A cost-utility analysis of 18F-fluorocholine-positron emission tomography imaging for localizing primary hyperparathyroidism in the United States.

BACKGROUND: Primary hyperparathyroidism historically necessitated bilateral neck exploration to remove abnormal parathyroid tissue. Improved localization allows for focused parathyroidectomy with lower complication risks. Recently, positron emission tomography using radiolabeled 18F-fluorocholine demonstrated high accuracy in detecting these lesions, but its cost-effectiveness has not been studied in the United States. METHODS: A decision tree modeled patients who underwent parathyroidectomy for primary hyperparathyroidism using single preoperative localization modalities: (1) positron emission tomography using radiolabeled 18F-fluorocholine, (2) 4-dimensional computed tomography, (3) ultrasound, and (4) sestamibi single photon emission computed tomography (SPECT). All patients underwent either focused parathyroidectomy versus bilateral neck exploration, with associated cost ($) and clinical outcomes measured in quality-adjusted life-years gained. Model parameters were informed by literature review and Medicare costs. Incremental cost-utility ratios were calculated in US dollars/quality-adjusted life-years gained, with a willingness-to-pay threshold set at $100,000/quality-adjusted life-year. One-way, 2-way, and threshold sensitivity analyses were performed. RESULTS: Positron emission tomography using radiolabeled 18F-fluorocholine gained the most quality-adjusted life-years (23.9) and was the costliest ($2,096), with a total treatment cost of $11,245 or $470/quality-adjusted life-year gained. Sestamibi single photon emission computed tomography and ultrasound were dominated strategies. Compared with 4-dimentional computed tomography, the incremental cost-utility ratio for positron emission tomography using radiolabeled 18F-fluorocholine was $91,066/quality-adjusted life-year gained in our base case analysis, which was below the willingness-to-pay threshold. In 1-way sensitivity analysis, the incremental cost-utility ratio was sensitive to test accuracy, positron emission tomography using radiolabeled 18F-fluorocholine price, postoperative complication probabilities, proportion of bilateral neck exploration patients needing overnight hospitalization, and life expectancy. CONCLUSION: Our model elucidates scenarios in which positron emission tomography using radiolabeled 18F-fluorocholine can potentially be a cost-effective imaging option for primary hyperparathyroidism in the United States. Further investigation is needed to determine the maximal cost-effectiveness for positron emission tomography using radiolabeled 18F-fluorocholine in selected populations.

Procedural and 12-month in-hospital costs of primary infra-popliteal bypass surgery, infrapopliteal best endovascular treatment, and major lower limb amputation for chronic limb threatening ischemia.

OBJECTIVE: Chronic limb-threatening ischemia (CLTI) is a growing global problem due to the widespread use of tobacco and increasing prevalence of diabetes. Although the financial consequences are considerable, few studies have compared the relative cost-effectiveness of different CLTI management strategies. The Bypass vs Angioplasty in Severe Ischaemia of the Leg (BASIL)-2 trial is randomizing patients with CLTI to primary infrapopliteal (IP) vein bypass surgery (BS) or best endovascular treatment (BET) and includes a comprehensive within-trial cost-utility analysis. The aim of this study is to compare over a 12-month time horizon, the costs of primary IP BS, IP best endovascular treatment (BET), and major limb major amputation (MLLA) to inform the BASIL-2 cost-utility analysis. METHODS: We compared procedural human resource (HR) costs and total in-hospital costs for the index admission, and over the following 12-months, in 60 consecutive patients undergoing primary IP BS (n = 20), IP BET (n = 20), or MLLA (10 transfemoral and 10 transtibial) for CLTI within the BASIL prospective cohort study. RESULTS: Procedural HR costs were greatest for BS (BS £2551; 95% confidence interval [CI], £1934-£2807 vs MLLA £1130; 95% CI, £1046-£1297 vs BET £329; 95% CI, £242-£390; P < .001, Kruskal-Wallis) due to longer procedure duration and greater staff requirement. With regard to the index admission, MLLA was the most expensive due to longer hospital stay (MLLA £13,320; 95% CI, £8986-£18,616 vs BS £8714; 95% CI, £6097-£11,973 vs BET £4813; 95% CI, £3529-£6097; P < .001, Kruskal-Wallis). The total cost of the index admission and in-hospital care over the following 12 months remained least for BET (MLLA £26,327; 95% CI, £17,653-£30,458 vs BS £20,401; 95% CI, £12,071-£23,926 vs BET £12,298; 95% CI, £6961-£15,439; P < .001, Kruskal-Wallis). CONCLUSIONS: Over a 12-month time horizon, MLLA and IP BS are more expensive than IP BET in terms of procedural HR costs and total in-hospital costs. These economic data, together with quality of life data from BASIL-2, will inform the calculation of incremental cost-effectiveness ratios for different CLTI management strategies within the BASIL-2 cost-utility analysis.

Ultrasound-Guided Injection Treatments Versus Surgical Neurectomy for Morton Neuroma: A Cost-Effectiveness Analysis.

