Financial Incentives and Treatment Outcomes in Adolescents With Severe Obesity: A Randomized Clinical Trial.

Importance: Adolescent severe obesity is usually not effectively treated with traditional lifestyle modification therapy. Meal replacement therapy (MRT) shows short-term efficacy for body mass index (BMI; calculated as weight in kilograms divided by height in meters squared) reduction in adolescents, and financial incentives (FIs) may be an appropriate adjunct intervention to enhance long-term efficacy. Objective: To evaluate the effect of MRT plus FIs vs MRT alone on BMI, body fat, and cardiometabolic risk factors in adolescents with severe obesity. Design, Setting, and Participants: This was a randomized clinical trial of MRT plus FIs vs MRT alone at a large academic health center in the Midwest conducted from 2018 to 2022. Participants were adolescents (ages 13-17 y) with severe obesity (≥120% of the 95th BMI percentile based on sex and age or ≥35 BMI, whichever was lower) who were unaware of the FI component of the trial until they were randomized to MRT plus FIs or until the end of the trial. Study staff members collecting clinical measures were blinded to treatment condition. Data were analyzed from March 2022 to February 2024. Interventions: MRT included provision of preportioned, calorie-controlled meals (~1200 kcals/d). In the MRT plus FI group, incentives were provided based on reduction in body weight from baseline. Main Outcomes and Measures: The primary end point was mean BMI percentage change from randomization to 52 weeks. Secondary end points included total body fat and cardiometabolic risk factors: blood pressure, triglyceride to high-density lipoprotein ratio, heart rate variability, and arterial stiffness. Cost-effectiveness was additionally evaluated. Safety was assessed through monthly adverse event monitoring and frequent assessment of unhealthy weight-control behaviors. Results: Among 126 adolescents with severe obesity (73 female [57.9%]; mean [SD] age, 15.3 [1.2] years), 63 participants received MRT plus FIs and 63 participants received only MRT. At 52 weeks, the mean BMI reduction was greater by -5.9 percentage points (95% CI, -9.9 to -1.9 percentage points; P = .004) in the MRT plus FI compared with the MRT group. The MRT plus FI group had a greater reduction in mean total body fat mass by -4.8 kg (95% CI, -9.1 to -0.6 kg; P = .03) and was cost-effective (incremental cost-effectiveness ratio, $39 178 per quality-adjusted life year) compared with MRT alone. There were no significant differences in cardiometabolic risk factors or unhealthy weight-control behaviors between groups. Conclusions and Relevance: In this study, adding FIs to MRT resulted in greater reductions in BMI and total body fat in adolescents with severe obesity without increased unhealthy weight-control behaviors. FIs were cost-effective and possibly promoted adherence to health behaviors. Trial Registration: ClinicalTrials.gov Identifier: NCT03137433.

Endoscopic Surveillance of Intestinal Metaplasia of the Esophagogastric Junction: A Decision Modeling Analysis.

INTRODUCTION: The incidence of esophagogastric junction adenocarcinoma (EGJAC) has been rising. Intestinal metaplasia of the esophagogastric junction (EGJIM) is a common finding in gastroesophageal reflux (irregular Z-line) and may represent an early step in the development of EGJAC in the West. Worldwide, EGJIM may represent progression along the Correa cascade triggered by Helicobacter pylori . We sought to evaluate the cost-effectiveness of endoscopic surveillance of EGJIM. METHODS: We developed a decision analytic model to compare endoscopic surveillance strategies for 50-year-old patients after diagnosis of non-dysplastic EGJIM: (i) no surveillance (standard of care), (ii) endoscopy every 3 years, (iii) endoscopy every 5 years, or (iv) 1-time endoscopy at 3 years. We modeled 4 progression scenarios to reflect uncertainty: A (0.01% annual cancer incidence), B (0.05%), C (0.12%), and D (0.22%). RESULTS: Cost-effectiveness of endoscopic surveillance depended on the progression rate of EGJIM to cancer. At the lowest progression rate (scenario A, 0.01%), no surveillance strategies were cost-effective. In moderate progression scenarios, 1-time surveillance at 3 years was cost-effective, at $30,989 and $16,526 per quality-adjusted life year for scenarios B (0.05%) and C (0.12%), respectively. For scenario D (0.22%), surveillance every 5 years was cost-effective at $77,695 per quality-adjusted life year. DISCUSSION: Endoscopic surveillance is costly and can cause harm; however, low-intensity longitudinal surveillance (every 5 years) is cost-effective in populations with higher EGJAC incidence. No surveillance or 1-time endoscopic surveillance of patients with EGJIM was cost-effective in low-incidence populations. Future studies to better understand the natural history of EGJIM, identify risk factors of progression, and inform appropriate surveillance strategies are required.

