Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity.
Abstract
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.
Type
Journal articleSubject
Science & TechnologyLife Sciences & Biomedicine
Medicine, General & Internal
General & Internal Medicine
HEALTH-CARE
BIOMEDICAL-RESEARCH
MORTALITY-RATES
GENE-THERAPY
PAIN CRISES
TRANSPLANTATION
IDENTIFICATION
IVACAFTOR
CHILDREN
SURVIVAL
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https://hdl.handle.net/10161/21139Published Version (Please cite this version)
10.1001/jamanetworkopen.2020.1737Publication Info
Farooq, Faheem; Mogayzel, Peter J; Lanzkron, Sophie; Haywood, Carlton; & Strouse,
John J (2020). 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, 3(3). pp. e201737. 10.1001/jamanetworkopen.2020.1737. Retrieved from https://hdl.handle.net/10161/21139.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
John J. Strouse
Associate Professor of Medicine
My research has focused on the epidemiology, risk factors, and prevention of the pulmonary
and central nervous system complications of sickle cell disease and includes retrospective
and prospective cohort studies and clinical trials. I received my Ph.D. in clinical
investigation from the Johns Hopkins Bloomberg School of Public Health for a series
of studies to identify predictors of cognitive function in children with sickle cell
disease. This work has expanded to the evaluation

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