High Cost of Sickle Cell Cure, Slows Insurance Reimbursement, Delaying Surcease of Sorrow

The high-cost of innovative treatments for dire diseases is largely driven by the high cost of government=mandated Phase 3 clinical trials. For relatively rare diseases, these costs must be spread over fewer patients, making the cost per patient astronomical. If mandates were repealed costs would fall, insurance would be more likely to cover them, and more patients would be cured more quickly.

Note also that one of the pharma firms developing a cure for sickle cell disease is Vertex. Vertex was chronicled in two books by Barry Werth. In the first Vertex was a mission-oriented startup. In the second it was growing by adding marketers, investment experts, legal experts, and regulator-whisperers–seemingly headed toward losing their mission and their passion.

How much of the original spirit of Vertex has managed to survive?

(p. A1) An estimated 100,000 people in the United States, most of them Black, have sickle cell disease.

Gene therapy dangles the prospect of normalcy for the estimated 20,000 people in the United States with the most severe forms of the disease — lives without constant pain and continuing damage to organs and bones and joints.

But all is not well in the world of sickle cell gene therapy.

Last December [2023], the Food and Drug Administration gave approval to two companies, Bluebird Bio of Somerville, Mass., and Vertex Pharmaceuticals of Boston, to sell the first gene therapies approved for sickle cell disease. After nine months, Kendric remains the first Bluebird patient to progress this far, with at least a few others advancing toward his pace.

. . .

(p. A20) “We all expected it to be much faster,” said Dr. Leo Wang of City of Hope Children’s Cancer Center in Los Angeles, which has so far sent cells from one patient to Vertex for the treatment, and is in the final stages of getting authorization from Bluebird.

. . . The treatment is labor intensive, requiring patients to spend at least a month in the hospital. City of Hope can treat at least one patient a month, Dr. Wang said. Other large medical centers said they could treat only 10 or fewer per year, and some say they can treat just five or six.

Then there is insurance. . . .

“Authorization and reimbursement are not the same thing,” said Dr. Stephan Grupp at Children’s Hospital of Philadelphia, which has not yet infused patients with treated cells.

The hospital, he says, has to buy the treatment for $2.2 million per patient from Vertex or $3.1 million from Bluebird. It is reimbursed after the therapy is delivered to the patient. Hospitals get nervous, Dr. Grupp said, because they have to lay out a lot of money. “They want to see that reimbursement happen,” he said.

Some hospitals decided to treat one patient at a time, limiting how much they commit up front.

Making the process even more cumbersome, a hospital has to negotiate separately with each patient’s insurer, said Dr. Julie Kanter of the University of Alabama at Birmingham. Her medical center has not yet started treating patients.

Most hospitals, she said, “don’t want to approve the treatment until they know what the payment plan looks like.”