Group-based trajectory analysis identifies varying diabetes-related cost trajectories among type 2 diabetes patients in Texas: an empirical study using commercial insurance.

BACKGROUND: The trend of Type 2 diabetes-related costs over 4 years could be classified into different groups. Patient demographics, clinical factors (e.g., A1C, short- and long-term complications), and rurality could be associated with different trends of cost. Study objectives are to: (1) understand the trajectories of cost in different groups; (2) investigate the relationship between cost and key factors in each cost trajectory group; and (3) assess significant factors associated with different cost trajectories. METHODS: Commercial claims data in Texas from 2016 to 2019 were provided by a large commercial insurer and were analyzed using group-based trajectory analysis, longitudinal analysis of cost, and logistic regression analyses of different trends of cost. RESULTS: Five groups of distinct trends of Type 2 diabetes-related cost were identified. Close to 20% of patients had an increasing cost trend over the 4 years. High A1C values, diabetes complications, and other comorbidities were significantly associated with higher Type 2 diabetes costs and higher chances of increasing trend over time. Rurality was significantly associated with higher chances of increasing trend over time. CONCLUSIONS: Group-based trajectory analysis revealed distinct patient groups with increased cost and stable cost at low, medium, and high levels in the 4-year period. The significant associations found between the trend of cost and A1C, complications, and rurality have important policy and program implications for potentially improving health outcomes and constraining healthcare costs.

Factors associated with higher hemoglobin A1c and type 2 diabetes-related costs: Secondary data analysis of adults 18 to 64 in Texas with commercial insurance.

OBJECTIVE: This study will identify factors associated with higher hemoglobin A1c (A1c) values and diabetes-related costs among commercially insured adults in Texas diagnosed with type 2 diabetes. RESEARCH DESIGN AND METHODS: This secondary data analysis was based on claims data from commercially insured individuals 18-64 years of age residing in Texas with diagnosed type 2 diabetes during the 2018-2019 study period. The final analysis sample after all the exclusions consisted of 34,992 individuals. Measures included hemoglobin A1c, diabetes-related costs, Charlson Comorbidity Index, diabetes-related complications, rurality and other socioeconomic characteristics. Longitudinal A1c measurements were modeled using age, sex, rurality, comorbidity, and diabetes-related complications in generalized linear longitudinal regression models adjusting the observation time, which was one of the 8 quarters in 2018 and 2019. The diabetes-related costs were similarly modeled in both univariable and multivariable generalized linear longitudinal regression models adjusting the observation time by calendar quarters and covariates. RESULTS: The median A1c value was 7, and the median quarterly diabetes-related cost was $120. A positive statistically significant relationship (p = < .0001) was found between A1c levels and diabetes-related costs, although this trend slowed down as A1c levels exceeded 8.0%. Higher A1c values were associated with being male, having diabetes-related complications, and living in rural areas. Higher costs were associated with higher A1c values, older age, and higher Charlson Comorbidity Index scores. CONCLUSION: The study adds updated analyses of the interrelationships among demographic and geographic factors, clinical indicators, and health-related costs, reinforcing the role of higher A1c values and complications as diabetes-related cost drivers.

Disease burden of prostate cancer from 2014 to 2019 in the United States: estimation from the Global Burden of Disease Study 2019 and Medical Expenditure Panel Survey.

OBJECTIVES: The aim of this study was to evaluate the disease burden of prostate cancer (PC) and assess key influencing factors associated with the disease expenditures of PC in the United States. METHODS: The total deaths, incidence, prevalence, and disability-adjusted life-years of PC were obtained from the Global Burden of Disease Study 2019. The Medical Expenditure Panel Survey was used to estimate healthcare expenditures and productivity loss and to investigate patterns of payment and use of healthcare resources in the United States. A multivariable logistic regression model was conducted to identify key factors influencing expenditures. RESULTS: For patients aged 50 and older, the burden for all age groups showed a modest increase over the 6-year period. Annual medical expenditures were estimated to range from US$24.8 billion to US$39.2 billion from 2014 to 2019. The annual loss in productivity for patients was approximately US$1,200. The top 3 major components of medical costs were hospital inpatient stays, prescription medicines, and office-based visits. Medicare was the largest source of payments for survivors. In terms of drug consumption, genitourinary tract agents (57.0%) and antineoplastics (18.6%) were the main therapeutic drugs. High medical expenditures were positively associated with age (p=0.005), having private health insurance (p=0.016), more comorbidities, not currently smoking (p=0.001), and patient self-perception of fair/poor health status (p<0.001). CONCLUSIONS: From 2014 to 2019, the national real-world data of PC revealed that the disease burden in the United States continued to increase, which was partly related to patient characteristics.

Avelumab Maintenance Treatment After First-line Chemotherapy in Advanced Urothelial Carcinoma-A Cost-Effectiveness Analysis.