Cost-Effectiveness of Liquid Biopsy for Colorectal Cancer Screening in Patients Who Are Unscreened.

Importance: Despite recommendations for universal screening, adherence to colorectal cancer screening in the US is approximately 60%. Liquid biopsy tests are in development for cancer early detection, but it is unclear whether they are cost-effective for colorectal cancer screening. Objective: To estimate the cost-effectiveness of liquid biopsy for colorectal cancer screening in the US. Design, Setting, and Participants: In this economic evaluation, a Markov model was developed to compare no screening and 5 colorectal cancer screening strategies: colonoscopy, liquid biopsy, liquid biopsy following nonadherence to colonoscopy, stool DNA, and fecal immunochemical test. Adherence to first-line screening with colonoscopy, stool DNA, or fecal immunochemical test was assumed to be 60.6%, and adherence for liquid biopsy was assumed to be 100%. For colonoscopy, stool DNA, and fecal immunochemical test, patients who did not adhere to testing were not offered other screening. In colonoscopy-liquid biopsy hybrid, liquid biopsy was second-line screening for those who deferred colonoscopy. Scenario analyses were performed to include the possibility of polyp detection for liquid biopsy. Exposures: No screening, colonoscopy, fecal immunochemical test, stool DNA, liquid biopsy, and colonoscopy-liquid biopsy hybrid screening. Main Outcomes and Measures: Model outcomes included life expectancy, total cost, and incremental cost-effectiveness ratios. A strategy was considered cost-effective if it had an incremental cost-effectiveness ratio less than the US willingness-to-pay threshold of $100 000 per life-year gained. Results: This study used a simulated cohort of patients aged 45 years with average risk of colorectal cancer. In the base case, colonoscopy was the preferred, or cost-effective, strategy with an incremental cost-effectiveness ratio of $28 071 per life-year gained. Colonoscopy-liquid biopsy hybrid had the greatest gain in life-years gained but had an incremental cost-effectiveness ratio of $377 538. Colonoscopy-liquid biopsy hybrid had a greater gain in life-years if liquid biopsy could detect polyps but remained too costly. Conclusions and Relevance: In this economic evaluation of liquid biopsy for colorectal cancer screening, colonoscopy was a cost-effective strategy for colorectal cancer screening in the general population, and the inclusion of liquid biopsy as a first- or second-line screening strategy was not cost-effective at its current cost and screening performance. Liquid biopsy tests for colorectal cancer screening may become cost-effective if their cost is substantially lowered.

Cost-Effectiveness of Pharmacotherapy for the Treatment of Obesity in Adolescents.

Importance: Antiobesity pharmacotherapy is recommended for adolescents ages 12 years and older with obesity. Several medications have been approved by the US Food and Drug Administration for adolescent use, but the most cost-effective medication remains unclear. Objective: To estimate the cost-effectiveness of lifestyle counseling alone and as adjunct to liraglutide, mid-dose phentermine and topiramate (7.5 mg phentermine and 46 mg topiramate), top-dose phentermine and topiramate (15 mg phentermine and 92 mg topiramate), or semaglutide among adolescent patients with obesity. Design, Setting, and Participants: This economic evaluation used a microsimulation model to project health and cost outcomes of lifestyle counseling alone and adjunct to liraglutide, mid-dose phentermine and topiramate, top-dose phentermine and topiramate, or semaglutide over 13 months, 2 years, and 5 years among a hypothetical cohort of 100 000 adolescents with obesity, defined as an initial body mass index (BMI; calculated as weight in kilograms divided by height in meters squared) of 37. Model inputs were derived from clinical trials, published literature, and national sources. Data were analyzed from April 2022 to July 2023. Exposures: Lifestyle counseling alone and as adjunct to liraglutide, mid-dose phentermine and topiramate, top-dose phentermine and topiramate, or semaglutide. Main Outcomes and Measures: The main outcome was quality-adjusted life years (QALYs), costs (2022 US dollars), and incremental cost-effectiveness ratios (ICERs), with future costs and QALYs discounted 3.0% annually. A strategy was considered cost-effective if the ICER was less than $100 000 per QALY gained. The preferred strategy was determined as the strategy with the greatest increase in QALYs while being cost-effective. One-way and probabilistic sensitivity analyses were used to assess parameter uncertainty. Results: The model simulated 100 000 adolescents at age 15 with an initial BMI of 37, of whom 58 000 (58%) were female. At 13 months and 2 years, lifestyle counseling was estimated to be the preferred strategy. At 5 years, top-dose phentermine and topiramate was projected to be the preferred strategy with an ICER of $56 876 per QALY gained vs lifestyle counseling. Semaglutide was projected to yield the most QALYs, but with an unfavorable ICER of $1.1 million per QALY gained compared with top-dose phentermine and topiramate. Model results were most sensitive to utility of weight reduction and weight loss of lifestyle counseling and top-dose phentermine and topiramate. Conclusions and Relevance: In this economic evaluation of pharmacotherapy for adolescents with obesity, top-dose phentermine and topiramate as adjunct to lifestyle counseling was estimated to be cost-effective after 5 years. Long-term clinical trials in adolescents are needed to fully evaluate the outcomes of pharmacotherapy, especially into adulthood.

