The approval of two gene therapies to treat sickle cell disease has given hope to patients that suffer from the debilitating disease, which overwhelmingly affects Black people and other people of color.
Health officials now face a challenge to find a approach to provide equitable access to the costly treatments.
The crippling episodes of pain from the genetic blood disorder make life unpredictable for patients like Michael Goodwin. Sickle cell has forced him to go away his job and at times taken him away from his family.
“I will be within the hospital 20 days out of the month sometimes,” said the 36-year-old Goodwin. “I have been in hospital lots more as I’ve gotten older, which hurts me, because I actually have a son now and I’m married.”
Still, he’s hesitant to try the brand new one-time gene therapies because they require months of intensive medical preparation, including chemotherapy, to arrange patients’ bone marrow stem cells for extraction and gene editing.
Goodwin also worries about the associated fee. Vertex Pharmaceuticals‘ gene therapy Casgevy lists for $2.2 million, while Bluebird Bio‘s treatment Lyfgenia lists for $3.1 million.
“I do have insurance, but … I have already got medical bills,” he said.
The therapies were cheered as a long-awaited breakthrough to treat the disease once they were approved in December. However the hurdles toward ensuring equitable access, and the dearth of infrastructure in place to manage the nascent treatments, have raised questions on just how many individuals will profit from them.
Blood sample of patient positive
tested for sickle cell.
Kitsawet Saethao | Istock | Getty Images
Goodwin’s hesitancy to pursue treatment is not any surprise to Dr. Julie Kanter, director of the Adult Sickle Clinic on the University of Alabama at Birmingham.
“My guess is even when 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 because the president of the National Alliance of Sickle Cell Centers. “And even that will be an excessive amount of for us to administer right this second.”
Greater than 100,000 Americans have sickle cell disease, based on Centers for Disease Control and Prevention estimates, and between 50% and 60% of them covered are covered by the federal and state insurance program Medicaid.
Kanter said it’ll take time to ramp up capability and to establish facilities across the country to treat patients at scale.
“We actually hope that having the National Alliance of Sickle Cell Centers will allow us to strengthen our centers to generally care higher for people living with this disease, which we have not been capable of do before because the associated fee is an issue,” she said.
High cost brings a recent payment model
As they work out learn how to ramp up treatment capability, state and federal officials are grappling with learn how to provide access to the costly recent treatments for the hundreds of patients covered by the Medicaid safety net program.
“It’s giving us a possibility to reply to folks with medical conditions for which there haven’t been very satisfying treatments. But I feel the immediate consideration is the associated fee may be very high. And state budgets simply cannot manage that on their very own,” said Kate McEvoy, executive director of the National Association of Medicaid Directors.
A University of Washington evaluation found that at a price of $2 million or less, the one-time gene therapy treatments would offer an appropriate value, offsetting the lifetime medical and quality-of-life costs for acute sickle cell patients. Many that suffer from the disease require multiple hospitalizations and blood transfusions, which might leave them unable to work.
However the researchers concluded that a cheaper price closer to $1 million would help ensure greater access.
The Biden administration is launching negotiations in the approaching weeks with Vertex and Bluebird Bio to acquire discounts for state Medicaid plans, with payments linked to patient health outcomes. It is an element of the Centers for Medicare & Medicaid Services’ Cell and Gene Therapy Access Model, which goals to make recent high-priced treatments easier to acquire. Approval of the sickle cell treatments prompted the administration to start implementing the brand new payment demonstration program one yr early, starting in January 2025.
“There are probably about 100 therapies within the pipeline on the FDA in a sophisticated stage of application … so it is a real-time priority by way of developing strategies which are going to undergird Medicaid programs capability to cover the treatments,” said McEvoy.
The direct talks with the sickle cell drugmakers come as large pharmaceutical firms like Merck, Eli Lilly and others are suing the Biden administration over the Inflation Reduction Act Medicare price negotiations, which got underway in February. Those talks could see sharply cheaper price offers on the primary 10 drugs chosen for negotiation.
But on Vertex’s quarterly earnings call this month, executives expressed confidence concerning the negotiation process on this case. They said discussions with individual state Medicaid agencies will help ensure wide access and address long-standing inequities of care within the sickle cell disease community.
“We’re not waiting for the demo before we secure access for patients who’re covered by Medicaid,” Steve Arbuckle, Vertex executive vice chairman and chief operating officer, told analysts. “Should you have a look at the profile of Casgevy, it’s so incredibly strong that actually we’re talking about an outcomes-based agreement which is whether a really, very small variety of patients may not respond.”
Employers are taking note
Private employer health plans are also grappling with learn how to pay for an increasing variety of novel treatments with seven-figure price tags, said Morgan Health CEO Dan Mendelson, whose firm focuses on workplace health programs.
“Many employers have a look at cell and gene therapies, see the prices, and are carving them out of their advantages. They know the therapies are precious,” Mendelson said. But for smaller firms, “one case could exceed the associated fee of insuring a complete population in the middle of a yr and the employer doesn’t even know if the worker goes to stay around.”
Morgan Health is exploring recent risk-sharing payment models that might help small- and medium-sized businesses cover the rising costs of specialty treatments coming onto the market.
Goodwin is roofed under his wife’s employer medical insurance. He hasn’t explored what sort of coverage her plan will provide for the brand new sickle cell treatments, because he’s still undecided whether or not they’re right for him.
“In the event that they could guarantee me the consequence — that I would not have sickle cell … I might do it in a heartbeat. In a heartbeat,” he said.
Along with discussions over payments, Vertex and Bluebird Bio are taking steps to coach doctors and patient communities concerning the advantages of their recent treatments.
Vertex expects its first industrial patient to start treatment in the approaching weeks. Bluebird said it anticipated its first patient would start Lyfgenia in the primary quarter.