Congestion is associated with adverse outcomes in heart failure (HF) patients. We characterized congestion in patients hospitalized for HF and examined the association between congestion severity at ...admission and postdischarge outcomes. Using the OPTIMIZE-HF registry linked to Medicare claims, we analyzed patients ≥65 years old hospitalized for HF from 2003 to 2004. Congestion severity was measured using a 15-point scale that scores dyspnea, orthopnea, fatigue, jugular venous pressure, rales, and edema. Patient characteristics and outcomes were described by congestion strata. Proportional hazards models were fit to examine associations between congestion and 1-year outcomes. Congestion scores for the 24,724 patients ranged from 0 to 14, with a median of 5 (Q1, Q3: 3, 7). At baseline, patients with the highest scores (≥7) had the highest rates of recent HF hospitalizations, EF ≤40%, and co-morbidities, including arrhythmias, diabetes mellitus, and renal insufficiency. Adjusting for patient characteristics, a 3-point congestion score increase was positively associated with mortality (hazard ratio HR 1.06, 95% confidence interval CI 1.03, 1.09), all-cause rehospitalization (HR 1.02, 95% CI 1.00, 1.04), and HF rehospitalization (HR 1.09, 95% CI 1.06, 1.12), but not emergency department visits (HR 0.99, 95% CI 0.97, 1.01). In conclusion, for patients hospitalized with HF, congestion was associated with rehospitalization and mortality.
The global pharmaceutical industry portfolio is skewed towards cancer and rare diseases due to more predictable development pathways and financial incentives. In contrast, drug development for major ...chronic health conditions that are responsible for a large part of mortality and disability worldwide is stalled. To examine the processes of novel drug development for common chronic health conditions, a multistakeholder Think Tank meeting, including thought leaders from academia, clinical practice, non-profit healthcare organizations, the pharmaceutical industry, the Food and Drug Administration (FDA), payors as well as investors, was convened in July 2022. Herein, we summarize the proceedings of this meeting, including an overview of the current state of drug development for chronic health conditions and key barriers that were identified. Six major action items were formulated to accelerate drug development for chronic diseases, with a focus on improving the efficiency of clinical trials and rapid implementation of evidence into clinical practice:1.Involve implementation science in the early phases of drug development.2.Involve regulatory agencies early in drug development, simplify clinical trial conduct and improve inter-agency collaboration.3.Involve payors early in drug development.4.Investigate novel implementation strategies.5.Increase focus on and funding for implementation science to develop strategies that improve utilization of proven effective treatments.6.Change public perception.
Regulators and Health Technology Assessment (HTA) bodies are increasingly familiar with, and publishing guidance on, external controls derived from real‐world data (RWD) to generate real‐world ...evidence (RWE). We recently conducted a systematic literature review (SLR) evaluating publicly available information on the use of RWD‐derived external controls to contextualize outcomes from uncontrolled trials submitted to the European Medicines Agency (EMA), the US Food and Drug Administration (FDA), and/or select HTA bodies. The review identified several key operational and methodological aspects for which more detailed guidance and alignment within and between regulatory agencies and HTA bodies is necessary. This paper builds on the SLR findings by delineating a set of key takeaways for the responsible generation of fit‐for‐purpose RWE. Practical methodological and operational guidelines for designing, conducting, and reporting RWD‐derived external control studies are explored and discussed. These considerations include: (i) early engagement with regulators and HTA bodies during the study planning phase; (ii) consideration of the appropriateness and comparability of external controls across multiple dimensions, including eligibility criteria, temporality, population representation, and clinical evaluation; (iii) ensuring adequate sample sizes, including hypothesis testing considerations; (iv) implementation of a clear and transparent strategy for assessing and addressing data quality, including data missingness across trials and RWD; (v) selection of comparable and meaningful endpoints that are operationalized and analyzed using appropriate analytic methods; and (vi) conduct of sensitivity analyses to assess the robustness of findings in the context of uncertainty and sources of potential bias.