BACKGROUND. Morton neuroma is a common, painful disorder of the foot with multiple treatment options of varying cost and effectiveness. OBJECTIVE. The aim of this study was to determine the most cost-effective treatment pathway for symptomatic Morton neuromas when conservative management has failed. METHODS. An incremental cost-utility analysis was performed comparing a direct to surgical neurectomy strategy with three selective injection strategies in which one or more ultrasound-guided injection therapies was tried first before surgery for patients who did not respond to treatment. The three selective injection strategies were selective steroid injection, selective alcohol injection, and selective steroid/alcohol injection in which both steroid injections and alcohol sclerosing injections were trialed successively before surgical neurectomy. The direct-to-surgery approach was compared with the three different selective injection strategies and with a no-treatment strategy in a decision-analytic model for a hypothetical group of patients with symptomatic Morton neuroma in whom conservative management had failed. Model parameters, including treatment costs, effectiveness, complication rates, and health utility states, were estimated from the literature, reimbursement databases, and expert opinion. The outcome was cost per quality-adjusted life year (QALY) with a time horizon of 3 years. A societal cost perspective was adopted with a willingness-to-pay threshold of $100,000/QALY. Sensitivity analyses for key model parameters were performed. RESULTS. For the base input values, the steroid/alcohol selective injection strategy was dominant and yielded an incremental cost-effectiveness ratio of $4401.61/QALY compared with no treatment. The probabilistic sensitivity analysis supported this strategy in 74% of 10,000 simulated trials. Results were robust with low sensitivity to most input parameters. However, when the probability of successful alcohol injection treatment dropped below 40%, the steroid selective injection strategy became most cost-effective. CONCLUSION. A trial of ultrasound-guided injection therapies for Morton neuroma is a cost-effective strategy compared with proceeding directly to surgical neurectomy. CLINICAL IMPACT. Ultrasound-guided injection therapies are indicated as first-line treatment of patients with symptomatic Morton neuromas when conservative management fails.

Characteristics of Cost-effectiveness Studies for Oncology Drugs Approved in the United States From 2015-2020.

Importance: Increasingly, cost-effectiveness analyses are being done to determine the value of rapidly increasing oncology drugs; however, this assumes that these analyses are unbiased. Objective: To analyze the characteristics of cost-effectiveness studies and to determine characteristics associated with whether an oncology drug is found to be cost-effective. Design, Setting, and Participants: This retrospective cross-sectional study included 254 cost-effectiveness analyses for 116 oncology drugs that were approved by the US Food and Drug Administration from 2015 to 2020. Exposures: Each drug was analyzed for the incremental cost-effectiveness ratio per quality-adjusted life year, the funding of the study, the authors' conflict of interest, the threshold of willingness-to-pay, from what country's perspective the analysis was done, and whether a National Institute for Health and Care Excellence cost-effectiveness analysis had been done. Main Outcomes and Measures: The main outcome was the odds of a study concluding that a drug was cost-effective. Results: There were 116 drug approvals with 254 studies and country perspectives. Of the country perspectives, 132 (52%) were from the US. Forty-seven of 78 drugs with cost-effective studies had been shown to improve overall survival, whereas 15 of 38 of drugs without a cost-effectiveness study had been shown to improve overall survival. Having a study funded by a pharmaceutical company was associated with higher odds of a study concluding that a drug was cost-effective than studies without funding (odds ratio, 41.36; 95% CI, 11.86-262.23). Conclusions and Relevance: In this cross-sectional study, pharmaceutical funding was associated with greater odds that an oncology drug would be found to be cost-effective. These findings suggest that simply disclosing potential conflict of interest is inadequate. We encourage cost-effectiveness analyses by independent groups.

How to Prevent Vaccines Falling Victim to Their Own Success: Intertemporal Dependency of Incidence Levels on Indirect Effects in Economic Reevaluations.

OBJECTIVES: Incremental cost-effectiveness analyses may inform the optimal choice of healthcare interventions. Nevertheless, for many vaccines, benefits fluctuate with incidence levels over time. Reevaluating a vaccine after it has successfully decreased incidences may eventually cause a disease resurgence if switching to a vaccine with lower indirect benefits. Decisions may successively alternate between vaccines alongside repeated rises and falls in incidence and when indirect effects from historic use are ignored. Our suggested proposal aims to prevent suboptimal decision making. METHODS: We used a conceptual model of demand to illustrate alternating decisions between vaccines because of time-varying levels of indirect effects. Similar to the concept of subsidies, we propose internalizing the indirect effects achievable with vaccines. In a case study over 60 years, we simulated a hypothetical 10-year reevaluation of 2 oncogenic human papillomavirus vaccines, of which only 1 protects additionally against anogenital warts. RESULTS: Our case study showed that the vaccine with additional warts protection is initially valued higher than the vaccine without additional warts protection. After 10 years, this differential decreases because of declines in warts incidence, which supports switching to the nonwarts vaccine that causes a warts resurgence eventually. Instead, pricing the indirect effects separately supports continuing with the warts vaccine. CONCLUSIONS: Ignoring how the observed incidences depend on the indirect effects achieved with a particular vaccine may lead to repeated changes in vaccines at successive reevaluations, with unintended resurgences, economic inefficiencies, and eroding vaccine confidence. We propose internalizing indirect effects to prevent vaccines falling victim to their own success.