BACKGROUND: Recently, a clinical trial (NCT02603432) showed that avelumab maintenance treatment, post first-line chemotherapy, can significantly prolong the overall survival of patients with advanced urothelial carcinoma (UC), however, the treatment was very expensive. This study aimed to determine the cost-effectiveness of avelumab maintenance therapy in advanced or metastatic UC from the US taxpayer perspective. METHODS: Based on the data of the JAVELIN Bladder 100 clinical trial (NCT02603432), a Markov multi-state model was constructed to investigate the costs and clinical outcomes of avelumab maintenance after platinum-based chemotherapy versus best supportive care (BSC) for advanced or metastatic UC. Parameters of the model came from the 2020 Average Sales Price Drug Pricing Files and published literature. The main outputs were costs, life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). Robustness was tested by deterministic and probabilistic sensitivity analyses. The analysis was stratified to include both the overall population and a subset of programmed death-ligand 1 (PD-L1)-positive patients. RESULTS: Avelumab maintenance therapy was estimated to generate an additional 0.26 QALYs (1.46 vs. 1.20 QALYs) and costs $183,271 ($278,323 vs. $95,052) more compared to BSC alone in the overall population, yielding an ICER of $699,065/QALY. For the PD-L1-positive population, avelumab produced a 0.42 increase in QALYs (1.74 vs. 1.32 QALYs) and raised costs to $223,238 ($320,355 vs. $97,117), resulting in an ICER of $521,850/QALY for this population. Both ICERs were above the willingness-to-pay (WTP) threshold of $200,000/QALY. Sensitivity analyses manifested that the model was robust. CONCLUSION: From the perspective of the US taxpayer, avelumab maintenance therapy is considered cost-ineffective for patients with advanced or metastatic UC at a WTP threshold of $200,000/QALY in the overall population as well as in PD-L1-positive population.

Physicians' interest in different strategies for supporting pain -management and opioid prescribing: A cross-sectional study.

OBJECTIVE: The purpose of this study was to explore physicians' attitudes toward different strategies for supporting pain management and opioid prescribing and to identify factors related to their attitudes toward the support strategies. Design/setting/participants/measures: This preliminary cross-sectional study collected and analyzed online survey responses from physicians in Texas and Minnesota (N = 69) between December 2017 and February 2018. Primary outcomes were physicians' interest in online continuing medical education (CME), mHealth patient monitoring system, and short, non-CME YouTube informational briefs about pain management and opioid prescribing. Multiple logistic regression models were used to examine the associations between physicians' characteristics, attitudes, training, experience, practice setting, and their interest in three different support strategies. RESULTS: About 51-58 percent of physicians indicated moderate-to-extreme interest in online CME (54 percent), mHealth monitoring (58 percent), and short, non-CME YouTube informational briefs (51 percent). Physicians, who practiced in a medium or large practice setting, were less likely to be interested in online CME or short, non-CME YouTube informational briefs. Physicians who prescribed a small number of Schedule II opioids were more likely to be interested in short, non-CME YouTube informational briefs and mHealth monitoring. CONCLUSIONS: Findings suggest that physicians may have different preferences in strategies for supporting their pain management and opioid prescribing practices. Future studies are needed to better understand the mechanisms underlying physicians' interest in different support strategies.

Cost-effectiveness analysis of primary treatments for localised prostate cancer: A population-based Markov analysis using real-world evidence.

OBJECTIVE: We evaluate cost-effectiveness of primary treatments for localised prostate cancer by uniquely combining prospectively collected real-world outcomes and costs from UCSF Cancer of Prostate Strategic Urologic Research Endeavor (CaPSURE™). METHODS: Markov models assessed cost-effectiveness of radical prostatectomy (RP), brachytherapy, electron beam radiation therapy (EBRT) and brachytherapy with EBRT by risk from US payers perspective over 8 years. Treatment costs included office visits, hospitalisation, procedures, medication and long-term care. Patients' surveyed HRQoL were mapped into utilities. Incremental cost-effectiveness ratios (ICERs) used cost per quality-adjusted life years (QALYs) and willingness-to-pay of $150,000/QALY. RESULTS: Cost-effectiveness analysis (CEA) showed for low-risk prostate cancer, EBRT dominated the lowest cost brachytherapy, but RPns and brachytherapy plus EBRT were cost-effective compared to brachytherapy with ICERs of $18,926 and $41,662 per QALY. In medium-risk patients, RP, EBRT and brachytherapy plus EBRT all were cost-effective compared with brachytherapy, with ICERs of $30,604, $22,588 and $21,627/QALY. In high-risk, brachytherapy dominated all treatments. Procedure cost and utility are driving ICER, but probabilistic sensitivity analysis showed the model was robust across variables. CONCLUSION: This first CEA combining prospective real-world evidence for HRQOL outcomes with costs shows cost-effectiveness of treatments vary by risk groups, providing new evidence for treatment decisions.

