Idiopathic pulmonary fibrosis in the United States: time to diagnosis and treatment.

OBJECTIVE: Create a timeline of diagnosis and treatment for IPF in the US. DESIGN, SETTING, AND PARTICIPANTS: A retrospective analysis was performed in collaboration with the OptumLabs Data Warehouse using an administrative claims database of Medicare Fee for Service beneficiaries. Adults 50 and over with IPF were included (2014 to 2019). EXPOSURE: To focus on IPF, the following diagnoses were excluded: post-inflammatory fibrosis, hypersensitivity pneumonitis, rheumatoid arthritis, sarcoidosis, scleroderma, and connective tissue disease. MAIN OUTCOMES AND MEASURES: Data were collected from periods prior, during, and following initial clinical diagnosis of IPF. This included prior respiratory diagnoses, number of respiratory-related hospitalizations, anti-fibrotic and oxygen use, and survival. RESULTS: A total of 44,891 with IPF were identified. The most common diagnoses prior to diagnosis of IPF were upper respiratory infections (47%), acute bronchitis (13%), other respiratory disease (10%), chronic obstructive pulmonary disease and bronchiectasis (7%), and pneumonia (6%). The average time to a diagnosis of IPF was 2.7 years after initial respiratory diagnosis. Half of patients had two or more respiratory-related hospitalizations prior to IPF diagnosis. Also, 37% of patients were prescribed oxygen prior to diagnosis of IPF. These observations suggest delayed diagnosis. We also observed only 10.4% were treated with anti-fibrotics. Overall survival declined each year after diagnosis with median survival of 2.80 years. CONCLUSIONS AND RELEVANCE: Our retrospective cohort demonstrates that IPF is often diagnosed late, usually preceded by other respiratory diagnoses and hospitalizations. Use of available therapies is low and outcomes remain poor.

Methods and Study Design for Cancer Health Economics Research: Summary of Discussions From a Breakout Session.

The legitimacy of findings from cancer health economics research depends on study design and methods. A breakout session, Methods and Study Design for Cancer Health Economics Research, was convened at the Future of Cancer Health Economics Research Conference to discuss 2 commonly used analytic tools for cancer health economics research: observational studies and decision-analytic modeling. Observational studies include analysis of data collected with the primary purpose of supporting economic evaluation or secondary use of data collected for another purpose. Modeling studies develop a parametrized structure, such as a decision tree, to estimate hypothetical impact. Whereas observational studies focus on what has happened and why, modeling studies address what may happen. We summarize the discussion at this breakout session, focusing on 3 key elements of high-quality cancer health economics research: study design, analytical methods, and addressing uncertainty.

Expenditures in Young Adults with Hodgkin Lymphoma: NCI-Designated Comprehensive Cancer Centers versus Other Sites.

BACKGROUND: Outcomes among Hodgkin lymphoma (HL) patients diagnosed between 22 and 39 years are worse than among those diagnosed <21 years, and have not seen the same improvement over time. Treatment at an NCI-designated Comprehensive Cancer Center (CCC) mitigates outcome disparities, but may be associated with higher expenditures. METHODS: We examined cancer-related expenditures among 22- to 39-year-old HL patients diagnosed between 2001 and 2016 using deidentified administrative claims data (OptumLabs Data Warehouse; CCC: n = 1,154; non-CCC: n = 643). Adjusting for sociodemographics, clinical characteristics, and months enrolled, multivariable general linear models modeled average monthly health-plan paid (HPP) expenditures, and incidence rate ratios compared CCC/non-CCC monthly visit rates. RESULTS: In the year following diagnosis, CCC patients had higher HPP expenditures ($12,869 vs. $10,688, P = 0.001), driven by higher monthly rates of CCC nontreatment outpatient hospital visits (P = 0.001) and per-visit expenditures for outpatient hospital chemotherapy ($632 vs. $259); higher CCC inpatient expenditures ($1,813 vs. $1,091, P = 0.001) were driven by 3.1 times higher rates of chemotherapy admissions (P = 0.001). Out-of-pocket expenditures were comparable (P = 0.3). CONCLUSIONS: Young adults with HL at CCCs saw higher health-plan expenditures, but comparable out-of-pocket expenditures. Drivers of CCC expenditures included outpatient hospital utilization (monthly rates of non-therapy visits and per-visit expenditures for chemotherapy). IMPACT: Higher HPP expenditures at CCCs in the year following HL diagnosis likely reflect differences in facility structure and comprehensive care. For young adults, it is plausible to consider incentivizing CCC care to achieve superior outcomes while developing approaches to achieve long-term savings.

Expenditures among young adults with acute lymphoblastic leukemia by site of care.