Estimating the Theoretical Cost Implications of Funding New Drugs Considered Not to Be Cost-Effective.

This study aims to estimate the theoretical excess expenditure that would be incurred by the Irish state-payer, should drugs be reimbursed at their original asking ("list") price rather than at a price at which the drug is considered cost-effective. In Ireland, all new drugs are evaluated by the National Centre for Pharmacoeconomics. For this study, drugs that were submitted by pharmaceutical companies from 2012 to 2017 and considered not cost-effective at list price were reviewed. A total of 43 such drugs met our inclusion criteria, and their pharmacoeconomic evaluations were further assessed. The price at which the drug could be considered cost-effective (cost-effective price) at the upper cost-effectiveness threshold used in Ireland (€ 45 000/quality adjusted life-year) was estimated for 18 drugs with an available cost-effectiveness model. Then, for each drug, the list price and cost-effective price (both per unit) were both individually applied to 1 year of national real-world drug utilization data. This allowed the estimation of the expected expenditures under the assumptions of list price paid and cost-effective price paid. The resulting theoretical excess expenditure, the expenditure at list price minus the expenditure at the cost-effective price, was estimated to be €108.2 million. This estimate is theoretical because of the confidentiality of actual drug prices. The estimation is calculated using the list price and likely overestimates the actual excess expenditure, which would reduce to zero if cost-effective prices are agreed. Nevertheless, this estimate illustrates the importance of a process to assess the value of new drugs so that potential excess drug expenditure is identified.

Cost-Effectiveness of a Serum Biomarker Test for Risk-Stratified Liver Ultrasound Screening for Hepatocellular Carcinoma.

OBJECTIVES: Risk-stratified ultrasound screening for hepatocellular carcinoma (HCC), informed by a serum biomarker test, enables resources to be targeted to patients at the highest risk of developing cancer. We aimed to investigate the cost-effectiveness of risk-stratified screening for HCC in the Australian healthcare system. METHODS: A Markov cohort model was constructed to test 3 scenarios for patients with compensated cirrhosis: (1) risk-stratified screening for high-risk patients, (2) all-inclusive screening, and (3) no formal screening. Probabilistic sensitivity analyses were undertaken to determine the impact of uncertainty. Scenario analyses were used to assess cost-effectiveness in Australia's Aboriginal and Torres Strait Islander peoples and to determine the impact of including productivity-related costs of mortality. RESULTS: Both risk-stratified screening and all-inclusive screening programs were cost-effective compared with no formal screening, with incremental cost-effectiveness ratios of A$39 045 and A$23 090 per quality-adjusted life-year (QALY), respectively. All-inclusive screening had an incremental cost-effectiveness ratio of A$4453 compared with risk-stratified screening and had the highest probability of being cost-effective at a willingness-to-pay (WTP) threshold of A$50 000 per QALY. Risk-stratified screening had the highest likelihood of cost-effectiveness when the WTP was between A$25 000 and A$35 000 per QALY. Cost-effectiveness results were further strengthened when applied to an Aboriginal and Torres Strait Islander cohort and when productivity costs were included. CONCLUSIONS: Cirrhosis population-wide screening for HCC is likely to be cost-effective in Australia. Risk-stratified screening using a serum biomarker test may be cost-effective at lower WTP thresholds.

Comparison of Cap-Assisted vs Conventional Endoscopic Technique for Management of Food Bolus Impaction in the Esophagus: Results of a Multicenter Randomized Controlled Trial.

INTRODUCTION: "Push" or "pull" techniques with the use of snares, forceps, baskets, and grasping devices are conventionally used to manage esophageal food bolus impaction (FBI). A novel cap-assisted technique has recently been advocated to reduce time taken for food bolus (FB) removal. This study aimed to compare the effectiveness of the cap-assisted technique against conventional methods of esophageal FB removal in a randomized controlled trial. METHODS: Consecutive patients with esophageal FBI requiring endoscopic removal, from 3 Australian tertiary hospitals between 2017 and 2019, were randomized to either the cap-assisted technique or the conventional technique. Primary outcomes were technical success and FB retrieval time. Secondary outcomes were technical success rate, en bloc removal rate, procedure-related complication, length of hospital stay, and cost of consumables. RESULTS: Over 24 months, 342 patients with esophageal FBI were randomized to a cap-assisted (n = 171) or conventional (n = 171) technique. Compared with the conventional approach, the cap-assisted technique was associated with (i) shorter FB retrieval time (4.5 ± 0.5 minutes vs 21.7 ± 0.9 minutes, P < 0.001), (ii) shorter total procedure time (23.0 ± 0.6 minutes vs 47.0 ± 1.3 minutes, P < 0.0001), (iii) higher technical success rate (170/171 vs 160/171, P < 0.001), (iv) higher rate of en bloc removal (159/171 vs 48/171, P < 0.001), and (v) lower rate of procedure-related mucosal tear and bleeding (0/171 vs 13/171, P < 0.001). There were no major adverse events or deaths within 30 days in either group. The total cost of consumables was higher in the conventional group (A$19,644.90 vs A$6,239.90). DISCUSSION: This multicenter randomized controlled trial confirmed that the cap-assisted technique is more effective and less costly than the conventional approach and should be first-line treatment for esophageal FBI.