Is adding ribociclib to fulvestrant cost-effective in treating postmenopausal women with HR+/HER2- advanced or metastatic breast cancer? A US payer perspective cost utility analysis.

BACKGROUND: Breast cancer is the most prevalent type of cancer in women in the United States. Ribociclib plus fulvestrant combination therapy gained US Food and Drug Administration approval to treat postmenopausal women with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) advanced or metastatic breast cancer in 2018. OBJECTIVE: To determine the cost-effectiveness of ribociclib plus fulvestrant vs placebo plus fulvestrant therapy in the target population from a US payer perspective. METHODS: A partitioned survival analysis model composed of 3 health states (progression free, progressed disease, and death) was constructed to evaluate the cost-effectiveness of ribociclib plus fulvestrant vs placebo plus fulvestrant. The progression-free survival and the overall survival data points were extracted from published Kaplan-Meier curves in the MONALEESA-3 study and fitted to parametric curves. The safety and efficacy of the treatment was referenced from the MONALEESA-3 trial. Costs were obtained from standard sources including the Red Book for medication costs, Medicare Clinical Laboratory/Physician Fee Schedule for clinical utilization, and the literature for costs of managing adverse events, subsequent therapy, and end-of-life care. Utility and disutility values were obtained from literature to calculate quality-adjusted life-years (QALYs). One-way and probabilistic sensitivity analyses were conducted to test the model robustness. Several scenario analyses were also investigated. RESULTS: In the base case, the ribociclib plus fulvestrant arm was associated with $522,844 and 3.25 QALYs compared with $50,395 and 2.14 QALYs in the placebo plus fulvestrant arm, leading to an incremental cost-effectiveness ratio of $425,951/QALY. The cost of ribociclib had the biggest impact on the model and constituted 84% of the total cost for the ribociclib plus fulvestrant arm. The probabilistic sensitivity analysis projected that the ribociclib plus fulvestrant treatment would have a net benefit over the placebo plus fulvestrant therapy at a willingness-to-pay (WTP) threshold of $405,600/QALY. CONCLUSIONS: At a WTP threshold of $150,000/QALY, the addition of ribociclib to fulvestrant is not considered to be cost-effective in postmenopausal women with HR+/HER2- advanced or metastatic breast cancer. The findings send a strong price signal to the manufacturer and can be used to facilitate payers with price negotiation in making coverage decisions.

Cost-Effectiveness Analysis of Atezolizumab Versus Durvalumab as First-Line Treatment of Extensive-Stage Small-Cell Lung Cancer in the USA.

BACKGROUND AND OBJECTIVES: Durvalumab and atezolizumab are approved as first-line therapy in extensive-stage small-cell lung cancer. Although cost-effectiveness analyses compared these immunotherapy drugs with standard chemotherapy-alone regimens, no head-to-head cost-effectiveness comparisons for these treatments exist. The aim of the present analysis is to determine the cost-effectiveness of durvalumab and atezolizumab as first-line therapy for extensive-stage small-cell lung cancer from the US payers' perspective. METHODS: This study is based on two placebo-controlled, phase 3 clinical trials: CASPIAN and IMpower133. A Markov model was developed to simulate the three health states: progression-free survival, progressed disease, and death in patients with extensive-stage small-cell lung cancer. Transition probabilities were estimated from the clinical trial survival curves and extended with life-time modelling. Health utilities and direct costs of adverse event treatment were included. Main outcome was the incremental cost-effectiveness ratio (ICER) using quality-adjusted life-years saved (QALYS). Sensitivity analysis was performed to assess the impact of variables on the ICER. RESULTS: Durvalumab group has a cost of $187,503 with an effectiveness of 1.08 while atezolizumab has a cost of $160,219 and an effectiveness of 0.932. Durvalumab is not cost-effective compared to atezolizumab with an ICER of $165,182 QALYS, which is over the willingness-to-pay threshold of $150,000. The model was most sensitive to durvalumab cost and the cost of treating durvalumab adverse effects. CONCLUSIONS: With the ICER of durvalumab treatment group being very close to $150,000, setting a higher willingness-to-pay threshold or decreasing the drug cost through contract pricing can increase the cost-effectiveness of durvalumab compared to atezolizumab.

Real-World Data on Liposomal Bupivacaine and Inpatient Hospital Costs After Colorectal Surgery.

