costs and effects is unclear, not least due to high ARAT costs. To assess treatment cost-effectiveness, we developed a Markov cohort model with health states of progression-free disease, progressive disease and death for men with newly diagnosed mHSPC, with a 30-year time horizon. Survival data, adverse events and utilities were informed by randomized controlled trial results, our meta-analysis of re-created individual patient survival data, and publicly available sources of unit costs. We applied a Swiss healthcare payer perspective and discounted costs and effects by 3%. We found a significant overall survival benefit for ADT+abiraterone versus ADT+docetaxel. The corresponding incremental cost-effectiveness ratio (ICER) was predicted to be EUR 39,814 per quality-adjusted life-year (QALY) gained. ADT+apalutamide and ADT+enzalutamide incurred higher costs and lower QALYs compared to ADT+abiraterone. For all ARATs, drug costs constituted the most substantial cost component. Results were stable except for a large univariable reduction in the pre-progression utility under ADT+abiraterone and very large variations in drug prices. Our model projected ADT+abiraterone to be cost-effective compared to ADT+docetaxel at a willingness-to-pay threshold of EUR 70,400/QALY (CHF 100,000 applying purchasing power parities). Given lower estimated QALYs for ADT+apalutamide and ADT+enzalutamide compared to ADT+abiraterone, the former only became cost-effective (the preferred) treatment option(s) at substantial 75-80% (80-90%) price reductions.

Once-weekly semaglutide versus insulin aspart for the treatment of type 2 diabetes in the UK: A long-term cost-effectiveness analysis based on SUSTAIN 11.

To evaluate the long-term cost-effectiveness of once-weekly semaglutide 1 mg versus insulin aspart in the UK. Long-term outcomes were projected over patients' lifetimes using the IQVIA CORE Diabetes Model (vers 9.0). SUSTAIN 11 was used to inform baseline cohort characteristics and treatment effects. Patients were modelled to receive once-weekly semaglutide plus basal insulin for 3 years before intensifying to basal-bolus insulin, compared with basal-bolus insulin for lifetimes in the aspart arm. Costs were accounted from a healthcare payer perspective in the UK, expressed in 2021 pounds sterling (GBP). Once-weekly semaglutide 1 mg was associated with improvements in quality-adjusted life expectancy of 0.18 quality-adjusted life years (QALYs) versus insulin aspart, due to a reduced incidence and delayed time to onset of diabetes-related complications. Direct costs were estimated to be GBP 800 higher with semaglutide, with higher treatment costs partially offset by cost savings from avoidance of diabetes-related complications. Once-weekly semaglutide 1 mg was therefore associated with an incremental cost-effectiveness ratio of GBP 4457 per QALY gained versus insulin aspart. Based on a willingness-to-pay threshold of GBP 20 000 per QALY gained, once-weekly semaglutide 1 mg was projected to be highly cost-effective versus insulin aspart for the treatment of type 2 diabetes in the UK.

Budget Impact Analysis of Empagliflozin in the Treatment of Patients With Type 2 Diabetes With Established Cardiovascular Disease in South Africa

This study aimed to estimate the budget impact and affordability of empagliflozin added to usual care compared with usual care alone, in a diabetic population with established cardiovascular disease, from a private healthcare payer perspective in South Africa. A budget impact model was adapted and localized. Epidemiological data were obtained from the South African Council for Medical Schemes. Clinical event rates were sourced from the EMPA-REG OUTCOME trial and drug costs from list prices. Clinical event costs were derived from a claims data analysis of the South African private healthcare sector and microcosting. Scenario analyses were performed on select inputs. The modeled outcomes included annual budget impact of empagliflozin, the incremental cost per life per month, cardiovascular deaths averted, and incremental cost per life saved, over 3 years. A total of 9 503 patients were eligible for empagliflozin (year 1), 12 670 (year 2), and 16 947 (year 3). The incremental cost was $1 272 297, $1 764 705, and $2 455 235, for years 1 to 3, respectively. The incremental cost per beneficiary per month was calculated as $0.012 (year 1), $0.016 (year 2), and $0.023 (year 3). The model estimated a 38.6% reduction in cardiovascular deaths, 305 lives saved, and an incremental cost per life saved of $17 999. Adding empagliflozin to usual care has a marginal budget implication and is highly affordable for private healthcare payers, with an acceptable incremental cost based on clinical outcomes.

