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Cascade of Costs Could Push Gene Therapy Over $1 Million Per Patient

Sophisticated management of life-threatening complications could double cost of care

Outrage over the cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the  first U.S. gene therapy, tisagenlecleucel (Kymriah, Novartis), a leukemia treatment approved in August. But total costs for tisagenlecleucel and 21 similar drugs in development—known as CAR T-cell therapies—will be far higher than many imagine, reaching $1 million or more per patient, leading cancer experts say.

Although the tisagenlecleucel price tag has “shattered oncology drug pricing norms,” said Leonard Saltz, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, “the sticker price is just the starting point.” These therapies lead to a cascade of costs, propelled by serious side effects that require sophisticated management, Saltz said. For such drugs, Saltz advised consumers to “think of the $475,000 as parts, not labor.”

Dr. Hagop Kantarjian, a leukemia specialist and professor at the University of Texas MD Anderson Cancer Center, estimates tisagenlecleucel’s total cost could reach $1.5 million.

The next CAR T-cell drug could be approved as soon as November. Such therapy is expensive because of the unique way that it works. Doctors harvest patients’ immune cells, genetically alter them to rev up their ability to fight cancer, then reinfuse them into patients.

Taking the brakes off the immune system, Kantarjian said, can lead to life-threatening complications that require lengthy hospitalizations and expensive medications, which are prescribed in addition to conventional cancer therapy, rather than in place of it.

Dr. Keith Eaton, a Seattle oncologist, said he ran up medical bills of $500,000 when he participated in a clinical trial of CAR T cells in 2013, even though all patients in the study received the medication for free. Eaton, who suffered from leukemia, spent nearly two months in the hospital.

Like Eaton, nearly half of patients who receive CAR T cells develop a severe or life-threatening complication called “cytokine storm,” in which the immune system overreacts, causing dangerously high fevers and sudden drops in blood pressure. These patients are typically treated in the intensive care unit. Other serious side effects include stroke-like symptoms and coma.

Although Eaton recovered, he wasn’t done with treatment. His doctors recommended a bone-marrow transplant, another harrowing procedure, at a cost of hundreds of thousands of dollars. Eaton said he feels fortunate to be healthy today, with tests showing no evidence of leukemia. His insurer paid for almost everything.

The debate about tisagenlecleucel’s pricing is far from over. Some doctors say the therapy, which could be used by about 600 patients a year, offers an incalculable benefit for desperately ill young people. Other cancer doctors, however, say the treatment’s price is no bargain because about 36% of patients who go into remission with tisagenlecleucel relapse within one year; many of them will need additional treatment.

Novartis has addressed the cost issue by introducing an “outcomes-based pricing” plan, which will require payment for the drug only if patients go into remission within one month of treatment. The company is still “working through the specific details” of the plan.

Source: Kaiser Health News; October 17, 2017.

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