INTRODUCTION: This study compared costs of care among colorectal surgery patients who received liposomal bupivacaine versus those who did not (control) from a health institution perspective. MATERIAL AND METHODS: This pharmacoeconomic evaluation was conducted among adults undergoing open or minimally invasive colorectal resection at an academic medical center from May 2016 to February 2018. Healthcare resource utilization was derived from the electronic health record. Total cost of care (2018 USD) was analyzed using a generalized linear model adjusted for American Society of Anesthesiologists score, enhanced recovery after surgery management, open surgery, opioid use before surgery, height, cancer, and age. The primary analysis used public costs. A sensitivity analysis used internal costs from the hospital to maximize internal validity. RESULTS: Of 486 included patients, 286 (59%) received liposomal bupivacaine. Total cost of care using public costs included perioperative local anesthetics (mean ± standard deviation [SD]: $392 ± 74 liposomal bupivacaine versus $8 ± 13 control), analgesics within 48 h after surgery (mean ± SD: $132 ± 99 liposomal bupivacaine versus $117 ± 127 control), postoperative ileus management (mean ± SD: $5 ± 51 liposomal bupivacaine versus $65 ± 284 control), and hospital length of stay (mean ± SD: $4459 ± 3576 liposomal bupivacaine versus $7769 ± 7082 control). Liposomal bupivacaine was associated with an adjusted absolute difference in total cost of care of -$1435 (95% confidence interval -$2401 to -$470; P = 0.004) using public costs and -$1345 (95% confidence interval -$2215 to -$476; P = 0.002) using internal costs. CONCLUSIONS: Use of liposomal bupivacaine in colorectal surgery was associated with a significant reduction in total cost of care that was predominately driven by reduced costs for hospital stay and postoperative ileus management despite higher medication costs.

A Cost-effectiveness Analysis Comparing Pembrolizumab-Axitinib, Nivolumab-Ipilimumab, and Sunitinib for Treatment of Advanced Renal Cell Carcinoma.

OBJECTIVES: The US Food and Drug Administration (FDA) approved nivolumab-ipilimumab and pembrolizumab-axitinib as first-line treatments for metastatic, clear-cell, renal cell carcinoma (mRCC) based on results from CheckMate 214 and KEYNOTE-426. Our objective was to compare the adjusted, lifetime cost-effectiveness between nivolumab-ipilimumab, pembrolizumab-axitinib, and sunitinib for patients with mRCC. MATERIALS AND METHODS: A 3-state Markov model was developed comparing nivolumab-ipilimumab and pembrolizumab-axitinib to each other and sunitinib, over a 20-year lifetime horizon from a US medical center perspective. The clinical outcomes of nivolumab-ipilimumab and pembrolizumab-axitinib were compared using matching-adjusted indirect comparison. Costs of drug treatment, adverse events, and utilities associated with different health states and adverse events were determined using national sources and published literature. Our outcome was incremental cost-effectiveness ratio (ICER) using quality-adjusted life years (QALY). One-way and probabilistic sensitivity analyses were conducted. RESULTS: Nivolumab-ipilimumab was the most cost-effective option in the base case analysis with an ICER of $34,190/QALY compared with sunitinib, while the pembrolizumab-axitinib ICER was dominated by nivolumab-ipilimumab and was not cost-effective (ICER=$12,630,828/QALY) compared with sunitinib. The mean total costs per patient for the nivolumab-ipilimumab and pembrolizumab-axitinib arms were $284,683 and $457,769, respectively, compared with sunitinib at $241,656. QALY was longer for nivolumab-ipilimumab (3.23 QALY) than for adjusted pembrolizumab-axitinib (1.99 QALY), which was longer than sunitinib's (1.98 QALY). These results were most sensitive to treatment cost in both groups, but plausible changes did not alter the conclusions. CONCLUSIONS: The base case scenario indicated that nivolumab-ipilimumab was the most cost-effective treatment option for mRCC compared with pembrolizumab-axitinib and sunitinib.

Evaluation of Tucatinib in HER2-Positive Breast Cancer Patients With Brain Metastases: A United States-Based Cost-Effectiveness Analysis.

PURPOSE: To evaluate the cost-effectiveness of tucatinib in human epidermal growth factor receptor 2 (HER2)-positive breast cancer (BC) patients with brain metastases (BMs) and the subgroup of active BMs from the United States (US) payer perspective. MATERIALS AND METHODS: A 3-state Markov model was developed to compare the cost-effectiveness of 2 regimens in HER2-positive BC patients with BMs: (1) tucatinib, trastuzumab, and capecitabine (TTC); (2) placebo, trastuzumab, and capecitabine (PTC). And subgroup analysis of active BMs was also performed. Lifetime costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER) and incremental net-health benefit (INHB) were estimated. The willingness-to-pay (WTP) threshold was $200,000/QALY. The robustness of the model was tested by sensitivity analyses. Additional scenario analysis was also performed. RESULTS: Compared with PTC, the ICER yielded by TTC was $418,007.01/QALY and the INHB was -1.08 QALYs in patients with BMs. In the subgroup of active BMs, the ICER and the INHB were $324,465.03/QALY and -0.71 QALY, respectively. The results were most sensitive to the cost of tucatinib. Probabilistic sensitivity analyses suggested that the cost-effective probability of TTC was low at the current WTP threshold in the patients with BMs and the subgroup of active BMs. CONCLUSION: Tucatinib is unlikely to be cost-effective in HER2-positive BC patients with BMs from the US payer perspective but shows better economics in patients with active BMs. Selecting a favorable population, reducing the price of tucatinib or offering appropriate drug assistance policies might be considerable options to optimize the cost-effectiveness of tucatinib.

