Sickle cell disease and cystic fibrosis are both rare, genetic diseases. While there are three times as many people (predominantly of African ancestry) in the U.S. with sickle cell disease compared to people (predominantly of European ancestry) with cystic fibrosis, research dollars are the same, after tallying federal funding from the National Institutes of Health and disease-specific foundations.
That per-patient funding gap has been known for decades. A new study, published in JAMA Network Open, translated those differing amounts into results: funding for researchers, scientific publications presenting discoveries, and drugs to treat patients. A team led by Duke hematologist John Strouse found a similar number of clinical trials but more scientific papers and drugs for cystic fibrosis.
Strouse, whose 20-year interest in improving the care of people with sickle cell disease dates back to medical school, talked to STAT about these results and what impact he’d like them to have. This interview has been condensed and lightly edited.
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The X factor that was not analyzed was Bob Beall, former CEO of Cystic Fibrosis Foundation. Bob did more to change the trajectory of care and innovative therapies for CF than anyone has in patient advocacy for any disease. Under his leadership CFF created continuous quality improvement at CF centers, established a therapeutics development network, and funded innovative research. Any disease would be fortunate to have an advocate with the vision and tenacity of Bob.
Cystic Fibrosis Foundation has a very successful Venture Philanthropy model which has leveraged their donor dollars and enabled more significant research investment. I’m the CEO of CureDuchenne; since 2004, we’ve also been able to leverage philanthropic donations and multiply our research through successful Venture Philanthropy. A significant measure of a non-profit’s ability to fill the drug development pipeline, is a solid business model. This should be included as a factor in comparing disease outcomes.