Cost-Effectiveness Analysis of Early versus Late Debridement of Superficial Triangular Fibrocartilage Complex Tears

BackgroundWhile initial nonoperative management is the conventional approach for superficial triangular fibrocartilage complex (TFCC) tears, a substantial portion of these cases go on to require surgery, and the optimal duration of nonoperative treatment is unknown. In this study, we evaluate the cost-effectiveness of early versus late arthroscopic debridement for the treatment of superficial TFCC tears without distal radioulnar joint (DRUJ) instability. MethodsWe created a decision tree to compare the following strategies from a healthcare payer perspective: immediate arthroscopic debridement versus immobilization for 4 or 6 weeks with late debridement as needed. Costs were obtained from the Centers for Medicaid and Medicare Services and a national administrative claims database. Probabilities and health-related quality-of-life measures were obtained from published sources. We conducted sensitivity analyses on model inputs, including a probabilistic sensitivity analysis consisting of 10,000 Monte Carlo simulations. ResultsImmobilization for 6 weeks while reserving arthroscopic debridement for refractory cases was both the least costly and most effective strategy. Immediate arthroscopic debridement became cost-effective when success rates of immobilization for 4 or 6 weeks were less than 7.7 or 10.5%, respectively. Our probabilistic sensitivity analysis showed that immobilization for 6 weeks was preferred 97.6% of the time, and immobilization for 4 weeks was preferred 2.4% of the time. ConclusionAlthough various early and late debridement strategies can be used to treat superficial TFCC tears without DRUJ instability, immobilization for 6 weeks while reserving arthroscopic debridement for refractory cases is the optimal strategy from a cost-effectiveness standpoint.

Cost-effectiveness analysis of current non-mandatory hepatitis B vaccination coverage vs expanding coverage among healthcare workers in Ethiopia

BackgroundEthiopia is a country with high endemicity in Hepatitis B (HepB) virus infection. However, only 14% of healthcare workers (HCWs) are currently immunized via a non-mandatory strategy in the country. Hence, this study aimed to estimate the cost-effectiveness of the current vaccination coverage and increasing coverage among HCWs in Ethiopia.MethodsBased on current practice, the study considered a monovalent HepB vaccine, which has a 90% protection rate with a complete three-dose series for lifelong protection. Markov model for current coverage (14%) and expanding vaccination coverage to 80% (as per World Health Organization (WHO) recommendation) was simulated based on the data got from both primary and secondary data. Secondary data, particularly cost and effectiveness data, were gained from published articles, WHO guidelines, and Ethiopian Federal Ministry of Health documents. Cost-related data for vaccination and chronic HepB treatment were also gathered by interviewing expertise from Tikur Anbesa specialized hospital. We conducted the study from a healthcare payer perspective, with a 3% discount rate of cost and health outcome as recommended by the WHO. The primary health outcome was measured by the Incremental Cost-Effectiveness Ratio (ICER). We employed deterministic analysis and tornado diagrams to manage parameter uncertainty and show a plausible range of cost and effectiveness of variables.ResultsCurrent vaccination program is more expensive (USD 29.99) with a more additional cost of USD 1.32 and with reduced effectiveness of 0.08 Life Years (LYs) compared to the expanded HepB vaccination strategy which costs USD 28.67 and gives a relatively high total LY gain of 28.62. The resulting ICER was USD-16.23 per LYs gained. The negative ICER shows that the expanded HepB vaccination strategy dominated the current vaccination strategy. A one-way sensitivity analysis also revealed that the current vaccine coverage was dominated by an increase in the risk of infection among unvaccinated individuals.ConclusionsExpanded vaccination coverage (to 80%) was found the most cost-effective strategy in Ethiopian HCWs compared to the current non-mandatory vaccine coverage (14%). In addition, the results of one-way and two-way sensitivity analysis reveal the robustness of our model conclusion.