The economic burden of systemic sclerosis-A systematic review.

AIM: Systemic sclerosis (SSc) is a rare, chronic autoimmune disease associated with a substantial economic burden. This study aimed to assess the costs associated with SSc and to identify major cost drivers. METHODS: A systematic search was conducted in MEDLINE and Embase to identify relevant studies. Two independent reviewers evaluated studies based on inclusion/exclusion criteria and performed data extraction. Costs were converted into 2017 US dollars by purchasing power parity. The review was conducted following the Preferred Reporting Items for Systematic Review and Meta-analysis (PRISMA) guideline. RESULTS: The original literature search identified 113 potentially relevant citations, of which 10 articles met all the inclusion/exclusion criteria and were included in the data extraction and analysis. The identified studies evaluated costs associated with SSc in 11 countries from North America, Europe, and Australia published between 2009 and 2018. Eight studies reported direct costs and seven studies reported indirect costs. Direct costs varied from $3356 (Hungary) to $27 032 (Germany) with hospitalization and medication being two of the biggest components of direct medical costs in most studies. The indirect costs for lost productivity varied from $2433 (Italy) to $20 663 (UK), accounting for a significant portion of the total economic burden. CONCLUSIONS: Large variations existed in annual costs of SSc, but all studies found that SSc imposed a substantial economic burden on patients and their families. The identified studies were mostly from high-income countries and there is still a knowledge gap regarding the cost of SSc in other parts of the world.

Cost-effectiveness of adding daratumumab or bortezomib to lenalidomide plus dexamethasone for newly diagnosed multiple myeloma.

BACKGROUND: Multiple myeloma survival rates are steadily increasing due to availability of new drug classes used in combination with corticosteroids and chemotherapy. The latest treatments are daratumumab or bortezomib in combination therapy with lenalidomide and dexamethasone (Rd). Daratumumab, a CD38-targeted, human IgG1k monoclonal antibody, and bortezomib, a proteasome inhibitor, are both approved as regimens for transplant-ineligible relapsed/refractory multiple myeloma (RRMM). There have been cost-effectiveness analyses for daratumumab and bortezomib use in RRMM, but there are limited data regarding cost-effectiveness for daratumumab or bortezomib use in newly diagnosed multiple myeloma patients who are ineligible for stem cell transplantation. OBJECTIVE: To compare the cost-effectiveness of 3 separate regimens-(1) daratumumab, lenalidomide, and dexamethasone triple therapy (DRd); (2) bortezomib and lenalidomide plus dexamethasone triple therapy (VRd); and (3) lenalidomide plus dexamethasone (Rd)-in patients with multiple myeloma ineligible for autologous stem cell transplant. METHODS: A 2-state Markov model was developed using a US health system perspective and lifetime time horizon. Transition probabilities were calculated from the latest progression-free survival data reported in two phase 3 randomized controlled trials-MAIA and SWOG S0777-and extrapolated using a Weibull distribution based on the Hoyle Henley method. National data sources were used to obtain costs in 2019 US dollars, discounted by 3%. Health state utilities from available literature were applied to each health state. Utility decrements for adverse events were individualized in each choice branch with utility decrement weighted by the percentage of patients who experienced the adverse event in the MAIA and SWOG S0777 trials. We assumed a treatment would be cost-effective at a willingness to pay (WTP) of $150,000 per progression-free quality-adjusted life-year ($/PFQALY). One-way and probabilistic sensitivity analyses were conducted. RESULTS: Rd standard therapy had the lowest overall cost at $329,867, followed by VRd at $385,434 and DRd with the highest overall total cost at $626,900. Rd was estimated to result in the least amount (1.24) of PFQALYs, followed by VRd at 1.35 PFQALYs and DRd at 1.52 PFQALYs. With a WTP threshold of $150,000 per PFQALY, VRd was not cost-effective compared with Rd standard therapy, with an incremental cost-effectiveness ratio (ICER) of $530,256 per PFQALY. DRd was not cost-effective compared with VRd (ICER = $1,396,318 per PFQALY), nor as compared with Rd standard therapy (ICER = $1060,832). One-way sensitivity analysis showed that our model was sensitive to cost of DRd, VRd, and Rd drugs. Probabilistic sensitivity analysis showed that only at a WTP threshold of $550,000 was VRd cost-effective for 40% of iterations. There were no reasonable WTP thresholds, up to $800,00, where DRd became more cost-effective than VRd. CONCLUSIONS: This study is the first analysis to directly compare the cost-effectiveness of 3 acceptable chemotherapy treatment regimens for patients with multiple myeloma ineligible for autologous stem cell transplant. Neither DRd nor VRd triple therapy were found to be cost-effective vs Rd. Further cost-effectiveness analyses that include overall survival data for daratumumab and bortezomib triple therapies are needed to demonstrate an ICER in QALYs. DISCLOSURES: No funding was received for this study. At the time of this study, Narsipur was a UCSF-Actelion Clinical Research and Medical Communications Fellow, unrelated to this study. The other authors have nothing to disclose.

