State lawmakers have budgeted nearly $15 million over two years to help Virginia Medicaid cover gene therapy treatments for people with sickle cell disease, a move providers said could help patients access this complex treatment more easily.
The funding is tied to a federal model designed to help Medicaid programs pay for expensive cell and gene therapies. It would help cover treatment for patients on Medicaid and other state-sponsored insurance plans and allow Virginia to pay separately for the gene therapy drug and the care needed to deliver it.
Sickle cell disease is an inherited blood disorder that can cause severe pain and organ damage. Gene therapy is a new treatment option for sickle cell disease that can help some patients produce healthier blood cells. But it is a long and intensive process that requires chemotherapy first, which can affect fertility.
Madeeha Deo, medical director at the Sentara-EVMS Comprehensive Sickle Cell Program, said the region has the largest adult sickle cell population in Virginia, with an estimated 800 to 1,200 adults living with the disease.
The program, which launched in October 2024, now serves more than 200 active adult patients. Deo said the clinic is still growing, seeing about three to five new patients a week.
Before the program opened, many Hampton Roads patients had to travel to Richmond or North Carolina for specialty care.
“They were either seeking care with their primary care doctors or hematologists, you know, that may not see a lot of sickle cell patients,” Deo said. “And when they weren’t receiving care at a sickle cell center, they were having to rely on local emergency rooms and hospitals for their acute care.”
Two gene therapies for sickle cell disease were approved by the FDA in late 2023. Sentara-EVMS does not currently provide the treatment, but Deo said the program screens patients who may be eligible and refers them to VCU Health in Richmond.
Elizabeth Krieger, the director of pediatric stem cell transplantation at Children's Hospital of Richmond at VCU, said they are the only gene therapy site for sickle cell patients in Virginia.
The treatment is not a simple infusion. Krieger said patients first go through consultations, organ function testing and insurance approval. Their blood-forming cells are collected and sent out for processing, which can take months.
Once the modified cells return, patients receive several days of chemotherapy to make room in the bone marrow before the cells are infused back into the body.
Krieger said patients may spend about 30 to 40 days in the hospital during part of the process. From the time a patient decides to pursue gene therapy, she said the full process can take about a year.
Deo said cost is a major barrier because many sickle cell patients are covered by Medicaid, an issue the new funding is meant to help with.
But other barriers remain, including eligibility, travel to Richmond, time away from work, family support and the risk that chemotherapy can affect fertility.
Krieger said the gene therapy access model "standardizes the access criteria for patients and the reimbursement structure for hospitals."
"This will increase the number of patients that can be impacted,” Kreiger said.
She noted fertility preservation had been a major gap for Medicaid patients pursuing gene therapy. She said the model will help address that by supporting coverage for fertility preservation, including freezing and storage.
The model also allows states enter agreements with drug manufacturers to pay less if a treatment does not meet expected clinical outcomes.
Deo said the budget change is important, but it will not replace the need for continued investment in comprehensive sickle cell care.
“There will be a lot of patients who will not be genetic therapy candidates and will still need access to good comprehensive sickle cell care,” Deo said.
