Medication adherence among patients with advanced prostate cancer using oral therapies.

Aims: In light of the extended overall survival and improved quality of life provided by advanced prostate cancer (PC) oral therapies, this study aimed to describe treatment adherence to advanced PC oral therapies and evaluate associated patient characteristics and subsequent healthcare resource utilization (HRU). Patients & methods: Patients with advanced PC initiating apalutamide, enzalutamide or abiraterone acetate were identified from administrative data (October 1, 2014-September 30, 2019). Adherence and persistence at six months postinitiation were used to evaluate patient factors and HRU. Results: Aged ≥75 years, Black race, chemotherapy use and higher pharmacy paid amounts were associated with poor adherence/persistence, which translated to higher HRU. Conclusions: Strategies to increase adherence and persistence may improve patient outcomes and associated HRU.

Lay abstract This study included 27,262 patients with advanced prostate cancer who started taking one of three oral cancer medications (apalutamide, enzalutamide or abiraterone acetate) between October 2014 and September 2019. Patients who were black, aged 75 years or older, who had chemotherapy or who had higher prescription costs had the most difficulty following dosing guidelines or staying on treatment. Patients who did not follow dosing guidelines required more healthcare services. In light of the extended survival and improved quality of life that oral cancer medication for advanced prostate cancer provides, helping patients to take the correct medication dose, at the right time, and for the recommended length of time may improve their outcomes and reduce medical costs.

Management of Patients with Metastatic Castration-Sensitive Prostate Cancer in the Real-World Setting in the United States.

PURPOSE: This study provides a contemporary assessment of the treatment patterns, health care resource utilization (HRU) and costs among metastatic castration-sensitive prostate cancer (mCSPC) patients in the U.S. MATERIALS AND METHODS: Adults with mCSPC were selected from Optum's de-identified Clinformatics® Data Mart Database (Commercial insurance/Medicare Advantage [COM/MA]; January 1, 2014-July 31, 2019) or Medicare Fee-for-Service (FFS; January 1, 2014-December 31, 2017). The index date was the first metastatic disease diagnosis date on/after the first prostate cancer diagnosis (without prior evidence of castration resistance). Patients were observed for 12 months pre-index (baseline) through their mCSPC period (from index until castration resistance or followup end). First-line (1L) mCSPC therapy was assessed during the mCSPC period; all-cause HRU and health plan-paid costs per-patient-per-year (PPPY) were measured during baseline and mCSPC periods. RESULTS: Among 6,517 COM/MA and 13,324 Medicare-FFS mCSPC patients over ∼10 months (median mCSPC period), 38% and 48% remained untreated/deferred treatment, and 45% and 46% received 1L androgen deprivation therapy (ADT) monotherapy, respectively. 1L abiraterone acetate and docetaxel were used among 7% and 6% of COM/MA and 1% and 2% of Medicare-FFS patients, respectively. HRU increased from baseline to mCSPC period, resulting in increased health plan-paid costs from $21,201 to $108,767 in COM/MA and from $16,819 to $69,639 PPPY in Medicare-FFS. CONCLUSIONS: This study highlights the limited use of newer therapies that improve survival in men with mCSPC in the U.S. HRU and costs increased substantially after onset of metastasis. Given the emergence of newer effective mCSPC therapies, further evaluation of future real-world mCSPC treatment patterns and outcomes is warranted.

A real-world study of treatment patterns and outcomes in US managed-care patients with type 2 Diabetes initiating injectable therapies.

AIMS: Examine real-world outcomes in patients with type 2 diabetes mellitus (T2DM) initiating injectable therapy as part of the Initiation of New Injectable Treatment Introduced after Antidiabetic Therapy with Oral-only Regimens (INITIATOR) study. MATERIALS AND METHODS: Linked insurance claims and medical record data were collected from 2 large US health insurers (April 1, 2010 to March 31, 2012) of T2DM adults initiating treatment with glargine (GLA) or liraglutide (LIRA). Baseline characteristics were examined and changes in 12-month follow-up outcomes were described for both treatment groups: HbA1c, weight change, hypoglycaemia, persistence, healthcare utilisation and costs. RESULTS: A total of 4490 patients were included (GLA, 2116; LIRA, 2374). At baseline, GLA patients had significantly higher HbA1c vs LIRA patients (9.72% vs 8.19%; P < .001), lower likelihood of having HbA1c < 7% (7.1% vs 23.8%; P < .001), lower bodyweight (100.9 kg vs 110.9 kg, P < .001), higher Charlson Comorbidity Index score (0.88 vs 0.63; P < .001), and higher diabetes-related costs ($3492 vs $2089; P < .001), respectively. During 12-months of follow-up, treatment persistence was 64%, mean HbA1c reduction was -1.24% and weight change was + 1.17 among GLA patients, and was 49%, -0.51% and -2.74 kg, respectively, among LIRA patients. Diabetes-related costs increased significantly from baseline to follow-up for LIRA patients ($2089 vs $3258, P < .001) but not for GLA patients ($3492 vs $3550, P = .890). CONCLUSIONS: There were clinically relevant baseline differences in both groups, suggesting that GLA and LIRA are prescribed for different patient groups, and highlighting that efficacy results from clinical trials do not always translate into real-world practice. Significant increases in healthcare costs were observed in the LIRA group, warranting further cost-effectiveness analysis.