Effectiveness and economic impact of a diabetes education program among adults with type 2 diabetes in South Texas.

BACKGROUND: The long-term growth and sustained high prevalence of obesity in the US is likely to increase the burden of Type 2 diabetes. Hispanic individuals are particularly burdened by a larger share of diabetes than non-Hispanic White individuals. Given the existing health disparities facing this population, we aimed to examine the effectiveness and potential cost savings of the Diabetes Education Program (DEP) offered as part of Healthy South Texas, a state-legislated initiative to reduce health disparities in 27 counties in South Texas with a high proportion of Hispanic adults. METHODS: DEP is an 8-h interactive workshop taught in English and Spanish. After the workshop, participants receive quarterly biometric screenings and continuing education with a health educator for one year. Data were analyzed from 3859 DEP participants with Type 2 diabetes living in South Texas at five time points (baseline, 3-months, 6-months, 9-months, 12-months). The primary outcome variable of interest for study analyses was A1c. A series of independent sample t-tests and linear mixed-model regression analyses were used to identify changes over time. Two methods were then applied to estimate healthcare costs savings associated with A1c reductions among participants. RESULTS: The majority of participants were ages 45-64 years (58%), female (60%), Hispanic (66%), and had a high school education or less (75%). At baseline, the average hemoglobin A1c was 8.57%. The most substantial reductions in hemoglobin A1c were identified from baseline to 3-month follow-up (P < 0.001); however, the reduction in A1c remained significant from baseline to 12-month follow-up (P < 0.001). The healthcare cost savings associated with improved A1c for the program was estimated to be between $5.3 to $5.6 million over a two to three year period. CONCLUSION: Findings support the effectiveness of DEP with ongoing follow-up for sustained diabetes risk management. While such interventions foster clinical-community collaboration and can improve patient adherence to recommended lifestyle behaviors, opportunities exist to complement DEP with other resources and services to enhance program benefits. Policy makers and other key stakeholders can assess the lessons learned in this effort to tailor and expand similar initiatives to potentially at-risk populations. TRIAL REGISTRATION: This community-based intervention is not considered a trial by ICMJE definitions, and has not be registered as such.

Is olaparib cost effective in metastatic castration-resistant prostate cancer patients with at least one favorable gene mutation in BRCA1, BRCA2 or ATM?

Aim: To compare the cost-effectiveness of olaparib versus control treatment in metastatic castration-resistant prostate cancer patients with at least one gene mutation in BRCA1, BRCA2 or ATM from the US payer perspective. Methods: A Markov model was constructed to assess the quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios. Sensitivity analyses and scenario analyses were conducted to explore the impact of uncertainties. Results: The base-case result indicated that, for patients with specific gene mutations, olaparib gained 1.26 QALYs and USD$157,732 total cost. Compared with control treatment, the incremental cost-effectiveness ratio of olaparib was USD$248,248/QALY. The price of olaparib was the most influential parameter. Conclusion: Olaparib is not cost effective in comparison with control treatment in metastatic castration-resistant prostate cancer patients with specific gene mutations.

Cost-Effectiveness of Adding Ribociclib to Endocrine Therapy for Patients With HR-Positive, HER2-Negative Advanced Breast Cancer Among Premenopausal or Perimenopausal Women.

PURPOSE: To evaluate the cost-effectiveness of adding ribociclib to endocrine therapy for pre/perimenopausal women with hormone receptor-positive (HR+), human epidermal receptor 2-negative (HER2-) advanced breast cancer from the US payer perspective. METHODS: A partitioned survival analysis model with three health states (progression-free, progressed disease, and death) was developed to compare the cost and effectiveness of ribociclib in combination with endocrine therapy versus endocrine therapy alone based on clinical data from the MONALEESA-7 phase 3 randomized clinical trials. Life years (LYs), quality-adjusted life-years (QALYs), and total costs were estimated and used to calculate incremental cost-effectiveness ratio (ICER) over a lifetime. Deterministic and probabilistic sensitivity analyses were conducted to test the uncertainties of model inputs. Additional scenario analyses were performed. RESULTS: In the base-case, ribociclib plus endocrine therapy was more effective than endocrine therapy with an additional 1.39 QALYs but also more costly with an ICER of $282,996/QALY. One-way deterministic sensitivity analysis showed that overall survival associated with the treatments and the cost of ribociclib had the greatest impact on the ICER. The probabilistic sensitivity analysis showed that only beyond a willingness-to-pay (WTP) threshold of $272,867, ribociclib plus endocrine therapy would surpass endocrine therapy alone as a cost-effective option. CONCLUSIONS: From the US payer perspective, ribociclib plus endocrine therapy for pre/perimenopausal patients with HR+/HER2- advanced breast cancer is not cost-effective at a WTP threshold of $100,000 or $150,000 per QALY in comparison of endocrine therapy alone.