BACKGROUND: Individuals diagnosed with acute lymphoblastic leukemia (ALL) between the ages of 22 and 39 years experience worse outcomes than those diagnosed when they are 21 years old or younger. Treatment at National Cancer Institute-designated Comprehensive Cancer Centers (CCC) mitigates these disparities but may be associated with higher expenditures. METHODS: Using deidentified administrative claims data (OptumLabs Data Warehouse), the cancer-related expenditures were examined among patients with ALL diagnosed between 2001 and 2014. Multivariable generalized linear model with log-link modeled average monthly health-plan-paid (HPP) expenditures and amount owed by the patient (out-of-pocket [OOP]). Cost ratios were used to calculate excess expenditures (CCC vs non-CCC). Incidence rate ratios (IRRs) compared CCC and non-CCC monthly visit rates. Models adjusted for sociodemographics, comorbidities, adverse events, and months enrolled. RESULTS: Clinical and sociodemographic characteristics were comparable between CCC (n = 160) and non-CCC (n = 139) patients. Higher monthly outpatient expenditures in CCC patients ($15,792 vs $6404; P < .001) were driven by outpatient hospital HPP expenditures. Monthly visit rates and per visit expenditures for nonchemotherapy visits (IRR = 1.6; P = .001; CCC = $8247, non-CCC = $1191) drove higher outpatient hospital expenditures among CCCs. Monthly OOP expenditures were higher at CCCs for outpatient care (P = .02). Inpatient HPP expenditures were significantly higher at CCCs ($25,918 vs $13,881; ꞵ = 0.9; P < .001) before accounting for adverse events but were no longer significant after adjusting for adverse events (ꞵ = 0.4; P = .1). Hospitalizations and length of stay were comparable. CONCLUSIONS: Young adults with ALL at CCCs have higher expenditures, likely reflecting differences in facility structure, billing practices, and comprehensive patient care. It would be reasonable to consider CCCs comparable to the oncology care model and incentivize the framework to achieve superior outcomes and long-term cost savings. LAY SUMMARY: Health care expenditures in young adults (aged 22-39 years) with acute lymphoblastic leukemia (ALL) are higher among patients at National Cancer Institute-designated Comprehensive Cancer Centers (CCC) than those at non-CCCs. The CCC/non-CCC differences are significant among outpatient expenditures, which are driven by higher rates of outpatient hospital visits and outpatient hospital expenditures per visit at CCCs. Higher expenditures and visit rates of outpatient hospital visits among CCCs may also reflect how facility structure and billing patterns influence spending or comprehensive care. Young adults at CCCs face higher inpatient HPP expenditures; these are driven by serious adverse events.

Health Care Cost Associated With Contemporary Chronic Myelogenous Leukemia Therapy Compared With That of Other Hematologic Malignancies.

PURPOSE: Given the widespread introduction of tyrosine kinase inhibitors (TKIs), we evaluated the cost associated with chronic myelogenous leukemia (CML) care compared with the cost of care for patients with hematologic malignancies (HEM) and for patients without cancer (GEN), to aid with resource allocation and clinical decision making. METHODS: A retrospective cohort was constructed from the OptumLabs Data Warehouse using claims from 2000 to 2016. Eligible patients had ≥ 2 CML claims and were enrolled continuously for ≥ 6 months before diagnosis and ≥ 1 year afterward (n = 1,909). Patients with CML were frequency matched 4:1 with HEM and GEN cohorts and were observed through October 2017. We used generalized linear models to assess the variation in total mean annualized health care costs in the 3 cohorts and to examine the influence of factors associated with costs. RESULTS: Mean annualized costs for CML were $82,054 (ie, $25,471 [95% CI, $20,808 to $30,133] more than those for HEM and $74,993 [95% CI, $70,818 to $79,167] more than those for GEN); these differences were driven by pharmacy costs in the CML group. The cost of CML care exceeded that for HEM and GEN for all index years in this study and increased over each diagnostic interval until 2015, peaking at $91,990. The mean annual cost of all TKIs increased. Imatinib's mean annualized cost was $41,546 in the period 2000-2004 but increased to $105,069 in the period 2015-2017. In multivariable analysis, percent days on TKIs had the greatest influence on cost: ≥ 75% of the time versus none showed a difference in cost of $108,716 (95% CI, $99,193 to $118,239). CONCLUSION: Contemporary CML costs exceeded the cost of treatment of other hematologic malignancies. Cost was primarily driven by TKIs, whose cost continued to increase over time.

Trends in opioid use in commercially insured and Medicare Advantage populations in 2007-16: retrospective cohort study.