Endoscopic Screening Program for Control of Esophageal Adenocarcinoma in Varied Populations: A Comparative Cost-Effectiveness Analysis.

BACKGROUND & AIMS: Guidelines suggest endoscopic screening for esophageal adenocarcinoma (EAC) among individuals with symptoms of gastroesophageal reflux disease (GERD) and additional risk factors. We aimed to determine at what age to perform screening and whether sex and race should influence the decision. METHODS: We conducted comparative cost-effectiveness analyses using 3 independent simulation models. For each combination of sex and race (White/Black, 100,000 individuals each), we considered 41 screening strategies, including one-time or repeated screening. The optimal strategy was that with the highest effectiveness and an incremental cost-effectiveness ratio <$100,000 per quality-adjusted life-year gained. RESULTS: Among White men, 536 EAC deaths were projected without screening, and screening individuals with GERD twice at ages 45 and 60 years was optimal. Screening the entire White male population once at age 55 years was optimal in 26% of probabilistic sensitivity analysis runs. Black men had fewer EAC deaths without screening (n = 84), and screening those with GERD once at age 55 years was optimal. Although White women had slightly more EAC deaths (n = 103) than Black men, the optimal strategy was no screening, although screening those with GERD once at age 55 years was optimal in 29% of probabilistic sensitivity analysis runs. Black women had a very low burden of EAC deaths (n = 29), and no screening was optimal, as benefits were very small and some strategies caused net harm. CONCLUSIONS: The optimal strategy for screening differs by race and sex. White men with GERD symptoms can potentially be screened more intensely than is recommended currently. Screening women is not cost-effective and may cause net harm for Black women.

Endoscopic Balloon Dilation Is Cost-Effective for Crohn's Disease Strictures.

BACKGROUND: Endoscopic balloon dilation (EBD) has emerged as an alternative intervention to manage Crohn's disease (CD) strictures. We determined the cost-effectiveness of EBD versus resection surgery for patients with short (< 4-5 cm) primary or secondary/anastomotic small or large bowel strictures. METHODS: A microsimulation state-transition model analyzed the benefits and risks of EBD and resection surgery for patients with primary or anastomotic CD strictures. Our primary outcome was quality-adjusted life years (QALYs) over ten years, and strategies were compared using a willingness to pay of $100,000/QALY from a societal perspective. Costs (2021 $US) and incremental cost-effectiveness ratios (ICER) were calculated. Deterministic 1-way and probabilistic analyses assessed model uncertainty. RESULTS: The EBD strategy cost $19,822 and resulted in 6.18 QALYs while the surgery strategy cost $41,358 and resulted in 6.37 QALYs. Surgery had an ICER of $113,332 per QALY, making EBD a cost-effective strategy. The median number of EBDs was 5 in the EBD strategy and 0 in the surgery strategy. The median number of surgeries was 2 in the surgery strategy and 1 in the EBD strategy. Of individuals who initially received EBD, 50.4% underwent subsequent surgery. One-way sensitivity analyses showed that the probabilities of requiring repeated interventions, surgery mortality (< 0.7%), and quality of life after interventions were the most influential model parameters. Probabilistic sensitivity analyses favored EBD in 50.9% of iterations. CONCLUSIONS: EBD is a cost-effective strategy for managing CD strictures. Differences in patient risk and quality of life after intervention impact cost-effectiveness. Intervention decisions should consider cost-effectiveness, patient risks, and quality of life.

Cost-effectiveness of neoadjuvant FOLFIRINOX versus gemcitabine plus nab-paclitaxel in borderline resectable/locally advanced pancreatic cancer patients.

