Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country鈥檚 first gene therapy, Novartis鈥 Kymriah, a leukemia treatment .
But the total costs of Kymriah and the in development 鈥 known as CAR T-cell therapies 鈥 will be far higher than many have imagined, reaching $1 million or more per patient, according to leading cancer experts. The next CAR T-cell drug could be approved as soon as November.
Although Kymriah鈥檚 price tag has 鈥渟hattered oncology drug pricing norms,鈥 said Leonard Saltz, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, 鈥渢he sticker price is just the starting point.鈥
These therapies lead to a cascade of costs, propelled by serious side effects that require sophisticated management, Saltz said. For this class of drugs, Saltz advised consumers to 鈥渢hink of the $475,000 as parts, not labor.鈥
Dr. Hagop Kantarjian, a leukemia specialist and professor at the University of Texas MD Anderson Cancer Center, estimates Kymriah鈥檚 total cost could reach $1.5 million.
CAR T-cell therapy is expensive because of the unique way that it works. Doctors harvest patients鈥 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, like nearly half of patients who receive CAR T-cell therapy, developed a life-threatening complication in which his immune system overreacted. He says he feels fortunate to be healthy today. (Courtesy of Dr. Keith Eaton)
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 called in which the immune system overreacts, causing dangerously high fevers and . These patients are typically treated in the intensive care unit. Other include .
The cytokine storm felt like 鈥渢he worst flu of your life,鈥 said Eaton, now 51. His fever spiked so high that a hospital nurse assumed the thermometer was broken. Eaton replied, 鈥淚t鈥檚 not broken. My temperature is too high to register on the thermometer.鈥
Although Eaton recovered, he wasn鈥檛 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.
Kymriah鈥檚 sticker price is especially 鈥渙utrageous鈥 given its relatively low manufacturing costs, said Dr. Walid Gellad, co-director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh.
The gene therapy process used to create Kymriah costs about $15,000, according to a 2012 presentation by Dr. Carl June, who pioneered CAR T-cell research at the University of Pennsylvania. June could not be reached for comment.
To quell unrest about price, Novartis has offered patients and insurers a new twist on the money-back guarantee.
Novartis will charge for the drug only if patients go into within one month of treatment. In a key , 83 percent of the children and young adults treated with Kymriah went into remission within three months. Novartis calls the plan 鈥渙utcomes-based pricing.鈥
Novartis is 鈥渨orking through the specific details鈥 of how the pricing plan will affect the Centers for Medicare & Medicaid Services, which pays for care for many cancer patients, company spokeswoman Julie Masow said. 鈥淭here are many hurdles鈥 to this type of pricing plan but, Masow said, 鈥淣ovartis is committed to making this happen.鈥
Masow said that Kymriah鈥檚 manufacturing costs are much higher than $15,000, although she didn鈥檛 cite a specific dollar amount. She noted that Novartis has invested heavily in the technology, designing 鈥渁n innovative manufacturing facility and process specifically for cellular therapies.鈥
As for Kymriah-related hospital and medication charges, 鈥渃osts will vary from patient to patient and treatment center to treatment center, based on the level of care each patient requires,鈥 Masow said. 鈥淜ymriah is a one-time treatment that has shown remarkable early, deep and durable responses in these children who are very sick and often out of options.鈥
Some doctors said Kymriah, which could be used by about 600 patients a year, offers an incalculable benefit for desperately ill young people. Kymriah is for children and young adults with a type of acute lymphoblastic leukemia and already have been treated with at least two other cancer therapies.
鈥淎 kid鈥檚 life is priceless,鈥 said Dr. Michelle Hermiston, director of pediatric immunotherapy at UCSF Benioff Children鈥檚 Hospital San Francisco. 鈥淎ny given kid has the potential to make financial impacts over a lifetime that far outweigh the cost of their cure. From this perspective, every child in my mind deserves the best curative therapy we can offer.鈥
Other cancer doctors say the Novartis plan is no bargain.
About who go into remission with Kymriah relapse within one year, said Dr. Vinay Prasad, an assistant professor of medicine at Oregon Health & Science University. Many of these patients will need additional treatment, said Prasad, who wrote an editorial about Kymriah鈥檚 price Oct. 4聽in
鈥淚f you鈥檝e paid half a million dollars for drugs and half a million dollars for care, and a year later your cancer is back, is that a good deal?鈥 asked Saltz, who co-wrote a
Dr. Steve Miller, chief medical officer for Express Scripts, a pharmacy benefit manager, said it would be more fair to judge Kymriah鈥檚 success after six months of treatment, rather than one month. Prasad goes even further. He said Novartis should issue refunds for any patient whose leukemia relapses within three years.
A consumer advocate group called 聽also has said that Kymriah costs too much, given that the federal government spent more than聽聽over two decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.
Rep. Lloyd Doggett, D-Texas, to the Medicare program鈥檚 director last month asking for details on how the Novartis payment deal will work.
鈥淎s Big Pharma continues to put price gouging before patient access, companies will point more and more proudly at their pricing agreements,鈥 Doggett wrote. 鈥淏ut taxpayers deserve to know more about how these agreements will work 鈥 whether they will actually save the government money, defray these massive costs, and ensure that they can access life-saving medications.鈥
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