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Objectives To assess the evolving landscape of low-density lipoprotein cholesterol-lowering therapies (LLTs) and quantify their effect on cardiovascular disease (CVD)-related mortality and morbidity. Study design Secondary data came from LLT clinical trials and 1999-2014 National Health and Nutrition Examination Survey (NHANES) data. 1996-2016 Medical Expenditure Panel Survey (MEPS) data were used to estimate LLT spending. Nonfatal CVD events prevented by LLTs were calculated from clinical trials and NHANES. The value of nonfatal events prevented was calculated as the product of event treatment costs and the number of events prevented. The value of mortality reduction was calculated as the product of a value of a life-year and the life expectancy gain from LLTs. This was compared with LLT spending estimated using MEPS. Methods Total LLT expenditures were calculated based on MEPS LLT utilization and expenditure data. Values of prevented hospitalizations, prevented CVD events, and other LLT utilization-related outcomes were pulled from the published literature. Results Combined, statins and ezetimibe prevented 2.8 million nonfatal heart attacks and 1.7 million nonfatal strokes from 1999 to 2014. Statin use generated $2.6 trillion in societal value through CVD deaths avoided from 1987 to 2014, and 85% accrued to patients. Conclusions LLTs have yielded significant societal value, and the majority of this value has accrued to patients.

Objective:This study compared health care use and costs among patients with treatment-resistant versus treatment-responsive depression across Medicaid, Medicare, and commercial payers.Methods:A ret...

Information on paroxysmal supraventricular tachycardia (PSVT) patient characteristics and the associated economic burden of the disease is limited. Therefore, we sought to characterize newly diagnosed PSVT patients and quantify their healthcare resource use and expenditures. We used enrollment, demographic, and claims data from IBM MarketScan Research Database and Medicare Limited Data Set (LDS) to identify patients newly diagnosed with PSVT (ICD-9: 427.0; ICD-10: I47.1) from 10/1/2012 to 9/30/2016. Patients were required to be observable 1-year before and after index diagnosis. Patients were stratified by age (

Background Uncomplicated urinary tract infection (uUTI) is often treated empirically without antibiotic (AB) susceptibility testing; however, antimicrobial-resistant bacteria could lead to suboptimal treatment and progression to complicated UTI (cUTI). We examined the likelihood of uUTI progression to cUTI in patients with susceptible and non-susceptible uropathogens. Methods We performed a retrospective cohort study using data from a large Mid-Atlantic US integrated delivery network’s electronic health records from July 1, 2016 to March 31, 2020. Patients included were female, aged ≥ 12 years with incident uUTI (diagnosis code or urine culture), and given an oral AB ± 5 days of diagnosis and ≥ 1 antibiotic susceptibility test. The primary outcome was progression to cUTI, defined as: new fever, nausea, or vomiting, in addition to uUTI symptoms; or receipt of intravenous antibiotic 3–28 days after index uUTI. Probability of progression to cUTI was assessed comparing patients with non-susceptible and susceptible isolates, with 1:1 propensity score matching. Patients retained for analysis had a nonzero predicted probability of being in the case and control group and were retained for analysis only if there were patients in the mirror group with similar propensity scores. Data were analyzed with logistic regression. Sensitivity analyses were performed to test the robustness of the primary analysis (Table). Results A total of 2565 patients were included: 1030 (40.2%) had non-susceptible isolates and 1535 (59.8%) had susceptible isolates. Mean age was 43.5 years and 59.5% of the cohort was White. After propensity score matching, patients with non-susceptible isolates were more than twice as likely to progress to cUTI versus patients with sensitive isolates (10.7% versus 4.9%; odds ratio, 2.35; p < 0.001; Figure). In sensitivity analyses, patients with non-susceptible isolates remained significantly more likely to progress to cUTI (p ≤ 0.009), excluding those receiving fluoroquinolones only (Table). Figure. Probability of progression to cUTI Table. Sensitivity analyses of the probability of uUTI progressing to cUTI in patients with non-susceptible versus susceptible isolates (matched population) Conclusion Patients with uUTI and AB-resistant isolates were significantly more likely to progress to cUTI than those with susceptible isolates. This finding highlights the need for greater understanding of antimicrobial resistance and has implications for the clinical management of uUTI. Disclosures Jason Shafrin, PhD, Precision Medicine Group (Employee, Former employee of Precision Medicine Group, which received funding from GlaxoSmithKline plc. to conduct this study) Alen Marijam, MSc, GlaxoSmithKline plc. (Employee, Shareholder) Ashish V. Joshi, PhD, GlaxoSmithKline plc. (Employee, Shareholder) Fanny S. Mitrani-Gold, MPH, GlaxoSmithKline plc. (Employee, Shareholder) Katie Everson, MSc, Precision Medicine Group (Employee, Employee of Precision Medicine Group, which received funding from GlaxoSmithKline plc. to conduct this study) Rifat Tuly, MPH, Precision Medicine Group (Employee, Employee of Precision Medicine Group, which received funding from GlaxoSmithKline plc. to conduct this study) Peter Rosenquist, MSc, Precision Medicine Group (Employee, Employee of Precision Medicine Group, which received funding from GlaxoSmithKline plc. to conduct this study) Michael Gillam, MD, MedStar Health (Employee, Employee of MedStar Health and received funding from GlaxoSmithKline plc. through Precision Medicine Group to conduct this study) Maria Elena Ruiz, MD, Nothing to disclose

