A checklist for ascertaining study cohorts in oncology health services research using secondary data: report of the ISPOR oncology good outcomes research practices working group.

OBJECTIVES: The ISPOR Oncology Special Interest Group formed a working group at the end of 2010 to develop standards for conducting oncology health services research using secondary data. The first mission of the group was to develop a checklist focused on issues specific to selection of a sample of oncology patients using a secondary data source. METHODS: A systematic review of the published literature from 2006 to 2010 was conducted to characterize the use of secondary data sources in oncology and inform the leadership of the working group prior to the construction of the checklist. A draft checklist was subsequently presented to the ISPOR membership in 2011 with subsequent feedback from the larger Oncology Special Interest Group also incorporated into the final checklist. RESULTS: The checklist includes six elements: identification of the cancer to be studied, selection of an appropriate data source, evaluation of the applicability of published algorithms, development of custom algorithms (if needed), validation of the custom algorithm, and reporting and discussions of the ascertainment criteria. The checklist was intended to be applicable to various types of secondary data sources, including cancer registries, claims databases, electronic medical records, and others. CONCLUSIONS: This checklist makes two important contributions to oncology health services research. First, it can assist decision makers and reviewers in evaluating the quality of studies using secondary data. Second, it highlights methodological issues to be considered when researchers are constructing a study cohort from a secondary data source.

Health-related quality of life and economic impact of urinary incontinence due to detrusor overactivity associated with a neurologic condition: a systematic review.

BACKGROUND: Patients with neurologic diseases often have neurogenic detrusor overactivity (NDO), which can result in a loss of voluntary bladder control and uncontrollable urinary incontinence (UI).The impact of UI due to NDO on patients' lives has not been well studied. The objective of this review was to assess the health-related quality of life (HRQoL) and economic burden in patients with urgency UI due to NDO in select countries in North America, the European Union, Asia, and Australia. METHODS: Systematic literature searches and reviews of articles published in English (January 2000 to February 2011) were conducted using MEDLINE®, EMBASE®, and the Cochrane Library. Studies assessing the impact of UI on HRQoL of patients with an underlying neurologic condition of interest (i.e., multiple sclerosis, spinal cord injury, Parkinson's disease, stroke, or spina bifida) were included. Economic studies in urgency UI also were included. RESULTS: Of 876 citations generated in the initial search, a total of 27 articles were deemed relevant: 16 articles presented HRQoL data and 11 articles presented information on the economic burden of UI. Humanistic studies used a range of HRQoL instruments to measure HRQoL burden, and the economic studies included different cost components to quantify the economic burden, making meaningful comparisons challenging. Despite this heterogeneity, the literature suggests that HRQoL in patients with UI due to NDO is worse than patients with UI in general or those with the same underlying neurologic condition without UI. In addition, urgency UI also results in substantial economic costs. CONCLUSIONS: Incontinent patients with underlying neurologic conditions have impaired HRQoL as well as substantial economic burden attributable to UI due to NDO. There is a need for urgency UI treatments that improve HRQoL of these patients and alleviate the economic burden of this condition.

Cost-offset analysis: bimatoprost versus other prostaglandin analogues in open-angle glaucoma.

OBJECTIVES: To develop a cost-offset model from a US payer perspective comparing glaucomatous progression and costs among primary open-angle glaucoma (POAG) patients using bimatoprost, latanoprost, or travoprost. STUDY DESIGN: Cost-offset model. METHODS: A Markov cohort model was used to estimate glaucomatous progression for POAG patients over 7 years. The model assumed bimatoprost-treated patients had lower resulting intraocular pressure (IOP) (by 1 mm Hg) for all presenting IOP categories than latanoprost- or travoprost-treated patients. Patients with lower IOP were assumed to have lower probability of progression. Those that progressed were assumed to do so at a rate of -0.6 dB per year. Direct costs associated with mean deviation score categories were applied to each treatment cohort to calculate the expected 7-year costs of treating patients with each prostaglandin analogue (PGA). Literature was used to support assumptions. A budget impact analysis was conducted where all travoprost patients switched to generic latanoprost and where all bimatoprost patients switched to generic latanoprost. The base case market share was 22% bimatoprost, 23% travoprost, and 55% latanoprost. RESULTS: Model results demonstrate that for a managed care plan with 9500 PGA-treated glaucoma patients, exclusive bimatoprost use would prevent progression in 136 additional individuals compared with exclusive travoprost or latanoprost treatment. Model results demonstrate that greater IOP reduction from bimatoprost is associated with increased cost savings compared with latanoprost or travoprost treatments. CONCLUSIONS: Model results demonstrate that greater IOP reduction from bimatoprost could reduce managed care spending.

