Estimated Prevalence and Testing for Albuminuria in US Adults at Risk for Chronic Kidney Disease.

Importance: Albuminuria testing is crucial for guiding evidence-based treatments to mitigate chronic kidney disease (CKD) progression and cardiovascular morbidity, but it is widely underutilized among persons with or at risk for CKD. Objective: To estimate the extent of albuminuria underdetection from lack of testing and evaluate its association with CKD treatment in a large US cohort of patients with hypertension or diabetes. Design, Setting, and Participants: This cohort study examined adults with hypertension or diabetes, using data from the 2007 to 2018 National Health and Nutrition Examination Surveys (NHANES) and the Optum deidentified electronic health record (EHR) data set of diverse US health care organizations. Analyses were conducted from October 31, 2022, to May 19, 2023. Main Outcomes and Measures: Using NHANES as a nationally representative sample, a logistic regression model was developed to estimate albuminuria (urine albumin-creatinine ratio ≥30 mg/g). This model was then applied to active outpatients in the EHR from January 1, 2017, to December 31, 2018. The prevalence of albuminuria among those with and without albuminuria testing during this period was estimated. A multivariable logistic regression was used to examine associations between having albuminuria testing and CKD therapies within the subsequent year (prescription for angiotensin-converting enzyme inhibitor [ACEi] or angiotensin II receptor blocker [ARB], prescription for sodium-glucose cotransporter 2 inhibitor [SGLT2i], and blood pressure control to less than 130/80 mm Hg or less than 140/90 mm Hg on the latest outpatient measure). Results: The total EHR study population included 192 108 patients (mean [SD] age, 60.3 [15.1] years; 185 589 [96.6%] with hypertension; 50 507 [26.2%] with diabetes; mean [SD] eGFR, 84 [21] mL/min/1.73 m2). There were 33 629 patients (17.5%) who had albuminuria testing; of whom 11 525 (34.3%) had albuminuria. Among 158 479 patients who were untested, the estimated albuminuria prevalence rate was 13.4% (n = 21 231). Thus, only 35.2% (11 525 of 32 756) of the projected population with albuminuria had been tested. Albuminuria testing was associated with higher adjusted odds of receiving ACEi or ARB treatment (OR, 2.39 [95% CI, 2.32-2.46]), SGLT2i treatment (OR, 8.22 [95% CI, 7.56-8.94]), and having blood pressure controlled to less than 140/90 mm Hg (OR, 1.20 [95% CI, 1.16-1.23]). Conclusions and Relevance: In this cohort study of patients with hypertension or diabetes, it was estimated that approximately two-thirds of patients with albuminuria were undetected due to lack of testing. These results suggest that improving detection of CKD with albuminuria testing represents a substantial opportunity to optimize care delivery for reducing CKD progression and cardiovascular complications.

Poor adherence to clinical practice guidelines: A call to action for increased albuminuria testing in patients with type 2 diabetes.

We describe the substantial shortfall in adherence to guideline-recommended albumin-to-creatinine ratio (uACR) testing for people in the United States with type 2 diabetes. Poor compliance with current guidelines leads to delays in diagnosis-and treatment- of chronic kidney disease, which adversely affects clinical outcomes and contributes to incremental economic burden.

Unmet needs and opportunities for optimal management of patients with type 2 diabetes and chronic kidney disease.

This brief report utilizes EHR data from a large US health system to summarize unmet needs in patients with type 2 diabetes and chronic kidney disease and identifies areas of opportunity to optimize management within this patient population from treatment, screening and monitoring, and health care resource use perspectives.

Descriptive study of the economic burden among patients with type 2 diabetes mellitus, chronic kidney disease, and chronic kidney disease and type 2 diabetes mellitus in a large US commercially insured population.