BACKGROUND: The 2020 National Comprehensive Cancer Network guidelines recommend neoadjuvant FOLFIRINOX or neoadjuvant gemcitabine plus nab-paclitaxel (G-nP) for borderline resectable/locally advanced pancreatic ductal adenocarcinoma (BR/LA PDAC). AIM: The purpose of our study was to compare treatment outcomes, toxicity profiles, costs, and quality-of-life measures between these two treatments to further inform clinical decision-making. METHODS AND RESULTS: We developed a decision-analytic mathematical model to compare the total cost and health outcomes of neoadjuvant FOLFIRINOX against G-nP over 12 years. The model inputs were estimated using clinical trial data and published literature. The primary endpoint was incremental cost-effectiveness ratios (ICERs) with a willingness-to-pay threshold of $100 000 per quality-adjusted-life-year (QALY). Secondary endpoints included overall (OS) and progression-free survival (PFS), total cost of care, QALYs, PDAC resection rate, and monthly treatment-related adverse events (TRAE) costs (USD). FOLFIRINOX was the cost-effective strategy, with an ICER of $60856.47 per QALY when compared to G-nP. G-nP had an ICER of $44639.71 per QALY when compared to natural history. For clinical outcomes, more patients underwent an "R0" resection with FOLFIRINOX compared to G-nP (84.9 vs. 81.0%), but FOLFIRINOX had higher TRAE costs than G-nP ($10905.19 vs. $4894.11). A one-way sensitivity analysis found that the ICER of FOLFIRINOX exceeded the threshold when TRAE costs were higher or PDAC recurrence rates were lower. CONCLUSION: Our modeling analysis suggests that FOLFIRNOX is the cost-effective treatment compared to G-nP for BR/LA PDAC despite having a higher cost of total care due to TRAE costs. Trial data with sufficient follow-up are needed to confirm our findings.

Estimated Cost-effectiveness of Medical Therapy, Sleeve Gastrectomy, and Gastric Bypass in Patients With Severe Obesity and Type 2 Diabetes.

Importance: Bariatric surgery is recommended for patients with severe obesity (body mass index ≥40) and type 2 diabetes (T2D). However, the most cost-effective treatment remains unclear and may depend on the patient's T2D severity. Objective: To estimate the cost-effectiveness of medical therapy, sleeve gastrectomy (SG), and Roux-en-Y gastric bypass (RYGB) among patients with severe obesity and T2D, stratified by T2D severity. Design, Setting, and Participants: This economic evaluation used a microsimulation model to project health and cost outcomes of medical therapy, SG, and RYGB over 5 years. Time horizons varied between 10 and 30 years in sensitivity analyses. Model inputs were derived from clinical trials, large cohort studies, national databases, and published literature. Probabilistic sampling of model inputs accounted for parameter uncertainty. Estimates of US adults with severe obesity and T2D were derived from the National Health and Nutrition Examination Survey. Data analysis was performed from January 2020 to August 2021. Exposures: Medical therapy, SG, and RYGB. Main Outcomes and Measures: Quality-adjusted life-years (QALYs), costs (in 2020 US dollars), and incremental cost-effectiveness ratios (ICERs) were projected, with future cost and QALYs discounted 3.0% annually. A strategy was deemed cost-effective if the ICER was less than $100 000 per QALY. The preferred strategy resulted in the greatest number of QALYs gained while being cost-effective. Results: The model simulated 1000 cohorts of 10 000 patients, of whom 16% had mild T2D, 56% had moderate T2D, and 28% had severe T2D at baseline. The mean age of simulated patients was 54.6 years (95% CI, 54.2-55.0 years), 61.6% (95% CI, 60.1%-63.4%) were female, and 65.1% (95% CI, 63.6%-66.7%) were non-Hispanic White. Compared with medical therapy over 5 years, RYGB was associated with the most QALYs gained in the overall population (mean, 0.44 QALY; 95% CI, 0.21-0.86 QALY) and when stratified by baseline T2D severity: mild (mean, 0.59 QALY; 95% CI, 0.35-0.98 QALY), moderate (mean, 0.50 QALY; 95% CI, 0.25-0.88 QALY), and severe (mean, 0.30 QALY; 95% CI, 0.07-0.79 QALY). RYGB was the preferred strategy in the overall population (ICER, $46 877 per QALY; 83.0% probability preferred) and when stratified by baseline T2D severity: mild (ICER, $36 479 per QALY; 73.7% probability preferred), moderate (ICER, $37 056 per QALY; 85.6% probability preferred), and severe (ICER, $98 940 per QALY; 40.2% probability preferred). The cost-effectiveness of RYGB improved over a longer time horizon. Conclusions and Relevance: These findings suggest that the effectiveness and cost-effectiveness of bariatric surgery vary by baseline severity of T2D. Over a 5-year time horizon, RYGB is projected to be the preferred treatment strategy for patients with severe obesity regardless of baseline T2D severity.

