It’s been a month since the Food and Drug Administration authorized two gene therapies for people battling sickle cell disease. The news opened a glimmer of hope, but many questioned the accessibility of the treatments.
For Andrea Hall, the new authorization of breakthrough treatments, Casgevy by Vertex Pharmaceuticals and Lyfgenia by Bluebird bio, is a stride in the right direction, especially for Black people, who are disproportionately impacted by the disease.
“Sickle cell disease primarily affects people of color,” Dr. Nadia Taylor of Florida’s Banyan Health Systems told Atlanta Black Star. “It’s a disease of the hemoglobin, which is an oxygen-carrying molecule in your blood, specifically your red blood cells. It causes the red blood cells when they’re under stress, specifically, low oxygen conditions…to turn into a half-moon shape. And that’s important because your red blood cells go everywhere in your body and circulate throughout.”
The disorder can cause pain and significant health problems for those living with it. Hall’s daughter, Shakira Martin, passed away at age 30 from complications of the disease in August 2016. She is continuing her daughter’s legacy through Shak’s Hope. This nonprofit foundation advocates for and helps improve the lives of those with sickle cell disease, specifically in Florida and Jamaica.
“I’m excited about the therapies. However, we are still concerned about cost and accessibility. Who has access and how will the cost be covered for a drug that’s over a million dollars,” Hall told Atlanta Black Star during a phone interview.
The cost of the historic treatments is high. The pharmaceutical and biotech giants behind the therapies, Vertex and Bluebird bio, have priced it at $2.2 million and $3.1 million, respectively, BioPharma Dive reported.
Both companies hinted at looking into “outcome contracts,” which would reimburse patients based on how effectively the treatment worked, according to the report. For Bluebird specifically, it is prepping a similar contract opportunity with Medicaid agencies. Black people with the disease who had Medicaid were nearly 90 percent of the insurance program’s enrollees in 2021, an analysis suggests.
“Will Medicaid be able to cover for those that are not able to work, that are not able to get insurance, that may be on disability, which a lot of them are?” Hall asked. “I’m excited for the community. As we move forward, I have to be because this is growth. For us, I know not everybody qualifies.”
‘It’s How She Lived’
To Hall, Shakira was a miracle. Before she was born, doctors informed her that she may not live past 12 years old; however, giving up was not an option for Hall. Her daughter went on to compete in beauty pageants — winning Miss Jamaica Universe in 2011 — and was a preschool teacher as well as the brainchild behind Shak’s Hope. She transcended all the barriers against her.
“She really was very resilient, even young. It’s not how she died, it’s how she lived,” Hall said.
For a parent dealing with a child with sickle cell, there were also many challenges. The journey included lengthy hospital stays and tackling side effects from medications. Hall recalls when Shakira’s friends visited to distract her from the discomfort. Throughout her life, she took a drug called hydroxyurea and had to receive several blood transfusions.
“It’s the fact that you can’t bear pain for your child, that you can’t take away any of it,” Hall added. “She required a lot of attention.”
Other people like Obi Ariguzo, who have also taken hydroxyurea and other medications, are optimistic about a potential new solution.
“At this point, if anything can help kind of limit the amount of pain crises I’m having with how frequently they’re happening, I’m all up for it,” Ariguzo, 39, told NBC News. “I feel like I’ve tried everything at this point.”
Hall has stressed the importance of supporting individuals grappling with the disease.
“I would just encourage people to show compassion because they need it. These patients need it. And I find that it’s lacking all around in the general public and lacking in the health care system.”