The Use of Decomposition Methods in Real-World Treatment Benefits Evaluation for Patients with Type 2 Diabetes Initiating Different Injectable Therapies: Findings from the INITIATOR Study.

BACKGROUND: Determining characteristics of patients likely to benefit from a particular treatment could help physicians set personalized targets. OBJECTIVES: To use decomposition methodology on real-world data to identify the relative contributions of treatment effects and patients' baseline characteristics. METHODS: Decomposition analyses were performed on data from the Initiation of New Injectable Treatment Introduced after Antidiabetic Therapy with Oral-only Regimens (INITIATOR) study, a real-world study of patients with type 2 diabetes started on insulin glargine (GLA) or liraglutide (LIRA). These analyses investigated relative contributions of differences in baseline characteristics and treatment effects to observed differences in 1-year outcomes for reduction in glycated hemoglobin A1c (HbA1c) and treatment persistence. RESULTS: The greater HbA1c reduction seen with GLA compared with LIRA (-1.39% vs. -0.74%) was primarily due to differences in baseline characteristics (HbA1c and endocrinologist as prescribing physician; P < 0.050). Patients with baseline HbA1c of 9.0% or more or evidence of diagnosis codes related to mental illness achieved greater HbA1c reductions with GLA, whereas patients with baseline polypharmacy (6-10 classes) or hypogylcemia achieved greater reductions with LIRA. Decomposition analyses also showed that the higher persistence seen with GLA (65% vs. 49%) was mainly caused by differences in treatment effects (P < 0.001). Patients 65 years and older, those with HbA1c of 9.0% or more, those taking three oral antidiabetes drugs, and those with polypharmacy of more than 10 classes had higher persistence with GLA; patients 18 to 39 years and those with HbA1c of 7.0% to less than 8.0% had higher persistence with LIRA. CONCLUSIONS: Although decomposition does not demonstrate causal relationships, this method could be useful for examining the source of differences in outcomes between treatments in a real-world setting and could help physicians identify patients likely to respond to a particular treatment.

Validation of disability status, a claims-based measure of functional status for cancer treatment and outcomes studies.

BACKGROUND: In prior research, we developed a claims-based prediction model for poor patient disability status (DS), a proxy measure for performance status, commonly used by oncologists to summarize patient functional status and assess ability of a patient to tolerate aggressive treatment. In this study, we implemented and validated the DS measure in 4 cohorts of cancer patients: early and advanced non-small cell lung cancers (NSCLC), stage IV estrogen receptor-negative (ER-) breast cancer, and myelodysplastic syndromes (MDS). DATA AND METHODS: SEER-Medicare data (1999-2007) for the 4 cohorts of cancer patients. Bivariate and multivariate logistic regression tested the association of the DS measure with designated cancer-directed treatments: early NSCLC (surgery), advanced NSCLC (chemotherapy), stage IV ER- breast cancer (chemotherapy), and MDS (erythropoiesis-stimulating agents). Treatment model fit was compared across model iterations. RESULTS: In both unadjusted and adjusted results, predicted poor DS was strongly associated with a lower likelihood of cancer treatment receipt in all 4 cohorts [early NSCLC (N=20,280), advanced NSCLC (N=31,341), stage IV ER- breast cancer (N=1519), and MDS (N=6058)] independent of other patient, contextual, and disease characteristics, as well as the Charlson Comorbidity Index. Inclusion of the DS measure into models already controlling for other variables did not significantly improve model fit across the cohorts. CONCLUSIONS: The DS measure is a significant independent predictor of cancer-directed treatment. Small changes in model fit associated with both DS and the Charlson Comorbidity Index suggest that unobserved factors continue to play a role in determining cancer treatments.

The effect of supplemental medical and prescription drug coverage on health care spending for Medicare beneficiaries with cancer.

OBJECTIVES: To examine whether patients with newly diagnosed cancer respond differently to supplemental coverage than the general Medicare population. METHODS: A cohort of newly diagnosed cancer patients (n = 1,799) from the 1997-2007 Medicare Current Beneficiary Survey and a noncancer cohort (n = 9,726) were identified and matched by panel year. Two-year total medical care spending was estimated by using generalized linear models with gamma distribution and log link-including endogeneity-corrected models. Interactions between cancer and type of insurance allowed testing for differential effects of a cancer diagnosis. RESULTS: The cancer cohort spent an adjusted $15,605 more over 2 years than did the noncancer comparison group. Relative to those without supplemental coverage, beneficiaries with employer-sponsored insurance, other private with prescription drug coverage, and public coverage had significantly higher total spending ($3,510, $2,823, and $4,065, respectively, for main models). For beneficiaries with cancer, supplemental insurance effects were similar in magnitude yet negative, suggesting little net effect of supplemental insurance for cancer patients. The endogeneity-corrected models produced implausibly large main effects of supplemental insurance, but the Cancer × Insurance interactions were similar in both models. CONCLUSIONS: Medicare beneficiaries with cancer are less responsive to the presence and type of supplemental insurance than are beneficiaries without cancer. Proposed restrictions on the availability of supplemental insurance intended to reduce Medicare spending would be unlikely to limit expenditures by beneficiaries with cancer, but would shift the financial burden to those beneficiaries. Policymakers should consider welfare effects associated with coverage restrictions.