Comparison of Levonorgestrel-releasing Intrauterine System (LNG-IUS) against Laparoscopic Assisted Supracervical Hysterectomy (LASH) for menorrhagia treatment: An economic evaluation.

BACKGROUND: This economic evaluation and literature review was conducted with the primary aim to compare the cost-effectiveness of laparoscopic assisted supracervical hysterectomy (LASH) with NICE's gold-standard treatment of Levonorgestrel-releasing intrauterine system (LNG-IUS) for menorrhagia. MATERIALS AND METHODS: A cost-utility analysis was conducted from an NHS perspective, using data from two European studies to compare the treatments. Individual costs and benefits were assessed within one year of having the intervention. An Incremental Cost-Effectiveness Ratio (ICER) was calculated, followed by sensitivity analysis. Expected Quality Adjusted Life Years (QALYS) and costs to the NHS were calculated alongside health net benefits (HNB) and monetary net benefits (MNB). RESULTS: A QALY gain of 0.069 was seen in use of LNG-IUS compared to LASH. This yielded a MNB between -£44.99 and -£734.99, alongside a HNB between -0.0705 QALYs and -0.106 QALYS. Using a £20,000-£30,000/QALY limit outlined by NICE,this showed the LNG-IUS to be more cost-effective than LASH, with LASH exceeding the upper bound of the £30,000/QALY limit. Sensitivity analysis lowered the ICER below the given threshold. CONCLUSIONS: The ICER demonstrates it would not be cost-effective to replace the current gold-standard LNG-IUS with LASH, when treating menorrhagia in the UK. The ICER's proximity to the threshold and its high sensitivity alludes to the necessity for further research to generate a more reliable cost-effectiveness estimate. However, LASH could be considered as a first line treatment option in women with no desire to have children.

Whole Grain Intakes Are Associated with Healthcare Cost Savings Following Reductions in Risk of Colorectal Cancer and Total Cancer Mortality in Australia: A Cost-of-Illness Model.

Whole grain consumption has been associated with the reduced risk of several chronic diseases with significant healthcare monetary burden, including cancer. Colorectal cancer (CRC) is one of the most common cancers globally, with the highest rates reported in Australia. Three servings of whole grains provide a 15% reduction in total cancer and 17% reduction in CRC risk; however, 70% of Australians fall short of this level of intake. The aim of this study was to assess the potential savings in healthcare costs associated with reductions in the relative risk of CRC and total cancer mortality following the whole grain Daily Target Intake (DTI) of 48 g in Australia. A three-step cost-of-illness analysis was conducted using input parameters from: (1) estimates of current and targeted whole grain intakes among proportions (5%, 15%, 50%, and 100%) of the Australian adult (≥20 years) population; (2) estimates of reductions in relative risk (with 95% confidence intervals) of CRC and total cancer mortality associated with specific whole grain intake from meta-analysis studies; and (3) estimates of annual healthcare costs of CRC and all cancers from disease expenditure national databases. A very pessimistic (5% of population) through to universal (100% of population) adoption of the recommended DTI in Australia were shown to potentially yield savings in annual healthcare costs equal to AUD 1.9 (95% CI 1.2-2.4) to AUD 37.2 (95% CI 24.1-48.1) million for CRC and AUD 20.3 (95% CI 12.2-27.0) to AUD 405.1 (95% CI 243.1-540.1) million for total cancers. As treatment costs for CRC and other cancers are increasing, and dietary measures exchanging whole grains for refined grains are not cost preclusive nor does the approach increase energy intake, there is an opportunity to facilitate cost-savings along with reductions in disease for Australia. These results suggest specific benefits of encouraging Australians to swap refined grains for whole grains, with greater overall adherence to suggestions in dietary guidelines.

A mixed-methods study to evaluate the effectiveness and cost-effectiveness of aerobic exercise for primary dysmenorrhea: A study protocol.

