Study Finds Funding Disparities Between Sickle Cell Disease, Cystic Fibrosis

Disparities in funding exist between sickle cell disease (SCD) and cystic fibrosis (CF), according to a new study published in JAMA Network Open. For SCD, the disparities may be associated with decreased research productivity and novel drug development. In this cross-sectional study, researchers compiled data on funding and outputs for CF and SCD. Specifically, they assessed whether expenditures by the National Institutes of Health (NIH) and each diseases’ national foundations are associated with the number of publications indexed in PubMed, US FDA drug approvals, and active clinical trials. NIH funding is allocated in accordance with disease burden. Because heart disease and cancer are associated with significant mortality and morbidity for millions of people, both conditions receive the largest amounts of funding. Both CF and SCD are inherited disorders that result in intermittent disease exacerbations, requiring hospitalizations. They are also associated with a substantial reduction in median life span. As patients with these diseases age, their rate of hospitalizations increases. “With the exception of recently developed cystic fibrosis transmembrane conductance regulator [CFTR] modulator therapies, approximately 80% of the health care costs associated with each disease is spent on hospital care,” the authors said. Although both diseases received a similar amount of federal government research funding between 2008 and 2018, the researchers note that SCD is 3 times as prevalent as CF. In addition, the US birth rate of SCD is 1 in 365 black individuals while the US birth rate of CF is 1 in 2500 white individuals. “Consideration of SCD as a black disease in the [United States] has permeated the experience for patients since the first description in the Western medical literature,” the researchers said. “The interaction of black individuals with the health care system is associated with distrust, given past ethical violations in the name of medical progress...This distrust between patient and practitioner can lead to conflict that results in suboptimal medical care and worsens patient medication adherence.” Using data from the NIH Report Database, the researchers compiled total NIH funding and career development awards between January 2008 and December 2017. Publicly available Internal Revenue Service tax returns were used to determine funding from disease-specific organizations. Included in the data set were forms from 11 nonprofit SCD organizations and 2 leading CF organizations: Cystic Fibrosis Foundation and Cystic Fibrosis Therapeutics. The study yielded the following results:

NIH funding per person with CF was greater than that for SCD (mean [SD], $2807 [$175] vs $812 [$147]; P <.001)
Philanthropic expenditures were significantly greater per person with CF compared with SCD (mean, $7690 [$3974] vs $102 [$13.7]; P <.001)
From 2008 to 2018, annual CF publications remained greater than those of SCD (mean, 1594 [225] vs 926 [157]; P <.001)
CF trials were significantly more likely to receive industry funding (mean, 15.6 [5.3] vs 6.8 [1.8]; P = .001)

“The funding disparity was markedly increased when factoring in disease-specific private foundation funding,” the researchers said. “The additional research support was associated with greater research productivity and pharmaceutical development for CF compared with SCD.” When it comes to disease-specific drug development, CF received 4 drug approvals during the study window while SCD received 1. In addition, “since 2012, the 3 novel disease-specific drugs that were approved for CF received 5 new indications. For SCD, only hydroxyurea received a new indication in 2017, and l-glutamine was initially approved for SCD the same year.” The researchers believe that because of the gap in private support and the association between funding with quality of life and survival, the federal government should increase its funding of SCD. However, “the more complex societal challenge involves overcoming mistrust and racism to empower and engage a community affected by the disease that has been historically disenfranchised.” Reference: Farooq F, Mogayzel PJ, Lanzkron S, Haywood C, Strouse JJ. Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity. JAMA Netw Open. 2020; 3(3):e201737. doi: 10.1001/jamanetworkopen.2020.1737.