For decades, sickle cell disease has forced patients into a lifetime of hospital visits, medications and blood transfusions to manage a painful genetic blood disorder that can shorten life expectancy by up to 20 years. The disease affects about 100,000 people in the U.S., more than 90 percent of whom are non-Hispanic Black or African American, according to the Centers for Disease Control and Prevention.
The FDA approval of Casgevy and Lyfgenia in December 2023 marked a breakthrough. Both therapies target the underlying genetic cause of sickle cell disease, offering the possibility of a functional cure for patients with severe forms of the condition.
“That opens up a whole world of individuals potentially being cured of sickle cell disease,” said Dr. Edward Donnell Ivy, chief medical officer of the Sickle Cell Disease Association of America.
But clinicians and patient advocates say the path to receiving the therapies is far from straightforward. Treatment involves a grueling process that includes chemotherapy and a stem cell transplant, requiring extended stays at specialized centers that are concentrated in a few regions.
“The patients’ priorities are actually a big part of really trying to figure out what the barriers to access are and how we overcome them,” said Dr. Alexias Thompson, an attending physician in the division of hematology at Children’s Hospital of Philadelphia and spokesperson for the American Society of Hematology.
The high cost of the therapies — priced at over $2 million per patient — and complex insurance coverage further limit access. Many patients live far from treatment centers, and the healthcare system has historically underinvested in sickle cell care. Patient advocacy groups are calling for a coordinated effort to expand access, including better reimbursement models, more treatment sites and support for patients navigating the process.