BACKGROUND: Chronic kidney disease (CKD) is a major public health concern that affects 37 million adults in the United States. It is well known that CKD presents a large economic burden, especially in the Medicare population. However, studies of the economic burden of CKD in younger populations are scarce. In particular, there is a gap in understanding how the presence of type 2 diabetes mellitus (T2DM) affects the burden of CKD in commercially insured populations. OBJECTIVE: To describe the economic and health care resource utilization (HCRU) burden of CKD within 3 patient groups (T2DM only, CKD only, and CKD and T2DM) aged 45-64 years overall and by Kidney Disease Improving Global Outcomes (KDIGO) CKD estimated glomerular filtration rate-based stage categories. METHODS: A descriptive, observational retrospective cohort study was conducted using administrative medical and pharmacy claims integrated with laboratory results data available in the HealthCore Integrated Research Database from January 1, 2017, to December 31, 2019. Three mutually exclusive groups of commercially insured patients aged 45-64 years were identified: T2DM only, CKD only, and CKD and T2DM. All-cause and disease-specific HCRU and costs in total, by medical and pharmacy benefits and across all places of service, were described for each of these groups 12 months after index date. For the CKD only and CKD and T2DM groups, costs were also described by KDIGO CKD stage. RESULTS: The CKD and T2DM group (n = 13,052) had numerically higher 12-month post-index all-cause and CKD/T2DM-related HCRU across all places of service. Mean 12-month all-cause costs for this group were $35,649, whereas costs for the CKD only group (n = 7,876) were $25,010 and costs for the T2DM only group (n = 120,364) were $16,121. Costs also tended to increase as CKD stage increased, with the greatest increases beginning at KDIGO stage 3b and higher. Mean 12-month all-cause costs for the CKD and T2DM group ranged from $29,993 to $41,222 for stages 1 to 3a and from $46,796 to $119,944 for stages 3b to 5. CONCLUSIONS: Commercially insured patients aged 45-64 years with CKD, especially those who also have T2DM, present a substantial burden in terms of elevated HCRU and costs. Costs tend to increase across KDIGO CKD stages and increase most rapidly at stage 3b and later. Therefore, there is an opportunity to reduce the burden of CKD in this population by investing in interventions to prevent or delay CKD disease progression. DISCLOSURES: HealthCore, Inc, received funding to perform this research, as well as funding from multiple pharmaceutical companies to perform various research studies outside of the submitted work. Mr Crowe and Dr Willey are employees of HealthCore, Inc., a wholly owned subsidiary of Elevance Health, Inc. Ms Chung was an employee of HealthCore, Inc., a wholly owned subsidiary of Elevance Health, Inc, at the time of study performance. Ms Chung and Dr Willey are shareholders of Elevance Health, Inc. Dr Kong, Dr Singh, Mr Farej, Dr Elliot, and Dr Williamson are employees of Bayer US, LLC. Dr Singh is a shareholder of Bayer US, LLC.

Improved Uranium Particle Analysis by SIMS using O3 - Primary Ions.

We have investigated the use of negative molecular oxygen primary ion beams (i.e., O2 - and O3 -) to determine the benefits of using such beams for uranium particle SIMS analyses. Typically, O- is the most practical negative primary ion species from the conventional duoplasmatron ion source for both age dating and uranium isotopic analysis of particles. Newer RF plasma ion sources make it possible to use O2 - and O3 - due to higher brightness and primary ion fluence, and the increased abundance of molecular species in the plasma relative to the duoplasmatron. We have determined that by using an O3 - beam, the ionization yield can be increased by a factor of approximately two over an O- beam, up to 4.7%, a substantial improvement that positively impacts measurement precision and detection limits. We also investigated the effect of the molecular oxygen beams on uranium isotope mass fractionation and the Th/U relative sensitivity factor for SIMS analyses in comparison to O- beams. We found that O3 - reduced instrumental mass fractionation and matrix/substrate effects relative to the other negative ion beams. Particle measurements using O3 - were improved compared to conventional O- beam analyses due to higher yields, smaller corrections, and reduced substrate effects.

Comparative effectiveness of larotrectinib versus entrectinib for the treatment of metastatic NTRK gene fusion cancers.

Aim: To extrapolate clinical trial results to estimate and compare expected progression-free and overall life years (LYs) and quality-adjusted LYs (QALYs) for larotrectinib and entrectinib in patients with colorectal cancer (CRC), soft tissue sarcoma (STS) and brain metastases prior to treatment with larotrectinib or entrectinib. Methods: A naive direct comparison of larotrectinib versus entrectinib was made using partitioned survival modeling methods from clinical trial data. Results: Larotrectinib resulted in an additional 1.58 LYs (1.17 QALYs), 5.81 LYs (2.02 QALYs) and 1.01 LYs in CRC, STS and baseline brain metastases, respectively, compared with entrectinib. Conclusion: Larotrectinib provided life expectancy and QALY gains compared with entrectinib. Additional studies will be beneficial as more patients are treated and survival data develop to better inform comparative effectiveness results.

The potential long-term comparative effectiveness of larotrectinib vs standard of care for treatment of metastatic TRK fusion thyroid cancer, colorectal cancer, and soft tissue sarcoma.

