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Novartis Wins Historic FDA Nod, Prices CAR-T For Leukemia at $475,000

Xconomy National — 

Ahead of schedule, Novartis has received the first-ever approval of a genetically modified living cell therapy called CAR-T, which uses a cancer patient’s own immune cells as the medicine. Within days, children and young adults with a severe form of acute lymphoblastic leukemia could receive the treatment at a handful of centers across the U.S.

The CAR-T therapy tisagenlecleucel, now branded Kymriah, will cost $475,000. It is a one-time treatment. Novartis (NYSE: NVS) said it would not charge patients who fail to respond to the treatment within one month. Officials on a conference call Wednesday also said they were open to other “value based arrangements” through negotiations with federal Medicare and state government Medicaid insurance programs. They did not give more details about those potential pricing structures.

Vinay Prasad, a blood cancer specialist at Oregon Health & Science University in Portland who is also a vocal critic of high drug prices, pointed out immediately on Twitter that the one-month cutoff is not the best medical measure of success. “1 month response is not an outcome. 18 (or 24 or 36) [months] free of relapse without allo Tx”—a reference to bone marrow transplant, the only current way to cure this type of leukemia—“should be the reimbursable outcome.”

In clinical studies from Novartis and the University of Pennsylvania, where the treatment was developed, 25 percent of patients who responded initially saw their leukemia come back within six months. The 12-month relapse rate was 36 percent.

On the call, Novartis officials framed the price tag, which does not include associated costs such as hospital stays, clinic visits, and emergency treatment for side effects, as reasonable. “We believe it will support sustainability and access while allowing Novartis a return on its investment,” said Novartis CEO of Oncology Bruno Strigini.

How insurance companies react to the cost, and whether they can shoulder it all at once, remains to be seen. Novartis said it would help patients without adequate insurance, or any at all, and allay related costs by paying for patients’ travel and board when getting treatment. “We’re committed to do everything we can within the constraints of the system,” said the firm’s U.S. oncology head Bill Hinshaw.

While many in the healthcare field have called for more creative price structures to alleviate the high cost of drugs, schemes based on a patient’s health outcome have not gotten much traction. Praise for the approval today was tempered with caution about CAR-T safety, because side effects could severely limit the promising field, and affordability.

“Many questions must be addressed before we can herald immunotherapeutic approaches to cancer an unqualified success,” wrote National Institutes of Health director Francis Collins in a blog post Wednesday. “There are still too many severe reactions, too many non-responses or relapses, and, potentially, a very high price tag for their widespread use, which will be truly challenging to scale up. But we’re off to a promising start.”

David Maloney, the medical director of cellular immunotherapy at the Fred Hutchinson Cancer Research Center in Seattle, called the FDA approval a “milestone” but issued a statement calling for “an increase in industry partnerships, more corporate investments and continued government funding” to help improve the safety, availability, and affordability of CAR-T treatments. The Hutch, as it is often called, is responsible for other CAR-T treatments currently in human studies but not yet approved. Its main corporate partner is Juno Therapeutics (NASDAQ: JUNO).

A major pricing question around CAR-T for blood cancers is whether they will provide a cure or simply beat back cancer enough for a patient to get a bone marrow transplant. CAR-T as cure—and an alternative to transplant—would be worth more. Novartis said today it priced tisagenlecleucel below the price range of a transplant and associated one-year costs in the U.S., $540,000 to $800,000. Although a transplant often provides a cure, it is both expensive and risky, with higher death rates than experimental CAR-T treatments.

The U.K. national health service’s price watchdog issued a report in 2015 that gave a rough estimate, with many caveats, that the relative value of CAR-T as cure was worth one-third more than CAR-T as a bridge to transplant.

Pediatric ALL is a rare disease. The Novartis product is only approved for the most desperate cases that have relapsed or failed to respond to several other treatments, including a transplant. Novartis’s Strigini said today Novartis expects about 600 patients a year to be eligible for tisagenlecleucel. The company is working to train staff at medical centers around the country, and it hopes to have 32 to 35 treatment centers ready by the end of the year, Strigini said. That’s no small feat: CAR-T is a complex process with potentially severe side effects that require a range of medical expertise on hand.

The tisagenlecleucel approval is not a surprise. Less than two months ago, a panel of advisors gave a unanimous recommendation that the FDA approve the drug.

An FDA decision wasn’t expected so soon, however. It also comes two days after Gilead Sciences (NASDAQ: GILD) announced an $11.9 billion acquisition of CAR-T developer Kite Pharma (NASDAQ: KITE). Kite’s lead product, for adults with a severe form of non-Hodgkin lymphoma (NHL), is also under FDA review. A decision is expected by the end of November but, given the promising data Kite has produced, approval could come sooner.

The FDA under new commissioner Scott Gottlieb has been approving drugs at an unprecedented rate in 2017. Novartis is also vying to have tisagenlecleucel approved in the same adult NHL population. It will submit its formal request for approval to the FDA by the end of this year, officials said today. It will also ask European regulators to consider tisagenlecleucel for pediatric ALL and adult NHL by the end of the year.

T cell image by NIAID via Creative Commons license.