CONTEXT Emerging technologies, changing diagnostic and treatment patterns, and changes in Medicare reimbursement are contributing to increasing use of imaging in cancer. Imaging is the fastest ...growing expense for Medicare but has not been examined among beneficiaries with cancer. OBJECTIVE To examine changes in the use of imaging and how those changes contribute to the overall cost of cancer care. DESIGN, SETTING, AND PATIENTS Analysis of a nationally representative 5% sample of claims from the US Centers for Medicare & Medicaid Services from 1999 through 2008. Patients were Medicare beneficiaries with incident breast cancer, colorectal cancer, leukemia, lung cancer, non-Hodgkin lymphoma, or prostate cancer. MAIN OUTCOME MEASURES Use and cost of imaging by modality, year, and cancer type. RESULTS There were 100 954 incident cases of breast cancer, colorectal cancer, leukemia, lung cancer, non-Hodgkin lymphoma, and prostate cancer from 1999 through 2006. Significant mean annual increases in imaging use occurred among all cancer types for positron emission tomography (35.9%-53.6%), bone density studies (6.3%-20.0%), echocardiograms (5.0%-7.8%), magnetic resonance imaging (4.4%-11.5%), and ultrasound (0.7%-7.4%). Conventional radiograph rates decreased or stayed the same. As of 2006, beneficiaries with lung cancer and beneficiaries with lymphoma incurred the largest overall imaging costs, exceeding a mean of $3000 per beneficiary within 2 years of diagnosis. By 2005, one-third of beneficiaries with breast cancer underwent bone scans and half of beneficiaries with lung cancer or lymphoma underwent positron emission tomography scans. Mean 2-year imaging costs per beneficiary increased at a rate greater than the increase in mean total costs per beneficiary for all cancer types. CONCLUSION Imaging costs among Medicare beneficiaries with cancer increased from 1999 through 2006, outpacing the rate of increase in total costs among Medicare beneficiaries with cancer.
Translational research is a data-driven process that involves transforming scientific laboratory- and clinic-based discoveries into products and activities with real-world impact to improve ...individual and population health. Successful execution of translational research requires collaboration between clinical and translational science researchers, who have expertise in a wide variety of domains across the field of medicine, and qualitative and quantitative scientists, who have specialized methodologic expertise across diverse methodologic domains. While many institutions are working to build networks of these specialists, a formalized process is needed to help researchers navigate the network to find the best match and to track the navigation process to evaluate an institution's unmet collaborative needs. In 2018, a novel analytic resource navigation process was developed at Duke University to connect potential collaborators, leverage resources, and foster a community of researchers and scientists. This analytic resource navigation process can be readily adopted by other academic medical centers. The process relies on navigators with broad qualitative and quantitative methodologic knowledge, strong communication and leadership skills, and extensive collaborative experience. The essential elements of the analytic resource navigation process are as follows: (1) strong institutional knowledge of methodologic expertise and access to analytic resources, (2) deep understanding of research needs and methodologic expertise, (3) education of researchers on the role of qualitative and quantitative scientists in the research project, and (4) ongoing evaluation of the analytic resource navigation process to inform improvements. Navigators help researchers determine the type of expertise needed, search the institution to find potential collaborators with that expertise, and document the process to evaluate unmet needs. Although the navigation process can create a basis for an effective solution, some challenges remain, such as having resources to train navigators, comprehensively identifying all potential collaborators, and keeping updated information about resources as methodologists join and leave the institution.
CONTEXT Practice guidelines recommend implantable cardioverter-defibrillator (ICD) therapy for patients with heart failure and left ventricular ejection fraction of 30% or less. The influence of sex ...and race on ICD use among eligible patients is unknown. OBJECTIVE To examine sex and racial differences in the use of ICD therapy. DESIGN, SETTING, AND PATIENTS Observational analysis of 13 034 patients admitted with heart failure and left ventricular ejection fraction of 30% or less and discharged alive from hospitals in the American Heart Association's Get With the Guidelines–Heart Failure quality-improvement program. Patients were treated between January 2005 and June 2007 at 217 participating hospitals. MAIN OUTCOME MEASURES Use of ICD therapy or planned ICD therapy at discharge. RESULTS Among patients eligible for ICD therapy, 4615 (35.4%) had ICD therapy at discharge (1614 with new ICDs, 527 with planned ICDs, and 2474 with prior ICDs). ICDs were used in 375 of 1329 eligible black women (28.2%), 754 of 2531 white women (29.8%), 660 of 1977 black men (33.4%), and 2356 of 5403 white men (43.6%) (P < .001). After adjustment for patient characteristics and hospital factors, the adjusted odds of ICD use were 0.73 (95% confidence interval, 0.60-0.88) for black men, 0.62 (95% confidence interval, 0.56-0.68) for white women, and 0.56 (95% confidence interval, 0.44-0.71) for black women, compared with white men. The differences were not attributable to the proportions of women and black patients at participating hospitals or to differences in the reporting of left ventricular ejection fraction. CONCLUSIONS Less than 40% of potentially eligible patients hospitalized for heart failure received ICD therapy, and rates of use were lower among eligible women and black patients than among white men.
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