BACKGROUND: Larotrectinib is approved for patients with metastatic TRK fusion cancers, including differentiated thyroid (DTC), colorectal cancer (CRC), and soft tissue sarcoma (STS). Given the basket clinical trial design of larotrectinib, direct comparisons against standard of care in each of the mentioned cancers have not been assessed. Also, owing to the limited duration of follow-up in clinical trials, long-term outcomes for treatments are generally not known or estimated. OBJECTIVE: To compare expected life-years (LYs) and quality-adjusted life-years (QALYs) for patients with metastatic DTC, CRC, and STS who are eligible to receive larotrectinib against patients with unknown NTRK gene fusion status receiving standard-of-care therapy. METHODS: We developed a partitioned survival model to estimate the long-term comparative effectiveness of larotrectinib and standard of care for 3 tumor types. Larotrectinib survival data, assessed by independent review committee, were derived from an updated July 2020 analysis of 19, 8, and 23 adult patients (aged ≥ 18 years) with metastatic TRK fusion DTC, CRC, and STS, respectively. The DTC survival data also included 2 patients aged less than 18 years for a total of 21 patients. Survival estimates for standard of care were derived from published clinical trials. Progressionfree and overall survival for all treatments were estimated using survival distributions (Exponential, Weibull, Log-logistic, and Lognormal) fit to the available data. The final exponential form was selected based on goodness-of-fit and clinical plausibility. QALYs were estimated by adjusting the time spent in the preprogression and postprogression health states by utility weights derived from publicly available literature. RESULTS: Patients receiving larotrectinib experienced more LYs and QALYs compared with those receiving standard-of-care treatments across all 3 assessed cancer types. In DTC, patients receiving larotrectinib had 7.15-8.26 additional LYs (5.87-6.12 QALYs); in CRC, patients receiving larotrectinib had 1.26-1.27 additional LYs (1.00 QALYs); and in STS, patients receiving larotrectinib had 5.56 additional LYs (1.99 QALYs). CONCLUSIONS: Compared with standard of care in metastatic TRK wild-type cancers, larotrectinib is estimated to result in improved LY and QALY outcomes based on parametric extrapolations of intrial survival data. Because patient-level data were unavailable for adjusted analyses, a cross-trial comparison was performed. Given the limitations of this analytic approach and the small sample size for larotrectinib in trials, future studies should reassess the comparative effectiveness of larotrectinib vs standard of care as treated patients accrue and long-term survival data mature. DISCLOSURES: K. Suh, J. Carlson, and S. Sullivan report consulting fees from Bayer US LLC. F. Xia and T. Williamson are employees of Bayer US LLC. This study was funded by Bayer US LLC. The sponsor had no role in the design of the study and did not have any role in the execution, analyses, interpretation of the data, or decision to submit results.

Quantifying the economic burden of unintended pregnancies due to drug-drug interactions with hormonal contraceptives from the United States payer perspective.

Background: In the United States of America (USA), nearly 10 million women use oral contraceptives (OCs). Concomitant administration of certain medications can result in contraceptive failure, and consequently unintended pregnancies due to drug-drug interactions (DDIs). The objective of this analysis was to estimate the economic impact of unintended pregnancies due to DDIs among women of reproductive age using an OC alone or in combination with an enzyme inducer co-medication in the USA from a payer perspective. Methods: A Markov model using a cohort of 1,000 reproductive-age women was developed to estimate costs due to contraceptive failure for OC alone versus OC with concomitant enzyme inducer drugs. All women were assumed to begin an initial state, continuing until experiencing an unintended pregnancy. Unintended pregnancies could result in birth, induced abortion, spontaneous abortion, or ectopic pregnancy. The cohort was analyzed over a time horizon of 1 year with a cycle length of 1 month. Estimates of costs and probabilities of unintended pregnancy outcomes were obtained from the literature. Probabilities from the Markov cohort trace was used to estimate number of pregnancy outcomes. Results: On average, enzyme inducers resulted in 20 additional unintended pregnancies with additional unadjusted and adjusted costs median (range) of USD136,304 (USD57,436-USD320,093) and USD65,146 (USD28,491-USD162,635), respectively. The major component of the direct cost is attributed to the cost of births. Considering the full range of events, DDIs with enzyme inducers could result in 16-25 additional unintended pregnancies and total unadjusted and adjusted costs ranging between USD46,041 to USD399,121 and USD22,839 to USD202,788 respectively. Conclusion: The direct costs associated with unintended pregnancies due to DDIs may be substantial and are potentially avoidable. Greater awareness of DDI risk with oral contraceptives among payers, physicians, pharmacists and patients may reduce unintended pregnancies in at-risk populations.

THE AUTHORS RESPOND.

DISCLOSURES: Funding for the study referred to in this letter was contributed by Bayer Healthcare. Xia and Williamson are employees of Bayer Healthcare. Roth, Carlson, and Sullivan are consultants to Bayer Healthcare. Carlson also reports fees from Adaptive Biotechnologies, unrelated to the study. Roth reports consulting fees from BMS, unrelated to the study.

Rosacea Treatment Satisfaction: Matching Adjusted Indirect Treatment Comparison Analysis of Metronidazole Gel or Cream vs Azelaic Acid Foam.