Prolonged prophylaxis with valganciclovir is cost effective in reducing posttransplant cytomegalovirus disease within the United States.

BACKGROUND: Cytomegalovirus (CMV) disease in transplant patients is known to have a substantial clinical and economic burden, and its prevention is expected to have long-term benefits. Evidence from the Improved Protection Against CMV in Transplant trial proved that prolonged prophylaxis of 200 days with valganciclovir compared with 100 days significantly reduces the incidence of CMV in high-risk kidney transplant seropositive donors/seronegative recipients. The aim of this study was to develop a cost-effectiveness model to evaluate prolonged prophylaxis of 200 days with valganciclovir and its long-term economic impact. METHODS: An economic model was designed to simulate long-term costs and outcomes of prolonged prophylaxis with valganciclovir (200 vs. 100 days) in a cohort of 10,000 high-risk renal transplant patients over 5 and 10 years. The first year of the model was based on the results of the Improved Protection Against CMV in Transplant trial and the extension to the long-term periods (5 and 10 years); and quality of life data were based on evidence retrieved through a systematic literature search. This analysis was conducted from the US healthcare payer perspective. RESULTS: For the 5-year time horizon, the incremental cost-effectiveness ratio of US $14,859/quality-adjusted life year (QALY) suggests that 200-day valganciclovir prophylaxis is cost effective over the 100-day regimen considering a threshold of US $50,000/QALY. The 10-year analysis revealed the 200-day prophylaxis as cost saving with a 2380 QALY gain and simultaneously lower cost. CONCLUSION: Prolonged prophylaxis with valganciclovir reduces the incidence of events associated with CMV infection in high-risk kidney transplant recipients and is a cost-effective strategy in CMV disease management.

Prevalence of stroke/transient ischemic attack among patients with acute coronary syndromes in a real-world setting.

BACKGROUND: Atherothrombosis is a systemic disease that may manifest as acute ischemic events in multiple vascular beds. Patients who have experienced an atherothrombosis-related ischemic event in 1 vascular bed are at risk for developing ischemic events in other vascular beds. Antiplatelet therapy demands an understanding of the balance between arterial thrombosis benefit and adverse event risk. Clinical trials indicate that dual antiplatelet therapy with aspirin and the newer thienopyridines increases the risk of bleeding in patients with acute coronary syndromes (ACS) with prior cerebrovascular events. Informed clinical decision making requires a better understanding of the real-world prevalence of cerebrovascular events. OBJECTIVE AND PURPOSE: To estimate the prevalence of stroke and/or transient ischemic attack (TIA) among patients with ACS within US health plan populations. METHODS: A retrospective, observational cohort study was conducted of patients with ACS in 5 health care claims databases. The index event was defined as the first documented inpatient health care claim for myocardial infarction or unstable angina. Patients with ≥12 months of pre-index medical care encounter information were included. Stroke/TIA was identified by the first health care claim for these conditions any time prior to or within 90 days following the index ACS event. RESULTS: Across all databases, between 3.8% and 15.7% of patients with ACS had prior stroke/TIA and between 3.4% and 11.7% of patients with ACS with no history of cerebrovascular events had documented stroke/TIA following the index ACS hospitalization. CONCLUSION: Despite important differences between the various database populations, there is a high prevalence of documented stroke/TIA in patients with ACS both prior to and following the ACS event. These real-world findings, set within the context of the increased bleeding risk observed with the newer thienopyridines, are important considerations when selecting antiplatelet therapy for patients with ACS.