BACKGROUND AND PURPOSE: Several studies have evaluated the effects of high-intensity aerobic training (HIAT) on pain severity and quality of life (QoL) among women with primary dysmenorrhea. However, to date, no studies have evaluated the effectiveness of HIAT on academic performance or absenteeism or examined the cost-effectiveness of HIAT relative to other treatments in women with primary dysmenorrhea. Furthermore, the mechanisms underlying aerobic exercise-induced analgesia in primary dysmenorrhea remain unclear. The objectives of this study are to: (1) evaluate the effects of HIAT on absenteeism and academic performance among university students, (2) identify the underlying mechanisms associated with aerobic exercise-induced analgesia in primary dysmenorrhea, and (3) determine the cost-effectiveness of HIAT compared with a wait-list control (WLC) group receiving usual care. METHODS: A sequential, embedded, mixed-methods study design, including a crossover, randomised controlled trial (RCT) and semi-structured focus groups, will be conducted alongside an economic evaluation. A total of 130 women aged 18-24 years will be randomised into either HIAT (n = 65) or wait-list control (n = 65) groups. Primary outcomes will include average pain intensity, absenteeism from university, and academic performance. Primary mediators will include salivary progesterone and prostaglandin F2α levels. Outcome and meditator variables will be assessed at baseline and post-treatment, at 12 and 28 weeks. An economic analysis will be conducted from the societal and healthcare perspective of Hong Kong. Semi-structured focus groups will be conducted at 32 weeks. Of the 130 participants included in the RCT, 70 will be included in the focus groups. STATISTICAL ANALYSIS: All statistical analyses will be performed on an intention-to-treat basis, using SPSS (version 24). Preliminary analysis using an independent samples t-test and a two-sided, unpaired Student's t-test will be performed to exclude carryover effects and identify within-participant differences in outcome variables between the study periods, respectively. Treatment effects will be evaluated using analysis of variance via a mixed-effects model with fixed effects for intervention, period, and sequence. In all models, random effects will include the participants nested within the sequence as a sampling cluster. The mediation effects will be assessed using the Sobel test. The EQ-5D responses will be converted into utility scores to estimate the gain or loss of quality-adjusted life-years. Seemingly unrelated regression analyses will be used to estimate the total cost differences and effect differences. Qualitative data will be analysed using the process of thematic analysis.

Impact of assumptions on future costs, disutility and mortality in cost-effectiveness analysis; a model exploration.

INTRODUCTION: In cost-effectiveness analyses, the future costs, disutility and mortality from alternative causes of morbidity are often not completely taken into account. We explored the impact of different assumed values for each of these factors on the cost-effectiveness of screening for colorectal cancer (CRC) and esophageal adenocarcinoma (EAC). METHODS: Twenty different CRC screening strategies and two EAC screening strategies were evaluated using microsimulation. Average health-related expenses, disutility and mortality by age for the U.S. general population were estimated using surveys and lifetables. First, we evaluated strategies under default assumptions, with average mortality, and no accounting for health-related costs and disutility. Then, we varied costs, disutility and mortality between 100% and 150% of the estimated population averages, with 125% as the best estimate. Primary outcome was the incremental cost per quality-adjusted life-year (QALY) gained among efficient strategies. RESULTS: The set of efficient strategies was robust to assumptions on future costs, disutility and mortality from other causes of morbidity. However, the incremental cost per QALY gained increased with higher assumed values. For example, for CRC, the ratio for the recommended strategy increased from $15,600 with default assumptions, to $32,600 with average assumption levels, $61,100 with 25% increased levels, and $111,100 with 50% increased levels. Similarly, for EAC, the incremental costs per QALY gained for the recommended EAC screening strategy increased from $106,300 with default assumptions to $198,300 with 50% increased assumptions. In sensitivity analyses without discounting or including only above-average expenses, the impact of assumptions was relatively smaller, but best estimates of the cost per QALY gained remained substantially higher than default estimates. CONCLUSIONS: Assumptions on future costs, utility and mortality from other causes of morbidity substantially impact cost-effectiveness outcomes of cancer screening. More empiric evidence and consensus are needed to guide assumptions in future analyses.

Cost-Effectiveness of Vioptix versus Clinical Examination for Flap Monitoring of Autologous Free Tissue Breast Reconstruction.

BACKGROUND: Vioptix is a near-infrared spectroscopy tissue oximetry technology that allows for noninvasive monitoring of flap perfusion. Despite the reported benefits of Vioptix, the cost-effectiveness of this flap monitoring technology has not been compared to clinical examination alone. METHODS: A cost-effectiveness model, from the patient perspective, was constructed with two treatment arms: clinical examination versus clinical examination combined with Vioptix for flap monitoring after autologous, free flap breast reconstruction. Costs, utilities, and other model inputs were identified from the literature. One-way and probabilistic sensitivity analyses were performed. Gamma distributions were created for cost variables, and beta distributions were created for probability variables. An incremental cost-effectiveness ratio under $50,000 per quality-adjusted life-year (QALY) was considered cost-effective. All analyses were performed using TreeAge Pro (Williamstown, Mass.). RESULTS: Mean cost of autologous free tissue transfer breast reconstruction with clinical examination-based flap monitoring was found to be $37,561 with an effectiveness of 0.79, whereas the mean cost of clinical examination with Vioptix for flap monitoring was $39,361 with effectiveness of 0.82. This yielded an incremental cost-effectiveness ratio of $60,507 for clinical examination combined with Vioptix for flap monitoring. One-way sensitivity analysis revealed that clinical examination with Vioptix became cost-effective when the cost of Vioptix was less than $1487. Probabilistic sensitivity analysis found that clinical examination was cost-effective in 86.5 percent of cases. CONCLUSION: Although clinical examination combined with Vioptix is minimally more effective for flap monitoring after autologous, free flap breast reconstruction, clinical examination alone is the more cost-effective flap monitoring option.