OBJECTIVE: To describe trends in the rate and daily dose of opioids used among commercial and Medicare Advantage beneficiaries from 2007 to 2016. DESIGN: Retrospective cohort study of administrative claims data. SETTING: National database of medical and pharmacy claims for commercially insured and Medicare Advantage beneficiaries in the United States. PARTICIPANTS: 48 million individuals with any period of insurance coverage between 1 January 2007 and 31 December 2016, including commercial beneficiaries, Medicare Advantage beneficiaries aged 65 years and over, and Medicare Advantage beneficiaries under age 65 years (eligible owing to permanent disability). MAIN ENDPOINTS: Proportion of beneficiaries with any opioid prescription per quarter, average daily dose in milligram morphine equivalents (MME), and proportion of opioid use episodes that represented long term use. RESULTS: Across all years of the study, annual opioid use prevalence was 14% for commercial beneficiaries, 26% for aged Medicare beneficiaries, and 52% for disabled Medicare beneficiaries. In the commercial beneficiary group, quarterly prevalence of opioid use changed little, starting and ending the study period at 6%; the average daily dose of 17 MME remained unchanged since 2011. For aged Medicare beneficiaries, quarterly use prevalence was also relatively stable, ranging from 11% at the beginning of the study period to 14% at the end. Disabled Medicare beneficiaries had the highest rates of opioid use, the highest rate of long term use, and the largest average daily doses. In this group, both quarterly use rates (39%) and average daily dose (56 MME) were higher at the end of 2016 than the low points observed in 2007 for each endpoint (26% prevalence and 53 MME). CONCLUSIONS: Opioid use rates were high during the study period of 2007-16, with the highest rates in disabled Medicare beneficiaries versus aged Medicare beneficiaries and commercial beneficiaries. Opioid use and average daily dose have not substantially declined from their peaks, despite increased attention to opioid abuse and awareness of their risks.

Novel Type 2 Diabetes Medication Access and Effect of Patient Cost Sharing.

BACKGROUND: Although drug formulary restrictions may reduce use of prescription medication and pharmacy costs, the effect of patient cost sharing on medication adherence and health care utilization and cost is unclear. OBJECTIVE: To evaluate the relationship between patient cost sharing for novel type 2 diabetes mellitus (T2DM) medications and medication adherence, persistence, and health care utilization and cost. METHODS: This retrospective study used medical and pharmacy claims linked to pharmacy benefit plan design data. Patients with T2DM were identified via ICD-9-CM codes (medical claims), outpatient prescription fills (pharmacy claims), and pharmacy benefit design information. Patients with T2DM treated with novel T2DM medications (DPP4 or GLP-1) were enrolled in plans with fixed or coinsurance medication copayment structures and followed for 12-48 months. Endpoints included medication persistence and adherence and total all-cause health care cost. Multivariable regression analysis estimated the effect of benefit design parameters, adjusting for baseline patient characteristics. RESULTS: The integrated database included 36,475 patients with T2DM. The majority (83.1%) had fixed copayment plans, and 3-tier plans were common (93.1%). Higher third-tier copayment was associated with poorer medication adherence and persistence but not total health care cost during follow-up. A $10 higher third-tier copayment was associated with 11% greater risk of novel T2DM medication discontinuation and 3% lower adherence. A comparison of patients with fixed versus coinsurance plans found that fixed plans were associated with higher adjusted persistence and total all-cause health care costs. CONCLUSIONS: Higher medication copayment amounts were associated with lower patient medication adherence and persistence in T2DM but not total health care costs, as health plan costs decreased while patient out-of-pocket costs increased. We observed higher total all-cause health care costs among T2DM patients with a fixed copay (vs. coinsurance) pharmacy benefit. Additional research incorporating plan design information is needed to further examine this finding. DISCLOSURES: This study was funded by Janssen Scientific Affairs, which was involved in study design, interpretation of data, editing manuscript content, and had final approval of the manuscript before submission. Lopez and Bookhart are employed by Janssen Scientific Affairs. At the time of this study, Henk was employed by Optum HEOR, which was contracted by Janssen to conduct this study. Portions of this study were presented at the 21st Annual International Meeting, ISPOR; May 21-25, 2016; in Washington, DC.

Opioid Prescribing for Opioid-Naive Patients in Emergency Departments and Other Settings: Characteristics of Prescriptions and Association With Long-Term Use.

