Starting in his early teens, Deshawn “DJ” Chow wasn’t sure he’d ever have the ability to live a traditional life. Crushing pain episodes brought on by his sickle cell disease were getting progressively worse. Â Â
“It’s just been hard skipping school and all the time being out and in of the hospital,” the 19-year-old said. “And just severe pain in … my head and my lower back.”
When recent sickle cell gene therapies were approved by the Food and Drug Administration just over a 12 months ago, Chow’s adopted parents sought out City of Hope Kid’s Cancer Center in Los Angeles to get him access to the brand new treatment. To their relief, the middle accepted him as patient, and quickly secured authorization from the Chows’ employer-sponsored insurance.
“They’re covering just about all of this [at] almost no cost out of pocket for us. So, we’re really grateful for those advantages,” said DJ’s dad, Sean Chow. “I’m amazed.”
DJ Chow is one in all a handful of the hospital’s patients who’ve been treated with Casgevy, the sickle gene therapy produced by Vertex Pharmaceuticals, which costs greater than $2 million per patient. The treatment process involved multiple hospitalizations in addition to chemotherapy treatments at additional cost over the course of the past 12 months.
Sickle cell is a blood disorder during which an individual’s red blood cells develop into misshapen into crescent moons. It disproportionately affects Black people and causes severe pain episodes that may steadily land patients within the hospital.
Chow is one in all small variety of patients to finish treatment with recent gene therapies. After completing the complete course of Casgevy treatments in January, he’s beginning to let himself dream about doing the things he’s all the time desired to do.
“Learn the right way to snowboard and surf and do all this stuff … experiences I never really got to do due to my sickle cell,” he said.
Slow ramp up
While greater than 100,000 Americans suffer from sickle cell disease, younger patients whose organs haven’t been damaged by the disease are probably the most promising candidates to learn from the brand new treatments.
Still, the ramp up of capability to treat patients at scale has been slow. In the primary 12 months since two gene therapies for sickle cell were approved by the FDA, just over 100 patients have undergone treatment.
Vertex executives said on the corporate’s fourth quarter earnings call that fifty patients globally had received their first cell collections by the tip of last 12 months 2024. Meanwhile, executives at competitor Bluebird Bio said last fall that just about five dozen patients had undergone treatment with its drug Lyfgenia, which is priced at greater than $3 million per patient. One other 37 patients are slated to start treatment with Bluebird’s therapy by early 2025.
For the primary treatment centers to supply the brand new sickle gene therapies, coordinating with insurers on obtaining coverage has required a little bit of learning curve.
“It is far smoother today than it was after we first began getting patients in,” said Jennifer Cameron, executive director of patient access at Kid’s National Hospital, in Washington, D.C. “Again and again, we’ll send them the billing and coding guides which might be developed by the … manufacturer and we share that with the payer, in the event that they don’t find out about it.”
City of Hope’s Dr. Leo Wang, the pediatric hematologist-oncologist who works with Chow, also said the method has gotten smoother, but he worries that the worth of those treatments still poses hurdles for coverage.
“The challenges for the health care system are immense. It is a very expensive therapy,” he said, “For employer-based insurance policy it might be a bit bit difficult to accommodate those costs.”
Up to now, the slow ramp up of patients in treatment has made coverage of early cases manageable, said David Joyner, CEO of CVS Health, the parent company of health insurer Aetna. But with demand expected to ramp up, he said many within the industry are developing recent payment models for the sickle cell treatments and other gene therapies on the horizon. Â
“There are emerging risk pools being developed … sometimes on the state Medicaid levels, and sometimes collectively across larger payers,” Joyner explained, in order that the financial burden of the treatments is spread beyond only one state or one company.
“You’ve gotten to take into consideration a special payment model, because today’s payment model will not be constructed to spread the price,” he said. “But that takes time.”
A challenge for Medicaid
For state Medicaid programs, the challenge of affordability for the brand new sickle cell treatments could also be even greater. Greater than half of sickle cell patients are covered under the federal-state government health plan for low-income Americans.
Southern states like Georgia, Florida and Mississippi have some the most important concentrations of sickle cell patients, in response to a study by researchers on the University of Chicago.
The Biden administration developed a Cell and Gene Therapy payment model under the Centers for Medicare and Medicaid, which is able to provide states with an outcomes-based discounted price and supply some funding for the brand new drugs. The deadline for states to use for this system is Feb. 28, with the primary federal grants to assist pay for the drugs on target to start in June, in response to CMS officials. Â Â
Under the brand new payment model, states could receive as much as $9.5 million in federal funding, but even with discounted prices that will not begin to cover the prices of treating Medicaid patients in some cases.
Researchers at Oregon Health & Science University calculated that the 10 states with the most important sickle cell populations could see a mean budget impact of $30 million, based on an estimate of treatments priced at slightly below $1.9 million.Â
Those increased costs would come at a time when the Trump administration and the Republican-controlled Congress are in search of ways to chop federal spending. The administration has already begun to chop staffing at health agencies, and federal funding for state Medicaid programs is anticipated to be on the table in upcoming budget proposals.
Health and Human Services Secretary Robert F. Kennedy Jr. said during his confirmation hearings last month that he’s committed to maintaining staffing to supply coordination of sickle cell coverage across the department and other agencies, without specifically discussing funding for the brand new gene therapies or Medicaid overall.
“I even have many friends who’ve sickle cell. I’ve seen the suffering they endure,” Kennedy said. “There at the moment are promising gene therapies. They’re very, very expensive, but it surely’s something that [National Institutes of Health] must be enthusiastically supporting — that sort of research.”
Sean Chow said he’s grateful to the researchers who developed the gene therapy, which he hopes will allow his son to have a more normal future, without debilitating episodes of pain. He wants other families to have the identical opportunity to access the high-priced gene therapies for his or her family members. Â
“Having a toddler with sickle cell has been heartbreaking,” he said. “I’m hoping as increasingly patients get the therapy, the price might be driven down.”