Direct costs of antineoplastic and supportive treatment for progressive multiple myeloma in a tax-based health system.

Aim: To estimate current real-world costs of drugs and supportive care for the treatment of multiple myeloma in a tax-based health system. Methods: Forty-one patients were included from a personalized medicine study (2016-2019). Detailed information was collected from patient journals and hospital registries to estimate the total and mean costs using inverse probability weighting of censored data. Results: Total observed (censored) costs for the 41 patients was €8.84 million during 125 treatment years, with antineoplastic drugs as the main cost driver (€5.6 million). Individual costs showed large variations. Mean 3-year cost per patient from first progression was €182,103 (€131,800-232,405). Conclusion: Prediction of real-world costs is hindered by the availability of detailed costing data. Micro-costing analyses are needed for budgeting and real-world evaluation of cost-effectiveness.

Lay abstract In recent years, there has been a dramatic improvement in the treatment of multiple myeloma due to the introduction of new drugs. These drugs have significantly increased survival but have also had an immense impact on healthcare budgets. In this study, we used detailed treatment information for multiple myeloma patients in combination with billing data from the hospital pharmacy at a Danish hospital to calculate individual cost histories for both drugs and supportive care. Using these data, we estimated the mean 3-year cost of a multiple myeloma patient to be €182.103, but we also found large variation between patients, causing an uncertainty of €50.000 in either direction. We believe that detailed costing studies, similar to the present one, are necessary for evaluation of cost-effectiveness of drugs in clinical practice.

Cost-effectiveness of bariatric surgery and non-surgical weight management programmes for adults with severe obesity: a decision analysis model.

OBJECTIVES: To determine the most cost-effective weight management programmes (WMPs) for adults, in England with severe obesity (BMI ≥ 35 kg/m2), who are more at risk of obesity related diseases. METHODS: An economic evaluation of five different WMPs: 1) low intensity (WMP1); 2) very low calorie diets (VLCD) added to WMP1; 3) moderate intensity (WMP2); 4) high intensity (Look AHEAD); and 5) Roux-en-Y gastric bypass (RYGB) surgery, all compared to a baseline scenario representing no WMP. We also compare a VLCD added to WMP1 vs. WMP1 alone. A microsimulation decision analysis model was used to extrapolate the impact of changes in BMI, obtained from a systematic review and meta-analysis of randomised controlled trials (RCTs) of WMPs and bariatric surgery, on long-term risks of obesity related disease, costs, quality adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) measured as incremental cost per QALY gained over a 30-year time horizon from a UK National Health Service (NHS) perspective. Sensitivity analyses explored the impact of long-term weight regain assumptions on results. RESULTS: RYGB was the most costly intervention but also generated the lowest incidence of obesity related disease and hence the highest QALY gains. Base case ICERs for WMP1, a VLCD added to WMP1, WMP2, Look AHEAD, and RYGB compared to no WMP were £557, £6628, £1540, £23,725 and £10,126 per QALY gained respectively. Adding a VLCD to WMP1 generated an ICER of over £121,000 per QALY compared to WMP1 alone. Sensitivity analysis found that all ICERs were sensitive to the modelled base case, five year post intervention cessation, weight regain assumption. CONCLUSIONS: RYGB surgery was the most effective and cost-effective use of scarce NHS funding resources. However, where fixed healthcare budgets or patient preferences exclude surgery as an option, a standard 12 week behavioural WMP (WMP1) was the next most cost-effective intervention.

Pembrolizumab and lenvatinib versus carboplatin and paclitaxel as first-line therapy for advanced or recurrent endometrial cancer: A Markov analysis.

OBJECTIVE: To determine the cost effectiveness of pembrolizumab/lenvatinib (P/L) versus standard-of-care carboplatin/paclitaxel (C/T) as first-line systemic therapy for patients with advanced/recurrent endometrial cancer. METHODS: We designed a Markov model to simulate treatment outcomes for advanced/recurrent endometrial cancer patients whose tumors are either microsatellite stable (MSS) or have high microsatellite instability (MSI-high). We adopted a healthcare sector perspective for the analysis. Model inputs for costs, health utility, and clinical estimates were obtained from the literature including data from GOG0209 and KEYNOTE-146. Primary outcomes included costs of care, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER). The time-horizon was three years and the discount rate was 3% annually. RESULTS: In a MSS cohort, compared to C/T, first-line treatment with P/L increased treatment costs by $212,670 and decreased QALYs by 0.28 per patient. In a MSI-high cohort, compared to C/T, P/L increased costs by $313,487 and increased QALYs by 0.11 per patient, representing an ICER of $2,849,882 per QALY. Sensitivity analyses found that the price of the new drugs was the most important determinant of the ICER and that the price of the new drugs would need to decrease by 85% to $2817 per cycle to reach a $150,000/QALY threshold. CONCLUSION: In the MSS model, we found that first-line therapy for advanced or recurrent endometrial cancer with P/L increased costs and worsened outcomes compared to C/T. In the MSI-high model, P/L improved survival and QALYs compared to C/T but was not cost-effective at the current cost of the drugs.