Characteristics of US Medicare Beneficiaries with Chronic Cough vs. Non-Chronic Cough: 2011-2018.

Background: Chronic cough (CC), characterized as a cough lasting >8 weeks, is a common multi-factorial syndrome in the community, especially in older adults. Methods: Using a pre-existing algorithm to identify patients with CC within the 2011-2018 Medicare beneficiaries, we examined trends in gabapentinoid use through repeated cross-sectional analyses and identified distinct utilization trajectories using group-based trajectory modeling (GBTM) in a retrospective cohort study. Individuals without CC but with any respiratory conditions related to cough served as a comparator group. Results: Among patients with CC, gabapentinoid use increased from 18.6% in 2011 to 24.1% in 2018 (p = 0.002), with a similar upward trend observed in the non-CC cohort but with overall lower usage (14.7% to 18.4%; p < 0.001). Patients with CC had significantly higher burdens of respiratory and non-respiratory comorbidities, as well as greater healthcare service and medication use compared to the non-CC cohort. The GBTM analyses identified three distinct gabapentinoid utilization trajectories for CC and non-CC patients: no use (77.3% vs. 84.5%), low use (13.9% vs. 10.3%), and high use (8.8% vs. 5.2%). Conclusions: Future studies are needed to evaluate the safety and effectiveness of gabapentinoid use in patients with refractory or unexplained CC in real-world settings.

Out-of-pocket health care expenditure burden for Medicare beneficiaries with cancer.

BACKGROUND: There is increasing concern regarding the financial burden of care on cancer patients and their families. Medicare beneficiaries often have extensive comorbidities and limited financial resources, and may face substantial cost sharing even with supplemental coverage. In the current study, the authors examined out-of-pocket (OOP) spending and burden relative to income for Medicare beneficiaries with cancer. METHODS: This retrospective, observational study pooled data for 1997 through 2007 from the Medicare Current Beneficiary Survey linked to Medicare claims. Medicare beneficiaries with newly diagnosed cancer were selected using claims-based diagnoses. Generalized linear models were used to estimate OOP spending. Logistic regression models identified factors associated with a high OOP burden, defined as spending > 20% of one's income during the cancer diagnosis and subsequent year. RESULTS: The cohort included 1868 beneficiaries with and 10,047 without cancer. Compared with the noncancer cohort, cancer patients were older, had more comorbidities, and were more likely to lack supplemental coverage. The mean OOP spending for cancer patients was $4727. Cancer patients faced an adjusted $976 (P < .01) incremental OOP spending. Greater than one-quarter (28%) of beneficiaries with cancer experienced a high OOP burden compared with 16% of beneficiaries without cancer (P < .001). Supplemental insurance and higher income were found to be protective against a high OOP burden, whereas assets, comorbidity, and receipt of cancer-directed radiation and antineoplastic therapy were associated with a higher OOP burden. CONCLUSIONS: Medicare beneficiaries with cancer face a higher OOP burden than their counterparts without cancer; some of the higher burden was explained by the higher comorbidity burden and lack of supplemental insurance noted among these patients. Financial pressures may discourage some elderly patients from pursuing treatment.

Treatment and outcomes for chronic myelomonocytic leukemia compared to myelodysplastic syndromes in older adults.

