Novartis develops a cell therapy for $500,000 per infusion

Is this cell therapy better or equivalent to autologous stem cell infusions, no one knows yet.

New Cancer Treatments Top $500,000 And Raise Daunting Questions About How To Pay07:31

Kymriah, a recently approved CAR-T cell drug, has a price tag of $475,000. (Courtesy Novartis)
Kymriah, a recently approved CAR-T cell drug, has a price tag of $475,000. (Courtesy Novartis)

It’s one of the most effective cancer treatments so far. And one of the most expensive cancer drugs ever.
That’s why cancer specialists are both excited about so-called CAR-T cell therapy — and worried about what it portends for the affordability of cancer care.
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Dr. Peter Bach, who studies drug pricing, says the price tag for two recently approved CAR-T cell drugs — one is $373,000 for a single dose, the other $475,000 — are benchmarks on the road to ever-higher cancer drug price tags.
“The one truth about cancer drugs is that prices are always higher than they were the year before,” says Bach, director of the Drug Pricing Lab at Memorial Sloan-Kettering Cancer Center in New York. “Now, every time we get a new drug that costs more, it signals that you can get away with charging even more.”
The drugs’ sticker price is only the beginning. It doesn’t include pre-treatment tests and monitoring, costly tests such as PET scans to monitor the patients’ response, and sophisticated management of toxic side effects, which sometimes require administration of an antidote that itself costs tens of thousands of dollars a dose.
The total package can easily exceed a half-million dollars, and some estimate it might add up to $750,000 or more. At this rate, if all the patients who might be helped by the new $373,000 drug got it, the national cost could be $5.6 billion — more than the entire health budgets of many nations. And that’s for only about 7,500 patients.
CAR-T cell therapy gives new urgency to a long-simmering question: Is there any ceiling on cancer drug prices?
But there’s a parallel truth about new cancer treatments that dramatically raise the chances of longer life and perhaps even cure. Once they’re approved, desperate patients and eager doctors start lining up to get them. And, understandably, no one wants to say no.
‘A Huge Leap’
“This is a game-changing technology,” says Dr. Jeffrey Barnes of Massachusetts General Hospital, which began moving CAR-T cell therapy from purely research to mainstream status over the past few weeks. The therapy reengineers patients’ own blood cells so they can lead an attack on cancer cells.
“To take someone who has a chance of improvement in their disease of 5 percent and be able to offer them 75 or 80 percent…it’s a huge leap,” Barnes says.
Barnes is alluding to a landmark study on CAR-T cell therapy published last month in the New England Journal of Medicine. Among 101 patients with an advanced blood cancer called B-cell lymphoma who got CAR-T cells, four out of five saw their cancers retreat. That happens in only one in 20 patients who get older treatments.
Most striking, more than half the CAR-T cell patients saw their lymphomas disappear completely. And more than a year later, 42 percent were still apparently cancer-free.
“The idea in theory isn’t just control, it’s permanent control,” Barnes says. “If you can harness this power and these cells can persist, you might be able to take an incurable disease and make it a curable disease.”
“Cure” isn’t a word that cancer specialists use with abandon, especially in talking about patients with advanced disease that’s beyond all other treatment.
No Guarantees
Barbara Kearney, a 71-year-old retired medical technician, reached for that hope. Last month she became the very first Mass. General patient, and one of the first in the nation, to get CAR-T cell therapy outside of a research study. She was diagnosed with B-cell lymphoma in 2006.
She had already gone through four different treatment regimens, including a bone marrow transplant. None of them worked.
When Kearney’s doctor told her about CAR-T cell therapy last fall, she was eager to try it, despite a high probability of serious side effects that can be life-threatening.
“I’m not afraid of dying at all,” she said in an interview in her Dorchester apartment in late November. “My thing is to help people become aware and let them know that, you know, there’s still a chance.”
The MGH doctors extracted cells from Kearney’s bloodstream and sent them to a California lab where technicians did some fancy genetic engineering. It caused a distinctive protein to appear on the surface of the cancerous blood cells. The marker protein acts as a beacon for  immune cells to home in on and destroy the cancer cells. The protein is called a chimeric antigen receptor, or CAR. The immune cells are called T-cells — thus CAR-T cell therapy.
The California lab, owned by a company called Gilead Sciences, grew up millions of Kearney’s custom-made CAR-T cells and shipped them back to Boston. On Dec. 11, MGH doctors dripped a clear liquid containing 160 million CAR-T cells into Kearney’s veins.
For 48 hours nothing happened. Then Kearney developed a fever — a common side-effect that signals the stepped-up immune activity caused by the CAR-T cells. But unfortunately, Barnes says, by the time she got the infusion, her aggressive lymphoma had already invaded her lungs and liver.
The hyperactive immune activity caused Kearney’s blood pressure to fluctuate wildly, damaging her kidneys. She suffered confusion and other neurologic symptoms, as two-thirds of CAR-T patients do. Ultimately she developed multiple organ failure.
“Despite heroic efforts, she passed away on Dec. 29,” Barnes says.
Kearney’s story shows that not even high-priced therapy comes with any guarantees.
Forging Ahead Despite Uncertainties
The Mass. General doctors are disappointed, but not deterred. They’ve done five more CAR-T infusions in the past few weeks. And more last-ditch patients like Kearney are lined up to get CAR-T treatments there, at the Dana-Farber Cancer Institute, and at 15 other hospitals around the nation.
Mara Bloom, director of the Mass. General Cancer Center, says the hospital isn’t sure how CAR-T cell therapy is going to be paid for. Hospital officials are negotiating with insurers on a case-by-case basis. (Kearney’s insurer said it would cover her treatment.) Medicare, the most important cancer payer, must by law cover FDA-approved cancer drugs, but the program has yet to say how much it will pay.
Some hospitals are holding back from treating CAR-T cell patients because of reimbursement uncertainties. But Bloom says “we can’t wait,” given many patients’ deteriorating status.
“I will tell you one thing: This type of price tag is not sustainable for the long term.”

