The recent approval of two gene therapies for treating sickle cell disease has provided renewed hope for patients affected by this debilitating condition, which predominantly impacts Black individuals and people of color.
However, health officials are now faced with the challenge of ensuring equitable access to these expensive treatments.
For patients like Michael Goodwin, who suffers from the severe pain and unpredictability caused by sickle cell disease, the new gene therapies represent a potential breakthrough.
Goodwin, who is 36, has experienced significant disruptions in his life due to the disease, including leaving his job and frequent hospitalizations.
“I can be in the hospital 20 days out of the month sometimes,” he shared. “I’ve been in the hospital much more often as I’ve gotten older, which is painful because I have a son now and I’m married.”
Despite the potential benefits, Goodwin is hesitant to pursue the new therapies due to the extensive medical preparation required, including chemotherapy to prepare his bone marrow stem cells for gene editing.
Additionally, he is concerned about the high costs of these treatments, with Vertex Pharmaceuticals’ Casgevy priced at $2.2 million and Bluebird Bio’s Lyfgenia at $3.1 million. “I do have insurance, but … I already have medical bills,” Goodwin said.
The approval of these therapies in December was celebrated as a major advancement, but questions remain about how to ensure that they are accessible to all who need them, given the high costs and the current lack of infrastructure for administering these treatments.
Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham, is not surprised by Goodwin’s reluctance.
“My guess is even if we opened the gates today to everybody getting this therapy, at most only 10% of those individuals affected by sickle cell would want this therapy,” said Kanter, who also serves as the president of the National Alliance of Sickle Cell Centers. “And even that would be too much for us to manage right this second.”
According to the Centers for Disease Control and Prevention, more than 100,000 Americans have sickle cell disease, with 50% to 60% of them covered by Medicaid. Kanter noted that building capacity and establishing treatment facilities across the country will take time.
“We really hope that having the National Alliance of Sickle Cell Centers will allow us to strengthen our centers to generally care better for people living with this disease, which we haven’t been able to do before because the cost is a problem,” she said.
As officials work to expand treatment capacity, they are also grappling with how to provide access to these costly new therapies for the thousands of Medicaid beneficiaries.
“It’s giving us an opportunity to respond to folks with medical conditions for which there have not been very satisfying treatments. But I think the immediate consideration is the cost is very high.
And state budgets simply cannot manage that on their own,” said Kate McEvoy, executive director of the National Association of Medicaid Directors.
A University of Washington analysis found that gene therapy treatments priced at $2 million or less would be considered acceptable, given their potential to offset the lifetime medical and quality-of-life costs associated with sickle cell disease.
Many patients require frequent hospitalizations and blood transfusions, which can impact their ability to work. However, a price closer to $1 million would ensure broader access.
The Biden administration is set to begin negotiations with Vertex and Bluebird Bio to secure discounts for state Medicaid plans, with payments tied to patient health outcomes.
This is part of the Centers for Medicare & Medicaid Services’ Cell and Gene Therapy Access Model, which aims to facilitate access to high-priced treatments.
The approval of the sickle cell therapies has accelerated the implementation of this new payment demonstration program, which will start 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 support Medicaid programs’ capacity to cover the treatments,” McEvoy said.
This initiative comes amid ongoing lawsuits from large pharmaceutical companies, such as Merck, challenging the Biden administration’s Medicare price negotiations under the Inflation Reduction Act. These negotiations could lead to significantly lower prices for the first 10 drugs selected.
During Vertex’s quarterly earnings call, executives expressed optimism about the negotiation process, emphasizing that discussions with state Medicaid agencies would help ensure broad access and address existing disparities in sickle cell care.
“We’re not waiting for the demo before we secure access for patients who are covered by Medicaid,” said Steve Arbuckle, Vertex’s executive vice president and chief operating officer.
“If you look at the profile of Casgevy, it is so incredibly strong that really we’re talking about an outcomes-based agreement, which looks at whether a very small number of patients may not respond.”
Private employer health plans are also considering how to manage the costs associated with these high-priced treatments.
Dan Mendelson, CEO of Morgan Health, which focuses on workplace health programs, noted that many employers are opting to exclude such therapies from their benefits due to the high costs.
For smaller companies, “one case could exceed the cost of insuring an entire population in the course of a year, and the employer doesn’t even know if the employee will stay,” Mendelson said.
Morgan Health is looking through new risk-sharing payment models to help small- and medium-sized businesses manage the rising costs of specialty treatments.
Goodwin, covered under his wife’s employer health insurance, has not yet explored the extent of her plan’s coverage for the new sickle cell treatments, as he remains uncertain about pursuing them.
“If they could guarantee me the outcome — that I wouldn’t have sickle cell … I would do it in a heartbeat. In a heartbeat,” he said.
In addition to payment discussions, Vertex and Bluebird Bio are working to educate doctors and patient communities about the benefits of their new treatments.
Vertex expects to start its first commercial patient in the coming weeks, while Bluebird anticipates initiating its first patient with Lyfgenia in the first quarter.
