The time has come for the U.S. Food and Drug Administration (FDA) to decide on a new sickle cell treatment. The FDA has until Dec. 8 to decide the fate of the drug.
As previously reported by BLACK ENTERPRISE, Exa-cel, the one-time gene therapy created by Vertex Pharmaceuticals and CRISPR therapy, has the potential to change lives. As the public awaits the FDA’s decision, concerns are being raised about the drug’s cost, accessibility, and transparency regarding potential side effects. The sickle cell treatment would be the first of its kind, in that would use CRISPR, a gene editing tool that alters one’s DNA. But its side effects are unknown, according to NBC News.
The newness of the drug, as well as a distrust of healthcare held by the Black community, may lead to hesitation in receiving the treatment.
Derek Robertson, president of Maryland Sickle Cell Disease Association, has two sons with sickle cell. He said, “We are aware that there are a number of individuals who are still a little bit hesitant. But overall, we’re excited mainly because it’s another option.”
Robertson, whose wife works in patient advocacy at Vertex, believes the focus should be on establishing a drug that is effective, and to “get it to the people who need it.” Later, the focus can turn to finding ways to ensure affordability and access.
As Dr. Cecelia Calhoun, a hematologist-oncologist and medical director of the Adult Sickle Cell Program at Smilow Cancer Hospital at the Yale New Haven Health System in Connecticut, discussed the disease process, saying sickle cell affects the “quality of life and ability to just function as normal humans.”
Kayla Smith Owens, a 25-year-old graduate of Bowie State University, shared her experience living with sickle cell disease. She was diagnosed at age 12 after experiencing mild pain and fatigue during puberty. Those symptoms were exacerbated following her matriculation in 2016.
“I had no college experience at all,” she said. “My time in college consisted of being in the hospital for seven days, coming home, catching up, and then going right back to the hospital.”
Living with the disease drastically altered her college experience. “I couldn’t join clubs. I didn’t have time to make friends because my time was spent in the hospital,” Owens said.
After graduating with a degree in criminal justice, Owens said she has not had the opportunity to work because she is constantly in pain and “always sick.” Owens feels the new drug “would be huge and change [her] life tremendously.”
Dr. Jeffrey Glassberg, director of the Mount Sinai Center for Sickle Cell Disease, had some patients who participated in the Exa-cel and Blue-bird clinical trials. (Blue-bird is an additional sickle cell drug under FDA review.) Glassberg said his patients have “normal blood counts” and are able to run three miles a day because “they don’t have sickle cell anymore.”
To receive the treatment, patients must undergo chemotherapy. Dr. Glassberg attributes the risks associated with chemotherapy to why less than 10% of people being treated for sickle cell at Mount Sinai will pursue the drug as treatment. However, he said the risks have “nothing to do with gene therapy.”
The cost of the treatment is an additional concern among doctors and patients. The drug could cost up to $2 million, according to the Institute for Clinical and Economic Review.
According to an analysis by NORC, a research organization associated with the University of Chicago, 87% of people who are both enrolled in Medicaid and have been diagnosed with sickle cell disease are Black.
As far as coverage and insurance costs are concerned, Dr. Glassberg said, “My experience has been
nobody really knows what’s going to happen until it just hits the market and then stuff starts to happen. Then we sort of figure out how to make it work.”Dr. Glassberg acknowledged the Black community’s distrust of the healthcare industry and said he and his colleagues have worked to build “foundations of trust and gain confidence” with their patients through support groups as well as a process where a group of patients assess and “approve” research studies.
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