 Mara Bloom, director of the MGH Cancer Center

“I will tell you one thing,” Bloom says. “This type of price tag is not sustainable for the long term. And it is my hope that there will be ways for us to have a better price tag, because this will be very tough.”
Bloom’s fear, widely shared in the cancer care community, is that hospitals may have to say no to CAR-T cell therapy — along with future expensive treatments. In other words, to ration cancer care.
“I hope we never have to go there,” Bloom says.
But a quiet type of rationing already occurs behind the scenes. Dr. Philip Amrein, who has treated cancer patients at MGH for 40 years, says he and his colleagues work hard to get increasingly expensive drugs covered. But, he says, they don’t always succeed.
In those cases, Amrein says, “we may just do without the medicine. Or we may use some other medicine that helps the situation but is not exactly to the point like the expensive medicine might be.”
Charging What The Market Will Bear
“The trajectory of spending on drugs is extremely worrisome,” says Bach, the drug pricing researcher.
“It’s not that the CAR-Ts themselves will break the bank,” he says. “It’s that they are in a pack of new drugs that companies have now realized they can charge half-a-million or a million dollars for. That’s how profit-seeking companies work. That’s not a good thing or a bad thing. That’s capitalism.”
Pharma giant Gilead Sciences snapped up the small company that developed the CAR-T cell treatment Kearney got, paying nearly $12 billion for it. (Eric Risberg/AP)
Pharma giant Gilead Sciences snapped up the small company that developed the CAR-T cell treatment Kearney got, paying nearly $12 billion for it. (Eric Risberg/AP)
Last summer, pharma giant Gilead Sciences snapped up the small company that developed the CAR-T cell treatment Kearney got, paying nearly $12 billion for it. Gilead was in the news four years ago for its decision to charge $1,000 a pill for a drug that cures hepatitis-C — $84,000 for a full course of treatment.
Nathan Kaiser, a Gilead spokesman, writes in an email that its high price for Yescarta, its CAR-T cell drug, is justified because it’s seven times more effective than older treatments for advanced B-cell lymphoma. The price tag was set after doing market research with insurers, government payers and cancer centers, he says.
Asked if the price of Yescarta might come down in the foreseeable future, Kaiser responded: “We do not have a comment at this time.”
Painful Tradeoffs
With CAR-T cell therapy exceeding a half-million dollars — considering the whole package — Bach says the core problem is whether a cancer drug should cost that much.
“That is a half-million dollars being taken away from something else we could be doing with our health care dollars — whether it’s treating people who are addicted to opiates or making sure that patients get their medication for HIV or even more simple things like making sure that kids with asthma have adequate control of it so they can go to school,” Bach says.
The problem could become more acute if Congress cuts back on Medicare, Medicaid and Affordable Care Act insurance subsidies — to offset the tax cuts it just enacted. And it will become still more urgent as researchers devise costly new ways to treat the big cancers, such as those of the lung, breast, prostate and colon. They’re working on that now.
In the meantime, Mass. General expects to do around 10 CAR-T cell infusions a month in 2018 – at a cost of perhaps $60 million to $90 million — while hoping insurers will come up with the money.

This segment aired on January 11, 2018.


Richard Knox Senior Correspondent, CommonHealth
Richard Knox is a senior correspondent for WBUR’s CommonHealth.

Novartis’ just-approved chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah) is going to be introduced on the market at a price of $500,000 for a single infusion, an amount that is within the range anticipated by oncologists and that Novartis characterized as well below a price level that could be justified on cost.

Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy. Each dose is a customized treatment created with a patient’s own T-cells, which are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein—a chimeric antigen receptor or CAR—that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

Novartis estimates that the turnaround time for processing the cells is 22 days, and initially the FDA has approved one manufacturing site for conducting this work: Novartis’ Morris Plains, New Jersey, laboratory. Whereas the time involved in processing is considered critical because of the need for rapid treatment of patients, Novartis has a cryogenic process that enables it to freeze and store samples of patients’ blood cells for processing at more convenient times and earlier during the treatment cycle.

“[Tisagenlecleucel] is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

The price may be high for CAR T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive. When children and young adults have their whole lives at stake, it’s easier to justify the expense of this new treatment.

Without CAR T-cell therapy, the costs of care, including a potential additional bone marrow transplant, are already “outrageously expensive,” Nichols said. “The real question is going to come when this therapy is poised to be expanded into other patients who are older, and providing the chance for 60 or 70 years of [additional] life is not what you’re talking about, and then we’re really going to have some tough value questions that we should be prepared to ask.”

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