Cost Effectiveness of Adding Pembrolizumab to Platinum and Fluoropyrimidine-Based Chemotherapy as First-Line Treatment for Advanced Esophageal Cancer: A US Healthcare Payer's Perspective.

Pembrolizumab plus cisplatin and fluorouracil demonstrated superior efficacy and comparable safety compared with fluorouracil and cisplatin (FP) as first-line treatment for locally advanced unresectable or metastatic carcinoma of the esophagus and gastroesophageal junction adenocarcinoma in a phase III trial (KEYNOTE-590). This study evaluated the cost effectiveness of pembrolizumab plus FP versus FP and versus a blended chemotherapy comparator including FP, carboplatin plus paclitaxel, FOLFOX, FOLFIRI, docetaxel plus FP, trastuzumab plus FP, and trastuzumab plus FOLFOX from a US healthcare payer's perspective. A partitioned survival model was developed with three health states (progression-free, progressive disease, and death). Overall survival, progression-free survival, time on treatment, and adverse events were informed by patient-level data from KEYNOTE-590. The blended chemotherapy comparator reflected the current US treatment landscape and was assumed to have similar efficacy and safety as FP. Health utilities were estimated using linear mixed-effects models based on EQ-5D-5L data from the trial. Resource use and cost inputs (2020 US dollars) were based on US standard sources and literature. Costs, life-years, and quality-adjusted life-years (QALYs) discounted at 3.0% per year and incremental cost-effectiveness ratio were outcomes in the model. Sensitivity and scenario analyses were conducted to assess the robustness of base-case results. Compared with FP, pembrolizumab plus FP produced a mean gain of 0.86 life-year and 0.77 QALY with additional costs of $112,630 over 37.6 years, yielding an incremental cost-effectiveness ratio of $147,097 per QALY. Results were similar when the intervention was pembrolizumab plus alternative chemotherapies or when blended chemotherapy became the comparator. Results were most sensitive to different overall survival extrapolation approaches. Our analysis suggests that pembrolizumab plus chemotherapy extended life-years and QALYs and is cost effective compared with chemotherapy alone as a first-line treatment for advanced esophageal cancer in the USA given a willingness-to-pay threshold of $150,000 per QALY.

The long-term cost-effectiveness of once-weekly semaglutide 1 mg vs. dulaglutide 3 mg and 4.5 mg in the UK

AimsOnce-weekly semaglutide and dulaglutide represent two highly efficacious treatment options for type 2 diabetes. A recent indirect treatment comparison (ITC) has associated semaglutide 1 mg with similar and greater improvements in glycated haemoglobin (HbA1c) and body weight, respectively, vs. dulaglutide 3 mg and 4.5 mg. The present study aimed to evaluate the long-term cost-effectiveness of semaglutide 1 mg vs. dulaglutide 3 mg and 4.5 mg in the UK.Materials and methodsThe IQVIA CORE Diabetes Model (v9.0) was used to project outcomes over patients’ lifetimes. Baseline cohort characteristics were sourced from SUSTAIN 7, with changes in HbA1c and body mass index applied as per the ITC. Modelled patients received semaglutide or dulaglutide for 3 years, after which treatment was intensified to basal insulin. Costs (expressed in 2020 pounds sterling [GBP]) were accounted from a healthcare payer perspective.ResultsSemaglutide 1 mg was associated with improvements in quality-adjusted life expectancy of 0.05 and 0.04 quality-adjusted life years (QALYs) vs. dulaglutide 3 mg and 4.5 mg, respectively, due to a reduced incidence of diabetes-related complications with semaglutide. Direct costs were estimated to be GBP 76 lower and GBP 8 higher in the comparisons with dulaglutide 3 mg and 4.5 mg, respectively. Overall outcomes were similar, but favoured semaglutide, and based on modelled mean outcomes it was considered dominant vs. dulaglutide 3 mg and associated with an incremental cost-effectiveness ratio of GBP 228 per QALY gained vs. dulaglutide 4.5 mg.ConclusionsSemaglutide 1 mg represents a cost-effective treatment vs. dulaglutide 3 mg and 4.5 mg for type 2 diabetes from a healthcare payer perspective in the UK.

