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.

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.

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.

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.

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 Analysis of Hepatic Arterial Infusion of FOLFOX Combined Sorafenib for Advanced Hepatocellular Carcinoma With Portal Vein Invasion.

BACKGROUND: Hepatic arterial infusion (HAI) of oxaliplatin, leucovorin, and fluorouracil (FOLFOX) plus sorafenib has a more desirable effect versus sorafenib for hepatocellular carcinoma (HCC) patients with portal vein invasion. However, considering the high cost of hepatic arterial infusion of chemotherapy (HAIC), this study evaluated the cost-effectiveness of HAIC plus sorafenib (SoraHAIC) versus standard care for HCC patients from the Chinese health system perspective. METHODS: A Markov multi-state model was constructed to simulate the disease course and source consumption of SoraHAIC. Costs of primary therapeutic drugs were calculated based on the national bid price, and hepatic artery catheterization fee was collected from the Fujian Provincial Price Bureau. Clinical data, other costs, and utility values were extracted from references. Primary outcomes included life-years (LYs), quality-adjusted life years (QALYs) and incremental cost-effectiveness ratio (ICER). The robustness of model was verified by uncertainty sensitivity analyses. RESULTS: SoraHAIC gained 1.18 QALYs (1.68 LYs) at a cost of $65,254, while the effectiveness and cost of sorafenib were 0.52 QALYs (0.79 LYs) and $14,280, respectively. The ICER of SoraHAIC vs sorafenib was $77,132/QALY ($57,153/LY). Parameter that most influenced the ICER was utility of PFS state. The probabilistic sensitivity analysis (PSA) showed that SoraHAIC was not cost-effective in the WTP threshold of 3*Gross Domestic Product (GDP) per capita of China ($30,492/QALY). But about 38.8% of the simulations were favorable to SoraHAIC at the WTP threshold of 3*GDP per capita of Beijing ($72,000/QALY). When 3*GDP per capita of Fujian ($47,285/QALY) and Gansu Province ($14,595/QALY) were used as WTP threshold, the acceptability of SoraHAIC was 0.3% and 0%, respectively. CONCLUSIONS: The study results indicated that SoraHAIC was not cost-effective in medium-, and low-income regions of China. In developed areas of China (Beijing), there was a 38.8% probability that the SoraHAIC regimen would be cost-effective.

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.

Clinical and Economic Burden of Valley Fever in Arizona: An Incidence-Based Cost-of-Illness Analysis.

BACKGROUND: Coccidioidomycosis, ie, Valley fever, is an important fungal infection in the Southwest, with half to two thirds of all cases occurring in Arizona. This endemic respiratory disease can range from primary uncomplicated pneumonia to disseminated infection such as meningitis with chronic pulmonary complications. Valley fever diagnoses have risen over recent years and cause substantial morbidity and economic burden in Arizona. METHODS: We estimated the lifetime cost-of-illness associated with all cases of Valley fever diagnosed in 2019 in Arizona. Natural history of the disease was determined from literature and expert opinion and assigned costs from national data sources to determine lifetime direct and indirect costs (work loss). RESULTS: Total lifetime costs of $736 million were estimated for the 10 359 cases of Valley fever diagnosed in Arizona in 2019. Direct costs of $671 million accounted for over 90% of expenditures, with $65 million in indirect costs. Disseminated infection produces the highest economic burden at $1.26 million direct and $137 400 indirect costs per person. The lowest Valley fever lifetime costs were for cases of primary uncomplicated pneumonia with $23 200 in direct costs and $1300 in lost wages. The average lifetime direct costs across all Valley fever manifestations are $64 800 per person diagnosed in Arizona in 2019 and $6300 for indirect costs. CONCLUSIONS: Valley fever is responsible for substantial economic burden in Arizona. Our estimates underscore the value of supporting research into developing more rapid diagnostic tests, better therapies, and ultimately a preventative vaccine to address this important public health problem in Arizona.

A cost-effectiveness analysis comparing a conventional mechanical needle to a radiofrequency device for transseptal punctures.

