Pioneering Cancer Drug to Cost $475,000 — and Analysts Say It's a Bargain

 (Victor Segura Ibarra and Rita Serda, NCI, NIH)

The Food and Drug Administration on Wednesday approved a futuristic new approach to treating cancer, clearing a Novartis therapy that has produced unprecedented results in patients with a rare and deadly cancer. The price tag: $475,000 for a course of treatment.

That sounds staggering to many patients — but it’s far less than analysts expected.

The therapy, called a CAR-T, is made by harvesting patients’ white blood cells and rewiring them to home in on tumors. Novartis’s product is the first CAR-T therapy to come before the FDA, leading a pack of novel treatments that promise to change the standard of care for certain aggressive blood cancers.

Novartis’s therapy is approved to treat children and young adults with relapsed acute lymphoblastic leukemia. It will be marketed as Kymriah.

The treatment’s approval has looked a foregone conclusion for months, but its potential price has been the subject of speculation and debate. Novartis picked the $475,000 price tag in an effort to balance patient access to Kymriah while giving the company a return on its investment, said Bruno Strigini, Novartis’s head of oncology, in a conference call Wednesday. The cost is below Wall Street analyst expectations, which reached as high as $750,000 for a dose. And it’s considerably cheaper than the roughly $700,000 price tag that U.K. regulators said would be fair considering Kymriah’s potential benefits.

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Novartis also said it is working with Medicare on a system in which the government would only pay for CAR-T treatment if patients respond within a month.

In a clinical trial, a single dose of Kymriah left 83 percent of participants cancer-free after three months, results oncologists have hailed as a major advance for patients with few other options. The most frequent side effect was an inflammatory storm called cytokine release syndrome, a reaction to CAR-T that can prove fatal in some patients but is commonly controlled with immunosuppressant drugs.

“I think this is most exciting thing I’ve seen in my lifetime,” said Dr. Tim Cripe, an oncologist with Nationwide Children’s Hospital, at an FDA meeting on Kymriah in July.

Unlike well-understood pills and commonly injected biotech drugs, CAR-T presents a radical new paradigm for doctors, regulators, and payers. Each dose is custom-tailored for an individual patient, requiring a complex process in which human cells must be safely ferried across the country, reliably re-engineered, and soundly returned.

That creates logistical issues unseen with previous drugs. To get Kymriah, patients will have to travel to one of just 32 sites around the country. From there, doctors harvest patients’ white blood cells and ship them off to a Novartis facility in New Jersey where they can be edited and mailed back. The entire process takes about 22 days, the company said. And the $450,000 price tag covers only Novartis’s role, not the costs of travel, hospitalization, or any drugs needed to tamp down Kymriah’s side effects.

And it remains unclear just how lucrative a business opportunity Kymriah presents. There are about 3,100 new cases of ALL each year, but roughly 70 percent can be pushed into remission by standard therapy. That could leave just a few hundred patients who might be eligible for Novartis’s therapy, casting doubt on whether the company can get an outsize return on what will be a substantial manufacturing investment.

But CAR-T’s potential goes far beyond Wednesday’s approval.

Novartis is developing Kymriah for use in lymphoma, and its pipeline includes other CAR-T therapies targeting an array of blood cancers. Kite Pharma, soon to be acquired by Gilead Sciences, is awaiting FDA approval for a lymphoma therapy and is, like Novartis, developing a bevy of cell therapies it hopes can treat tumors liquid and solid. Juno Therapeutics, which slipped into a third place after its lead CAR-T ran into safety problems, has a similar focus.

This story was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.

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