Estimating the Economic and Clinical Value of Introducing Ceftazidime/Avibactam into Antimicrobial Practice in Japan: A Dynamic Modelling Study.

BackgroundAntimicrobial resistance (AMR) is one of the most serious public health challenges worldwide, including in Japan. Globally, research and development of new antimicrobials has stalled due to unfavorable market conditions, which undervalue antimicrobials. Furthermore, Japan faces the additional challenge of delayed commercialization for a number of recently approved treatments.ObjectiveThis study aims to examine the impact on AMR of introducing a new anti-infective treatment, ceftazidime/avibactam, into current treatment strategies. It reports the resulting clinical and economic outcomes from the perspective of healthcare payers in Japan.MethodsA previously published and validated dynamic disease transmission model was adapted to the Japanese setting. The model estimated health economic outcomes for treating three Gram-negative hospital-acquired infections, under different treatment strategies, from a healthcare payers’ perspective. Outcomes were assessed over a 10-year time horizon with a willingness-to-pay threshold of ¥5,000,000 (US$45,556) per quality-adjusted life-year (QALY) gained and an annual discount rate of 2% applied to costs and benefits.ResultsIntroducing ceftazidime/avibactam in the framework of a diversification strategy with piperacillin/tazobactam is associated with reducing 798,640 bed days, equating to ¥21.0 billion (US$190.9 million) savings in hospitalization costs, and a gain of 363,034 life-years, or 308,641 QALYs. This translates into a monetary benefit of ¥1.56 trillion (US$14.3 billion) to Japanese healthcare payers.DiscussionIntroducing a new antimicrobial agent into clinical practice is associated with considerable clinical and economic benefits. This analysis demonstrates that the approach taken to incorporate a new antimicrobial agent into clinical practice impacts on the scale of these clinical and economic benefits; greater benefits are associated with earlier use of antimicrobials as part of an antimicrobial stewardship program.ConclusionThis analysis shows that changing the way in which a new antimicrobial is used within a treatment strategy has the potential for additional significant clinical and economic value.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41669-022-00368-w.

Cost-effectiveness of MicroRNA for Pancreatic Cancer Screening in Patients With Diabetes

The study aimed to assess the cost-effectiveness of microRNA compared with carbohydrate antigen 19-9, abdominal ultrasound, magnetic resonance imaging, endoscopic ultrasound, computed tomography, positron emission tomography, and no screening for pancreatic cancer (PC) screening in patients with diabetes. We developed a state-transition model from a health care payer perspective and a lifetime horizon. We targeted 3 hypothetical cohorts of patients with long-standing type 2 diabetes (LSD), new-onset diabetes (NOD), and LSD having intraductal papillary mucinous neoplasm (IPMN), aged 40, 50, 60, and 70 years. The main outcomes were costs, quality-adjusted life-years, life expectancy life-years, incremental cost-effectiveness ratios, and deaths from PC. In the base-case analysis, abdominal ultrasound in patients with LSD and microRNA in patients with NOD and LSD having IPMN were the most cost-effective for all age groups. Cost-effectiveness was sensitive to PC incidence and the cost of microRNA. Probabilistic sensitivity analysis showed that microRNA was 42% to 54% cost-effective for NOD and 76% to 78% cost-effective for LSD having IPMN at a willingness-to-pay level of US $100,000 per quality-adjusted life-year gained. MicroRNA prevented 30,641 PC deaths in diabetic patients compared with no screening. In patients with NOD and LSD having IPMN, microRNA-based PC screening is cost-effective and recommended for early PC detection.