INTRODUCTION: Transseptal puncture is an integral step in various catheter-based cardiac procedures and can be performed with either the conventional mechanical needle or an FDA-cleared device utilizing radiofrequency (RF) energy. Although a previous randomized trial suggested that the RF transseptal device may be faster and more often successful, the increased equipment costs may dissuade operators from routine use. This analysis compares the cost-effectiveness of the mechanical needle to the RF device during pulmonary vein isolation. METHODS: The rates of successful transseptal punctures for each device and transseptal-related complications were determined from the peer-reviewed medical literature. Procedural, equipment, and complication costs were obtained from peer-reviewed medical literature and the Healthcare Cost and Utilization Project. The effectiveness was defined as the probability of 30-day survival following a successful transseptal puncture. Monte Carlo probabilistic analyses tested variable effects of costs and complication rates on the incremental cost-effectiveness ratio. RESULTS: The 30-day effectiveness of the RF device vs the mechanical needle was 99.7% and 98.8%, respectively. After accounting for all costs of performing a single transseptal puncture, the cost at 30 days associated with the RF device was $41 less than the mechanical needle ($21 096 vs $21 137). The RF device was similarly dominant to the mechanical needle in double transseptal puncture scenarios. Finally, the RF device was more cost-effective than the mechanical needle at any willingness-to-pay in Monte Carlo probabilistic sensitivity analyses. CONCLUSIONS: Despite greater equipment costs, the RF device costs less and provides better effectiveness at 30 days than the conventional mechanical needle.

Robust Health Utility Assessment Among Long-term Survivors of Prostate Cancer: Results from the Cancer of the Prostate Strategic Urologic Research Endeavor Registry.

BACKGROUND: Valid health utility values are essential for comparative effectiveness analyses. However, subjective utilities in long-term survivors of prostate cancer (PCa) with various oncological and functional outcomes have not been well described. OBJECTIVE: To quantify utilities in long-term survivors of PCa using the standard gamble method, generally regarded as the approach best grounded in economic theory. DESIGN, SETTING, AND PARTICIPANTS: We performed a cross-sectional study nested within a prospective cohort-Cancer of the Prostate Strategic Urologic Research Endeavor (CaPSURE). Overall, 1884 (59.7%) of 3155 active participants across all disease states returned the questionnaire. INTERVENTION: Various primary treatments for PCa. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Utility values for PCa health, sexual function, urinary function, bowel function, and overall health were measured, based on patients' conditions at the time of the survey. Bias correction methods were employed. RESULTS AND LIMITATIONS: After exclusion of incomplete or disqualified data, 1740 (92.3% of responding) patients were included in the final analysis. The mean age was 73.1 ±â€¯8.2 yr at a median of 9 yr (interquartile range: 6-11) since diagnosis. Mean utilities for PCa health and overall health were 0.934 ±â€¯0.120 and 0.960 ±â€¯0.100, respectively. After bias correction by probability weighting function, utilities were 0.866 ±â€¯0.154 and 0.897 ±â€¯0.142, and by mixed model correction, 0.845 ±â€¯0.186 and 0.884 ±â€¯0.176, respectively. Measured utilities were similarly high for specific functional outcomes, even with bias corrections. Survivorship bias and skewed proportion of disease status due to natural history of PCa were potential limitations. CONCLUSIONS: Standard gamble-based utilities in long-term survivors of PCa were much higher than those determined previously. The results indicate substantial human resilience: most PCa patients adapt to their health status over time even if they experience incomplete functional recovery and would not take risk in pursuit of better quality of life. PATIENT SUMMARY: We elicited health utilities (measures of quality of life) among long-term survivors of prostate cancer using the most robust method. These were much higher than previously reported values that were based on theoretical scenarios or indirect methods. Long-term survivors of prostate cancer may adapt well to their health conditions over time even if they experience disease-specific or functional problems.

Utilization, Cost, and Pricing Scheme of Compounded Medications for Public Health System Patients: The California Workers' Compensation System, 2011-2013.

