Since his teenage years, Deshawn “DJ” Chow isn’t sure if he can live a normal life. The pain attacks caused by his sickle cell disease gradually worsened.
“It’s just hard to skip school and always go in and out of the hospital,” the 19-year-old said. “Just… the severe pain in my head and lower back.”
More than a year ago, when the Food and Drug Administration approved a new sickle cell gene therapy, Joe’s adoptive parents sought out the City of Hope Children’s Cancer Center in Los Angeles to give him a new treatment. To lift their relief, the center accepted his patience and quickly gained authorization from Chows’ employer-sponsored insurance.
“They cover almost all of this (AT) and spend almost no money. So we really appreciate the benefits,” said Sean Chow, DJ’s father. “I was surprised.”
DJ Chow is one of the few patients in the hospital who received Casgevy treatment, which is Pinpoint Drugsthe cost per patient is over $2 million. The treatment process involves multiple hospitalizations and chemotherapy treatments, over the past year, at an additional cost.
Sickles are a blood disease in which one of the people’s red blood cells become a crescent satellite. It disproportionately affects black people and causes severe pain attacks that often land in hospitals.
Chow is one of the few patients who have completed treatment using new gene therapy. After completing the full process of Casgevy’s treatment in January, he began to dream of doing what he had always wanted to do.
“Learn how to ski and surf, do all these things … go through sickle cells that I never really did,” he said.
Slow ramp
While more than 100,000 Americans suffer from sickle cell disease, young patients who have not yet been damaged by the disease are the most promising candidates to benefit from new therapies.
Nevertheless, the improvement in the ability to treat patients on a large scale is still slow. In the first year since the two gene therapy used in sickle cell, the FDA has approved more than 100 patients.
Vertex executives said in the company’s fourth-quarter revenue call that 50 patients worldwide received their first cell collections by the end of last year. Meanwhile, competitor executives Bluebird creature It said last fall that nearly five patients treated their drug, Yejing, which costs more than $3 million per patient. Another 37 patients are scheduled to begin treatment with Bluebird treatment in early 2025.
For the first treatment centers to offer new sickle gene therapy, coordination with insurance companies to gain coverage requires some learning curve.
“Today is smoother than when we first started recruiting patients.” Jennifer Cameron, Executive Director of Patient Visits at Children’s National Hospital in Washington, D.C. This was developed by… manufacturers, if they don’t know, we have a relationship with the payer. shared.”
Dr. Leo Wang, a pediatric hematologist who works with Chow, also said the process has become smoother, but he is concerned that the price of these treatments still presents obstacles.
“The challenges to the healthcare system are enormous. It’s a very expensive therapy and it can be a bit difficult for employer-based insurance plans to adapt to these costs,” he said.
David Joyner, CEO of CVS Health, parent company of health insurer Aetna, said that so far the slow rise in patients in treatment has brought coverage of early cases. But as demand is expected to increase, he said many in the industry are considering developing new payment models for sickle cell therapy and other gene therapies.
“The emerging risk pool was developed … sometimes at the state Medicaid level, sometimes collectively developed among large payers,” Joyner explained.
“You have to think about different payment models because today’s payment models are not for the purpose of spreading costs,” he said. “But that takes time.”
Medicaid Challenges
For the National Medicaid, the affordability challenge of new sickle cell therapy may be greater. More than half of sickle cell patients cover federal government health programs targeting low-income Americans.
According to Georgia, southern states such as Florida and Mississippi, the highest concentrations of sickle cell patients. Research by University of Chicago researchers.
The Biden administration has developed a cell and gene therapy payment model under the Medicare and Medicaid Centers that will provide states with results-based discount prices and provide some funding for new drugs. According to CMS officials, states have a deadline to apply for the program on February 28, first receiving federal grants to help pay for drugs starting in June.
Under the new payment model, states can receive Up to $9.5 million in federal fundingbut even the discounted price may not start covering the cost of treating Medicaid patients in some cases.
Researchers at Oregon University of Health and Sciences have calculated 10 states with the largest population may see an average budget impact of $30 millionbased on estimates for valuation estimates are less than $1.9 million.
These increased costs will occur at a time when the Trump administration and Republican-controlled Congress are looking for ways to reduce federal spending. The government has begun cutting staffing in health agencies, and federal funding for state Medicaid is expected to be listed in the upcoming budget proposals.
Secretary of Health and Human Services Robert F. Kennedy Jr. comprehensive.
“I have a lot of friends with sickle cells. I’ve seen the pain they endure,” Kennedy said. “There are promising gene therapies right now. They’re very, very expensive, but that’s (National Institutes of Health) Such research should be enthusiastically supported.”
Sean Chow said he thanked the researchers who developed gene therapy and he hoped his son would have a more normal future without debilitating. He hopes other families have the same opportunity to get high-priced gene therapy for their loved ones.
“It’s heartbreaking to have a child with sickle cells,” he said. “I hope that as more and more patients are treated, the cost can be reduced.”