Objective: Characterize variations in healthcare resource use (HRU), costs, and ablation prevalence in younger women and men newly diagnosed with paroxysmal supraventricular tachycardia (PSVT). Methods: Commercially insured patients age 18-40 from the Truven Health MarketScan database with newly diagnosed PSVT (ICD-9: 427.0; ICD-10: I47.1) from October 2012 to September 2016, were included when observable one year before and after index diagnosis. The outcomes were mean annual per patient HRU and costs paid by insurers 1 year pre- and post-diagnosis. Results: Among 5,466 study patients, a majority were women (N=3,655; 66.9%). Newly diagnosed women were older (mean age: 30.4y vs. 29.4y; PFigure ]. Conclusion: Healthcare spending increases significantly in younger patients newly diagnosed with PSVT. Despite the fact that emergency department visits and hospital admissions are more common in women than men after a PSVT diagnosis, spending after a PSVT diagnosis is significantly lower for women, reflecting lower ablation rates and spending for services with a PSVT diagnosis.

Background and Objectives: In 2013-14, approximately 30% of adults (20 years of age or older) in the United States (US) had high low-density lipoprotein cholesterol (LDL-C, LDL-C≥ 200 mg/dL). Elevated LDL-C is associated with an increased risk of cardiovascular disease (CVD) related events and death, and a key target for therapy. The lipid lowering treatment (LLT) landscape has evolved significantly over the course of the last 40 years with the introduction of statins, fibrates, ezetimibe, CETP inhibitors, and PCSK9 inhibitors (PCSK9i). Currently, statins are first-line therapy for LDL-C reduction. This study quantified the effect of LLT use since 1987 on CVD-related mortality and morbidity. Methods: Descriptive analyses of the rates of LLT use and LLT spending over time were conducted using both NHANES (1999-2014) and MEPS data (1996-2016), and the association between LDL-C levels and mortality and hospitalizations were identified in the literature. By using hazard ratios from published clinical trials, and NHANES data on the number of LLT users and frequency of non-fatal CVD events among these users, the total number of non-fatal CVD events prevented over time was calculated. Events avoided were calculated as the difference between CVD events observed among LLT users and events that would have occurred in the absence of LLT use. The latter was calculated using the difference in events between treated and untreated patients in LLT trials. The value associated with the prevention of non-fatal CVD events due to LLT use was calculated as the product of the costs associated with treating CVD events and the total number of CVD events prevented. The value of mortality reduction was calculated as the product of the value of a statistical life year ($150,000) and the life expectancy gain from the WOSCOPS and ODYSSEY clinical trials. This value was compared with LLT spending. Results: The uptake of statins and ezetimibe between 1999 and 2014 prevented an estimated total of 2.8M myocardial infarctions (MIs) and 1.7M strokes in the US. The cumulative value derived from MI and stroke events prevented through statin and ezetimibe use, though largely driven by statin use, from 1999–2012 amounted to approximately $258B (2018 USD) and $37B, respectively. The use of statins from 1999–2012 resulted in an additional 12.6 million life-years at a value of $1.9T, and we estimate that statin use could generate an additional $953.1B in value to society from 2018–2025 through reductions in CVD mortality, with patients retaining 94% of the value. Conclusions: Innovation in the LLT landscape, particularly through the introduction and uptake of statins, ezetimibe and PCSK9is, yields significant value to society through the reduction of costs associated with both fatal and non-fatal CVD events. Of the value that accrues to society from the utilization of statins, patients retain approximately 95%.