BACKGROUND: Although medically necessary in some cases, there is growing concern that compounded medications are being overprescribed, leading to questions about safety and necessity for high use and cost. Safety concerns regarding compounded medications were highlighted by the 2013 contamination of steroid injections by the New England Compounding Center, which caused serious infections and other injuries to at least 751 patients and resulted in at least 64 patient deaths. A study contributed to our understanding of compounded medication use and cost, finding in a sample of commercially insured population that the average ingredient cost for compounded medication prescriptions was $710.36, which is 130% higher than for noncompounded medication prescriptions. The literature on use and cost of compounded medications in noncommercially insured populations and related regulations, however, is sparse. The California Workers' Compensation System (CAWCS)-the largest U.S. workers' compensation system and a public health system experiencing high compounded medication costs-provided an opportunity for additional analysis of these issues. Furthermore, CAWCS data on compounded medication use and cost allow for the exploration of alternative pricing mechanisms that may control costs. OBJECTIVES: To (a) examine use, cost, and billing and reimbursement practices for compounded medications in a public health system-CAWCS- and (b) evaluate regulations and recommend an alternative pricing mechanism that could control costs in California. METHODS: Descriptive statistics for use, cost, and reimbursement patterns of all compounded medication prescriptions included in CAWCS's Workers Compensation Information System claims datasets from 2011 to 2013 were determined. This study coded a unique dataset that (a) identified compounded medications at the ingredient level; (b) grouped compounded medications from ingredient level to compounded medications as a whole; and (c) categorized compounded medications into applicable Colorado pricing categories. T-tests assessed if regulation AB 378, which targets compounded medications, was associated with a difference in mean cost. The Colorado pricing scheme was applied to estimate cost and provide recommendations. RESULTS: Despite the AB 378 requirement for compounded medications to be billed at the ingredient level for reimbursement, 15% of pharmacy-dispensed and 6% of physician-dispensed medications were not billed at the ingredient level. For pharmacy-dispensed compounded medications billed at the ingredient level, mean amount paid (SD) per ingredient was $45.40 (195.97), and for those medications billed at the single compounded medication level, mean amount paid (SD) per medication was $95.20 (326.33) over all years. For physician-dispensed medications billed at the ingredient level, mean amount paid (SD) per ingredient was $75.47 (205.51), and when billed at the single medication level was $204.83 (221.01). T-tests showed a mean increase in compounded medication mean amount paid between pre- and post-AB 378 groups of $12.27 (P < 0.001) for pharmacy-dispensed medications and $11.34 (P < 0.001) for physician-dispensed medications, suggesting that AB 378 did not curb compounded medication mean amount paid. CONCLUSIONS: The average cost of CAWCS pharmacy- and physician-dispensed compounded medications consistently increased. Various factors may have influenced this increase, but AB 378 did not achieve its full regulatory intent to standardize billing and reimbursement and control cost. Grouping of ingredients into compounded medications allowed for application of the Colorado pricing scheme to CAWCS claims data. Adoption of Colorado pricing would save 46% of current compounded medication cost for less complicated medications, while increasing cost for more complicated medications. The analyses recommended a revised Colorado pricing scheme, which would provide improved incentives for accurate billing and lead to savings for CAWCS. DISCLOSURES: Funding for this study was provided by the California Workers' Compensation System. The authors had final control regarding study design, study conduct, and writing of the manuscript. The authors report no conflicts of interest.

The costs of dementia subtypes to California Medicare fee-for-service, 2015.

INTRODUCTION: Dementia is among the costliest of medical conditions, but it is not known how these costs vary by dementia subtype. METHODS: The effect of dementia diagnosis subtype on direct health care costs and utilization was estimated using 2015 California Medicare fee-for-service data. Potential drivers of increased costs in Lewy body dementia (LBD), in comparison to Alzheimer's disease, were tested. RESULTS: 3,001,987 Medicare beneficiaries were identified, of which 8.2% had a dementia diagnosis. Unspecified dementia was the most common diagnostic category (59.6%), followed by Alzheimer's disease (23.2%). LBD was the costliest subtype to Medicare, on average, followed by vascular dementia. The higher costs in LBD were explained in part by falls, urinary incontinence or infection, depression, anxiety, dehydration, and delirium. DISCUSSION: Dementia subtype is an important predictor of health care costs. Earlier identification and targeted treatment might mitigate the costs associated with co-occurring conditions in LBD.

The Rise of Valley Fever: Prevalence and Cost Burden of Coccidioidomycosis Infection in California.