Prior studies have investigated patients' characteristics, treatments, and outcomes for older adults with myelodysplastic syndromes, but most failed to distinguish chronic myelomonocytic leukemia. Recognizing potentially important differences between the diseases, we undertook a population-based comparison of baseline characteristics, treatments, and outcomes between older adults with chronic myelomonocytic leukemia and myelodysplastic syndromes. The patients' data were obtained from Surveillance Epidemiology and End Results registry data from 2001-2005, linked to Medicare claims. Baseline characteristics, treatment (red blood cell transfusions, hematopoietic growth factors, hypomethylating agents, chemotherapy or transplantation), progression to acute myeloid leukemia, and overall survival were compared using bivariate techniques. Multivariate logistic regression estimated differences in treatments received. Cox proportional hazard models estimated the effects of chronic myelomonocytic leukemia relative to myelodysplastic syndromes on progression-free survival. A larger proportion of patients with chronic myelomonocytic leukemia (n=792), compared to patients with myelodysplastic syndromes (n=7,385), failed to receive any treatment (25% versus 15%; P<0.0001), or only received red blood cell transfusions (19.8% versus 16.7%; P=0.037). A larger percentage of patients with chronic myelomonocytic leukemia progressed to acute myeloid leukemia (42.6% versus 15.5%, respectively; P<0.0001), with shorter time to progression. Chronic myelomonocytic leukemia patients had a shorter median survival (13.3 versus 23.3 months; P<0.0001) and lower 3-year survival rate (19% versus 36%; P<0.0001). Adjusted estimates, controlling for baseline characteristics and selected treatments, indicate that chronic myelomonocytic leukemia was associated with an increased risk of progression to acute myeloid leukemia or death (HR 2.22; P<0.0001), compared to myelodysplastic syndromes. In conclusion, chronic myelomonocytic leukemia is less frequently treated in older adults and is associated with worse outcomes, even after controlling for the patients' baseline characteristics and selected treatments. Our data suggest the need for continued evaluation of the biological differences between these diseases and clinical trials targeting chronic myelomonocytic leukemia.

Interdisciplinary collaboration in the diagnosis and management of chronic cough: the role and importance of primary care providers.

Chronic cough (CC) is associated with many conditions, so identifying contributing causes poses a diagnostic challenge. However, guidelines written for US physicians do not explicitly outline suggested roles for primary care providers (PCPs) in the approach to patients with CC, including refractory or unexplained CC. The objective of this review is to describe the role of PCPs in the diagnosis and treatment of CC in adults. This narrative review draws upon literature (identified via a PubMed search performed January 9, 2023, using primary care/disease state-related terms) and expertise from specialist physicians to provide recommendations for CC management in primary care. Cough is one of the top reasons patients seek care from PCPs; accordingly, PCPs are often the first physicians to conduct workup and initiate treatment. Patients with CC often experience a burdensome cough that lasts for years, have high healthcare resource utilization (HCRU), undergo multiple or failed treatment trials, and have limited success finding an etiology. Although specialist referral may be needed for many diagnostic tests, initial aspects of CC workup and management should be completed in primary care. Often more accessible than specialists, real-world evidence on HCRU suggests PCPs are important stakeholders in diagnosing and managing CC, including during initial workup and treatment for the most common causes of CC (i.e. upper-airway cough syndrome, asthma, noneosinophilic asthmatic bronchitis, and gastroesophageal reflux disease). Thorough workup at the primary care level may facilitate earlier identification of CC cause(s), improving patient journey to diagnosis and management.

Patterns of Cough Medication Prescribing among Patients with Chronic Cough in Florida: 2012-2021.

Among patients with chronic cough (CC) in the 2012-2021 statewide OneFlorida Clinical Research Consortium database, we examined trends in cough medication (CM) prescribing prevalence over time in repeated cross-sectional analyses and identified distinct CM utilization trajectories using group-based trajectory modeling (GBTM) in a retrospective cohort study. Among eligible adults (≥18 years) without cancer/benign respiratory tumor diagnoses, we identified CC patients and non-CC patients with any cough-related diagnosis. In the GBTM analysis, we calculated the number of monthly prescriptions for any CMs (excluding gabapentinoids) during the 12 months from the first qualifying cough event to identify distinct utilization trajectories. From 2012 to 2021, benzonatate (9.6% to 26.1%), dextromethorphan (5.2% to 8.6%), and gabapentinoid (5.3% to 14.4%) use increased among CC patients, while opioid antitussive use increased from 2012 to 2015 and decreased thereafter (8.4% in 2012, 14.7% in 2015, 6.7% in 2021; all p < 0.001). Of 15,566 CC patients and 655,250 non-CC patients identified in the GBTM analysis, CC patients had substantial burdens of respiratory/non-respiratory comorbidities and healthcare service and concomitant medication use compared to non-CC patients. Among CC patients, GBTM identified three distinct CM utilization trajectories: (1) no CM use (n = 11,222; 72.1%); (2) declining CM use (n = 4105; 26.4%); and (3) chronic CM use (n = 239; 1.5%). CC patients in Florida had limited CM use with increasing trends in use of benzonatate, dextromethorphan, and gabapentinoids and a decreasing trend in opioid antitussive use. CC patients, particularly with chronic prescription CM use, experienced substantial disease burden.

Patient and physician characteristics associated with erythropoiesis-stimulating agent use in patients with myelodysplastic syndromes.