Initiation of Somatostatin analogues for neuroendocrine tumor patients: a cost-effectiveness analysis.

BACKGROUND & AIMS: Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are heterogeneous neoplasms. Although some have a relatively benign and indolent natural history, others can be aggressive and ultimately fatal. Somatostatin analogues (SSAs) improve both quality of life and survival for these patients once they develop metastatic disease. However, these drugs are costly and their cost-effectiveness is not known. METHODS: A decision-analytic model was developed and analyzed to compare two treatment strategies for patients with Stage IV GEP-NETs. The first strategy had all patients start SSA immediately while the second strategy waited, reserving SSA initiation until the patient showed signs of progression. Sensitivity analysis was performed to explore model parameter uncertainty. RESULTS: Our model of patients age 60 with metastatic GEP-NETs suggests empiric initiation of SSA led to an increase 0.62 unadjusted life-years and incremental increase in quality-adjusted life years (QALYs) of 0.44. The incremental costs were $388,966 per QALY and not cost-effective at a willingness-to-pay threshold of $100,000. Death was attributed to GEP-NETs for 94.1% of patients in the SSA arm vs. 94.9% of patients in the DELAY SSA arm. Sensitivity analysis found that the model was most sensitive to costs of SSAs. Using probabilistic sensitivity analysis, the SSA strategy was only cost-effective 1.4% of the time at a WTP threshold of $100,000 per QALY. CONCLUSIONS: Our modeling study finds it is not cost-effective to initiate SSAs at time of presentation for patients with metastatic GEP-NETs. Further clinical studies are needed to identify the optimal timing to initiate these drugs.

On All Accounts: Cost-Effectiveness Analysis of Limited Preoperative Optimization Efforts Before Colon Cancer Surgery.

BACKGROUND: Reports suggest that preoperative optimization of a patient's serious comorbidities is associated with a reduction in postoperative complications. OBJECTIVE: The purpose of this study was to assess the cost and benefits of preoperative optimization, accounting for total costs associated with postoperative morbidity. DESIGN: This study is a decision tree cost-effectiveness analysis with probabilistic sensitivity analysis (10,000 iterations). SETTING: This is a hypothetical scenario of stage II colon cancer surgery. PATIENT: The simulated 65-year-old patient has left-sided, stage II colon cancer. INTERVENTION: Focused preoperative optimization targets high-risk comorbidities. OUTCOMES: Total discounted (3%) economic costs (US $2018), effectiveness (quality-adjusted life-years), incremental cost-effectiveness ratio (incremental cost-effectiveness ratio, cost/quality-adjusted life-years gained), and net monetary benefit. RESULTS: We calculated the per individual expected health care sector total cost of preoperative optimization and sequelae to be $12,395 versus $15,638 in those not optimized (net monetary benefit: $1.04 million versus $1.05 million). A nonoptimized patient attained an average 0.02 quality-adjusted life-years less than one optimized. Thus, preoperative optimization was the dominant strategy (lower total costs; higher quality-adjusted life-years). Probabilistic sensitivity analysis demonstrated 100% of simulations favoring preoperative optimization. The breakeven cost of optimization to remain cost-effective was $6421 per patient. LIMITATIONS: Generalizability must account for the lack of standardization among existing preoperative optimization efforts, and decision analysis methodology provides guidance for the average patient or general population, and is not patient-specific. CONCLUSIONS: Although currently not comprehensively reimbursed, focused preoperative optimization may reduce total costs of care while also reducing complications from colon cancer surgery. See Video Abstract at http://links.lww.com/DCR/B494. EN TODO CASO ANLISIS DE RENTABILIDAD DE LOS ESFUERZOS LIMITADOS DE OPTIMIZACIN PREOPERATORIA ANTES DE LA CIRUGA DE CNCER DE COLON: ANTECEDENTES:Los informes sugieren que la optimización preoperatoria de las comorbilidades graves de un paciente se asocia con una reducción de las complicaciones postoperatorias.OBJETIVO:El propósito de este estudio fue evaluar el costo y los beneficios de la optimización preoperatoria, teniendo en cuenta los costos totales asociados con la morbilidad postoperatoria.DISEÑO:Análisis de costo-efectividad de árbol de decisión con análisis de sensibilidad probabilístico (10,000 iteraciones).AJUSTE ENTORNO CLINICO:Escenario hipotético Cirugía de cáncer de colon en estadio II.PACIENTE:Paciente simulado de 65 años con cáncer de colon en estadio II del lado izquierdo.INTERVENCIÓN:Optimización preoperatoria enfocada dirigida a comorbilidades de alto riesgo.RESULTADOS:Costos económicos totales descontados (3%) (US $ 2018), efectividad (años de vida ajustados por calidad [AVAC]), relación costo-efectividad incremental (ICER, costo / AVAC ganado) y beneficio monetario neto (NMB).RESULTADOS:Calculamos que el costo total esperado por sector de atención médica individual de la optimización preoperatoria y las secuelas es de $ 12,395 versus $ 15,638 en aquellos no optimizados (NMB: $ 1.04 millones versus $ 1.05 millones, respectivamente). Un paciente no optimizado alcanzó un promedio de 0.02 AVAC menos que uno optimizado. Por lo tanto, la optimización preoperatoria fue la estrategia dominante (menores costos totales; mayores AVAC). El análisis de sensibilidad probabilístico demostró que el 100% de las simulaciones favorecían la optimización preoperatoria. El costo de equilibrio de la optimización para seguir siendo rentable fue de $ 6,421 por paciente.LIMITACIONES:La generalización debe tener en cuenta la falta de estandarización entre los esfuerzos de optimización preoperatorios existentes y esa metodología de análisis de decisiones proporciona una guía para el paciente promedio o la población general, no específica del paciente.CONCLUSIONES:Si bien actualmente no se reembolsa de manera integral, la optimización preoperatoria enfocada puede reducir los costos totales de la atención y al mismo tiempo reducir las complicaciones de la cirugía de cáncer de colon. Consulte Video Resumen en http://links.lww.com/DCR/B494.