Coccidioidomycosis (CM) is a fungal infection endemic in the southwestern United States (US). In California, CM incidence increased more than 213% (from 6.0/100,000 (2014) to 18.8/100,000 (2017)) and continues to increase as rates in the first half of 2018 are double that of 2017 during the same period. This cost-of-illness study provides essential information to be used in health planning and funding as CM infections continue to surge. We used a "bottom-up" approach to determine lifetime costs of 2017 reported incident CM cases in California. We defined CM natural history and used a societal approach to determine direct and discounted indirect costs using literature, national datasets, and expert interviews. The total lifetime cost burden of CM cases reported in 2017 in California is just under $700 million US dollars, with $429 million in direct costs and $271 million in indirect costs. Per person direct costs were highest for disseminated disease ($1,023,730), while per person direct costs were lowest for uncomplicated CM pneumonia ($22,039). Cost burden varied by county. This is the first study to estimate total costs of CM, demonstrating its huge cost burden for California.

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.

Assigning value to preparation for prostate cancer decision making: a willingness to pay analysis.

BACKGROUND: The Personal Patient Profile-Prostate (P3P) is a web-based decision support system for men newly diagnosed with localized prostate cancer that has demonstrated efficacy in reducing decisional conflict. Our objective was to estimate willingness-to-pay (WTP) for men's decisional preparation activities. METHODS: In a multicenter, randomized trial of P3P, usual care group participants received typical preparation for decision making plus referral to publicly-available, educational websites. Intervention group participants received the same, plus online P3P educational media specific to the user's personal preferences and values, and a communication coaching component tailored to race\ethnicity, age and language. WTP data were collected one week after physician consultation. An iterative bidding direct contingent valuation survey format was used, randomly assigning participants to high or low starting values (SV). Tobit models were used to explore associations between SV-adjusted WTP and age, education, marital and work-status, insurance, decision-control preference and decision-making stage. RESULTS: Of 392 participants enrolled, 141 P3P and 107 usual care (UC) provided a WTP value. Men were willing to pay a median $25 (IQR $10-100) for P3P in addition to usual care preparation materials. In the final multivariable tobit regression model, SV, marital status, stage of decision making and income were significantly associated with WTP for P3P. Decision control preference was considered marginally significant (p = 0.11). Men were WTP a median $30 (IQR $10-$200) for usual care material alone. In the final multivariable model, SV, education, and stage of decision making were significantly associated with WTP in usual care. CONCLUSION: WTP was similar for UC and for the addition of P3P to UC decision preparation. The WTP values were associated with demographic and preference variables. Findings can help focus decision support on future patients who would benefit most: those without strong support systems, at earlier stages of decision making, and open to a shared-decision style. TRIAL REGISTRATION: NCT NCT01844999 . Registered May 3, 2013.

A Practical First Step Using Needs Assessment and a Survey Approach to Implementing a Clinical Pharmacogenomics Consult Service.

INTRODUCTION: Genetic-guided selection of non-oncologic medications is not commonly practiced in general, and at University of California, San Francisco (UCSF) Health, specifically. Understanding the unique position of clinicians with respect to clinical pharmacogenetics (PG) at a specific institution or practice is fundamental for implementing a successful PG consult service. OBJECTIVES: To assess clinicians' current practices, needs, and interests with respect to clinical PG at UCSF Health, a large tertiary academic medical center. METHODS: A list of 42 target medications with clinical PG recommendations was complied. Clinical specialties that routinely used the target medications were identified. A 12-question survey focused on practice of PG for target medications was developed. Pharmacists and physicians were surveyed anonymously in several clinical specialties. Survey results were analyzed using descriptive statistics. RESULTS: Of the 396 clinicians surveyed, 76 physicians and 59 pharmacists participated, resulting in 27% and 50% average response rates, respectively. The current use of PG in clinical practice for physicians and pharmacists was 29% and 32%, respectively, however this number varied across clinical specialties from 0% to 80%. Of clinicians whom reported they do not currently apply PG, 63% of physicians and 54% of pharmacists expressed interest in integrating PG. However, the level of interest varied from 20% to 100% across specialties. Of the respondents, 64% of physicians and 56% of pharmacists elected to provide contact information to investigators to further discuss their interest related to clinical PG. CONCLUSIONS: While PG is not uniformly practiced at UCSF Health, there is considerable interest in utilizing PG by the respondents. Our approach was successful at identifying clinicians and services interested in PG for specific drug-gene pairs. This work has set a foundation for next steps to advance PG integration at UCSF Health. Clinicians can adopt our approach as preliminary work to build a clinical PG program at their institutions.

Financial Impact of Alternative Pricing Benchmarks for Physician-Dispensed Drugs in the California Workers' Compensation System.