OBJECTIVE: To assess differences in patient-reported treatment side effects and concerns associated with azelaic acid 15% foam (AAF) vs metronidazole cream (MC) and metronidazole gel (MG). METHODS: This study used matching-adjusted indirect comparison (MAIC) to compare patient-reported outcomes from survey data evaluating rosacea treatments. Outcomes of interest included percentages of patients reporting concerns and side effects and measures of importance of the concerns and tolerability of the side effects. Patients in each analysis (MG vs AAF and MC vs AAF) were matched using stabilized inverse propensity scores. RESULTS: When compared to AAF, MG-treated patients more frequently reported concerns with treatment efficacy (54% vs 4%), application (7% vs 3%), and treatment side effects. MC-treated patients more frequently reported concerns with treatment efficacy (61% vs 5%) and dryness (8% vs 5%). AAF-treated patients more frequently reported concerns with cost of treatment compared with MG (7% vs 1%) and MC (9% vs 4%). Among patients reporting concerns, level of importance associated with these concerns was similar for AAF-treated patients compared with MG- and MC-treated patients. When compared to AAF-treated patients, MG-treated patients more frequently reported side effects of dryness (26% vs 15%) and uneven skin tone (3% vs 0%), and MC-treated patients more frequently reported side effects of burning (7% vs 3%), itching (7% vs 5%), and redness (7% vs 5%). MG- and MC-treated patients indicated greater intolerance for reported side effects than AAF-treated patients. CONCLUSIONS: MG- and MC-treated patients more frequently reported treatment concerns and side effects than AAF-treated patients, and tolerability of those side effects was higher for patients treated with AAF. While treatment cost is a more frequent concern in patients treated with AAF, these patients less frequently reported concerns with treatment efficacy and reported similar or greater tolerance to side effects than patients treated with either MC or MG. J Drugs Dermatol. 2020;19(3): doi:10.36849/JDD.2020.3679.

The Potential Long-Term Comparative Effectiveness of Larotrectinib and Entrectinib for Second-Line Treatment of TRK Fusion-Positive Metastatic Lung Cancer.

BACKGROUND: Larotrectinib and entrectinib are FDA-approved therapies for patients with non-small cell lung cancer (NSCLC) with neurotrophic receptor tyrosine kinase gene fusion (TRK fusion-positive) whose cancer has metastasized and progressed. Early evidence indicates that these targeted therapies may offer dramatic survival benefits versus traditional cytotoxic regimens, but it remains uncertain how larotrectinib and entrectinib compare with each other. OBJECTIVE: To simulate and compare expected life-years and quality-adjusted life-years (QALYs) for both TRK inhibitors. METHODS: We developed a partitioned survival model to project the long-term comparative effectiveness of larotrectinib versus entrectinib in second-line treatment of metastatic NSCLC. Larotrectinib survival data were derived from a 13-month follow-up of 12 patients with TRK fusion-positive NSCLC in the NCT02122913 (phase 1) and NCT02576431 (NAVIGATE) trials. Entrectinib survival data were derived from a 13-month follow-up of 10 patients with TRK fusion-positive NSCLC in the ALKA-372-001, STARTRK-1, and STARTRK-2 trials. For larotrectinib and entrectinib progression-free survival and overall survival (OS), in-trial survival was extrapolated using parametric curve fits. Exponential fits were selected for all survival models based on minimal Bayesian information criteria and clinical plausibility. Lifetime survival curves were used to estimate expected mean/median survival. QALYs were estimated by applying preprogression and postprogression health state utilities derived from the literature. RESULTS: In the base case, treatment with larotrectinib and entrectinib resulted in 5.4 and 1.2 median preprogression life-years and 7.0 and 1.8 median total life-years, respectively. Mean preprogression life-years (QALYs) were 7.5 (5.0) and 1.9 (1.2), and mean total life-years (QALYs) were 9.2 (5.8) and 4.4 (2.4), respectively. CONCLUSIONS: Among TRK inhibitors for metastatic NSCLC, larotrectinib is estimated to provide improved life-year and QALY outcomes versus entrectinib based on parametric extrapolations of in-trial survival data. Our analysis is limited by lack of NSCLC-specific data on entrectinib OS, the small samples of patients with NSCLC in the trials, and a cross-trial comparison. Future studies should re-evaluate the comparative effectiveness of larotrectinib versus entrectinib as more patients are treated and as long-term survival data mature. DISCLOSURES: Funding for this study was contributed by Bayer Healthcare, which reviewed the manuscript drafts, and employees contributed to the manuscript as coauthors. Xia and Williamson are employees of Bayer Healthcare. Roth, Carlson, and Sullivan are consultants to Bayer Healthcare and retain rights to all final revisions to the manuscript. Carlson also reports fees from Adaptive Biotechnologies, unrelated to this work. Roth reports consulting fees from BMS, unrelated to this work.

Current status of biomarker testing in historically rare, high-unmet-need tumors: soft tissue sarcomas and thyroid cancers.