Total Shoulder Arthroplasty Is Cost-Effective Compared with Hemiarthroplasty: A Real-World Economic Evaluation.

BACKGROUND: Although outcome studies generally demonstrate the superiority of a total shoulder arthroplasty (TSA) over a hemiarthroplasty (HA), comparative cost-effectiveness has not been well studied. From a publicly funded health-care system's perspective, this study compared the costs and quality-adjusted life-years (QALYs) in patients who underwent TSA with those in patients who underwent HA. METHODS: We conducted a cost-utility analysis using a Markov model to simulate the costs and QALYs for patients undergoing either TSA or HA over a lifetime horizon to account for costs and medically important events over the patient lifetime. Subgroup analyses by age groups (≤50 or >50 years) were performed. A series of sensitivity analyses were performed to assess robustness of study findings. The results were presented in 2019 U.S. dollars. RESULTS: TSA was dominant as it was less costly ($115,785 compared with $118,501) and more effective (10.21 compared with 8.47 QALYs) than HA over a lifetime horizon. Changes to health utility values after TSA and HA had the largest impact on the cost-effectiveness findings. At a willingness-to-pay (WTP) threshold of $50,000 per QALY gained, HA was not found to be cost-effective. The probability that TSA was cost-effective was 100%. CONCLUSIONS: Based on a WTP of $50,000 per QALY gained, from the perspective of Canada's publicly funded health-care system, TSA was found to be cost-effective in all patients, including those ≤50 years of age, compared with HA. LEVEL OF EVIDENCE: Economic and Decision Analysis Level II. See Instructions for Authors for a complete description of levels of evidence.

Can Cost-effectiveness Analysis Inform Genotype-Guided Aspirin Use for Primary Colorectal Cancer Prevention?

BACKGROUND: Inherited genetic variants can modify the cancer-chemopreventive effect of aspirin. We evaluated the clinical and economic value of genotype-guided aspirin use for colorectal cancer chemoprevention in average-risk individuals. METHODS: A decision analytical model compared genotype-guided aspirin use versus no genetic testing, no aspirin. The model simulated 100,000 adults ≥50 years of age with average colorectal cancer and cardiovascular disease risk. Low-dose aspirin daily starting at age 50 years was recommended only for those with a genetic test result indicating a greater reduction in colorectal cancer risk with aspirin use. The primary outcomes were quality-adjusted life-years (QALY), costs, and incremental cost-effectiveness ratio (ICER). RESULTS: The mean cost of using genotype-guided aspirin was $187,109 with 19.922 mean QALYs compared with $186,464 with 19.912 QALYs for no genetic testing, no aspirin. Genotype-guided aspirin yielded an ICER of $66,243 per QALY gained, and was cost-effective in 58% of simulations at the $100,000 willingness-to-pay threshold. Genotype-guided aspirin was associated with 1,461 fewer polyps developed, 510 fewer colorectal cancer cases, and 181 fewer colorectal cancer-related deaths. This strategy prevented 1,078 myocardial infarctions with 1,430 gastrointestinal bleeding events, and 323 intracranial hemorrhage cases compared with no genetic testing, no aspirin. CONCLUSIONS: Genotype-guided aspirin use for colorectal cancer chemoprevention may offer a cost-effective approach for the future management of average-risk individuals. IMPACT: A genotype-guided aspirin strategy may prevent colorectal cancer, colorectal cancer-related deaths, and myocardial infarctions, while minimizing bleeding adverse events. This model establishes a framework for genetically-guided aspirin use for targeted chemoprevention of colorectal cancer with application toward commercial testing in this population.