New sickle cell gene therapies are a breakthrough, but figuring out how to pay their high prices is a struggle

The approval of two gene therapies to treat sickle cell disease has given hope to patients suffering from the debilitating disease, which overwhelmingly affects blacks and people of color.

Health officials now face the challenge of finding a way to provide equitable access to expensive treatments.

Paralyzing episodes of pain from the genetic blood disorder make life unpredictable for patients like Michael Goodwin. Sickle cell has forced him out of work and at times has taken him away from his family.

“Sometimes I can be in the hospital 20 days a month,” Goodwin, 36, said. “I’ve been in hospital a lot more as I’ve gotten older, which hurts, because now I have a kid and I’m married.”

Still, he’s hesitant to try new gene therapies all at once because they require months of intensive medical preparation, including chemotherapy, to prepare patients’ bone marrow stem cells for extraction and gene editing.

Goodwin also worries about the cost. Vertex PharmaceuticalsGene therapy Casgevy has $2.2 million, while Bluebird BioLyfgenia’s treatment has $3.1 million.

“I have insurance, but I already have medical bills,” she said.

The therapies were hailed as a long-awaited breakthrough in treating the disease when they were approved in December. But obstacles to ensuring equitable access and a lack of infrastructure to administer the nascent treatments have raised questions about how many people will benefit.

Sickle cell positive patient blood sample.

Kitsawet Saethao | Stock | Getty Images

Goodwin’s reluctance to pursue treatment isn’t a surprise to Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham.

“My guess is that even if we opened the doors today to everyone who gets this therapy, at most only 10 percent of individuals affected by sickle cell disease would want this therapy,” said Kanter, who also serves as president of the National Alliance of the Sickle. Cellular centers. “And even that would be too much for us to handle at this point.”

More than 100,000 Americans have sickle cell disease, according to estimates from the Centers for Disease Control and Prevention, and 50% to 60% of them are covered by the federal and state Medicaid insurance programs.

Kanter said it will take time to build capacity and set up facilities across the country to treat patients at scale.

“We’re really hoping that having the National Alliance of Sickle Cell Centers will allow us to strengthen our centers to better serve people living with this disease, which we haven’t been able to do before because cost is an issue,” he said. said

The high cost leads to a new payment model

As they figure out how to increase treatment capacity, state and federal officials are scrambling to give access to expensive new treatments to the thousands of patients covered by the Medicaid safety net program.

“It’s giving us an opportunity to respond to people with medical conditions for which there haven’t been very successful treatments. But I think the immediate consideration is that the cost is very high. And state budgets just can’t handle it alone,” said Kate. McEvoy, executive director of the National Association of Medicaid Directors.

A University of Washington analysis found that at a cost of $2 million or less, single gene therapy treatments would provide acceptable value, offsetting the lifetime medical and quality-of-life costs of anemia patients sickle Many who suffer from the disease require multiple hospitalizations and blood transfusions, which can leave them unable to work.

But the researchers concluded that a lower price closer to $1 million would help ensure greater access.

The Biden administration will begin negotiations in the coming weeks with Vertex and Bluebird Bio to obtain rebates for state Medicaid plans, with payments tied to patient health outcomes. It’s part of the Centers for Medicare and Medicaid Services’ Gene and Cell Therapy Access Model, which aims to make it easier to get high-priced new treatments. The approval of the sickle cell treatments prompted the administration to begin implementing the new payment demonstration program a year earlier, starting in January 2025.

“There are probably about 100 therapies in the pipeline at the FDA in an advanced stage of application, so this is a real-time priority in terms of developing strategies that will strengthen the ability of Medicaid programs to cover the treatments,” McEvoy said.

Direct talks with sickle cell drug makers come as big pharma Merck are suing the Biden administration over the Inflation Reduction Act’s Medicare price negotiations, which began in February. These talks could see much lower price offers for the first 10 drugs selected for negotiation.

But on Vertex’s quarterly earnings call this month, executives expressed confidence in the negotiation process in this case. They said discussions with individual state Medicaid agencies will help ensure broad access and address longstanding care disparities in the sickle cell disease community.

“We’re not waiting for demonstration before we guarantee access to patients covered by Medicaid,” Steve Arbuckle, Vertex’s executive vice president and chief operating officer, told analysts. “If you look at Casgevy’s profile, it’s so incredibly strong that we’re really talking about an outcome-based agreement that looks at whether a very, very small number of patients may not respond.”

Employers take note

Private employer health plans are also struggling with how to pay for a growing number of new treatments with seven-figure price tags, said Morgan Health CEO Dan Mendelson, whose company focuses on the programs of occupational health.

“Many entrepreneurs look at cell and gene therapies, see the costs and take their profits out of them. They know the therapies are valuable,” Mendelson said. But for smaller companies, “one case could exceed the cost of insuring an entire population over the course of a year, and the employer doesn’t even know if the employee will stay.”

Morgan Health is exploring new risk-sharing payment models that could help small and medium-sized businesses cover the rising costs of specialty treatments coming to market.

Goodwin is covered by his wife’s employer’s health insurance. He hasn’t explored what kind of coverage his plan will offer for the new sickle cell treatments, because he’s not yet sure if they’re right for him.

“If I could be guaranteed the result that I wouldn’t have sickle cell disease, I would do it in a heartbeat. In a heartbeat,” he said.

In addition to discussions about payments, Vertex and Bluebird Bio are taking steps to educate doctors and patient communities about the benefits of their new treatments.

Vertex expects its first commercial patient to begin treatment in the coming weeks. Bluebird said it expected its first patient to start Lyfgenia in the first trimester.

Correction: Eli Lilly is not suing the Biden administration over the Inflation Reduction Act. An earlier version of this story got it wrong.

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