Introduction: The development of precision medicine and targeted therapies have revolutionized cancer treatment. Historically, treatment was chosen based on the tumor-histology, but can now be tailored to patient-specific biomarkers. Investigations have shown up to 40% of cancer patients who undergo molecular testing have an actionable biomarker with a drug currently available, and that patients benefit from these drugs. In 2018, larotrectinib became the first drug developed and approved exclusively as a tumor-agnostic therapy. Because of advancement in precision medicine, biomarker testing has become a standard of care in a variety of tumors and its use is increasing.Areas covered: We discuss the landscape of biomarker testing in the context of soft tissue sarcomas and thyroid cancer, two rare diseases with historically high unmet needs in their subpopulations. We summarize historical data and contemporary applications.Expert opinion: The paradigm shift in oncology treatment toward precision medicine, including tumor-agnostic agents, is experiencing substantial momentum but still facing challenges. A gap exists between guideline recommendations for biomarker testing and clinical application, resulting in compromised access and suboptimal outcomes. Future progress will require routine access to testing and expanding treatment options coupled with awareness, predictability, and strategies to address resistance mechanisms.

Concerns and Treatment Satisfaction in Patients Being Treated With Azelaic Acid Foam for Rosacea

Objective: To describe patient characteristics, concerns, side effects, treatment satisfaction, and quality of life (QoL) of rosacea patients currently being treated with monotherapy azelaic acid foam based on patient-reported data. Methods: The study utilized a non-interventional, prospective, observational design. Patients were recruited in the United States and were eligible if the following criteria were met: diagnosed with rosacea by a medical professional, ≥18 years of age, currently receiving monotherapy with azelaic acid foam, and able to provide informed consent. Patients using other topical treatments for rosacea during enrollment were excluded. An online tool administered a survey of 3 questionnaires including the Rosacea Treatment Preference Questionnaire, Treatment Satisfaction with Medicines Questionnaire (SATMED-Q), and Dermatology Life Quality Index (DLQI). The survey collected demographics, clinical characteristics, treatment history, adverse events, and patient-reported outcomes related to treatment with azelaic acid foam and QoL with rosacea. Results: 54 patients met eligibility criteria. Participants were primarily female (90.7%), ranging from 26 to 63 years of age. The most common subtypes reported were erythematotelangiectatic and papulopustular (74.1% each) with 59.3% of participants reporting mild symptoms (16.7% "absent"; 24.1% "moderate") in the 4 weeks before enrollment. The majority reported no concerns (74.1%) with their treatment. The biggest concern was cost (11.1%), with a mean importance score (IS) on a 10-point scale of 9.3. A majority (77.8%) of patients reported no side effects. Side effects reported included dryness (13%; IS: 5.3), stinging (7.4%, IS: 2.5), itching (5.6%; IS: 4.7), or burning (3.7%; IS: 7.0). Global satisfaction (SATMED-Q) mean score was 79.0 and treatment effectiveness mean score was 70.8. QoL impact of rosacea was minimal (mean DLQI score: 2.35). In regression models, increasing dryness was significantly associated with worsening outcomes in SATMED-Q and DLQI. Conclusions: Patient characteristics of the study population closely mirror the distribution of rosacea by gender and subtype as in previous estimates. Findings indicate minimal patient concerns with azelaic acid foam and primarily pertained to cost. Patient-reported side effects were rare. Minor patient-reported side effects and concerns do not appear to affect rosacea-related QoL and medication satisfaction. Compared to a previously conducted study of similar design with patients using metronidazole gel and metronidazole cream, more patients in the current study reported no concerns with their treatment, while the number of patients reporting no side effects, as well as mean SATMED-Q and DLQI scores, were similar. Further research is necessary to directly compare the results of these 2 studies. J Drugs Dermatol. 2019;18(4):381-386.

Budget Impact of Appropriate Low-Dose Aspirin Use for Primary and Secondary Cardiovascular Event Prevention in the Managed Care Setting.