BACKGROUND: Pricing drugs in the California Workers' Compensation System (CAWCS) has become more difficult as there are increasingly fewer drugs listed in the Medi-Cal primary fee schedule, which is used as the source for CAWCS drug prices. This presents a challenge of providing timely and accurate CAWCS reimbursement. The objectives of this study are (1) to explore any trends in physician-dispensed drug prices; (2) to compare the proportion of drugs with and without a price and to determine the financial implications of repricing CAWCS physician-dispensed drugs with five alternative pricing benchmarks; and (3) to offer recommendations for the pricing benchmark to maximize pricing coverage and to remain budget neutral. METHODS: We evaluated physician-dispensed drugs at the transaction level, reimbursed in the CAWCS. Frequency, reimbursement rate, and total and average paid costs were reported. We matched each claim line in the CAWCS to the corresponding unit price of an alternative price benchmark including average wholesale price, wholesale acquisition cost, direct prices, national average drug acquisition cost, and Federal Upper Limit. RESULTS: Average wholesale price provided prices for 99.9% of physician-dispensed drug claims, while Medi-Cal, the current primary physician-dispensed drug benchmark provided prices for a lower percentage (92.7%) of claims. The CAWCS prices were equivalent to 49% of the average wholesale price, 95.5% of Medi-Cal, 126.7% of the wholesale acquisition cost, 266% of the Federal Upper Limit, 64.4% of direct prices, and 197% of national average drug acquisition cost-estimated prices. CONCLUSIONS: The CAWCS current Medi-Cal pricing for physician-dispensed drugs is better than all alternatives in terms of price availability, transparency, and budget neutrality, but pricing availability may decrease over time as Medi-Cal moves to managed care. National average drug acquisition cost is the next best alternative, but it requires combinations of pricing benchmarks to maximize its price availability.

A gray area for reimbursement: medical foods for non-inborn errors of metabolism.

OBJECTIVES: The use of medical foods (MFs) specifically for non-inborn errors of metabolism (non-IEM) is rising. Concomitantly, evidence for the safety and efficacy of these non-IEM MFs is lacking. We examined the current use and costs of non-IEM MFs and determined whether the scientific evidence supporting their effectiveness and medical utility is adequate to warrant public reimbursement. STUDY DESIGN: We employed a qualitative literature review analysis. METHODS: PubMed and MEDLINE databases were searched for all years using relevant keywords, including names of non-IEM MFs identified in the California Workers' Compensation System (CAWCS) claims dataset from 2011 to 2013. The quality of extracted data was scored with the Delfini Evidence Tool Kit. RESULTS: Only 2 (3.2%) of 62 studies were conducted with scientific rigor. These 2 studies were for dietary management of Alzheimer disease, which does not have a distinctive nutritional requirement necessitating an MF. Seventy-one percent of the studies of MFs used by patients in the CAWCS were considered to have uncertain validity. CONCLUSIONS: Most reviewed non-IEM MFs lack evidence to support their safety and efficacy. These non-IEM MFs do not abide by FDA draft guidance, as they do not address a distinct nutritional requirement for a disease and yet often have a National Drug Code or "Rx only" label. Consequently, these products do not meet the statutory definition of an MF. We recommend that CAWCS and other payers not provide insurance coverage for non-IEM MFs until more scientific evidence supports their safety, efficacy, and use for nutritional need of a disease.

Use and Costs of Medical Foods and Convenience-Packaged Drugs in the California Workers' Compensation System.

