From my early teens, I wasn’t sure if Deshawn “DJ” Chow could lead a normal life. The episodes of crushing pain caused by his sickle cell disease were gradually getting worse.
“It’s difficult to skip school and always get in and out of hospitals,” the 19-year-old said. “And the severe pain in my head and my lower back.”
When new sickle cell gene therapy was approved by the Food and Drug Administration more than a year ago, butterfly adoptive parents search for Hope Children’s Urban Cancer Center in Los Angeles to make access to new treatments. did. For their relief, the center accepted him as a patient and promptly secured approval from the insurance sponsored by Chaws’s employer.
“They rarely get everything out of their pockets for us, so we’re really grateful for the benefits of those,” said Sean Chow, the father of the DJ. “I’m surprised.”
DJ Chow is one of the hospital patients treated at Casgevy; Apex drugscosts more than $2 million per patient. The treatment process included multiple hospitalizations and chemotherapy treatments at additional costs over the past year.
Sickle red blood cells are blood disorders in which a person’s red blood cells collapse into a crescent moon. It disproportionately affects black people and causes episodes of intense pain that allow patients to frequently land in hospitals.
Chow is one of a handful of patients completing treatment with new gene therapy. After completing his full course of Casgevy treatment in January, he’s starting to make him dream of doing what he wants to do all the time.
“Learn how to snowboard and surf, do all these things… experiences I really didn’t do because of my sickle,” he said.
Slow ramp up
Over 100,000 Americans suffer from sickle cell disease, but young patients whose organs are not damaged by the disease are the most promising candidates to benefit from new treatments.
Still, the increase in our ability to treat large patients has slowed down. Over 100 patients have been treated in the first year since two gene therapies for sickle cells were approved by the FDA.
Vertex executives call the company’s fourth quarter revenues, which 50 patients worldwide received their first cell collection by the end of last year. Meanwhile, competitor executives Bluebird Bio Last fall, nearly 50 patients said they had been treated with the drug Rifgenia. Another 37 patients are expected to begin treatment for Bluebird therapy by early 2025.
The first treatment center requires a little learning curve to adjust to obtain coverage with insurance companies to provide new sickle gene therapy.
Jennifer Cameron, executive director of patient access at Children’s National Hospital in Washington, DC, sends billing and coding guides on multiple occasions. It’s … developed by the manufacturer and if they don’t know about it, they share it with the payer. ”
Dr. Leo Wang of Hope City of Hope, a pediatric hematologist oncologist who works with Chow, also said the process has become smoother, but the prices of these treatments still pose a coverage hurdle. I’m worried that it might happen.
“The challenges of the healthcare system are immeasurable. This is a very expensive treatment,” he said.
So far, slow ramp-ups in patients undergoing treatment have made early cases manageable, said David Joyner, CEO of CVS Health, the parent company of health insurance company Aetna. However, with demand expected to increase, he said many in the industry are considering developing new payment models for sickle cell therapy on the horizon and other gene therapy.
“New risk pools are being developed, sometimes at the Medicaid level in the state, sometimes crossing large payers,” explains Joyner, and the financial burden of treatment is one state or one company. It’s spreading beyond.
“Today’s payment models are not built to spread costs, so we need to think about another payment model,” he said. “But that takes time.”
Medicaid’s challenge
For the state’s Medicaid program, the affordable challenges for new sickle cell treatments may be even greater. More than half of sickle cell patients are covered by the federal government health plan for low-income Americans.
Southern states like Georgia, Florida and Mississippi have the highest concentrations of sickle cell patients, according to a study by researchers at the University of Chicago.
The Biden Bureau has developed a cell and gene therapy payment model under the Centers for Medicare and Medicaid. This will provide states with a base discount price and fund new drugs. According to CMS officials, the state’s deadline for applying for the program is February 28th, with the first federal grant to help pay for drugs that are on track in June.
With the new payment model, states can receive up to $9.5 million in federal funding, but in some cases there are discounted prices to avoid starting to cover medical costs for Medicaid patients.
Researchers at Oregon Health & Science University can see the impact of average budgets at $30 million based on 10 states with the largest sickle cell population, based on a treatment estimate of just under $1.9 million I calculated that.
These increases in costs will come as the Trump administration and Republican-controlled Congress are looking for ways to cut federal spending. The administration is already beginning to cut staffing at health agencies, and federal funding for the state’s Medicaid program is expected to appear on the table in upcoming budget proposals.
Health and Human Services Director Robert F. Kennedy Jr. will provide adjustments to sickle cell compensation in the department and other agencies without specifically discussing new gene therapy or Medicaid funding at a confirmation hearing last month. He said he is committed to maintaining staffing to the point. whole.
“I have a lot of friends who have sickle cells and I’ve seen the pain they endure,” Kennedy said. “There are promising gene therapies right now. They are very, very expensive, but that’s something (National Institutes of Health) should be enthusiastically supported and that kind of research.”
Sean Chow said he appreciates researchers who developed gene therapy. He hopes his son has a more normal future without debilitating episodes of pain. He hopes that other families have the same opportunity to access expensive gene therapy for their loved ones.
“It was heartbreaking to have a child with sickle cells,” he said. “I hope more and more patients will be treated and I hope they can keep costs down.”