Jason Shafrin,1 Katalin Bognar,1 Katie Everson,1 Michelle Brauer,2 Darius N Lakdawalla,3 Felicia M Forma4 1Policy and Economics, Precision Health Economics, Los Angeles, CA, USA; 2Policy and Economics, Precision Health Economics, Boston, MA, USA; 3School of Pharmacy, Sol Price School of Public Policy, Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California, Los Angeles, CA, USA; 4Health Economics and Outcomes Management, Otsuka Pharmaceutical Development & Commercialization, Inc., Princeton, NJ, USA Background: New digital technologies offer providers the promise of more accurately tracking patients’ medication adherence. It is unclear, however, whether access to such information will affect provider treatment decisions in the real world.Methods: Using prescriber-reported information on patient non-compliance from health insurance claims data between 2008 and 2014, we examined whether prescribers’ knowledge of non-compliance was associated with different prescribing patterns for patients with serious mental illness (SMI). We examined patients who initiated an oral atypical antipsychotic, but were later objectively non-adherent to this treatment, defined as proportion of days covered (PDC)

Introduction: Myelodysplastic syndromes (MDS) include a diverse group of bone marrow disorders associated with high mortality and morbidity. In individuals with MDS, the bone marrow produces dysfunctional blood cells with about one-third of patients progressing to acute myeloid leukemia (AML). Previous estimates of the economic burden of MDS are highly variable, with the existing clinical literature deficient of detailed economic data and existing economic studies lacking accurate information on MDS diagnostics. To address the issue, this study uses registry-linked claims data from SEER-Medicare to estimate the economic burden of MDS among elderly individuals in the US. Methods: We conducted a retrospective case-control study using SEER-Medicare data from January 1, 2010 to December 31, 2016. The study included two cohorts, both aged ≥66 years: (i) patients diagnosed with MDS between January 1, 2011 and December 31, 2015, and (ii) cancer-free controls from a 5% random sample of Medicare beneficiaries. Patients with MDS were followed from index date, defined as the date of initial MDS diagnosis, to death, study end date, or 24 months post-index, whichever occurred first. A 1-year pre-index lookback period was used to identify comorbidities. Except in the instance of death, all individuals had a minimum of 1-year post-index follow-up. We propensity score-matched MDS cases and controls using demographic factors and comorbidities. After examining standardized differences between the MDS and control groups under various caliper widths and allowed numbers of matches, a caliper width of 0.05 of the standard deviation of the propensity score with 25 matched neighbors with replacement was chosen, as this minimized standardized differences across covariates. We calculated average 30-day cost burden for each matched patient based on costs incurred over the number of days in their individual follow-up period. We then estimated incremental healthcare cost burden for patients with MDS relative to the matched controls, including inpatient, outpatient, and pharmaceutical drug costs. We used a generalized linear model (GLM) with a log link and adjusted for demographics and comorbidities. Results: Of the 18,780 patients diagnosed with MDS during the study period, 9,524 met the inclusion criteria. Prior to matching, the control group of 288,877 cancer-free patients was 40% male with a mean age of 75.8 years (SD=8.3), while the MDS group was 57% male with a mean age of 80.1 years (SD=7.4). In addition, standardized differences between the groups were >0.2 for 50% (8/16) of comorbidities considered. After matching, the number of matched controls fell to 109,075 individuals that were 57% male with a mean age of 80.6 (SD=8.4), and standardized differences were Conclusion: The economic burden of MDS is higher than that of the cancer-free Medicare population, with an estimated incremental adjusted cost burden of nearly $130,000 in the two years following MDS diagnosis. Medical costs make up the majority of this cost difference. Our results provide an updated estimate of the economic burden of MDS using the most recent data and MDS coding methodology, with detailed estimates of cost by component. Our results fall within the range of estimates of previous studies using SEER-Medicare data. Figure Disclosures Shafrin: Precision Medicine Group: Employment, Equity Ownership. Green:Precision Health Economics: Employment, Other: Employee of Precision Health Economics (PHE). PHE received funding from Breath Therapeutics for this research. . Murphy:Precision Medicine Group: Employment. Everson:Precision Medicine Group: Employment. Dennen:Precision Medicine Group: Employment. Price:Jazz Pharmaceuticals: Employment, Equity Ownership. Batt:Precision Medicine Group: Consultancy.

