Sickle Cell Gene Therapy Can Cure, But Costs Almost $3 Million. Who Will Pay?

• May 31, 2023
• Dennis Thompson HealthDay Reporter

An exceptionally pricey gene therapy cure for sickle cell disease could soon be available, but it's not clear whether insurance companies will balk at the cost and deny coverage.

On the surface, the gene therapy does not appear as cost-effective as the grinding medical care that sickle cell patients now receive, according to a new analysis.

Gene therapy applied just once to a sickle cell patient costs as much as $2.8 million, compared to an estimated average cost of about $1.2 million for the lifelong waves of hospitalizations, blood transfusions, and in some cases bone marrow transplants that now constitute standard care, the report says.

However, researchers argue that the cost of gene therapy can be justified if it's weighed against the value of improving the length and quality of life for people living with sickle cell disease, as well as addressing the systemic health inequities these patients face in the United States.

Gene therapy can provide about 10 more healthy years of life for the average patient, the researchers estimated. People with sickle cell disease currently expect only about 30 healthy years, versus 64 years for the average American.

"The therapy is not cost-effective by conventional standards. At the same time, in that context it does give patients an extra 10 years of healthy life, which is obviously huge,"said lead researcher Dr. George Goshua, an assistant professor of hematology with the Yale School of Medicine in New Haven, Conn.

Those extra 10 years would cost about $176,000 per year, which is greater than the standard U.S. cost-effectiveness threshold of $100,000 a year, according to the report.

But if the analysis also weighs the goal of health equity, then the gene therapy could be considered cost-effective for U.S. patients with sickle cell disease, the study authors concluded.

"A lot of people, when they hear the word cost-effectiveness, they just assume that means you're talking about cheapness, how cheap something can be,"Goshua said. "It has nothing to do with cheaper therapy. It all has to do with how much more value we get out of a given therapy per how much more money are we paying for that given therapy."

Sickle cell disease is an inherited disorder, and in the United States most patients are of African ancestry or identify themselves as Black, according to the U.S. National Institutes of Health. About 100,000 people in the United States have sickle cell disease.

Sickle cell disease affects the shape of a person's red blood cells. Normally, these cells are disc-shaped and flexible enough to move easily through blood vessels.

The red blood cells of a person with sickle cell disease are crescent-shaped, resembling a sickle. The cells are stiff and sticky, and cause pain and organ damage when they clump together in different parts of the body.

These problems are caused by a substance called hemoglobin, which is the part of a red blood cell that carries oxygen to tissues throughout your body. A faulty gene causes the body to produce defective hemoglobin that distorts the shape of the blood cells.

In the gene therapy, stem cells are removed from a person's blood-producing bone marrow. Lab technicians expose them to a virus that inserts into them a healthy copy of the faulty hemoglobin gene.

While this takes place, the patient's remaining bone marrow is killed off with chemotherapy. The lab-repaired stem cells are then implanted and start producing healthy hemoglobin.

"This is kind of like doing a bone marrow transplant into yourself," said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America.

The U.S. Food and Drug Administration is expected to consider approval of the gene therapy, which is called LentiGlobin, by the end of the year, Goshua said.

The projected cost of LentiGlobin is in line with other gene therapies that offer a one-time cure, which run between $2 million and $3.5 million, he added.

Based on this study, it is imperative that the therapy's developer, Bluebird Bio, make a strong case to insurance companies for coverage based on health equity, said Richard Cookson, co-director of the Equity in Health Policy research group at the University of York in the United Kingdom.

"Companies seeking an 'equity premium' for their products will need to demonstrate impact on reducing health inequality by providing rigorous evidence on distributional cost-effectiveness,"Cookson said. "Merely talking about equity, fairness and justice is not enough. As the old saying goes: 'In God we trust, all others must bring data.'"

Such analysis should include what having sickle cell disease costs a patient throughout their life, Hsu said.

"Having sickle cell disease puts a drag on your whole life, basically with absences, with impaired productivity, with making the whole family's schedule be constrained by what's happening with the sick person,"Hsu said. "That goes on and on and on, and those sorts of things haven't been calculated very well."

Hsu said other conditions also create these sort of "opportunity costs."He gave the example of Alzheimer's disease, which requires constant caregiving for the patient.

"Having somebody with any kind of chronic disease has much more burden than what the person sitting in the medical insurance office or the economics office can see,"Hsu said.

However, both Goshua and Hsu said they are doubtful whether health equity will weigh heavily in the decision whether or not to cover gene therapy for sickle cell disease.

"I have a big question mark in my mind with regards to how much they will value the equity component,"Goshua said of insurers. "But the best that we can do is give them those hard metrics, and it's up to them to decide how they want to use it, if at all."

Hsu also expressed "great skepticism about being able to right these health disparities."

"Because sickle cell disease disproportionately affects minority people, not completely but disproportionately, and because it has such an impact on quality of life and productivity, it tends to make people fall into a lower socioeconomic range,"Hsu said.

"It really pulls people down in socioeconomic status and, accordingly, their political clout,"he explained.

At the same time, Hsu said there's great excitement that a gene therapy could soon be available.

"This is culmination of decades of research. Ever since DNA was first discovered practically, sickle cell disease was recognized as a genetic condition,"Hsu said. "It's so, so, so gratifying to see this progress, and yet to have some things potentially taken away on cost concerns is also so frustrating. It's like having the golden ring there but not being able to grab it."

The new study was published online May 30 in the Annals of Internal Medicine.

More information

The U.S. Centers for Disease Control and Prevention has more about sickle cell disease.

SOURCES: George Goshua, MD, assistant professor, hematology, Yale School of Medicine, New Haven, Conn.; Richard Cookson, DPhil, co-director, Equity in Health Policy research group, University of York, United Kingdom; Lewis Hsu, MD, PhD, chief medical officer, Sickle Cell Disease Association of America; Annals of Internal Medicine, May 30, 2023, online

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