STUDY OBJECTIVE: We explore the emergency department (ED) contribution to prescription opioid use for opioid-naive patients by comparing the guideline concordance of ED prescriptions with those attributed to other settings and the risk of patients' continuing long-term opioid use. METHODS: We used analysis of administrative claims data (OptumLabs Data Warehouse 2009 to 2015) of opioid-naive privately insured and Medicare Advantage (aged and disabled) beneficiaries to compare characteristics of opioid prescriptions attributed to the ED with those attributed to other settings. Concordance with Centers for Disease Control and Prevention (CDC) guidelines and rate of progression to long-term opioid use are reported. RESULTS: We identified 5.2 million opioid prescription fills that met inclusion criteria. Opioid prescriptions from the ED were more likely to adhere to CDC guidelines for dose, days' supply, and formulation than those attributed to non-ED settings. Disabled Medicare beneficiaries were the most likely to progress to long-term use, with 13.4% of their fills resulting in long-term use compared with 6.2% of aged Medicare and 1.8% of commercial beneficiaries' fills. Compared with patients in non-ED settings, commercial beneficiaries receiving opioid prescriptions in the ED were 46% less likely, aged Medicare patients 56% less likely, and disabled Medicare patients 58% less likely to progress to long-term opioid use. CONCLUSION: Compared with non-ED settings, opioid prescriptions provided to opioid-naive patients in the ED were more likely to align with CDC recommendations. They were shorter, written for lower daily doses, and less likely to be for long-acting formulations. Prescriptions from the ED are associated with a lower risk of progression to long-term use.

A Retrospective Study to Examine Healthcare Costs Related to Cardiovascular Events in Individuals with Hyperlipidemia.

INTRODUCTION: Few studies have demonstrated the cost burden of cardiovascular events (CVEs) among patients with hyperlipidemia. The primary objective of this study was to determine the mean costs associated with CVEs among patients with hyperlipidemia by follow-up time period. Secondary objectives of this study included characterizing costs by CVE type and coronary heart disease (CHD) risk. METHODS: This retrospective cohort study used longitudinal claims to calculate payer costs according to CHD risk level and type of CVE, during several follow-up periods (acute and short-term, comprising year 1; plus years 2 and 3). RESULTS: There were 193,385 patients with hyperlipidemia with a CVE. Costs in the acute (30-day) period were highest ($22,404) driven by inpatient care (77%). Costs remained high ($15,133 in year 3) with ambulatory care (from 14% in acute to 37% in year 3) and pharmaceutical costs (from 2% in acute to 24% in year 3) representing a greater proportion. After second and third CVEs, acute costs were lower than for the first CVE. But in the post-acute periods, costs were higher after second and third CVEs than after first CVEs. Acute costs varied considerably by type of CVE ($9149 for transient ischemic attack to $54,251 for coronary artery bypass graft; P < 0.001), but post-acute costs were more similar across types. Costs differed by baseline CHD risk for all follow-up periods (P < 0.001), but less than by CVE type. As expected, patients without CVEs had significantly lower costs. CONCLUSION: Among patients with hyperlipidemia, the economic burden of CVEs is substantial up to 3 years after a CVE. Costs remain high after subsequent CVEs and actually increase for non-inpatient utilization. FUNDING: Amgen Inc.

Characteristics and short-term outcomes of patients with type 2 diabetes mellitus treated with canagliflozin in a real-world setting.

OBJECTIVE: Canagliflozin is a sodium glucose co-transporter 2 inhibitor that has been shown to improve glycemic control in type 2 diabetes mellitus (T2DM). This study aimed to describe the characteristics, treatment utilization, and outcomes of patients treated with canagliflozin in the real world within the first 6 months of it being commercially available. METHODS: This retrospective cohort study used a large US health plan database for commercial and Medicare Advantage enrollees. Patients aged 18 and over with T2DM who filled a canagliflozin prescription during 1 April 2013 to 30 September 2013 were eligible for inclusion. Patients were required to be enrolled for 6 months before (baseline period) and 3 months after (follow-up period) the first canagliflozin claim. RESULTS: Overall, 3234 patients met study criteria (mean age was 55.7 years; 43.4% were female). Among patients with available lab data at baseline and follow-up, mean HbA1c decreased from 8.54% at baseline to 7.76% at follow-up (p < 0.001); the proportion of patients with HbA1c ≥9.0% decreased by more than half (from 32.0% at baseline to 15.5% at follow-up, p < 0.001). Almost all (94.8%) patients received at least one baseline antihyperglycemic agent; among them, 33.6% received two and 41.5% received three or more agents. Compared to baseline, usage of antihyperglycemic agents during follow-up was lower for metformin, sulfonylureas, insulin, DPP-4 inhibitors, GLP-1 receptor agonists and thiazolidinediones. CONCLUSIONS: Patients treated with canagliflozin when first available in the US typically had poorly controlled HbA1c levels at baseline and had received multiple prior antihyperglycemic agents. Following the first canagliflozin claim, they had an improvement in HbA1c levels and used fewer antihyperglycemic agents. These study results should help clinicians and payers better understand the initial profile of patients receiving canagliflozin and short-term outcomes in the real world. Given the short follow-up time frame and the fact that HbA1c data was not available in all patients, future research on longer term outcomes is warranted.

Retrospective claims analysis of best supportive care costs and survival in a US metastatic renal cell population.