Background: Recent literature provides conflicting views on whether there is a negative value trend in pharmaceutical oncology treatments. A previous study (J Econ Perspect 2015;29:1) found that the price of pharmaceutical oncology treatments has outpaced the improvements in survival benefits over time, with most treatments developed since 1995 offering marginal improvements in survival with significant increases in costs. In contrast, other literature suggests pharmaceutical oncology treatments have produced significant value. Chimeric antigen receptor t-cell (CAR-T) therapy represents a novel approach for treating particular hematologic cancers-including pediatric acute lymphoblastic leukemia and diffuse large b-cell lymphoma-but a remaining question is whether CAR-T represents a continued trend of low-value innovation, or a significant break from past trends. In this study, we investigate three key questions: (i) what are the trends over the past 10 to 20 years in incremental quality-adjusted life years (QALYs) and incremental cost per incremental quality-adjusted life year (cost/QALY) for newly approved anti-cancer innovations? (ii) how do innovations for hematologic cancers differ from those for other cancers in terms of this value trend? and (iii) how does CAR-T therapy compare with the recent history of innovations for both hematologic and non-hematologic cancers? Methods: We identified all analyses of pharmaceutical treatments for cancer published since 2007 that appear in the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, a comprehensive database focusing on a subset of CEAs called cost-utility analyses (CUAs), which quantify health benefits in terms of QALYs. CUA results on CAR-T therapies came from an analysis conducted by the Institute for Clinical and Economic Review (2018). We limited the CUAs in our study to those conducted in the US context with at least one of the following measures of value: incremental QALYs or cost/QALY, and with intervention/indication approval year 1995 or later. We measured linear trend in the value measures as a function of approval year and compared interventions for non-hematologic cancers with non-CAR-T interventions for hematologic cancers and, in turn, with CAR-T therapy. Regression analysis was used to control for the potential influence of characteristics of a CUA such as discount rate chosen, time horizon, number of years between publication and approval of the intervention for the indication in question, and an indicator for rare diseases as defined by the Genetic and Rare Diseases Information Center of the NIH. Indicators for CAR-T therapy and for non-CAR-T treatments for hematologic cancers were included to test for differences from innovations for other cancer types. Results: 103 incremental QALY and 108 cost/QALY measures met our inclusion criteria. The figures show incremental QALYs and cost/QALY, respectively, by approval year. The regression analysis shows that -- for innovations as a whole -- incremental QALYs gained have declined with approval year by 0.079 per year (95% CI -0.128 to -0.030). Cost effectiveness has worsened somewhat over time although that trend is not statistically significant (cost/QALY increase of $36,147 per year, 95%CI -$29,575 to $101,868). CAR-T is substantially more effective than both pharmaceutical cancer innovations outside of hematology (5.17 more incremental QALYs, 95%CI 4.09 to 6.25) and other innovations within hematology (4.67 more incremental QALYs, 95%CI 3.44 to 5.90). Innovations in hematology other than CAR-T are somewhat more effective than innovations outside of hematology (0.5 more incremental QALYs, 95%CI -0.09 to 1.09), although the difference is not statistically significant. Differences in cost/QALY between CAR-T and other pharmaceutical interventions are not statistically significant. Conclusions: CAR-T therapy improved health outcomes (measured in QALYs) to a greater extent than both treatments for non-hematologic cancers and non-CAR-T treatments for hematologic cancers, with no significant differences in cost/QALY. Those improvements represent a break from a trend previously reported in the literature of negligible incremental effectiveness and rising costs per life-year gained. Disclosures Baumgardner: Precision Health Economics: Employment. Everson:Precision Health Economics: Employment. Brauer:Precision Health Economics: Employment. Zhang:Novartis Pharmaceuticals Corporation: Employment, Equity Ownership. Hao:Novartis Pharmaceuticals Corporation: Employment, Equity Ownership. Liu:Novartis Pharmaceuticals Corporation: Employment, Equity Ownership. Lakdawalla:Precision Health Economics: Consultancy; Novartis Pharmaceuticals Corporation: Other: PHE conducted the research underlying this study with funding from Novartis.; Precision Medicine Group: Equity Ownership.