• by Thom Forbes  @tforbes, August 31, 2017

In what it calls an “historic action,” the Food and Drug Administration yesterday approved the use of Kymriah (tisagenlecleucel), a cell-based gene therapy for the treatment of patients up to 25 years of age who have an aggressive type of B-cell acute lymphoblastic leukemia (ALL).

Basel, Switzerland-based Novartis, which developed Kymriah — previously known as CLT019 — has put a price of $475,000 on the treatment but is going out of its way to point out that it could charge a lot more, and that few families will bear the brunt of paying for its customized administration.

“We have taken a very responsible approach” to pricing, Bill Hinshaw, Novartis’ U.S. oncology head, tells Denise Roland and Peter Loftus of the Wall Street Journal. 

“He cited independent cost-effectiveness estimates that showed Kymriah could command a price of $600,000 to $750,000. He said that because patients will be children or young adults — who typically fall under their parents’ or caregivers’ private insurance plans, Medicaid or other federal plans aimed specifically at children — few patients would likely end up paying near the full price out of pocket. That cost will fall to the employers, insurers and taxpayer-funded government programs that fund health-care costs,” Roland and Loftus write.

Kymriah “is neither a pill nor an injection, but a personalized medicine service that functions as a ‘living drug.’ Patients would have their body’s own disease-fighting T cells fortified and multiplied in a lab, then get the cells back to help them fight their cancer,” writes Melissa Healy for the Los Angeles Times. “In clinical trials of 88 patients with a relapsing or treatment-resistant form of acute lymphoblastic leukemia, 73 went into remission after receiving the experimental treatment.”

Those tougher cases of the disease previously had low survival rates, Dr. John Cunningham, chairman of the department of pediatrics at University of Chicago Medicine Comer Children's Hospital, tells the Chicago Tribune’s Lisa Schencker. “In my 30 years of being an oncologist, that’s the most dramatic difference I've ever seen,” he says. “I've never seen something so spectacular.”

Kymriah is given just once to each patient “and must be made individually for each” writes Denise Grady for the New York Times. “Novartis said that if a patient does not respond within the first month after treatment, there will be no charge. The company also said it would provide financial help to families who were uninsured or underinsured.”

Bloomberg’s Anna Edney, James Paton and Caroline Chen point out that pricing controversies are not new (even exclusive of Martin Shkreli’s widely covered shenanigans that recently resulted in a conviction for securities fraud in federal court).

“In 2013, Gilead Science Inc. introduced an $84,000 cure for the viral disease hepatitis C, prompting a nationwide debate over drug costs. And new cancer treatments that have greatly extended some patients’ lives can cost $150,000 a year,” they write.

“Yet the almost half-million-dollar price tag on the Novartis CAR-T drug is a new benchmark, and more are likely to follow, with similar new therapies for blindness, blood disorders and other cancers. Spark Therapeutics Inc.’s gene therapy for a genetic disorder that causes childhood blindness is expected to get an agency decision by January.”

Kymriah — thankfully — has a very limited market. 

“It remains unclear as just how lucrative a business opportunity Kymriah presents, whatever the price. There are about 3,100 new cases of ALL each year, but roughly 70% 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,” observes Damian Garde for STAT.

But it’s also a peek at the future of sicknesscare.

“FDA Commissioner Scott Gottlieb, himself a survivor of blood cancer, predicted that this new approach to cancer treatment will ‘change the face of modern medicine,’” reports the LAT’s Healy.

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

“The University of Chicago Medicine is one of a limited number of U.S. sites working to offer [Kymriah],” reads a news release from that institution that goes on to point out that Sam Tinaglia, 18, of nearby Park Ridge, was one of the first 100 children in the country to participate in clinical trials. “In 2015, during his junior year of high school, Sam underwent CAR T-cell therapy at Children’s Hospital of Philadelphia. “Six weeks later, they sent me home and that was pretty much it,” said Sam.

And he, and his family, can’t put a price on that.