Posted by Kaiser Health News–on October 17, 2017
Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country’s first gene therapy, Novartis’s Kymriah, a leukemia treatment approved in August.
But the total costs of Kymriah and the 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, according to leading cancer experts. The next CAR T-cell drug could be approved as soon as November.
Although Kymriah’s 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.”
According to Saltz, the side effects of these therapies add to the cascade of costs with which consumers are confronted because they require sophisticated management. For this class of 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 Kymriah’s total cost could reach $1.5 million.
CAR T-cell therapy is expensive because it requires doctors to harvest patients’ immune cells, genetically alter them to amp up their ability to fight cancer, then reinfuse them into patients.
Kantarjian added that taking the brakes off the immune system 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.
Dr. 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, and they are often faced with serious side effects including stroke-like symptoms and coma.
Eaton, now 51, said the cytokine storm felt like “the worst flu of your life.” His fever spiked so high that a hospital nurse assumed the thermometer was broken, to which he replied, “It’s not broken. My temperature is too high to register on the thermometer.”
Following his recovery, Eaton was recommended for a bone-marrow transplant–another harrowing procedure—which cost hundreds of thousands of dollars. Tests now show no evidence of leukemia. His insurer paid for almost everything.
To subdue unrest about price, Novartis will charge for the drug only if patients go into remission within one month of treatment. In a key clinical trial, 83% of the children and young adults treated with Kymriah went into remission within three months. Novartis calls the plan “outcomes-based pricing.”
Company spokeswoman Julie Masow said, “costs will vary from patient to patient and treatment center to treatment center, based on the level of care each patient requires.” She added that, “Kymriah 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.”
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 approved for children and young adults with a type of acute lymphoblastic leukemia and already have been treated with at least two other cancer therapies.
“A kid’s life is priceless,” said Dr. Michelle Hermiston, director of pediatric immunotherapy at UCSF Benioff Children’s Hospital San Francisco. “Any 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.”
Other cancer doctors say the Novartis plan is no bargain.
About 36 percent of patients 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’s price Oct. 4 in Nature.
“If you’ve 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?” asked Saltz, who co-wrote a recent editorial on Kymriah’s price in JAMA.
I’ve been extremely fortunate to run marathons ranging from the breath-taking beauty of the “Big Sur” in Carmel, CA, to the New York City event, which meanders through all five boroughs. They’re both 26.219 miles long.
And everyone I’ve known who’s ever run a marathon eventually “hits the wall” toward the end. The body is tapped out of the fuel needed to continue running, it starts to feel like jello, and your whole world shrinks to the size of a ping-pong ball.
Boiling it down, the Kaiser report essentially says America is now, or very soon will be, hitting the same type of wall with the cost of immuno-oncology (IO) therapy.
And although the report doesn’t say it explicitly, this nation will not be able to afford to make this wondrous treatment available to everyone. Do the math per Kaiser’s whole cost-of-care calculation–not just the cost of the “medicine,”–and it becomes quite clear.
In simple terms, the dilemma, already faced in other “rich” nations like the UK and Canada, is we all want personalized medicine. But at a total cost-of-care price of around $1.5 million per patient, who is going to pay for it?
As I’ve written before, most insurance companies will only pay for the standard treatments, and they will only consider cellular therapy when all other options fail. Ideally, we use our own immune system to fight cancer, and the therapy would be a one-event cure as opposed to traditional treatments including checkpoint inhibitors, where the patient takes the med for a specific period of time and hopes the cancer is eradicated.
Adding insult to injury, a new study shows that many of these pricey cancer drugs don’t even work.
The payer determines who gets access to these extremely costly “wonder drugs.” That’s because insurance companies will not pay for a new therapy unless it is proven that it works and is in fact a “cure” (equals no relapse after initial remission) by their standards not just the FDA’s. It also must be proven to be safe and save the insurance company money.Steve's Take: We need a national solution to paying for expensive new #genetherapy treatments Click To Tweet
But in order to ascertain this, economic data must be collected for a predetermined period of time not just to demonstrate these factors, but also that the med saves the company money compared to “standard of care” treatment. This can be generic versions of the drug or biosimilar of a biologic drug.
If the insurance company will not cover the ultra-expensive, single-dose therapy, then the patient or family will. Most patients and families cannot afford these potential cures and they instead will be forced to rely upon the standard treatment(s) and cross their fingers.
One intractable dilemma, however, is that as people live longer, they require more health care because of additional diseases or disorders concurrent with a primary disease or disorder like cancer. These comorbidities, as they are known, automatically jack up the cost of care.
To prevent healthcare costs from mushrooming out of control, especially now with CAR-T and other budding programs, the entire US healthcare system has to shift in a fundamental manner not seriously addressed in either Obamacare or the recent GOP proposals to repeal and replace it.
As in the UK and Canada, among other nations, we need to alter our national mindset so everyone unconsciously practices preventive car and takes responsibility for their health, embraces the notion of treating patients with the right drug–with the freedom of advanced technology being covered by insurance.
We’re now “hitting the wall” on the cost issue. Whether it’s a universal payer system, a government-run and -funded system or a hybrid like it is currently, we’re much closer with these new gene therapies to making a national decision which way we’re going.