“This is an initiative the government has taken to responsibly shepherd resources, but at the same time, maintaining access. From a racial equity, health equity perspective, it’s really important that we make this work,” said Krishnamurti, whose sickle cell patients have participated in clinical trials testing gene therapies.

Some states, such as Michigan, are opting to form their own agreements with Bluebird Bio. The company said it’s in talks with Medicaid agencies in 15 other states and is reviewing the CMS framework. Bluebird spokesperson Jess Rowlands said the company looks “forward to working with the agency on an outcomes-based approach that enables access.”

In a statement released last week, Tom Klima, the company’s chief commercial and operating officer, said, “Our commercial approach is built on the principle that people with sickle cell disease insured through Medicaid deserve the same timely access to gene therapy as patients with other forms of insurance.”

In an earnings call last month, Vertex’s chief operating officer Stuart Arbuckle called the new model an “important additional path to access.”

Who is affected?

The majority of sickle cell patients in the U.S. are Black, but people of Hispanic, Middle Eastern and South Asian descent are also disproportionately affected. There are several types of sickle cell disease, but all of them affect hemoglobin, the protein inside red blood cells that carries oxygen. All types of the disease cause the body’s red blood cells to be deformed.

The disease affects an estimated 100,000 Americans. It can cause strokes, severe anemia and episodes of extreme pain, leading to repeated hospitalizations. Those with sickle cell disease have a life span more than two decades shorter than the U.S. average.

The two gene therapy treatments for sickle cell disease recently approved by the FDA, called Casgevy and Lyfgenia, cost $2.2 million and $3.1 million per patient, respectively. The therapies require several other procedures — including chemotherapy prior to the treatments, which involve removing blood cells from a patient and modifying the DNA before re-introducing them in the body.

In Atlanta, Mapillar Dahn’s three daughters have sickle cell disease.

Her oldest daughter, Amatullaah Tyler, who is 20, was hospitalized earlier this month for another pain crisis. At the end of last year, she underwent a hip replacement due to avascular necrosis, which is the death of bone tissue due to lack of blood supply and a complication of the disease.

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