Pricey new gene therapies for sickle cell pose access test

The first gene therapies for sickle cell disease may treat the inherited blood condition for many years, if not a lifetime. They will also cost several million dollars.

On Friday, Vertex Pharmaceuticals set the list price of Casgevy, the first CRISPR-based therapy approved by the Food and Drug Administration, at $2.2 million dollars. Bluebird bio, which also received FDA clearance for its genetic medicine Lyfgenia the same day, chose a higher price of $3.1 million.

The twin approvals make for a momentous day for sickle cell patients, who have had few treatment options to battle a disease that drugmakers long neglected in their hunt of new medicines. In testing, both Casgevy and Lyfgenia eliminated sickle cell’s characteristic pain crises, which can cause serious health problems, including death.

“This is thrilling,” said Alexis Thompson, hematology division chief at the Children’s Hospital of Philadelphia, in an interview Friday. “It could not be a more exciting day to not only have one, but two options to be considered.”

But the seven-figure price tags immediately raise questions about access, particularly for a patient population that’s regularly faced discrimination and barriers to care. While similar to the cost of gene therapies for other rare diseases, Casgevy and Lyfgenia are now among the most expensive medicines on the market.

Bluebird, in a statement, said it settled on Lyfgenia’s price “in recognition of the value the therapy may deliver through robust and sustained clinical benefits.” The company also cited the “lifetime impact” that reducing or eliminating pain crises could have on healthcare costs.

Those costs can be substantial. Pain crises can be serious enough to warrant long stays in the hospital, and the damage they wreak on the body leads to bone and organ complications. Both Bluebird and Vertex estimate the cost of managing sickle cell over a lifetime for someone with recurrent pain crises is between $4 million and $6 million.

To some insurers, preventing the regular hospital visits and other negative consequences associated with pain crises could be a compelling proposition. Bluebird and Vertex also say they are exploring “outcomes-based” contracts that link reimbursement to how well patients do on treatment.

However, many of the approximately 16,000 people Vertex estimates are eligible for Casgevy in the U.S. are covered by Medicaid. The government insurance program is limited in its ability to strike such outcomes-based deals by budget and price-reporting rules, although the Biden administration is planning a pilot to develop workarounds.

Bluebird plans to make a contract option available specifically to Medicaid agencies that’s able to tie Lyfgenia’s cost to performance. The biotechnology company said it is in “advanced discussions” with commercial payers and more than 15 state Medicaid agencies.

“We feel very confident in how we priced Lyfgenia,” Tom Klima, Bluebird bio’s chief commercial and operating officer, said in a conference call Friday. “We’ve already been engaged with payers and have not seen any significant pushback.”

Treatment can come with other costs, too. Both Casgevy and Lyfgenia are made from patients’ own stem cells, which are collected and genetically modified in such a way that, when reinfused, they can curb red blood cell sickling. The process is long and intensive, involving testing, a preconditioning chemotherapy regimen and follow-up monitoring. Patients who travel to treatment centers for the therapies may face out-of-pocket expenses, such as for nearby lodging.

“Cost is a worry, [as is] worrying about where to receive treatment,” said Victoria Gray, the first person with sickle cell to receive Casgevy in Vertex’s clinical trial.

“If [Casgevy] was being offered at a hospital I was mistreated at, I wouldn’t have done it,” added Gray, who traveled to Tennessee to enroll in the study. “People may feel more comfortable doing it outside of town rather than at their usual place where they have been judged.”

Vertex said they have nine treatment centers across seven states and Washington, D.C., ready to offer Casgevy, while Bluebird said there are 27 sites qualified to provide Lyfgenia once it’s avalible early next year.

The preparatory chemotherapy regimen can also cause infertility. While Vertex plans to offer fertility support to commercially insured patients, a spokesperson said the company is unable to do the same for people in Medicaid. Freezing eggs and sperm can cost thousands of dollars, and in vitro fertilization even more.

Such hurdles may slow uptake of both therapies. Additionally, Lyfgenia comes with an FDA warning about the risk of blood cancer, and as a result requires blood monitoring every six months.

“The dramatic difference in pricing, plus a black box warning, in our opinion, likely renders Casgevy the more appealing option for patients,” wrote Dae Gon Ha, an analyst at Stifel, in a note to clients. Analysts at RBC Capital Markets and Mizuho Securities also predicted Casgevy would have faster adoption, for similar reasons.

Shares in Bluebird fell 40% Friday, while Vertex’s stock was down 1%. Shares in CRISPR Therapeutics, which developed Casgevy in partnership with Vertex, slid by 8%.

Gwendolyn Wu contributed reporting

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