BACKGROUND: Cardiovascular disease remains the leading cause of death in adults in the United States and constitutes a substantial portion of overall national health expenditures. Aspirin is generally recommended for primary cardiovascular event prevention based on a given patient's underlying cardiovascular event risk profile, particularly for those aged 50-69 years with a 10-year risk of coronary heart disease of ≥ 10%. Evidence-based clinical guidelines are in agreement for secondary prevention consisting of lifelong, low-dose aspirin therapy following a cardiovascular event. Despite these recommendations, research suggests suboptimal concordance between guidelines and clinical practice. OBJECTIVE: To evaluate the budget impact of appropriate low-dose aspirin use for primary and secondary cardiovascular event prevention compared with current rates of low-dose aspirin use. METHODS: An economic model measuring budget spend for cardiovascular events, aspirin, and aspirin-related adverse events was developed from the perspective of a U.S. payer. The model compared current rates of aspirin use to appropriate rates of aspirin use according to guideline recommendations for both primary and secondary cardiovascular event prevention. RESULTS: For a hypothetical plan with 1 million members, an estimated 18,026 patients were on aspirin therapy for primary cardiovascular event prevention, while guidelines recommend that 55,788 patients should have been on aspirin therapy for this indication. Optimal aspirin use in the primary cardiovascular event prevention population reduced the number of nonfatal myocardial infarctions (MIs; -367), ischemic strokes (-232), and deaths (-60), with an increase in the number of gastrointestinal bleeds (169) and hemorrhagic strokes (98). Evidence-based guideline-compliant use of aspirin for primary cardiovascular event prevention resulted in total cost savings of approximately $4.2 million over a 5-year time horizon. For secondary cardiovascular event prevention, an estimated 48,663 patients were on aspirin, while clinical guidelines recommend that 71,316 patients should have been on aspirin therapy for this indication. Optimal aspirin use in secondary cardiovascular event prevention reduced the number of nonfatal MIs (-515), ischemic strokes (-375), and deaths (-217), with an increase in the number of gastrointestinal bleeds (98) and hemorrhagic strokes (58). Evidence-based guideline-compliant use of aspirin for secondary cardiovascular event prevention resulted in total cost savings of approximately $11 million over a 5-year time horizon. CONCLUSIONS: Appropriate low-dose aspirin use for primary and secondary cardiovascular event prevention can result in improved patient outcomes with significant cost savings for U.S. payers. As a simple and inexpensive prophylactic measure for cardiovascular event prevention, aspirin use should be carefully considered in all appropriate at-risk adult patients. DISCLOSURES: Development of this manuscript and the corresponding budget impact analysis was funded by Bayer. Coppolecchia, Williamson, and Cameron are employees of Bayer. Carlton, Lennert, and Moradi are employees of Xcenda, a consulting firm that received funding from Bayer to assist in the completion of this study. Khalaf-Gillard was an employee of Xcenda at the time of the study. The corresponding poster was presented at the Academy of Managed Care Pharmacy Nexus 2017; October 16-19, 2017; Dallas, TX.

Patient Preferences and Therapeutic Satisfaction with Topical Agents for Rosacea: A Survey-Based Study.

BACKGROUND: Rosacea is a chronic inflammatory skin disorder that primarily affects the convexities of the central face. Depending on the severity and type of rosacea, physicians may prescribe interventions such as behavioral changes, laser and intense pulsed light, as well as various pharmacologic therapies, including topical agents. The impact of side effects associated with topical treatments for rosacea on patient preferences and treatment satisfaction is not well-documented. OBJECTIVE: To assess patients' concerns, treatment satisfaction, and quality of life (QOL) associated with topical treatments for rosacea. METHODS: Patients were identified for participation in a one-time survey from electronic medical records between 2010 and 2015 from the largest privately held and physician-run multispecialty group practice in Massachusetts. Patients were eligible to participate in the survey if they were aged ≥18 years and had ≥1 diagnoses of rosacea, ≥1 prescriptions for topical metronidazole gel/cream or azelaic acid gel, ≥6 months of follow-up, and an active treatment record in 2014. Treatment-related concerns and their importance were assessed using a questionnaire developed for this study. Treatment satisfaction and QOL were evaluated using the Treatment Satisfaction with Medicines Questionnaire (SATMED-Q) and the Dermatology Life Quality Index (DLQI), respectively. RESULTS: Of the 900 eligible patients surveyed, 216 (24%) responded. Among the responders, 122 reported currently using a topical rosacea treatment. The most common treatment-related concerns were efficacy (64.8%), skin dryness (18.4%), unspecified side effects (9.6%), burning sensation (8.8%), and application technique (8.0%). The treatment-related concerns that were assessed as most important by responders included efficacy (mean score 9.1, on a 10-point scale), soreness (7.6), itching (7.5), burning (7.4), and dryness (7.3). Averaged across all the responders, treatment satisfaction was rated as neutral (mean SATMED-Q score, 56.5), whereas the impact of rosacea on QOL was minimal (mean DLQI score, 2.7). Increasing dryness was significantly associated with worsening QOL, and trends toward significance were observed for increasing soreness, itching, and burning sensations. CONCLUSIONS: The survey results suggest a need for novel topical therapies for patients with rosacea that have increased efficacy and tolerability, which may improve patient satisfaction and QOL.

Rate of Adverse Events and Healthcare Costs Associated with the Topical Treatment of Rosacea.