Economic consequences of severe bleeding in patients with acute coronary syndrome in the USA.

INTRODUCTION: Previous studies have demonstrated increased costs associated with bleeding in clinical trials, but none have yet examined the association of bleeding with costs/charges in a real-world setting. This study examines the association between health care charges and severe bleeding events among patients with acute coronary syndrome (ACS) in a real-world US setting. METHODS: This retrospective study of ACS patients enrolled in a regional, 570,000-member commercial health plan evaluated resource utilization for patients with and without severe bleeding using medical encounter data in health care administrative records. Inclusion criteria were continuous health plan enrollment in the 6 months before initial ACS-related hospitalization, age of at least 18 years, and an inpatient ACS claim between January 1995 and May 2007. Severe bleeding events were defined as having an in-hospital record for: (a) bleeding plus blood transfusion, (b) intracranial hemorrhage, or (c) blood transfusion followed by death. Hospitalizations in which the patient had a nonsevere bleeding event, defined as having an International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) code for bleeding without transfusion or death, were removed from analysis. Resource utilization was assessed using hospital charges. Multiple linear regression analyses controlling for key covariates were used to assess the association of severe bleeding during initial hospitalization with an ACS diagnosis/procedure with charges and length of stay (LOS). RESULTS: There were 11,266 ACS patients identified: 928 patients (8.2%) had severe bleeding during initial hospitalization. Severe bleeding events were associated with significantly higher hospital charges and increased LOS than hospitalizations without severe bleeding events. After adjusting for patient characteristics, in-hospital ACS-related procedures, and LOS, patients with severe bleeding incurred initial hospitalization charges that were $48,114 higher than those of patients without bleeding (P<0.001). CONCLUSION: In a real-world setting, hospitalizations with both severe bleeding and an ACS diagnosis or procedure are associated with significantly higher hospitalization charges and resource use compared with ACS-related hospitalizations without bleeding events. However, due to the limitations of retrospective analyses, no causal relationship can be determined as patient comorbidities represent a possible source of confounding.

Economic consequences of ACS-related rehospitalizations in the US.

OBJECTIVE: To examine economic consequences related to rehospitalization following initial acute coronary syndrome (ACS) treatment in United States managed care settings. STUDY DESIGN: Retrospective observational studies. RESEARCH DESIGN AND METHODS: Retrospective observational studies were conducted on two managed care populations to examine medical encounter insurance claims and charges for ACS-related rehospitalizations following an index hospitalization for new onset ACS (2002-2007). All charges were adjusted to year 2007 United States Dollars (USDs). MAIN OUTCOME MEASURES: The main outcomes for this study were the direct charges related to ACS rehospitalizations as captured in two separate medical encounter claims databases. RESULTS: Of the 11,266 ACS patients identified for analysis in the health system plan, 3588 (32%) had at least one ACS rehospitalization. Of the 97,177 ACS patients enrolled in the nationally representative managed care database, 32,578 (34%) had at least one ACS-related rehospitalization. Multivariate analyses demonstrated that coronary artery bypass graft (CABG) was the strongest predictor of increased charges during the recurrence in both populations (p < 0.0001). When controlling for length of stay (LOS) in the model, CABG remained a significant predictor of increased charges, while percutaneous coronary intervention (PCI) and stent insertion became even stronger predictors of increased charges. CONCLUSIONS: The costs associated with ACS-related rehospitalizations in a real-world setting are high, even when controlling for known cost drivers such as length of stay.

Clinical impact of early clopidogrel discontinuation following acute myocardial infarction hospitalization or stent implantation: analysis in a single integrated health network.