Mainstream germline genetic testing for patients with epithelial ovarian cancer leads to higher testing rates and a reduction in genetics-related healthcare costs from a healthcare payer perspective

ObjectiveGermline genetic testing is increasingly offered to patients with epithelial ovarian cancer by non-genetic healthcare professionals, so called mainstream genetic testing. The aim of this study was to evaluate the effect of implementing a mainstream genetic testing pathway on the percentage of newly diagnosed patients with epithelial ovarian cancer to whom genetic testing was offered and the genetics-related healthcare costs. MethodsThe possible care pathways for genetic counseling and testing and their associated costs were mapped. Patient files from all newly diagnosed patients with epithelial ovarian cancer before (March 2016 – September 2017) and after (April 2018 – December 2019) implementing our mainstream genetic testing pathway were analyzed. Based on this analysis, the percentage of newly diagnosed patients to whom genetic testing was offered was assessed and genetics-related healthcare costs were calculated using a healthcare payer perspective based on a Diagnosis-Related Group financing approach. ResultsWithin six months after diagnosis, genetic testing was offered to 56% of patients before and to 70% of patients after implementation of our mainstream genetic testing pathway (p = 0.005). Genetics-related healthcare costs decreased from €3.511,29 per patient before implementation to €2.418,41 per patient after implementation of our mainstream genetic testing pathway (31% reduction, p = 0.000). ConclusionThis study shows that mainstream genetic testing leads to a significantly higher proportion of newly diagnosed patients with epithelial ovarian cancer being offered germline genetic testing. In addition, it significantly reduces genetics-related healthcare costs per patient.

Value of the Immunization Programfor Children in the 2017 USBirth Cohort.

We evaluated the economic impact of routine childhood immunization in the United States, reflecting updated vaccine recommendations and recent data on epidemiology and coverage rates. An economic model followed the 2017 US birth cohort from birth through death; impact was modeled via a decision tree for each of the vaccines recommended for children by the Advisory Committee on Immunization Practices as of 2017 (with annual influenza vaccine considered in scenario analysis). Using information on historic prevaccine and vaccine-era incidence and disease costs, we calculated disease cases, deaths, disease-related healthcare costs, and productivity losses without and with vaccination, as well as vaccination program costs. We estimated cases and deaths averted because of vaccination, life-years and quality-adjusted life-years gained because of vaccination, incremental costs (2019 US dollars), and the overall benefit-cost ratio (BCR) of routine childhood immunization from the societal and healthcare payer perspectives. Over the cohort's lifetime, routine childhood immunization prevented over 17 million cases of disease and 31 000 deaths; 853 000 life years and 892 000 quality-adjusted life-years were gained. Estimated vaccination costs ($8.5 billion) were fully offset by the $63.6 billion disease-related averted costs. Routine childhood immunization was associated with $55.1 billion (BCR of 7.5) and $13.7 billion (BCR of 2.8) in averted costs from a societal and healthcare payer perspective, respectively. In addition to preventing unnecessary morbidity and mortality, routine childhood immunization is cost-saving. Continued maintenance of high vaccination coverage is necessary to ensure sustained clinical and economic benefits of the vaccination program.

Cost-minimization analysis of recombinant factor VIII Fc versus emicizumab for treating patients with hemophilia A without inhibitors in Europe

Background and objective A cost-minimization model was developed to compare recombinant factor VIII Fc (rFVIIIFc) and emicizumab as prophylaxis for hemophilia A without inhibitors. Methods The model was based on 100 patients from the healthcare payer perspective in the UK, France, Italy, Spain, and Germany (5-year time horizon). Costs included: drug acquisition; emicizumab wastage by bodyweight (manufacturer’s dosing recommendations); and additional FVIII for breakthrough bleeds. Scenario analyses (UK only): reduced emicizumab dosing frequency; and emicizumab maximum wastage. Results Total incremental 5-year savings for rFVIIIFc rather than emicizumab use range from €89,320,131 to €149,990,408 in adolescents/adults (≥12 years) and €173,417,486 to €253,240,465 in children (<12 years). Emicizumab wastage accounts for 6% of its total cost in adolescents/adults and 26% in children. Reducing the emicizumab dosing frequency reduces the incremental cost savings with rFVIIIFc, but these remain substantial (adolescents/adults, >€92 million; children >€32 million). Maximum emicizumab wastage increases by 86% and 106%, respectively, increasing the incremental cost savings with rFVIIIFc to €125,352,125 and €105,872,727, respectively. Conclusion Based on cost-minimization modeling, rFVIIIFc use for hemophilia A prophylaxis in patients without inhibitors is associated with substantial cost savings in Europe, reflecting not only higher acquisition costs of emicizumab, but also other costs including wastage related to available vial sizes.

