Low-income Floridians with sickle cell disease will have access to new gene therapies

The Florida Agency for Health Care Administration approved both sickle cell disease gene therapies for Medicaid coverage on June 14, 2024. (Photo by TEK IMAGE/SCIENCE PHOTO LIBRARY via Getty Images)Quality Journalism for Critical Times

Raising a child with sickle cell disease during the 1990s, there wasn’t much hope for Kemba Gosier, a Miami resident, that she could make her daughter’s unbearable pain go away.

Now, Floridians with sickle cell disease are closer to a functional cure after health care officials authorized Medicaid coverage for newly FDA-approved gene therapies.

Officials with the Florida Agency for Healthcare Administration voted Friday to cover both breakthrough therapies, offering relief for more than 7,000 low-income people with subsidized health insurance in the state, according to a report from the agency. The staggering cost of the therapies left people in the sickle cell medical community wondering how accessible the one-time treatment would be; it can cost up to $3.1 million.

The more than 7,000 Florida Medicaid patients represent about half of the state’s total population living with sickle cell. A 2023 study in Blood, a journal of the American Society of Hematology, placed the number of cases in Florida at 13,886, the highest in the nation. Most people living with sickle cell disease in the country are Black. In fact, 63 percent of Florida Medicaid recipients with the disease are Black, according to the AHCA report.

‘It gives us so much hope’

For Gosier, finding out the FDA had approved the gene therapies late last year felt unreal, she told Florida Phoenix in a video interview. She hoped AHCA would approve both therapies so families and their doctors could determine which one suited them best.

“There was a sense of, ‘My goodness, finally,’ because sickle cell has been documented since 1910,” Gosier said. “And even though it’s been documented that long, we only have four treatments as far as drug therapies, so the fact that we now have an actual curative treatment is unprecedented. It gives us so much hope and inspiration, especially for our younger children that are coming up.”

A 12-year-old boy in Washington, D.C., received the first treatment in May, according to The New York Times.

New way for states to cover pricey gene therapies will start with sickle cell disease

Despite the high cost, Deepakbabu Chellapandian, director of the bone marrow transplant unit at Johns Hopkins All Children’s Hospital in St. Petersburg, told AHCA officials during a Thursday meeting that Medicaid coverage of the therapies could result in overall savings.

“The initial cost of the gene therapy may be prohibitively high, but it is a one-time treatment compared to the ongoing lifelong medical care required for managing sickle cell symptoms. By potentially curing the disease, gene therapy can reduce the overall financial burden on the health care system, insurance providers, and patients,” he said.

In the meantime, a new federal program will make the life-changing treatments available to Medicaid patients starting next year.

Here’s how the therapies work

Vertex Pharmaceuticals and CRISPR Therapeutics developed Casgevy and Bluebird Bio developed Lyfgenia. Both work by removing blood stem cells from the bone marrow and sending them to the manufacturers to edit the cells, which are infused back into the body. Manufacturing the cells can take between 10 weeks and six months and the process requires the use of other drugs to condition the body for the procedure.

While AHCA approved coverage for both drugs, it classified Lyfgenia as the preferred treatment, meaning that additional steps are needed before specific patients can access Casgevy. Still, both require prior authorization. Only people 12 years or older who have frequent complications from sickle cell disease can undergo the therapies.

Additionally, the treatments can take place only at qualified health centers. A representative from Bluebird Bio who attended the AHCA meeting on Friday said the company is onboarding centers with experienced personnel to handle the procedure in Gainesville, Jacksonville, Orlando, and Miami.

“We can’t necessarily share every [qualified treatment center or QTC] that is in process, but, currently, we continue to expand and we’ll have a meaningful number of QTCs in the state,” he said.

A Vertex spokesperson told the Phoenix via email the company expects to open a treatment center in Florida in the coming weeks.

Community connections

Kemba Gosier of Miami established a nonprofit to help parents whose children have sickle cell disease. Photo courtesy of Grosier.

Before the development of the therapies, bone marrow transplant was the only cure. People with sickle cell disease also have to take strong pain medication. By the time Gosier’s daughter was 11 years old, she needed a hip replacement. She had endured more than 30 hospital visits because the sickle-shaped red blood cells in her body caused so much pain.

“There is no greater pain in the world for a parent to than to watch your child suffer, and there’s absolutely nothing you can do about it,” she said. “That’s like the worst feeling in the world: That you’re not able to take the pain away because, as a mom, you’re supposed to kiss the boo-boos and make it better. But when you have a serious health condition like sickle cell disease, you try your best but it’s very difficult to watch your child suffer.”

Gosier created the nonprofit Advancing Sickle Cell Advocacy Project in 2015 to help parents of children with sickle cell disease cope. When news of the new therapies spread, the nonprofit held a town hall with representatives of the manufacturers to answer the many questions floating in the community.

How are gene therapies different than bone marrow transplants?

University of Miami’s Dela Ziga, a pediatric hematology and oncology doctor, said his first patient set to undergo the gene therapy has beta-thalassemia, another inherited blood disorder. Ziga has been treating patients with sickle cell disease since 1990 in Ghana, and he has been performing bone marrow transplants since 2013, but he likened the FDA’s move to approve Casgevy and Lyfgenia at once to a lightning strike.

“When the announcement was made for these two products, I wanted to do a cartwheel. That’s how happy I was,” he said in a video interview with the Phoenix.

“My phone just went off the hook. Everyone was texting me and calling me. All my patients. So, it was very, very exciting news for us, and I will say a great day for the sickle cell population world. I call it a very rare event, similar to a lightning strike, because for two gene products to be approved on the same day for one disease is unbelievable.”

More patients could qualify for gene therapy than for bone marrow transplants because the gene editing uses the patient’s own cells rather than a donor’s.

Regarding Medicaid coverage of the gene therapies, Ziga said it would “be fantastic.”

Aside from the Medicaid coverage of breakthrough therapies in Florida, Gov. Ron DeSantis has approved a $10 million grant program for community-based projects increasing access to specialized sickle cell disease treatments in the 2024-25 fiscal year budget.

“I just think this is a new day for sickle cell disease. This is our time now. All eyes are on us, and we finally need to be put as a priority,” Gosier said.

The post Low-income Floridians with sickle cell disease will have access to new gene therapies appeared first on Florida Phoenix.