Cost-effectiveness of ribociclib plus endocrine therapy versus placebo plus endocrine therapy in HR-positive, HER2-negative breast cancer.

BACKGROUND: The 2015 American Society of Clinical Oncology guidelines recommend first-line treatment of hormone receptor (HR)-positive breast cancer with endocrine therapy plus or minus palbociclib, a selective cyclin-dependent kinase (CDK)4/6 inhibitor. In 2018, the U.S. Food and Drug Administration approved ribociclib, a new orally available selective CDK4/6 inhibitor. While gains in progression-free survival (PFS) and overall survival (OS) from ribociclib are important for clinical and treatment outcomes, trade-offs in adverse events (AEs) and additional costs necessitate cost-effectiveness analysis (CEA) to assist consideration by third-party payer systems, physicians, and patients. OBJECTIVES: To (a) develop a Markov model and (b) determine the cost-effectiveness of ribociclib plus endocrine therapy versus endocrine therapy alone as treatment for premenopausal and perimenopausal patients with HR-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer. METHODS: A lifetime 3-state Markov model ("stable," "progressed," and "dead" health states) was developed using a U.S. payer perspective. Transition probabilities were calculated based on OS and PFS outcomes from the randomized controlled phase 3 trial MONALEESA-7. These Kaplan-Meier curves were extended to lifetime by estimating best-fit distributions using loglogistic distribution for ribociclib curves and Weibull distribution for placebo curves. Costs were obtained from national data sources using 2019 U.S. dollars (USD) and discounted by 3%. Utilities were obtained via published breast cancer literature and were included for each health state and for time spent with each AE. Results were expressed as an incremental cost-effectiveness ratio (ICER) expressed as USD per quality-adjusted life-year (QALY) saved. Treatments were assumed to be cost-effective based on a willingness-to-pay (WTP) threshold of $150,000 per QALY gained. Base-case, 1-way sensitivity tornado diagrams and probabilistic sensitivity analyses demonstrated changes in the ICER and were driven by the cost of ribociclib and the utility of remaining in the stable health state. RESULTS: Ribociclib plus endocrine therapy was cost-effective at an ICER of $124,513 per QALY when compared with endocrine therapy alone at a WTP threshold of $150,000. The ribociclib plus endocrine therapy arm had an effectiveness of 5.28 QALYs and a total cost of $385,112, while placebo plus endocrine therapy provided only 2.46 QALYs at a lower total cost of $67.246. The model was sensitive to the cost of ribociclib and the utility of time spent in the stable health state. Probabilistic sensitivity analysis demonstrated that endocrine therapy alone was cost-effective until a WTP of $125,000 and was cost-effective 72% of the time at the WTP threshold. CONCLUSIONS: Ribociclib plus endocrine therapy is more cost-effective than endocrine therapy alone. Professionals in managed care settings should consider the pharmacoeconomic benefits of ribociclib for the treatment of HR-positive, HER2-negative breast cancer as they make value-based formulary decisions. Further CEAs should be considered as direct treatment comparison trials between CDK4/6 inhibitors are completed in the future. DISCLOSURES: No outside funding supported this study. The authors have nothing to disclose.

Cost-effectiveness of atezolizumab plus chemotherapy for advanced non-small-cell lung cancer.