BACKGROUND: Sales of medical foods (MFs) and convenience packages (CPs) are projected to exceed $2 billion in the United States, with an annual growth rate of 10%. Several studies have highlighted the rapid growth of MF use within the California Workers' Compensation System (CAWCS). To curb this growth, California implemented Assembly Bill 378 (AB 378) in 2012 to regulate physicians' incentives to dispense MFs and CPs. AB 378's regulation on only physician-dispensed MFs and CPs and not pharmacy-dispensed MFs and CPs generated a setting for evaluating the differential effect of the bill on MF and CP use and cost. OBJECTIVES: To (a) examine the use and cost of MFs and CPs in the CAWCS that are not for inborn errors of metabolism and (b) evaluate the regulatory effect of AB 378. METHODS: This study adopted a quantitative approach and employed descriptive statistics and t-tests. The analyses used the most recent complete annual claims data from the Workers' Compensation Information System dataset to evaluate MF and CP claims frequencies and dollar amounts reimbursed from 2011 to 2013 and to compare the difference between physician-dispensed and pharmacy-dispensed products. RESULTS: Of 151,107 MFs and CPs billed, 95,528 (63.2%) prescriptions were reimbursed. The reimbursed MFs and CPs accounted for approximately $19 million paid to pharmacies and physicians over 3 years. Physician-dispensed MFs, which were regulated by AB 378 in January 2012, experienced a reduction in mean amount reimbursed by $9.95 (P < 0.001)-from $195.64 to $185.68-compared with the mean amount reimbursed in 2011. Conversely, physician-dispensed CPs, as well as pharmacy-dispensed MFs and CPs, did not experience a decrease in mean amount reimbursed. CONCLUSIONS: The results indicated that AB 378 was associated with a statistically significant reduction in physician-dispensed MFs. Concomitantly, the results from t-tests showed no statistically significant difference in mean amount reimbursed for MFs and CPs to pharmacies before and after AB 378. The finding was expected and as hypothesized because AB 378 did not regulate pharmacy-dispensed MFs and CPs. Legislative measures, such as AB 378 in California, may influence rising costs and use of MFs and CPs in general. Other workers' compensation systems could adopt similar legislation to affect the behavior of physician prescribing of non-inborn errors of metabolism MFs and further test these findings. DISCLOSURES: Funding for this study was contributed by the California Workers' Compensation System. The authors have nothing to disclose.

The impact of alternative pricing methods for drugs in California Workers' Compensation System: Fee-schedule pricing.

INTRODUCTION: California's Workers' Compensation System (CAWCS) Department of Industrial Relations questioned the adequacy of the current Medi-Cal fee-schedule pricing and requested analysis of alternatives that maximize price availability and maintain budget neutrality. OBJECTIVES: To compare CAWCS pharmacy-dispensed (PD) drug prices under alternative fee schedules, and identify combinations of alternative benchmarks that have prices available for the largest percentage of PD drugs and that best reach budget neutrality. METHODS: Claims transaction-level data (2011-2013) from CAWCS were used to estimate total annual PD pharmaceutical payments. Medi-Cal pricing data was from the Workman's Compensation Insurance System (WCIS). Average Wholesale Prices (AWP), Wholesale Acquisition Costs (WAC), Direct Prices (DP), Federal Upper Limit (FUL) prices, and National Average Drug Acquisition Costs (NADAC) were from Medi-Span. We matched National Drug Codes (NDCs), pricing dates, and drug quantity for comparisons. We report pharmacy-dispensed (PD) claims frequency, reimbursement matching rate, and paid costs by CAWCS as the reference price against all alternative price benchmarks. RESULTS: Of 12,529,977 CAWCS claims for pharmaceutical products 11.6% (1,462,814) were for PD drugs. Prescription drug cost for CAWCS was over $152M; $63.9M, $47.9M, and $40.6M in 2011-2013. Ninety seven percent of these CAWCS PD claims had a Medi-Cal price. Alternative mechanisms provided a price for fewer claims; NADAC 94.23%, AWP 90.94%, FUL 73.11%, WAC 66.98%, and DP 14.33%. Among CAWCS drugs with no Medi-Cal price in PD claims, AWP, WAC, NADAC, DP, and FUL provided prices for 96.7%, 63.14%, 24.82%, 20.83%, and 15.08% of claims. Overall CAWCS paid 100.52% of Medi-Cal, 60% of AWP, 97% of WAC, 309.53% of FUL, 103.83% of DP, and 136.27% of NADAC. CONCLUSIONS: CAWCS current Medi-Cal fee-schedule price list for PD drugs is more complete than all alternative fee-schedules. However, all reimbursement approaches would require combinations of pricing benchmarks. We suggest keeping primary reimbursement at 100% of Medi-Cal and for drugs without a primary Medi-Cal price calculating the maximum fee as 60% of AWP and then 97% of WAC. Alternatively, we suggest using NADAC as a primary fee-schedule followed by either 60% AWP and 97% WAC or AWP-40% for drugs with no NADAC price. Fee-schedules may not offer the best price and a formulary approach may provide more flexibility.