BACKGROUND: Rosacea is a condition more common in women than in men, and in people aged ≥30 years than in younger patients. Adverse events associated with the use of topical medications for rosacea may lead to a lack of treatment adherence. Previous studies have reported low treatment adherence rates among patients with rosacea. OBJECTIVE: To describe the rate of treatment discontinuation resulting from adverse events and the associated healthcare costs among patients with rosacea who are receiving a topical medication. METHODS: We conducted a retrospective cohort study of patients diagnosed with rosacea based on International Classification of Diseases, Ninth Revision, Clinical Modification code 695.3 who were newly initiating topical treatment with metronidazole, azelaic acid, sodium sulfacetamide/sulfur, or benzoyl peroxide between January 1, 2009, and September 30, 2013. Patients were identified from the MarketScan Commercial Claims and Encounters database and the Medicare Supplemental database and had to be aged ≥30 years, have continuous coverage with medical and pharmacy benefits ≥12 months before treatment and ≥3 months after treatment inititation, and have no evidence of oral antibiotic use or ocular rosacea during the study period. The 3-month period immediately after the index date (ie, first topical rosacea treatment) was defined as the postindex period and was used to evaluate the outcome measures, which included the rate of adverse events, treatment patterns, and healthcare costs. RESULTS: The final cohort included 49,351 patients, with a mean age of 54 years, and 74.5% of the patients were female. Metronidazole was the most common (72.7%) treatment, followed by azelaic acid (21.7%), sodium sulfacetamide/sulfur (3.4%), and benzoyl peroxide (2.2%). A total of 6270 (12.7%) patients had a coded adverse event, of whom 199 (3.2%) continued treatment despite the adverse event, 466 (7.4%) switched to another treatment within 8.8 days, and 5605 (89.4%) discontinued therapy within 31.1 days. Patients with adverse events incurred, on average, a cost of $325 (medical, $143; pharmacy, $182) in rosacea-related costs; patients without adverse events incurred, on average, a cost of $172 (medical, $14; pharmacy, $157) in rosacea-related costs. CONCLUSIONS: The majority of adverse events associated with current topical drugs for rosacea resulted in treatment switch or discontinuation. Drugs with a different mechanism of action or new formulations of existing drugs may provide additional treatment options for patients and may lead to improved adherence and better symptom control.

Excess costs associated with patients with chronic thromboembolic pulmonary hypertension in a US privately insured population.

BACKGROUND: Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare and potentially fatal disease. Little is known about the economic burden associated with CTEPH patients in the US. OBJECTIVES: The objective of this study was to estimate excess direct costs associated with privately insured patients with CTEPH in the US. METHODS: From a privately insured claims database (>8 million beneficiaries, 2002-7), 289 CTEPH patients were identified using the criteria: two or more claims for pulmonary hypertension (PH), International Classification of Diseases, ninth edition, clinical modification (ICD-9-CM) code 416.0 or 416.8; one or more claim for pulmonary embolism (ICD-9-CM: 415.1, V12.51; ICD-9 procedure: 38.7; Current Procedural Terminology [CPT]-4 code: 36010, 37620, 75825, 75940; Healthcare Common Procedure Coding System [HCPCS] code: C1880) within 12 months prior or 1 month after the initial PH claim (index date); one or more claim for right heart catheterization (RHC) within 6 months prior to any PH claim or one or more claim for echocardiogram within 6 months prior to a specialist-diagnosed PH claim; aged 18-64 years. Patients with CTEPH were matched demographically to controls without PH. Patients were followed as long as continuously eligible; mean follow-up in CTEPH patients was 21.5 months. Chi-squared tests were used to compare baseline co-morbidities. Wilcoxon rank-sum tests were used to compare direct (medical and pharmaceutical) patient-month costs to insurers. RESULTS: The average age for CTEPH patients was 52.2 years, and 57.1% were women. Compared with controls, CTEPH patients had significantly higher baseline rates of co-morbidities (e.g. essential hypertension, congestive heart failure and chronic pulmonary disease) and a higher mean Charlson Co-morbidity Index score. Mean direct patient-month costs (year 2007 values) were $US4782 for CTEPH patients and $US511 for controls (p < 0.0001). Sensitivity analysis restricting the sample to patients diagnosed following RHC yielded a 15% increase in excess costs relative to the original sample. Regarding cost drivers, inpatient services accounted for 54%, outpatient and other services for 33% and prescription drugs for 11% of total direct healthcare costs per patient-month in CTEPH patients. Circulatory-/respiratory-related patient-month costs were $US2496 among CTEPH patients and $US128 among controls (p < 0.0001). CONCLUSIONS: CTEPH patients had substantially higher costs and co-morbidity than matched controls, with circulatory-/respiratory-related costs accounting for 55% of excess costs. The high burden of illness suggests opportunities for savings from improved management.

Excess costs associated with patients with pulmonary arterial hypertension in a US privately insured population.

