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Comparative Study
. 2020 Mar 2;3(3):e201737.
doi: 10.1001/jamanetworkopen.2020.1737.

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

Faheem Farooq  1 , Peter J Mogayzel  2 , Sophie Lanzkron  3 , Carlton Haywood  3   4 , John J Strouse  5   6
Affiliations
Comparative Study

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

Faheem Farooq et al. JAMA Netw Open. .

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.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Mogayzel reported receiving grant funding from Vertex Pharmaceuticals and being a member of the Cystic Fibrosis Foundation Board of Directors. Dr Lanzkron reported being a site principal investigator on research funded by Pfizer, Ironwood, and Global Blood Therapeutics. Dr Strouse reported being a consultant for Global Blood Therapeutics, serving as a site principal investigator on research funded by Takeda, and serving as treasurer of the Sickle Cell Adult Provider Network. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Number of Disease-Specific PubMed Listings and US Food and Drug Administration Drug Approvals Over Time
CF indicates cystic fibrosis; SCD, sickle cell disease.
Figure 2.
Figure 2.. Progression of Life Span for Cystic Fibrosis (CF) and Sickle Cell Disease (SCD) and Major Health Care Milestones
Life Span graphs adapted from Wailoo and Elborn. ALL indicates acute lymphocytic leukemia; CFF, Cystic Fibrosis Foundation; HSCT, hematopoietic stem cell transplant; and NIH, National Institutes of Health.
See this image and copyright information in PMC

References

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