Patient and physician characteristics associated with use of erythropoiesis-stimulating agents in myelodysplastic syndrome patients have not yet been described. Myelodysplastic syndrome patients diagnosed from 2001 to 2005 were identified from the Surveillance Epidemiology and End Results-Medicare database. Multivariate regressions examined the association between patient and physician characteristics and the probability of receiving any erythropoiesis-stimulating agents, and of receiving therapeutic-length (≥ 8 week) treatment episodes. Among the 6,588 myelodysplastic syndrome patients studied, 65% received erythropoiesis-stimulating agents. Use of erythropoiesis-stimulating agents was lower for blacks compared to whites (OR 0.78; 95% CI:0.61-0.99), single persons compared to married (OR 0.77; 95% CI:0.62-0.97), Medicaid recipients (OR 0.66; 95% CI:0.55-0.79), and those living in census tracts with lower educational attainment. Patients who did not consult a hematology-oncology specialist were less likely to receive erythropoiesis-stimulating agents. Specialist access, financial resources and mobility are key determinants of receipt of erythropoiesis-stimulating agents among myelodysplastic syndrome patients.

Erythropoiesis-stimulating agents are not associated with increased risk of thrombosis in patients with myelodysplastic syndromes.

BACKGROUND: There are limited reports of thrombosis among myelodysplastic syndrome patients exposed to erythropoiesis stimulating agents. It is not clear whether erythropoiesis stimulating agents are associated with an increased risk of thrombosis in myelodysplastic syndromes, as they are among patients with solid tumors. DESIGN AND METHODS: The association between use of erythropoiesis stimulating agent and transient thrombosis risk in patients with myelodysplastic syndromes was assessed in a case-crossover study nested within a cohort of incident myelodysplastic syndrome patients. Using the US Surveillance, Epidemiology, and End Results Medicare-linked database, cases with an incident diagnosis of deep vein thrombosis were identified. Using conditional logistical regression, the odds of exposure to erythropoiesis stimulating agents in the 12 weeks prior to the incident deep vein thrombosis (hazard period) was compared to the exposure odds in a prior 12-week comparison period. RESULTS: Within the cohort of eligibles with myelodysplastic syndromes (n = 5,673) there were 212 incident cases of deep vein thrombosis events. Mean age was 76.2 (standard deviation = ± 8.6) years. Use of erythropoiesis stimulating agents was not associated with deep vein thrombosis in the crude nor the adjusted models (OR = 1.21, 95% CI: 0.60, 2.43). Central venous catheter placement (OR = 6.47, 95% CI: 2.37, 17.62) and red blood cell transfusion (OR = 4.60, 95% CI: 2.29, 9.23) were associated with deep vein thrombosis. CONCLUSIONS: Despite the link between use of erythropoiesis stimulating agents and thrombosis among patients with solid tumors, this study provides evidence that their safety profile may be different among patients with myelodysplastic syndromes.

Evaluation of Cough Medication Use Patterns in Ambulatory Care Settings in the United States: 2003-2018.

Using 2003−2018 National Ambulatory Medical Care Survey data for office-based visits and 2003−2018 National Hospital Ambulatory Medical Care Survey data for emergency department (ED) visits, we conducted cross-sectional analyses to examine cough medication (CM) use trends in the United States (US) ambulatory care settings. We included adult (≥18 years) patient visits with respiratory-infection-related or non-infection-related cough as reason-for-visit or diagnosis without malignant cancer or benign respiratory tumor diagnoses. Using multivariable logistic regressions, we examined opioid antitussive, benzonatate, dextromethorphan-containing antitussive, and gabapentinoid use trends. From 2003−2005 to 2015−2018, opioid antitussive use decreased in office-based visits (8.8% to 6.4%, Ptrend = 0.03) but remained stable in ED visits (6.3% to 5.9%, Ptrend = 0.99). In both settings, hydrocodone-containing antitussive use declined over 50%. Benzonatate use more than tripled (office-based:1.6% to 4.8%; ED:1.5% to 8.0%; both Ptrend < 0.001). Dextromethorphan-containing antitussive use increased in ED visits (1.8% to 2.6%, Ptrend = 0.003) but stayed unchanged in office-based visits (3.8% to 2.7%; Ptrend = 0.60). Gabapentinoid use doubled in office-based visits (1.1% in 2006−2008 to 2.4% in 2015−2018, Ptrend < 0.001) but was negligible in ED visits. In US office-based and ED ambulatory care settings, hydrocodone-containing antitussive use substantially declined from 2003 to 2018, while benzonatate use more than tripled, and dextromethorphan-containing antitussive and gabapentinoid use remained low (<3%).

Identification of patients with metastatic castration-sensitive or metastatic castration-resistant prostate cancer using administrative health claims and laboratory data.