Cost-effectiveness Analysis of Combined Physical and Cognitive Exercises Programs Designed for Preventing Dementia among Community-dwelling Healthy Young-old Adults.

This study aims to estimate the cost-effectiveness of combined physical and cognitive programs designed to prevent community-dwelling healthy young-old adults from developing dementia. The analysis was conducted from a public healthcare and long-term care payer's perspective. Quality-adjusted life years (QALYs) and expenses for health services and long-term care services were described in terms of effectiveness and cost, respectively. A thousand community-dwelling healthy adults aged 65 years were generated through simulation and analyzed. The incremental cost-effectiveness ratio (ICER) of adults with preventive program intervention compared to those with nonintervention was simulated with a 10-year cycle Markov model. The data sources for the parameters to build the Markov models were selected with priority given to higher levels of evidence. The threshold for assessing cost-effectiveness was set as less than 5,000,000 Japanese yen/QALY. The ICER was estimated as -5,740,083 Japanese yen (US$-57,400)/QALY. A program targeting community-dwelling healthy young-old adults could be cost-effective.

Cost-Effectiveness of iGlarLixi Versus Premix BIAsp 30 in People with Type 2 Diabetes Suboptimally Controlled by Basal Insulin in the US.

IntroductionMany people with type 2 diabetes mellitus (T2DM) experience suboptimal glycemic control and require therapy advancement. This cost-effectiveness analysis was conducted to compare iGlarLixi (insulin glargine 100 U/mL plus lixisenatide) versus BIAsp 30 (biphasic insulin aspart 30) in people with T2DM suboptimally controlled with basal insulin.MethodsThe IQVIA Core Diabetes Model was used to estimate lifetime costs and outcomes for people with T2DM from a US healthcare payer perspective. Initial clinical data were based on the phase 3 randomized, open-label, active-controlled SoliMix clinical study, which compared the efficacy and safety of once-daily iGlarLixi with twice-daily BIAsp 30. Lifetime costs (US$) and quality-adjusted life-years (QALYs) were predicted, and the incremental cost-effectiveness ratio (ICER) for iGlarLixi versus BIAsp 30 was estimated; the willingness-to-pay threshold was considered to be $50,000. A subgroup analysis considered people with T2DM aged ≥ 65 years.ResultsEstimated QALYs gained were slightly higher with iGlarLixi compared with BIAsp 30 (9.3 vs. 9.2), with lower costs for iGlarLixi ($117,854 vs. $120,109); the ICER for iGlarLixi was therefore considered dominant over BIAsp 30 in the base case. Key drivers for cost savings were the higher dose and twice-daily administration for BIAsp 30 versus once-daily administration for iGlarLixi. The robustness of the base-case results was confirmed by sensitivity and scenario analyses. Results were similar in a subgroup of people with T2DM aged ≥ 65 years.ConclusionIn people with T2DM with suboptimal glycemic control on basal insulin, iGlarLixi confers improved QALYs and reduced costs compared with BIAsp 30.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13300-022-01300-5.

