USA TODAY US Edition

Revolution­ary gene therapy approved for leukemia — at $475,000 fee

New approach harvests cancer patients’ immune cells, but price tag is called excessive

- Liz Szabo

The nation’s first sanctioned gene therapy — approved Wednesday to fight leukemia that resists standard therapies — will cost $475,000 for a one-time treatment, its manufactur­er announced.

The Food and Drug Administra­tion approved the therapy, called Kymriah, in children and young adults with acute lymphoblas­tic leukemia whose disease has come back in spite of previous treatments. These patients typically have a poor prognosis, surviving three to nine months, according to Novartis, which makes the innovative therapy.

The Switzerlan­d-based company said the drug will cost nothing if patients fail to benefit in the first month.

Kymriah treats cancer in an entirely new way. The individual­ized approach involves harvesting cancer patients’ immune cells, geneticall­y engineerin­g them, then returning them to patients’ bodies. The genetic engineerin­g process aims to rev up patients’ immune systems to better fight cancer.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said Scott Gottlieb, the FDA commission­er. “New technologi­es such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractabl­e illnesses.”

Novartis is working with 20 hospitals to provide Kymriah within a month. Eventually, the therapy will be offered at 32 sites, the company said. The first patients could be treated within days. The company is training hospitals and staff to provide the treatment, which can cause a lifethreat­ening immune reaction, as well as long-term complicati­ons.

Novartis said it priced its drug based on several considerat­ions. British health authoritie­s have said a price of $649,000 for a onetime treatment would be justified given Kymriah’s significan­t benefits.

Novartis also considered the cost of bone-marrow transplant­s, which are given to many leukemia patients whose cancer relapses.

Those transplant­s can cost up to $800,000, Novartis said.

An advocacy group called Patients for Affordable Drugs recently met with officials at Novartis to ask it to set a “fair” price for the drug, whose early developmen­t was supported by $200 million in federal research grants.

David Mitchell, a multiple myeloma patient and president of the advocacy group, described the drug ’s price tag as “excessive.”

“Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more,” he said. “The drug pricing system in America is completely broken. Until policy in this country changes, the vicious cycle of pa- tients struggling under high drug prices will continue.”

The FDA also is considerin­g a CAR T-cell therapy from California-based Kite Pharma. Gilead Sciences — which has been criticized for the $84,000 price tag of its hepatitis C drug — announced Monday that it will buy Kite for $11.9 billion.

Novartis officials said they will offer a patient assistance program to help people with out-of-pocket costs.

Experts have noted that hidden costs could further add to patients’ financial burdens.

Beyond the cost of the procedure, patients would need to pay for traditiona­l chemothera­py, which is given before CAR T-cell therapy to improve its odds of success. They would also have to foot the bill for travel and lodging to one of the hospitals equipped to provide the high-tech treatment.

Because patients can develop life-threatenin­g side effects weeks after the procedure, doctors will ask patients to stay within two hours of the hospital for up to a month.

In New York, even budget hotels cost more than $200 a night — an expense not typically covered by insurance.

Patients who develop a dangerous complicati­on, in which the immune system overreacts and attacks vital organs, might need coverage for emergency room care.

“The drug pricing system in America is completely broken. Until policy in this country changes, the vicious cycle of patients struggling under high drug prices will continue.” David Mitchell, president of Patients for Affordable Drugs

Kaiser Health News, a non-profit health newsroom whose stories appear in news outlets nationwide, is an editoriall­y independen­t part of the Kaiser Family Foundation.

 ?? NOVARTIS, VIA BRENT STIRTON, AP ?? Human
T cells belonging to cancer patients arrive at Novartis Pharmaceut­icals’ facility in Morris Plains, N.J.
NOVARTIS, VIA BRENT STIRTON, AP Human T cells belonging to cancer patients arrive at Novartis Pharmaceut­icals’ facility in Morris Plains, N.J.

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