OBJECTIVE: To develop algorithms to identify metastatic castration-sensitive prostate cancer (mCSPC) patients and castration-resistant prostate cancer (mCRPC) patients, using health claims data and laboratory test results. METHODS: A targeted literature review summarized mCSPC and mCRPC patient selection criteria previously used in real-world retrospective studies. Novel algorithms to identify mCSPC and mCRPC were developed based on diagnosis codes indicating hormone sensitivity/resistance, prostate-specific antigen (PSA) test results, and claims for castration and mCRPC-specific treatments. These algorithms were applied to claims data from Optum Clinformatics Extended DataMart (Date of Death) Databases (commercial insurance/Medicare Advantage [COM/MA]; 01 January 2014-31 July 2019) and Medicare Fee-for-Service (Medicare-FFS; 01 January 2014-31 December 2017). RESULTS: Previous real-world studies identified mCSPC primarily based on metastasis diagnosis codes, and mCRPC based on mCRPC-specific drugs. Using the current study's algorithms, 7034 COM/MA and 19,981 Medicare-FFS patients were identified as having mCSPC, and 2578 COM/MA and 11,554 Medicare-FFS as having mCRPC. Most mCSPC patients were identified based on evidence of being hormone/castration-naive. Patients were identified as having mCRPC most commonly based on rising PSA (COM/MA), or at the metastasis diagnosis date if it occurred after castration (Medicare-FFS). Among patients with mCSPC, 14-17% had evidence of progression to castration resistance during a median 1-year follow-up period, mostly based on use of mCRPC-specific drugs. CONCLUSIONS: Comprehensive algorithms based on claims and laboratory data were developed to identify and distinguish patients with mCSPC and mCRPC. This will facilitate appropriate identification of mCSPC and mCRPC patients based on health claims data and better understanding of patient unmet needs in real-world settings.

Clinical characteristics, patterns of lipid-lowering medication use, and health care resource utilization and costs among patients with atherosclerotic cardiovascular disease.

PURPOSE: The aim of this study was to investigate real-world patient characteristics, medication use, and health care resource utilization (HCRU) and costs among patients with clinical atherosclerotic cardiovascular disease (ASCVD) as defined by 2013 American College of Cardiology/American Heart Association (ACC/AHA) guidelines, to examine burden of disease and unmet needs, such as potential undertreatment. PATIENTS AND METHODS: This retrospective cohort study utilized a nationally representative managed care database to identify newly diagnosed ASCVD patients between January 1, 2007, and November 30, 2012 (index = first ASCVD diagnosis date) in the USA. Patients had ≥12-month pre-index (baseline) and ≥12-month post-index (follow-up) health plan enrollment and no baseline lipid-lowering medication (LLM). Patient characteristics, LLM utilization patterns, HCRU, and costs were examined for all patients and by subgroups based on LLM use pattern and/or follow-up low-density lipoprotein cholesterol (LDL-C) levels. RESULTS: A total of 128,017 ASCVD patients were identified with a mean (SD) age of 59 (13) years, 43.1% female, and 48.8% with ≥36-month follow-up. Within 12-month follow-up, 10.6% had high-intensity statins and 56.9% had no LLM fills. Baseline mean (SD) all-cause costs were $8,852 ($25,608). At 12-month follow-up, mean (SD) all-cause and ASCVD-related costs were $31,443 ($54,040) and $20,289 ($45,159), respectively. The 36-month analyses showed similar distributions. Multivariable analyses showed that age, gender, region, health insurance type, baseline comorbidities, baseline use of specific medications, baseline lipid profiles, and index ASCVD type were significantly associated with all-cause and ASCVD-related health care costs. CONCLUSION: Patients have nonoptimal treatment for ASCVD and substantial HCRU and costs associated with residual risk. Unmet needs and cost burdens of ASCVD patients merit additional investigation.

Real-World Characteristics and Treatment Patterns in Patients with Urticaria Initiating Omalizumab in the United States.