OBJECTIVE: To determine the association between the discontinuation of clopidogrel therapy prior to 1 year and the risk of acute myocardial infarction (AMI) hospitalization, coronary intervention or all-cause mortality in a cohort of managed-care patients following AMI hospitalization or stent insertion. RESEARCH DESIGN AND METHODS: This observational cohort study included 1152 patients enrolled in the Health Alliance Plan who were hospitalized for AMI, or who underwent coronary stent placement. Clopidogrel use was assessed using pharmacy claims data. The association between discontinuation of clopidogrel prior to 1 year following the initial ACS event and the primary outcome of AMI hospitalization/procedure was assessed using Cox proportional hazards models. Additionally, an analysis was conducted to determine the association of discontinuation prior to 1 year with a secondary composite outcome of AMI hospitalization/coronary stent procedure or all-cause mortality. MAIN OUTCOME MEASURES: The primary outcome was AMI hospitalization or procedure. The secondary outcome was a composite of AMI hospitalization/ procedure, or all-cause mortality. RESULTS: Discontinuation of clopidogrel in the total cohort of patients was associated with a significantly higher risk of the primary outcome of AMI hospitalization/ coronary intervention (HR 2.712, 95% CI 1.634-4.502). Consistent with this finding, discontinuation of clopidogrel was also associated with a significantly higher risk of the secondary composite endpoint (HR 1.844, 95% CI 1.281-2.653). CONCLUSIONS: In patients enrolled in an integrated health network, clopidogrel discontinuation prior to 1 year following AMI hospitalization or stent placement is associated with adverse outcomes including greater risk of death, AMI hospitalization or coronary intervention. These results should be interpreted within the context and limitations of observational research, which cannot attribute causality.

The relationship of mean deviation scores and resource utilization among patients with glaucoma: a retrospective United States and European chart review analysis.

PURPOSE: To determine the relationship between mean deviation (MD) scores and resource utilization among glaucoma patients in the United States and Europe. METHODS: A retrospective analysis of patient in the United States (June 1990 to January 2003, N=151) and Europe (June 1994 to July 2003, N=194) was conducted. Office visits, pressure-lowering medications, visual field examinations, and glaucoma surgeries were recorded with age, intraocular pressure (IOP), and MD. Patients with missing MD and IOP were excluded; final sample sizes were 130 in the United States and 161 in Europe. Fixed effects and logistic regression models assessed the associations between MD and visits, medication, examinations, and surgery. RESULTS: Number of office visits and visual field examinations significantly increased but the number of glaucoma medications decreased as MD worsened and IOP increased. Odds of trabeculoplasty within 2 years decreased by 8.1% in the United States and 9.9% in Europe with every 1.0 dB improvement in MD. Odds of trabeculectomy within 2 years increased by 13.9% in the United States and by 18.6% in Europe with every 1 mm Hg increase in IOP. In the United States, odds of trabeculectomy within 5 years increased by 12.2% with each 1.0 dB worsening in MD. CONCLUSIONS: MD is associated with resource utilization in glaucoma patients. Evidence supporting use of MD to predict surgery is less conclusive, possibly due to the relative low frequency of surgeries. Therapies aimed at stabilizing visual field deterioration may reduce resource use and costs associated with glaucoma; however, more research is necessary to establish whether such treatments meet common standards of cost-effectiveness.

Recruitment of families for genetic studies of epilepsy.

PURPOSE: Study of families containing multiple affected individuals is essential for genetic research on the epilepsies, yet practically nothing has been published about methods for identification and recruitment of families or expected participation rates. Here we describe the strategy used for data collection in a genetic linkage study, to provide guidelines for efficient design of new studies. METHODS: Potentially eligible families were ascertained from private physicians, clinics, and self-referrals. Participation rates were examined at each step of the recruitment process, according to ascertainment source, initial contact method, gender, and ethnicity. RESULTS: Among 320 potentially eligible families identified, only 68 (21%) were successfully enrolled. Contact was established with an index subject in 83% of families, and a screen for eligibility was completed in 88% of these. However, only 54% of screened families were confirmed to be eligible, and of these, only 54% were enrolled. In eligible families, 79% of index subjects agreed to participate; the low family enrollment rates resulted largely from refusals by other family members whose participation was needed for linkage analysis. At each step in the recruitment process, the participation rate was higher in self-referred than in other families. CONCLUSIONS: Recruitment of families for genetic studies is labor-intensive; many potentially eligible families may have to be screened for each family enrolled. Recruitment is easier with self-referred families than with those identified through other methods. The introduction of standardized methods for identification of eligible families from clinical settings can improve efficiency.