Starting in his early teenage years, Deshawn “DJ” Chow was not sure if he could ever lead a normal life. Limitation of pain delivery caused by his sickle cell disease were getting worse.
“It has just been difficult to skip school and always be in and out of the hospital,” said the 19-year-old. “And just serious pain in … my head and lower back.”
When new sickle cell gene therapies were approved just more than a year ago by the Food and Drug Administration, Chow's adopted Parents City of Hope Children's Cancer Center in Los Angeles sought to gain access to the new treatment. To their relief, the center accepted him as patient and quickly took care of the authorization of the insurance sponsored by De Kauwelen.
'They cover all all this [at] Almost no costs from our own pocket for us. So we are very grateful for those benefits, “said DJ's father, Sean Chow.” I am surprised. “
DJ Chow is one of the handful of patients of the hospital treated with Casevy, the sickle therapy produced by Vertex -medicinesWhich costs more than $ 2 million per patient. The treatment process included several hospital admissions and chemotherapy treatments at extra costs in the course of the past year.
Sickle cell is a blood disorder in which the red blood cells of a person are deformed in Half Maan. It influences disproportionately black people and causes serious pain deliveries that patients can often land in the hospital.
Chow is one of the small number of patients to complete the treatment with new gene therapies. After completing the full course of Casevly treatments in January, he starts to dream of doing the things he always wanted to do.
“Learn how to snowboard and surf and do all these things … experiences I never really did because of my sickle cell,” he said.
Slow rises
Although more than 100,000 Americans suffer from sickle cell disease, younger patients whose organs are not damaged by the disease are the most promising candidates to take advantage of the new treatments.
Yet the rise of capacity to treat patients on scale was slow. In the first year since two gene therapies for sickle cells were approved by the FDA, just over 100 patients have undergone treatment.
Vertex leaders said that the profit of the company in the fourth quarter of the company that 50 patients had received their first cell collections worldwide by the end of last year 2024. Meanwhile, managers at competitor Bluebird Bio Last fall that nearly five dozen patients had undergone treatment with the drug lyffic, which costs more than $ 3 million per patient. Another 37 patients are planned to start treatment with the therapy of Bluebird in early 2025.
For the first treatment centers that the new Sikkel gene therapies offer, coordination with insurers about obtaining coverage requires a little learning curve.
“It's much smoother today than when we first started getting patients,” said Jennifer Cameron, executive director of the access patient access in the Children's National Hospital, in Washington, DC, “we often send them the invoicing and coding guides that are Developed by the … manufacturer and we share that with the payer if they know nothing about it. “
Dr. Leo Wang of City of Hope, the pediatric hematologist-oncologist who works with Chow, also said that the process has become smoother, but he is worried that the price of these treatments is still obstacles to cover.
“The challenges for the health care system are huge. This is a very expensive therapy,” he said, “it can be a bit difficult for employer-based insurance plans to accommodate those costs.”
So far, the slow driveway of patients in treatment has made the coverage of early cases manageable, said David Joyner, CEO of CVS Health, the parent company of health insurer Aetna. But with the demand expected, he said that many in the industry are looking at the development of new payment models for sickle cell treatments and other gene therapies on the horizon.
“Riskoles are being developed … sometimes at the state of Medicaid levels, and sometimes collectively in larger payers,” Joyner explained so that the financial burden of the treatments is only one or one company is distributed.
“You have to think about a different payment model, because today's payment model was not built to distribute the costs,” he said. “But that takes time.”
A challenge for Medicaid
For Medicaid programs of the State, the challenge of affordability for the new sickle cell treatments can be even greater. More than half of the sickle cell patients fall under the health plan of the federal state for Americans with a low income.
Southern states such as Georgia, Florida and Mississippi have some largest concentrations of sickle cell patients, according to a study by researchers from the University of Chicago.
The Biden administration developed a cell and gene therapy payment model among the Centers for Medicare and Medicare, which will offer states a price-based price and will provide some financing for the new medicines. The deadline for states to request the program is 28 February, with the first federal subsidies that can pay the medicines on the right track, according to CMS officials.
According to the new payment model, states up to $ 9.5 million can receive federal financing, but even with discount prices that may not start covering the costs of dealing with medicid patients in some cases.
Researchers from Oregon Health & Science University calculated that the 10 states with the largest sickle cell populations could see an average budget impact of $ 30 million, based on an estimate of treatments that cost a little less than $ 1.9 million.
Those higher costs would come at a time when the Trump government and the Republican controlled congress are looking for ways to lower federal expenses. The administration has already started reducing staff at health authorities and federal financing for State Medicaid programs is expected 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 is committed to maintaining staff to coordinate sickle cell coverage about the department and other agencies, without specifically discussing general.
“I have many friends who have sickle cell. I have seen the suffering they ended,” said Kennedy. “There are now promising gene therapies. They are very, very expensive, but it is something that [National Institutes of Health] Should enthusiastically support – that kind of research. “
Sean Chow said that he is grateful to the researchers who have developed gene therapy, which he hopes to enable his son to have a more normal future, without debilitating episodes of pain. He wants other families to get the same chance to gain access to the expensive gene therapies for their loved ones.
“Having a child with sickle cell has been heartbreaking,” he said. “I hope that as more and more patients get therapy, the costs can be driven out.”