INTRODUCTION: Survival and best supportive care (BSC) costs for patients with metastatic renal cell carcinoma (mRCC), after stopping therapy, are poorly characterized yet an important aspect of patient care. This study examined survival and costs associated with BSC after one or two lines of therapy (LOTs) for mRCC. METHODS: A retrospective cohort analysis used claims data from commercially insured or Medicare Advantage Prescription Drug (MAPD) plan enrollees of a large United States health plan with an index RCC diagnosis (ICD-9-CM 189.0) between January 1, 2007 and June 30, 2010; initiating any of the following therapies 30 days pre-index date through disenrollment from plan: sunitinib, temsirolimus, sorafenib, bevacizumab, everolimus, pazopanib, cytokines. LOT was identified using prescription fill and administration dates. Health care costs represent health plan- plus patient-paid amounts. RESULTS: The cohort (n = 274) was 73% male, with a mean age of 63.3 years (SD 11.1), with 80% commercially insured (20% MAPD), and 68% starting BSC following one LOT. Mean BSC duration was longer following one than two LOTs (223 [SD 260], 176 [SD 163] days). Median survival from the start of BSC was similar following one and two LOTs (126 and 118 days). Total BSC costs following one and two LOTs averaged US$50,188 (SD $96,984) and $37,295 (SD $51,102). Monthly costs for BSC following one and two LOTs ($10,151 and $10,566) were not substantially lower than costs while on treatment ($14,621 and $16,957). Inpatient hospital costs represented 47% and 49% following one and two LOTs, with ambulatory costs of approximately 36% following each LOT. CONCLUSION: Our study found similar survival and monthly costs for BSC following either one or two LOTs, with almost half of the cost reflecting inpatient care. Compared to costs on treatment ($14,621 to $16,957), BSC costs can be considerable ($10,151 to $10,566).

Pegfilgrastim prophylaxis is associated with a lower risk of hospitalization of cancer patients than filgrastim prophylaxis: a retrospective United States claims analysis of granulocyte colony-stimulating factors (G-CSF).

BACKGROUND: Myelosuppressive chemotherapy can lead to dose-limiting febrile neutropenia. Prophylactic use of recombinant human G-CSF such as daily filgrastim and once-per-cycle pegfilgrastim may reduce the incidence of febrile neutropenia. This comparative study examined the effect of pegfilgrastim versus daily filgrastim on the risk of hospitalization. METHODS: This retrospective United States claims analysis utilized 2004-2009 data for filgrastim- and pegfilgrastim-treated patients receiving chemotherapy for non-Hodgkin's lymphoma (NHL) or breast, lung, ovarian, or colorectal cancers. Cycles in which pegfilgrastim or filgrastim was administered within 5 days from initiation of chemotherapy (considered to represent prophylaxis) were pooled for analysis. Neutropenia-related hospitalization and other healthcare encounters were defined with a "narrow" criterion for claims with an ICD-9 code for neutropenia and with a "broad" criterion for claims with an ICD-9 code for neutropenia, fever, or infection. Odds ratios (OR) for hospitalization and 95% confidence intervals (CI) were estimated by generalized estimating equation (GEE) models and adjusted for patient, tumor, and treatment characteristics. Per-cycle healthcare utilization and costs were examined for cycles with pegfilgrastim or filgrastim prophylaxis. RESULTS: We identified 3,535 patients receiving G-CSF prophylaxis, representing 12,056 chemotherapy cycles (11,683 pegfilgrastim, 373 filgrastim). The mean duration of filgrastim prophylaxis in the sample was 4.8 days. The mean duration of pegfilgrastim prophylaxis in the sample was 1.0 day, consistent with the recommended dosage of pegfilgrastim - a single injection once per chemotherapy cycle. Cycles with prophylactic pegfilgrastim were associated with a decreased risk of neutropenia-related hospitalization (narrow definition: OR = 0.43, 95% CI: 0.16-1.13; broad definition: OR = 0.38, 95% CI: 0.24-0.59) and all-cause hospitalization (OR = 0.50, 95% CI: 0.35-0.72) versus cycles with prophylactic filgrastim. For neutropenia-related utilization by setting of care, there were more ambulatory visits and hospitalizations per cycle associated with filgrastim prophylaxis than with pegfilgrastim prophylaxis. Mean per-cycle neutropenia-related costs were also higher with prophylactic filgrastim than with prophylactic pegfilgrastim. CONCLUSIONS: In this comparative effectiveness study, pegfilgrastim prophylaxis was associated with a reduced risk of neutropenia-related or all-cause hospitalization relative to filgrastim prophylaxis.

Health care costs and resource utilization, including patient burden, associated with novel-agent-based treatment versus other therapies for multiple myeloma: findings using real-world claims data.

