Browsing by Subject "HEALTH-CARE"

Now showing 1 - 4 of 4
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    Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity.
    (JAMA network open, 2020-03-02) Farooq, Faheem; Mogayzel, Peter J; Lanzkron, Sophie; Haywood, Carlton; Strouse, John J
    Importance:Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal recessive disorders associated with intermittent disease exacerbations that require hospitalizations, progressive chronic organ injury, and substantial premature mortality. Research funding is a limited resource and may contribute to health care disparities, especially for rare diseases that disproportionally affect economically disadvantaged groups. Objective:To compare disease-specific funding between SCD and CF and the association between funding and research productivity. Design, Setting, and Participants:This cross-sectional study examined federal and foundation funding, publications indexed in PubMed, clinical trials registered in ClinicalTrials.gov, and new drug approvals from January 1, 2008, to December 31, 2018, in an estimated US population of approximately 90 000 individuals with SCD and approximately 30 000 individuals with CF. Main Outcomes and Measures:Federal and foundation funding, publications indexed in PubMed, clinical trial registrations, and new drug approvals. Results:From 2008 through 2018, federal funding was greater per person with CF compared with SCD (mean [SD], $2807 [$175] vs $812 [$147]; P < .001). Foundation expenditures were greater for CF than for SCD (mean [SD], $7690 [$3974] vs $102 [$13.7]; P < .001). Significantly more research articles (mean [SD], 1594 [225] vs 926 [157]; P < .001) and US Food and Drug Administration drug approvals (4 vs 1) were found for CF compared with SCD, but the total number of clinical trials was similar (mean [SD], 27.3 [6.9] vs 23.8 [6.3]; P = .22). Conclusions and Relevance:The findings show that disparities in funding between SCD and CF may be associated with decreased research productivity and novel drug development for SCD. Increased federal and foundation funding is needed for SCD and other diseases that disproportionately affect economically disadvantaged groups to address health care disparities.
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    Depression, quality of life, and medical resource utilization in sickle cell disease.
    (Blood advances, 2017-10-12) Adam, Soheir S; Flahiff, Charlene M; Kamble, Shital; Telen, Marilyn J; Reed, Shelby D; De Castro, Laura M
    Sickle cell disease (SCD) is a chronic, debilitating disorder. Chronically ill patients are at risk for depression, which can affect health-related quality of life (HRQoL), health care utilization, and cost. We performed an analytic epidemiologic prospective study to determine the prevalence of depression in adult patients with SCD and its association with HRQoL and medical resource utilization. Depression was measured by the Beck Depression Inventory and clinical history in adult SCD outpatients at a comprehensive SCD center. HRQoL was assessed using the SF36 form, and data were collected on medical resource utilization and corresponding cost. Neurocognitive functions were assessed using the CNS Vital Signs tool. Pain diaries were used to record daily pain. Out of 142 enrolled patients, 42 (35.2%) had depression. Depression was associated with worse physical and mental HRQoL scores (P < .0001 and P < .0001, respectively). Mean total inpatient costs ($25 000 vs $7487, P = .02) and total health care costs ($30 665 vs $13 016, P = .01) were significantly higher in patients with depression during the 12 months preceding diagnosis. Similarly, during the 6 months following diagnosis, mean total health care costs were significantly higher in depressed patients than in nondepressed patients ($13 766 vs $8670, P = .04). Depression is prevalent in adult patients with SCD and is associated with worse HRQoL and higher total health care costs. Efforts should focus on prevention, early diagnosis, and therapy for depression in SCD.
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    End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.
    (Blood advances, 2019-12) Farrell, Ann T; Panepinto, Julie; Carroll, C Patrick; Darbari, Deepika S; Desai, Ankit A; King, Allison A; Adams, Robert J; Barber, Tabitha D; Brandow, Amanda M; DeBaun, Michael R; Donahue, Manus J; Gupta, Kalpna; Hankins, Jane S; Kameka, Michelle; Kirkham, Fenella J; Luksenburg, Harvey; Miller, Shirley; Oneal, Patricia Ann; Rees, David C; Setse, Rosanna; Sheehan, Vivien A; Strouse, John; Stucky, Cheryl L; Werner, Ellen M; Wood, John C; Zempsky, William T
    To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.
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    Mapping the Dynamic Complexity of Chronic Disease Care in Singapore: Using Group Model Building in Knowledge Elicitation
    (Systems Research and Behavioral Science, 2018-11-01) Ansah, JP; Matchar, DB; Koh, V; Schoenenberger, L
    This study describes a group model building exercise that aims to develop a deeper understanding of the dynamic complexity of chronic disease care delivery within a primary care setting in Singapore, leveraging on the insights of stakeholders with personal and institutional knowledge of the health care system. A group model building exercise, which included 50 stakeholders, was used to develop the qualitative model. The qualitative model helped to bring a feedback perspective to understanding the dynamic complexity of chronic disease care delivery. The feedback perspective helped in identifying the systemic issues within chronic disease care delivery, which has the potential to inform system-wide interventions and policies to improve health. Enhancing chronic care in Singapore will require an enhancement of both the capacity and capability of the primary care sector. © 2018 John Wiley & Sons, Ltd.