Sickle Cell Disease (SCD) is a rare inherited blood disease caused by a mutation in the hemoglobin-Beta gene found on chromosome 11. It most commonly affects Black and African Americans in the United States, and has historically received less attention than other rare diseases.
CDC data shows that close to 100,000 Americans have SCD. These patients “have less access to comprehensive team care than people with genetic disorders such as hemophilia and cystic fibrosis,” according to the HHS Office of Minority Health.
There has also been a documented disparity between federal and private financing for SCD as compared with cystic fibrosis, another rare disease that impacts fewer people. One study showed that cystic fibrosis received roughly 440 times as much financial support per patient from national foundations in 2011.
Dr. Ted Love, CEO of Global Blood Therapeutics (GBT) described SCD as “the epitome of disparity.”
President Biden proclaimed this month to be the first annual September Sickle Cell Awareness month, and promised to fight these “systemic inequities in our health care system.” Biden urged Americans to “unite in our mission to enhance the quality of life for those diagnosed with SCD.”
Funding for SCD research has grown in the past few years, with a $100 million donation pledged by the Bill & Melinda Gates Foundation in 2019, followed by $534 million by the NIH Cure Sickle Cell Initiative. Research is also growing, and 894 clinical trials for SCD therapeutics are underway.
Funding research has been paying off, as GBT produced the first FDA-approved medicine that tackles the fundamental cause of sickle cell disease. GBT, which Pfizer is buying, recently received favorable FDA designations for two next-generation medicines in clinical studies; bluebird bio and Vertex also have medications in the pipeline.
The Sickle Cell Disease Treatment Centers Act introduced last week proposes a $535 million annual grant for SCD care at community health and outpatient centers “so patients can access care and other services closer to their homes.” Dr. Love praised the bill as “a significant step toward equitable access for the sickle cell community.”
Learn more about Sickle Cell Disease.