BACKGROUND: . Treatment of multiple myeloma has dramatically improved with the introduction of bortezomib (BOR), thalidomide (THAL), and lenalidomide (LEN). Studies assessing health care costs, particularly economic burden on patients, are limited. We conducted a claims-based, retrospective analysis of total health care costs as well as patient burden (patient out-of-pocket costs and number of ambulatory/hospital visits) associated with BOR/THAL/LEN treatment versus other therapies (OTHER). METHODS. Treatment episodes starting between January 1, 2005 and September 30, 2010 were identified from the claims database of a large U.S. health plan. Health care costs and utilization were measured during 1 year after initiation and analyzed per treatment episode. Multivariate analyses were used to adjust for patient characteristics, comorbidities, and line of treatment. RESULTS: A total of 4,836 treatment episodes were identified. Mean adjusted total costs were similar between BOR ($112,889) and OTHER ($111,820), but higher with THAL ($129,412) and LEN ($158,428). Mean adjusted patient out-of-pocket costs were also similar for BOR ($3,846) and OTHER ($3,900) but remained higher with THAL ($4,666) and LEN ($4,483). Mean adjusted rates of ambulatory visits were similar across therapies (BOR: 69.67; THAL: 66.31; LEN: 65.60; OTHER: 69.42). CONCLUSIONS: Adjusted analyses of real-world claims data show that total health care costs, as well as patient out-of-pocket costs, are higher with THAL/LEN treatment episodes than with BOR/OTHER therapies. Additionally, similar rates of ambulatory visits suggest that any perceived advantage in patient convenience of the orally administered drugs THAL/LEN is not supported by these data.

Patient survival and healthcare utilization costs after diagnosis of triple-negative breast cancer in a United States managed care cancer registry.

BACKGROUND: Triple-negative breast cancer (TNBC) makes up 10-17% of all breast cancers and, due to lack of receptor expression, is unresponsive to therapies that target hormonal receptors or HER2. Unique in its tumor aggression and high rates of recurrence, TNBC is less likely to be detected by mammogram and has a poorer prognosis than other breast cancer subtypes (non-TNBC). OBJECTIVES: To examine the survival, healthcare utilization, and healthcare cost for women with TNBC compared with non-TNBC breast cancer. METHODS: The study population was derived from a US managed care cancer registry linked to health insurance claims and social security mortality data. Based on initial type and stage at diagnosis, patients were divided into two cohorts: patients with TNBC and those with non-TNBC. Records were analyzed from initial diagnosis until death, disenrollment, or end of observation period. Survival and annual healthcare utilization and costs were estimated and compared between cohorts after adjusting for baseline demographic characteristics, comorbidities, and prior resource use. Subgroup analyses were performed in patients diagnosed with stage I-III and IV breast cancer. RESULTS: The study included women diagnosed with TNBC (n = 450) and non-TNBC (n = 1807). Median follow-up time for all patients was 716 days (688.5 and 733 days for TNBC and non-TNBC patients, respectively). After initial diagnosis, overall mortality risk for the TNBC cohort was twice as high as the non-TNBC cohort (HR = 2.02, p < 0.0001). Patients with TNBC had more annual hospitalizations, hospitalized days, and number of emergency room visits relative to non-TNBC. Despite similar annual total healthcare costs, adjusted inpatient costs for patients with non-TNBC averaged 77% higher ($8395 vs. $4745, p < 0.0001). Furthermore, payer reimbursements were higher for TNBC than non-TNBC patients ($8213 vs. $4486, p < 0.0001). CONCLUSIONS: While it does not control for race or socioeconomic status, this study found that in a US managed care setting, patients with TNBC compared with non-TNBC have significantly shorter survival, accompanied by higher inpatient utilization and healthcare costs.

Retrospective evaluation of the clinical benefit of long-term continuous use of zoledronic acid in patients with lung cancer and bone metastases.