Background Adding atezolizumab to carboplatin/nab-paclitaxel improved progression-free survival and overall survival in patients with advanced non-squamous non-small-cell lung cancer. However, estimating the economy of atezolizumab/carboplatin/nab-paclitaxel is urgent on account of the high cost of atezolizumab. Objective This study aimed to evaluate the cost-effectiveness of atezolizumab plus carboplatin/nab- paclitaxel for untreated advanced non-squamous non-small-cell lung cancer from the United States payer perspective. Setting This study was based on randomized clinical trial data from the IMpower130 (NCT02367781) published in Lancet Oncology (May 2019). Method A Markov model was constructed to estimate the health expenditure on atezolizumab in combination with carboplatin/nab-paclitaxel for advanced non-small-cell lung cancer treatment. Drug costs were collected from Red Book Wholesale Acquisition Cost, and health state utility values were obtained from the literature. Uncertainty was evaluated via one-way and probabilistic sensitivity analyses. Main outcome measure The main outcomes were cost, life years, quality-adjusted life years, and incremental cost-effectiveness ratio. Results Over a 10-year horizon, atezolizumab/carboplatin/nab-paclitaxel treatment was associated with an expected 1.76 life years and 0.99 quality-adjusted life years compared to the 1.21 life years and 0.67 quality-adjusted life years for carboplatin/nab-paclitaxel alone. Compared to carboplatin/nab-paclitaxel, atezolizumab/carboplatin/nab-paclitaxel produced an incremental cost of $105,617. The resultant incremental cost-effectiveness ratio was $333,199 per quality-adjusted life year, which exceeded the willingness-to-pay threshold of $180,000 per quality-adjusted life year. The price of atezolizumab and utility values were the parameters that greatly impacted the incremental cost-effectiveness ratio. Carboplatin/nab-paclitaxel exhibited 98.6% probability of being a cost-effective treatment option compared to atezolizumab/carboplatin/nab-paclitaxel at a willingness-to-pay of $180,000 per quality-adjusted life year. However, reducing atezolizumab acquisition cost by 43.4% could make atezolizumab/carboplatin/nab-paclitaxel more cost-effective than carboplatin/nab-paclitaxel. Conclusion Adding atezolizumab to carboplatin/nab-paclitaxel was not cost-effective for advanced non-squamous non-small-cell lung cancer in the base-case scenario. Decreasing atezolizumab acquisition cost might enhance the cost-effectiveness.

Cost-Utility Analysis of Pembrolizumab Versus Chemotherapy as First-Line Treatment for Metastatic Non-Small Cell Lung Cancer With Different PD-L1 Expression Levels.

To evaluate the cost-utility of pembrolizumab versus chemotherapy as the first-line setting for metastatic non-small cell lung cancer (NSCLC) from the US health care system perspective, a Markov model was developed to compare the lifetime cost and effectiveness of pembrolizumab versus chemotherapy for untreated metastatic NSCLC, based on the clinical data derived from phase III randomized controlled trial (KEYNOTE-042; ClinicalTrials.gov; NCT02220894). Weibull distribution was fitted to simulate the parametric survival functions. Drug costs were collected from official websites, and utility values were obtained from published literature. Total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were computed as primary output indicators. The impact of different PD-L1 expression levels on ICER was also evaluated. One-way and probabilistic sensitivity analyses were performed to assess the model uncertainty. Compared with chemotherapy, patients treated with pembrolizumab provided an additional 1.13, 1.01, and 0.59 QALYs in patients with PD-L1 expression levels of ≥50%, ≥20%, and ≥1%, with corresponding incremental cost of 53,784, 47,479, and 39,827, respectively. The resultant ICERs of pembrolizumab versus chemotherapy were 47,596, 47,184, and 68,061/QALY, in three expression levels of PD-L1, respectively, all of which did not exceed the WTP threshold of 180,000/QALY. Probability sensitivity analysis outcome supported that pembrolizumab exhibited evident advantage over chemotherapy to be cost-effective. One-way sensitivity analysis found that ICERs were most sensitive to utility value of pembrolizumab in progression survival state. All the adjustment of parameters did not qualitatively change the result. For treatment-naive, metastatic NSCLC patients with PD-L1+, pembrolizumab was estimated to be cost-effective compared with chemotherapy for all PD-L1 expression levels at a WTP threshold of 180,000/QALY in the context of the US health care system.

Longitudinal Comparison of Patient-Level Outcomes and Costs Across Prostate Cancer Treatments With Urinary Problems.

Prostate cancer (PCa) is the leading cancer in men in the United States. This study evaluated direct costs of treating urinary problems after PCa treatments and determined predictors of long-term costs for urinary problems. Data from the Cancer of Prostate Strategic Urologic Research Endeavor registry was analyzed for this study. Annual treatment costs for urinary problems for up to 14 years were compared among different primary PCa treatments, which included radical prostatectomy, external beam radiation therapy, brachytherapy, and watchful waiting. A multivariate generalized estimating equation (GEE) model with bootstrapping was estimated to identify the predictors associated with treatment costs for urinary problems. A total of 3,062 eligible patients were identified with a mean age of 65 years at diagnosis. Mean annual treatment cost for urinary problems across all patients with PCa was $118/patient. Those greater than 74 years old had the highest cost ($238/patient). Mean annual cost for urinary problems among only those with urinary problems was $432. Multivariate regression showed patients undergoing radical prostatectomy had significantly lower (-63%, p = .01) costs for urinary problems than those treated with watchful waiting. This study helps to understand the importance of treating urinary problems associated with different PCa treatments and highlights their medical care costs. The pattern of treatment costs for urinary problems across all PCa treatments suggests that clinicians need to offer treatment for urinary problems to all PCa patients over longer time periods, even to those choosing watchful waiting.