BACKGROUND: Omalizumab is indicated for the management of chronic idiopathic urticaria (CIU) in patients aged 12 years or older with persistent hives that are not adequately controlled by H1 antihistamines. While its safety and efficacy in CIU patients have been evaluated in multiple clinical trials, real-world use of omalizaumab in CIU has not been well characterized. OBJECTIVE: To assess demographics, clinical characteristics, and treatment patterns of CIU patients who initiated omalizumab to better understand the usage of this agent in CIU management in the real world. METHODS: This retrospective cohort study used medical and pharmacy claims data in the United States from the HealthCore Integrated Database to identify patients with CIU newly treated with omalizumab (≥ 4 omalizumab claims within 6 months of the initial claim) between March 21, 2014, and October 31, 2015 (study intake period). The index date was defined as the date of the first claim for omalizumab during the study intake period. Demographic and clinical characteristics were described for patients treated with omalizumab, as were treatment patterns associated with omalizumab and concomitant medications associated with CIU treatment. Descriptive and inferential statistics were reported. The Kaplan-Meier method was used to examine omalizumab treatment patterns. RESULTS: This study included 298 omalizumab-treated patients (mean [SD] age of 43.5 [13.64] years; 70.8% female); approximately 84% were seen by an allergist/immunologist. All patients had ≥ 12 months of continuous enrolment and a subset of 138 patients had ≥ 18 months of follow-up. For patients with ≥ 12 months of post-index follow-up, 12.1% (n = 36), 28.5% (n = 85), and 32.9% (n = 98) discontinued omalizumab within the 6-month, 12-month, and the entire post-index periods (mean 530 days), respectively; the mean number of days patients were continuously treated with omalizumab was 443.1 (95% CI = 425.0-461.3); the probabilities of continuous treatment (95% CI) were 0.879 (0.836-0.911), 0.711 (0.656-0.759), and 0.647 (0.585-0.703) for the 6-, 12-, and 18-month post-index periods, respectively. For the 98 patients who discontinued omalizumab during the entire post-index period, 28.6% restarted omalizumab after the first discontinuation within the post-index period (mean time from first discontinuation to first restart=329 days). Use of medications such as oral corticosteroids, montelukast, cyclosporine, and prescription H1 and H2 antihistamines decreased during the 1- to 6-month and 7- to 12-month post-index periods compared with those within the 6-month pre-index period. CONCLUSIONS: In this cohort of CIU patients who were newly prescribed omalizumab, the majority were treated by allergists/immunologists as expected, and approximately 60% of patients continued on therapy beyond 18 months. Concomitant medication use decreased after omalizumab initiation. These data on the real-world use of omalizumab for CIU may help to better inform decision-making processes for health care payers by quantifying omalizumab and concomitant medication treatment patterns over a longer time frame relative to previous studies. DISCLOSURES: This study was sponsored by Novartis Pharmaceuticals, which provided funding support for the conduct of the study. Kavati, Ortiz, and Paknis are employees of Novartis Pharmaceuticals. Ke, Wertz, Huang, Wang, Willey, and Stephenson are employees of HealthCore, an independent research organization that received funding from Novartis Pharmaceuticals for the conduct of this study. Beck is an employee of the University of Rochester Medical Center, who was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports grants from Genentech, outside the currently submitted work. Bernstein is affiliated with Bernstein Clinical Research Center, which was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports receiving grants and personal fees from Novartis Pharmaceuticals, grants and personal fees from Genentech outside of the submitted work, and is an author on the Joint Task Force for Practice Parameters for Urticaria and the GALEN international guidelines for urticaria under preparation. Selected study data were presented in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting on May 20-24, 2017, in Boston, MA. A poster based on this dataset was presented at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting on October 26-30, 2017, in Boston, MA.

Real-world Clinical Characteristics, Treatment Patterns, and Exacerbations in US Patients With Asthma Newly Treated With Omalizumab.

PURPOSE: The objective of this study was to examine patient characteristics, treatment patterns, and exacerbations among patients with asthma newly treated with omalizumab. METHODS: Data for this study were obtained from administrative claims and medical records. The index date was the date of the first claim for omalizumab. All patients had ≥12 months of continuous health plan eligibility before and after the index date. Demographic and clinical characteristics were obtained 12 months before the index date. Treatment patterns of asthma medications, including omalizumab, and asthma exacerbations were evaluated in the preindex and postindex periods. FINDINGS: The study included 1564 patients. Asthma-related medication use decreased from the preindex to the postindex periods (oral corticosteroids, 83.3%-66.4%, P < 0.001; inhaled corticosteroids [ICSs], 33.1%-26.8%, P < 0.001; long-acting ß2-adrenergic agonists [LABAs], 6.6%-5.2%, P = 0.009; ICS-LABA combination, 69.3%-64.3%, P < 0.001; leukotriene modifiers, 67.8%-59.7%, P < 0.001). The proportion of patients with any asthma exacerbations decreased by 33.6% (66.6%-44.2%, P < 0.001). Notably, the relative decreases in hospitalization and emergency department exacerbations were 79.3% and 72.2%, respectively. A total of 930 patients (59.5%) discontinued omalizumab treatment during the entire postindex period (maximum, 3400 days [approximately 9 years]), with 353 (38.0%) restarting omalizumab treatment. IMPLICATIONS: In this real-world analysis, patients newly initiating omalizumab therapy for allergic asthma used fewer concomitant asthma medications, while experiencing significant reductions in asthma exacerbations, especially hospitalization- and emergency department-specific exacerbations, from pre- to post-omalizumab treatment initiation periods.

Real-world treatment patterns and outcomes of omalizumab use in patients with chronic idiopathic urticaria.

OBJECTIVE: To examine treatment patterns, treatment response, and demographic and clinical characteristics of patients with chronic idiopathic urticaria (CIU) newly initiated on omalizumab therapy in real-world practice in the US. METHODS: This retrospective observational cohort study used US claims data from the HealthCore Integrated Research Database (HIRD®) augmented with medical record data to identify CIU patients newly-treated with omalizumab (≥4 omalizumab claims within 6 months of initial claim; index date = first omalizumab claim date) between March 21, 2014 and October 31, 2015 and with ≥6 months pre- and ≥12 months post-index health plan eligibility. Study outcomes were captured from medical records for up to 12 months pre-index and up to 24 months post-index. Descriptive statistics were reported. RESULTS: This study consisted of 88 patients with a mean (SD) age of 43.4 (± 13.46) years, 68.2% were female, and 36 patients had ≥18 months post-index eligibility. Among 69 patients with documented index dose, 75.4% received omalizumab 300 mg and 69.6% remained on index dose throughout follow-up. For 52 patients with documented index dosing frequency, 96.2% had every 4-week dosing frequency. Among 86 patients with omalizumab documentation, 83.7% reported CIU improvement after omalizumab initiation and 24.4% discontinued omalizumab. For all patients, the proportion using oral corticosteroids (OCS) decreased pre- to post-index (52.3% vs 39.8%). Similar results were observed for patients with ≥18 months post-index eligibility. CONCLUSIONS: In this real-world analysis, the majority of patients remained on omalizumab for ≥12 months, and had a positive response. OCS use decreased following omalizumab initiation.