Cost-effectiveness analysis of tumor molecular testing in stage III endometrial cancer (089)

<b>Objectives:</b> Treatment of advanced and recurrent endometrial cancer (EC) has traditionally been based on factors, such as histology and stage. Recent studies suggest that tumor molecular profiles may predict treatment outcomes and guide treatment choice. For example, pembrolizumab (PMB) use in patients with mismatch repair deficient (MMRd) tumors versus combined pembrolizumab and lenvatinib (PL) in patients with mismatch repair proficient tumors (MMRp). Furthermore, <i>POLE</i> mutated (<i>POLE</i>mut) tumors demonstrate a favorable prognosis even with minimal treatment. Prospective trials using molecular profiling for treatment assignment are ongoing, but tumor molecular testing (TMT) is costly and time-consuming. We, therefore, sought to evaluate the cost-effectiveness of TMT in stage III EC. <b>Methods:</b> A Markov decision model was created to compare two testing strategies in patients with stage III EC: TMT versus no testing (NT). TMT patients had sequential <i>POLE</i> next-generation sequencing (NGS), mismatch repair (MMR) immunohistochemistry (IHC), and p53 IHC. Adjuvant therapies included radiation (RT) alone for <i>POLE</i>mut and chemoradiation (CTRT) for NT, MMRd, p53 mutated (p53mut), and no specific molecular profile (NSMP) using PORTEC-3 data. All patients received six cycles of carboplatin and paclitaxel (CP) for first recurrence using GOG 209 outcomes. MMRd patients received PMB alone for the second recurrence, whereas all other patients received PL. National database and registry values, literature review, and expert input were used for cost and utility values. A healthcare payer perspective, willingness-to-pay (WTP) threshold of $100,000, and 5-year time horizon were used. Data analysis was done with TreeAge Pro Software 2021. Outcomes were reported in quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses tested model robustness, and a structural sensitivity analysis was performed in which controls underwent MMR IHC alone. <b>Results:</b> Compared to NT, TMT was less costly and more effective. While the model was most sensitive to median overall survival with PL, probability of MMRd status, and cost of lenvatinib, TMT remained cost-saving over all parameters tested. On Monte Carlo analysis, TMT was favored in 79% and 80% of iterations with a WTP of $50,000 and $100,000, respectively. When controls had MMR IHC alone, TMT was no longer cost-effective with an incremental cost of $3100 and incremental effectiveness of 0.017 QALYs, or $184,942/QALY gained. <b>Conclusions:</b> For patients with stage III EC, this model suggests TMT is associated with cost savings compared to NT. However, when compared to MMR IHC alone, a common clinical approach, TMT was no longer a cost-effective strategy at a WTP threshold of $100,000. Further prospective studies are needed to determine the prognostic value of molecular profiles in endometrial cancer. It will be essential to continue assessing the cost-effectiveness of these molecular tests as new data becomes available.

Economic Evaluation of Telerobotic Ultrasound Technology to Remotely Provide Ultrasound Services in Rural and Remote Communities.

Telerobotic ultrasound technology allows radiologists and sonographers to remotely provide ultrasound services in underserved areas. This study aimed to compare costs associated with using telerobotic ultrasound to provide ultrasound services in rural and remote communities to costs associated with alternate models. A cost-minimization approach was used to compare four ultrasound service delivery models: telerobotic ultrasound (Model 1), telerobotic ultrasound and an itinerant sonographer (Model 2), itinerant sonographer without telerobotic ultrasound (Model 3), and travel to another community for all exams (Model 4). In Models 1-3, travel was assumed when exams could not be successfully performed telerobotically or by an itinerant sonographer. A publicly funded healthcare payer perspective was used for the reference case and a societal perspective was used for a secondary non-reference case. Costs were based on the literature and experience using telerobotic ultrasound in Saskatchewan, Canada. Costs were expressed in 2020 Canadian dollars. Average cost per ultrasound exam was $342, $323, $368, and $478 for Models 1, 2, 3, and 4, respectively, from a publicly funded healthcare payer perspective, and $461, $355, $447, and $849, respectively, from a societal perspective. In one-way sensitivity analyses, Model 2 was the lowest cost from a payer perspective for communities with population >2075 people, distance >350 km from the nearest ultrasound facility, or >47% of the population eligible for publicly funded medical transportation. Health systems may wish to consider solutions such as telerobotic ultrasound and itinerant sonographers to reduce healthcare costs and improve access to ultrasound in rural and remote communities.