Cost-effectiveness of Venous Thromboembolism Prophylaxis After Hospitalization in Patients With Inflammatory Bowel Disease.

BACKGROUND: Patients with inflammatory bowel disease (IBD) have a 2- to 3-fold greater risk of venous thromboembolism (VTE) than patients without IBD, with increased risk during hospitalization that persists postdischarge. We determined the cost-effectiveness of postdischarge VTE prophylaxis among hospitalized patients with IBD. METHODS: A decision tree compared inpatient prophylaxis alone vs 4 weeks of postdischarge VTE prophylaxis with 10 mg/day of rivaroxaban. Our primary outcome was quality-adjusted life years (QALYs) over 1 year, and strategies were compared using a willingness to pay of $100,000/QALY from a societal perspective. Costs (in 2020 $USD), incremental cost-effectiveness ratios (ICERs) and number needed to treat (NNT) to prevent 1 VTE and VTE death were calculated. Deterministic 1-way and probabilistic analyses assessed model uncertainty. RESULTS: Prophylaxis with rivaroxaban resulted in 1.68-higher QALYs per 1000 persons compared with no postdischarge prophylaxis at an incremental cost of $185,778 per QALY. The NNT to prevent a single VTE was 78, whereas the NNT to prevent a single VTE-related death was 3190. One-way sensitivity analyses showed that higher VTE risk >4.5% and decreased cost of rivaroxaban ≤$280 can reduce the ICER to <$100,000/QALY. Probabilistic sensitivity analyses favored prophylaxis in 28.9% of iterations. CONCLUSIONS: Four weeks of postdischarge VTE prophylaxis results in higher QALYs compared with inpatient prophylaxis alone and prevents 1 postdischarge VTE among 78 patients with IBD. Although postdischarge VTE prophylaxis for all patients with IBD is not cost-effective, it should be considered in a case-by-case scenario, considering VTE risk profile, costs, and patient preference.

Cost-effectiveness and budget impact analyses of tisagenlecleucel in adult patients with relapsed or refractory diffuse large B-cell lymphoma from Singapore's private insurance payer's perspective.

BACKGROUND: Patients experiencing relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) have limited treatment options and poor prognosis. Tisagenlecleucel (TIS) has shown improved clinical outcomes, but at a high upfront cost. Singapore has a multi-payer healthcare system where private insurance is one of the major payers. This study evaluated the cost-effectiveness and budget impact of TIS against salvage chemotherapy regimen (SCR) for treating r/r DLBCL patients who have failed ≥2 lines of systemic therapy from Singapore's private insurance payer's perspective. METHODS: Over a life-time horizon, a partitioned survival model with three health-states was developed to evaluate the cost-effectiveness of TIS vs. SCR with or without hematopoietic stem cell transplantation (HSCT). Efficacy inputs for TIS and SCR were based on 43 months of observation data from pooled JULIET and UPenn trials, and CORAL extension studies respectively. Direct costs for pre-treatment, treatment, adverse events, follow-up, subsequent-HSCT, relapse, and terminal care were included. Incremental cost-effectiveness ratios (ICERs) were calculated as the total incremental costs per quality-adjusted life-year (QALY) gained. Additionally, the financial implication of introducing TIS in Singapore from a private payer's perspective was analyzed, comparing the current treatment pathway (without TIS) with a future scenario (with TIS) over 5 years. RESULTS: Compared with SCR, TIS was the dominant option, with cost savings of S$8,477 alongside an additional gain of 2.78 QALYs in privately insured patients who shifted from private to public hospitals for TIS treatment. Scenario analyses for patients starting in public hospitals show ICERs of S$99,623 (no subsidy) and S$133,261 (50% subsidy for SCR treatment, no subsidy for TIS), supporting the base case. The projected annual budget impact ranges from S$850,000 to S$3.4 million during the first 5 years. CONCLUSIONS: TIS for treating r/r DLBCL patients who have failed ≥2 lines of systemic therapies, is likely to be cost effective with limited budget impact.