Real-World Characteristics and Treatment Patterns in Patients with Urticaria Initiating Omalizumab in the United States.

BACKGROUND: Omalizumab is indicated for the management of chronic idiopathic urticaria (CIU) in patients aged 12 years or older with persistent hives that are not adequately controlled by H1 antihistamines. While its safety and efficacy in CIU patients have been evaluated in multiple clinical trials, real-world use of omalizaumab in CIU has not been well characterized. OBJECTIVE: To assess demographics, clinical characteristics, and treatment patterns of CIU patients who initiated omalizumab to better understand the usage of this agent in CIU management in the real world. METHODS: This retrospective cohort study used medical and pharmacy claims data in the United States from the HealthCore Integrated Database to identify patients with CIU newly treated with omalizumab (≥ 4 omalizumab claims within 6 months of the initial claim) between March 21, 2014, and October 31, 2015 (study intake period). The index date was defined as the date of the first claim for omalizumab during the study intake period. Demographic and clinical characteristics were described for patients treated with omalizumab, as were treatment patterns associated with omalizumab and concomitant medications associated with CIU treatment. Descriptive and inferential statistics were reported. The Kaplan-Meier method was used to examine omalizumab treatment patterns. RESULTS: This study included 298 omalizumab-treated patients (mean [SD] age of 43.5 [13.64] years; 70.8% female); approximately 84% were seen by an allergist/immunologist. All patients had ≥ 12 months of continuous enrolment and a subset of 138 patients had ≥ 18 months of follow-up. For patients with ≥ 12 months of post-index follow-up, 12.1% (n = 36), 28.5% (n = 85), and 32.9% (n = 98) discontinued omalizumab within the 6-month, 12-month, and the entire post-index periods (mean 530 days), respectively; the mean number of days patients were continuously treated with omalizumab was 443.1 (95% CI = 425.0-461.3); the probabilities of continuous treatment (95% CI) were 0.879 (0.836-0.911), 0.711 (0.656-0.759), and 0.647 (0.585-0.703) for the 6-, 12-, and 18-month post-index periods, respectively. For the 98 patients who discontinued omalizumab during the entire post-index period, 28.6% restarted omalizumab after the first discontinuation within the post-index period (mean time from first discontinuation to first restart=329 days). Use of medications such as oral corticosteroids, montelukast, cyclosporine, and prescription H1 and H2 antihistamines decreased during the 1- to 6-month and 7- to 12-month post-index periods compared with those within the 6-month pre-index period. CONCLUSIONS: In this cohort of CIU patients who were newly prescribed omalizumab, the majority were treated by allergists/immunologists as expected, and approximately 60% of patients continued on therapy beyond 18 months. Concomitant medication use decreased after omalizumab initiation. These data on the real-world use of omalizumab for CIU may help to better inform decision-making processes for health care payers by quantifying omalizumab and concomitant medication treatment patterns over a longer time frame relative to previous studies. DISCLOSURES This study was sponsored by Novartis Pharmaceuticals, which provided funding support for the conduct of the study. Kavati, Ortiz, and Paknis are employees of Novartis Pharmaceuticals. Ke, Wertz, Huang, Wang, Willey, and Stephenson are employees of HealthCore, an independent research organization that received funding from Novartis Pharmaceuticals for the conduct of this study. Beck is an employee of the University of Rochester Medical Center, who was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports grants from Genentech, outside the currently submitted work. Bernstein is affiliated with Bernstein Clinical Research Center, which was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports receiving grants and personal fees from Novartis Pharmaceuticals, grants and personal fees from Genentech outside of the submitted work, and is an author on the Joint Task Force for Practice Parameters for Urticaria and the GALEN international guidelines for urticaria under preparation. Study concept and design were primarily contributed by Kavati, Ortiz, and Paknis, with assistance from the other authors. Huang, Wang, and Ke took the lead in data collection, with assistance from Wertz, Willey, and Stephenson. Data interpretation was performed by Ke, Wertz, and Willey, assisted by the other authors. The manuscript was written by Stephenson, Ke, and Wang, along with Willey, Wertz, and Huang, and revised by Bernstein and Beck, with assistance from the other authors. Selected study data were presented in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting on May 20-24, 2017, in Boston, Massachusetts. A poster based on this dataset was presented at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting on October 26-30, 2017, in Boston, Massachusetts.