BACKGROUND: Pulmonary arterial hypertension (PAH) is a rare but fatal disease. Little is known about the economic burden associated with PAH patients in the US. OBJECTIVES: The objective of this study was to estimate excess direct costs associated with privately insured PAH patients in the US. METHODS: From a privately insured claims database (>8 million beneficiaries, 2002-7), 471 patients with PAH were identified using the criteria: two or more claims for primary pulmonary hypertension (PH), International Classification of Diseases, ninth edition, clinical modification (ICD-9-CM) code 416.0; no left heart disease, lung diseases, chronic thromboembolic PH or miscellaneous PH diagnoses within 12 months prior or 1 month after the initial PH claim (index date); one or more claim for right heart catheterization (RHC) within 6 months prior to any PH claim or one or more claim for echocardiogram within 6 months prior to a specialist-diagnosed PH claim; aged 18-64 years. Patients with PAH were matched demographically to controls without PH. Patients were followed as long as continuously eligible; mean follow-up of PAH patients was 24.8 months. Chi-squared tests were used to compare baseline co-morbidities. Wilcoxon rank-sum tests were used to compare direct (medical and pharmaceutical) patient-month costs to insurers. RESULTS: The average age for PAH patients was 52.2 years, and 55.8% were women. Compared with controls, PAH patients had significantly higher baseline rates of co-morbidities (e.g. essential hypertension, diabetes mellitus and congestive heart failure) and a higher mean Charlson Co-morbidity Index score. Mean direct patient-month costs (year 2007 values) were $US2023 for PAH patients and $US498 for controls (p < 0.0001), yielding excess costs of $US1525. Sensitivity analysis restricting the sample to patients diagnosed following RHC yielded a 64% increase in excess costs relative to the original sample. Regarding cost drivers, inpatient services accounted for 45%, outpatient and other services for 38% and prescription drugs for 15% of total direct healthcare costs per patient-month in PAH patients. Circulatory/respiratory system-related patient-month costs were $US724 among PAH patients and $US114 among controls (p < 0.0001). CONCLUSIONS: Patients with PAH had substantially higher costs and co-morbidity than controls, with circulatory/respiratory system-related costs accounting for 40% of excess costs. The high burden of illness suggests opportunities for savings from improved management.

Prevalence of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension in the United States.

BACKGROUND: The prevalence of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) in the US is largely unknown. Prior research has estimated PAH prevalence in Europe at ∼15-52 per million. METHODS: Using a privately insured claims database (1999-2007) for the under age 65 population and a Medicare claims database for the 65+ population, and following the current clinical classification of PH, CTEPH patients were identified as having: ≥2 claims for pulmonary hypertension (PH) [ICD-9-CM: 416.0, 416.8]; ≥1 claim for pulmonary embolism (PE) ≤12 months prior or 1 month after the initial PH claim (index date). PAH patients were identified: ≥2 claims for primary PH [416.0]; no left heart disease, lung diseases, CTEPH, or miscellaneous PH diagnoses ≤12 months prior or 1 month after the index date. Both cohorts were required to have ≥1 claim for right heart catheterization ≤6 months prior to any PH claim, or ≥1 claim for echocardiogram ≤6 months prior to a specialist-diagnosed PH claim. Age- and gender-standardized prevalence rates per million individuals (PMI) were calculated using appropriate population weights. RESULTS: Prevalence rates (95% CI) of CTEPH were estimated at 63 (34-91) PMI among the privately insured (<65), and 1007 (904-1111) PMI among the Medicare population (≥65). The corresponding estimates for PAH were 109 (71-146) PMI among the <65 population, and 451 (384-519) PMI for Medicare. LIMITATIONS: Identification of PAH and CTEPH patients in administrative claims data is challenging, due to lack of specific ICD-9-CM codes for the conditions and risk of misdiagnosis. CONCLUSIONS: Prevalence rates of CTEPH and PAH increase with age, and are higher among women. The increased risk of PE may explain the sharp age gradient for CTEPH prevalence. The estimated US prevalence of PAH is higher than existing estimates.

Prevalence rates and costs of metabolic syndrome and associated risk factors using employees' integrated laboratory data and health care claims.

OBJECTIVE: This study assessed the relative value of using laboratory, claims, or integrated laboratory-claims data to identify prevalence rates and costs of metabolic syndrome among Chevron Texaco Corporation, San Ramon, California, employees. METHODS: This study identified five metabolic syndrome risk factors by using three identification methods: (1) health-screening data, applying the National Cholesterol Education Program Adult Treatment Panel III and World Health Organization definitions; (2) employer-based claims data applying proxy International Classification of Diseases, 9th Revision definitions; and (3) integrated laboratory-claims data. Prevalence rates and costs of metabolic syndrome and associated risk factors were estimated. RESULTS: Laboratory-defined and claims-defined approaches underestimated metabolic syndrome prevalence rates compared with the integrated approach by 22.9% and 87.5%, respectively. Employees with metabolic syndrome had double the costs of those without any risk factors ($4603 vs $1859; P = 0.0384). CONCLUSIONS: Results suggest that integrating laboratory and claims data is a more balanced approach than either approach alone for identifying metabolic syndrome among Chevron employees.