BACKGROUND: For patients with bone metastases, skeletal-related events including fracture are common, can cause considerable morbidity, and may reduce overall survival (OS). This retrospective analysis assessed the effect of Zometa (zoledronic acid, ZOL), an intravenous bisphosphonate (IV-BP), on fracture risk and OS in patients with bone metastases from lung cancer (LC). (Zometa is a registered trademark of Novartis Pharmaceuticals Corporation, USA.) METHODS: A claims-based analysis using commercial and Medicare Advantage data from >45 US managed-care plans was used to evaluate the association between fracture risk and treatment persistency (31-90, 91-180, 181-365, and ≥366 days) and follow-up duration in LC patients diagnosed with bone metastases between 01/01/2001 and 12/31/2006 and treated with ZOL or without (no IV-BP). Persistency was defined as the absence of a >45-day gap between ZOL treatments. Analysis of variance tests were used to compare follow-up duration, a proxy for OS, between ZOL persistency groups. The effect of time to treatment with ZOL was also assessed. RESULTS: In 9874 LC patients with bone metastases (n = 1090 ZOL; n = 8784 no IV-BP) the unadjusted relative fracture risk was reduced by 40% with ZOL vs no IV-BP; fracture risk decreased consistently with increasing duration of ZOL treatment. Even short-term (31-90 days) ZOL significantly reduced fracture risk (47%) vs no IV-BP (p = 0.005) with adjustment for differences in demographic and clinical characteristics. Delaying ZOL until after bone metastases were diagnosed significantly increased fracture risk (p = 0.0017). For a sub-set of patients included in a survival analysis (n = 550 ZOL; n = 4512 no IV-BP), mortality was significantly lower (mean, 38.6 vs 46.8 deaths/100 person-years; p = 0.038) in those treated with ZOL vs no IV-BP. LIMITATIONS: Interpretation of this claims-based analysis must be tempered by the inherent limitations of observational data, such as limited clinical information and the ability to control for prognostic factors. CONCLUSIONS: This retrospective analysis demonstrates that LC patients with bone metastases receiving ZOL had significantly reduced risk of fracture (p = 0.005) and death (p < 0.038) vs patients receiving no IV-BP. Longer ZOL persistency consistently yielded better outcomes, with ≥12 months' treatment producing the greatest benefit.

Assessment of zoledronic acid treatment patterns and clinical outcomes in patients with bone metastases from genitourinary cancers.

BACKGROUND: Patients with bone metastases secondary to genitourinary (GU) cancer are at risk for skeletal-related events (SREs), including bone pain requiring palliative radiotherapy, fractures or surgery to bone, spinal cord compression, and hypercalcemia of malignancy. These SREs can be debilitating and potentially life-limiting. This study examined treatment practices and the association of treatment patterns with Zometa (zoledronic acid, ZOL), an intravenous bisphosphonate (IV-BP), with SREs and fractures. (Zometa is a registered trademark of Novartis Pharmaceuticals Corporation, USA.) METHODS: Retrospective analysis of commercial and Medicare Advantage enrollment and medical claims data was performed to evaluate IV-BP use and SRE patterns in adult patients with GU cancers. Criteria included diagnosis of ≥1 bone metastasis and prostate cancer (PC), renal cell carcinoma (RCC), or bladder cancer (BlC) between January 2001 and December 2006; continuous healthcare plan enrollment for ≥6 months before the index date; and no evidence of prior IV-BP use. Patients were followed until disenrollment from the healthcare plan or December 2007. RESULTS: Of 6347 patients (PC, n = 4976; RCC, n = 941; BlC, n = 430; mean [standard deviation] age: 68.9 [11.1] years), only approximately 23% received ZOL. The mean time between diagnosis of bone metastasis and ZOL initiation was approximately 108 days. Among patients with PC, fracture risk was significantly smaller for ZOL vs no IV-BP (incidence rate ratio = 0.70; p < 0.001), and 2-year survival was significantly longer for ZOL-treated vs no IV-BP patients (p = 0.007). Patients with longer persistency on ZOL had a smaller fracture risk than patients with shorter persistency. Sub-set analyses were not performed for RCC and BIC because the proportion of patients treated was too low. LIMITATIONS: Interpretation of this claims-based analysis must be tempered by the inherent limitations of observational data, such as limited and accurate available information, and unavailable information including clinical or disease-specific parameters. CONCLUSIONS: Intravenous BP therapy is not always received in patients with bone metastases secondary to GU cancers, and, when used, there are typically long time periods before treatment initiation. Without IV-BPs, PC patients have significantly larger risks of fracture and death compared with ZOL-treated patients, and benefits appear to be larger with increasing persistency on ZOL.

Retrospective database analysis of the effect of zoledronic acid on skeletal-related events and mortality in women with breast cancer and bone metastasis in a managed care plan.

BACKGROUND: Bone metastases are common in patients with advanced breast cancer, and place patients at risk for skeletal-related events (SREs) including pathologic fracture, spinal cord compression, hypercalcemia of malignancy, and the need for radiotherapy and/or surgery to bone. These SREs are associated with reduced survival and quality-of-life. The nitrogen-containing bisphosphonates Zometa (zoledronic acid, ZOL) and Aredia (pamidronate disodium, PAM) reduce SRE risk in patients with bone metastases from breast cancer. This database analysis compared SRE and mortality rates in a real-life setting in women with breast cancer receiving ZOL and PAM, and assessed long-term ZOL benefit. METHODS: A retrospective, claims-based analysis was conducted using commercial and Medicare Advantage data from >45 US managed-care plans. Eligible adult patients had diagnoses for breast cancer and bone metastasis between 01/01/01 and 12/31/06, continuous enrollment in the health plan, and no evidence of bone metastasis or intravenous bisphosphonate (IV-BP) use for 6 months before their first ZOL or PAM infusion. Patients were followed until disenrollment (including mortality) or end of the analysis period (12/31/07). Persistency was defined as absence of a >45-day gap between IV-BP treatments. RESULTS: Of 8757 patients (mean age, 58.1 [SD 12.4] years), approximately 30% were treated with ZOL, 15% with PAM, and 55% with no IV-BP. Patients treated with ZOL had a moderately lower incidence of SREs (mean, 36.2 vs 40.0 SREs/100 person-years; p = 0.0707) and significantly lower mortality (mean, 6.5 vs 11.2 deaths/100 person-years; p < 0.001) compared with PAM-treated patients. Longer persistency with ZOL was associated with lower risk of fracture and all SREs (trend-test p = 0.0076 and p = 0.0200, respectively). LIMITATIONS: Interpretation of this claims-based analysis must be tempered by the inherent limitations of observational data, such as imbalances in patient populations and the potential for bias in treatment selection. CONCLUSIONS: This analysis suggests that fewer than half of breast cancer patients with bone metastases receive IV-BPs. Longer persistence with ZOL was associated with lower SRE risk, and ZOL-treated patients had longer survival and a non-significant trend toward fewer SREs compared with PAM.

Incidence and costs of cervical intraepithelial neoplasia in a US commercially insured population.

OBJECTIVE: This study aimed to estimate incidence, cost per episode of care, and US population burden of cervical intraepithelial neoplasia (CIN). MATERIALS AND METHODS: For women continuously enrolled in a US health plan from January 1, 1999 to December 31, 2004, medical claims were used to identify potential CIN diagnosis. Presence and grade of CIN (CIN 1, CIN 2,3, or no CIN) were verified in medical records for a randomly selected subset (n = 254). Incidence, costs, and population burden were calculated. RESULTS: Annual incidence for CIN 1 and CIN 2,3 was 1.6 and 1.2 per 1,000 women, respectively. Incidence was highest among women aged 21 to 30 years (3.3 and 3.6 per 1,000) and women aged 31 to 40 years (2.9 and 2.7 per 1,000). Costs per episode of care were higher for CIN 2,3 ($1,634 vs $1,084). Estimated annual burden per 1,000 US women was $1,059 for CIN 1 and $1,803 for CIN 2,3. CONCLUSIONS: We estimate that 412,000 women in the United States are diagnosed with CIN annually, with an associated cost of approximately $570 million.

Estimation of economic costs associated with transfusion dependence in adults with MDS.

OBJECTIVE: To examine the economic burden of myelodysplastic syndromes (MDS) and the incremental cost of transfusion dependence. RESEARCH DESIGN AND METHODS: Adults with evidence of MDS were identified between 05/01/2000 and 09/30/2003 from a longitudinal, retrospective claims database for a large, geographically diverse US health plan and their medical histories were followed for at least 6 months. Patients were classified as transfusion-dependent (MDS-TD) or transfusion-independent (MDS-TI). MAIN OUTCOME MEASURES: Variables were categorized as demographic, health status, utilization, or cost. Utilization (inpatient hospitalizations, outpatient facility visits, emergency department visits, and physician office visits) is reported as the mean and median numbers of each specified encounter per subject. Costs were measured as the sum of patient and plan liability. All variables were analyzed descriptively, and appropriate statistical tests were used to compare the MDS-TD and MDS-TI cohorts. Pharmacy, medical, and total health care costs, adjusted for demographics and comorbidity, were estimated using gamma regression with a log link. RESULTS: The MDS-TI cohort consisted of 2864 patients, and the MDS-TD cohort comprised 336 patients. Mean age for the entire study sample was 70.2 years. The MDS-TI cohort tended to receive most of its medical care at physicians' offices, whereas the MDS-TD cohort received nearly as much medical care at outpatient facilities (e.g., infusion clinics, hospital outpatient clinics) as it did in physicians' offices. The MDS-TD cohort had significantly higher mean annual costs: pharmacy, $4457 vs. $2926; medical, $50,663 vs. $17,469; total, $51,066 vs. $19,811 (p < 0.001 for all comparisons). Thus, transfusion dependence was associated with an incremental cost of $31,255 per patient per year. Some limitations inherent to using claims data and diagnosis codes for research apply to this study. CONCLUSIONS: This study demonstrated that an important consequence of transfusion dependence for MDS patients was markedly greater use of, and consequently higher costs associated with, inpatient and outpatient services. Continued research and efforts to develop biologic and pharmaceutical therapies may help more patients achieve transfusion independence, thereby reducing the financial burden of MDS.