Novartis meeting before CAR-T approval leaves concerned about pricing

David Mitchell was pleasantly surprised when Novartis’oncology chief, Bill Hinshaw, agreed his request for a meeting to discuss what a reasonable price might be for tisagenlecleucel (CTL019), its upcoming CAR-T leukemia treatment that's made from the immune cells of individual patients.

The meeting was held just hours before the FDA's surprising early approval of tisagenlecleucel.

Mitchell, who is battling multiple myeloma, believes CAR-T is a genuine breakthrough, but he wanted to urge Novartis executives to consider the fact that taxpayers invested hundreds of millions of dollars in CAR-T before Novartis jumped in to commercialize it.

But after visiting Novartis Tuesday—just hours before the FDA's surprising early approval of tisagenlecleucel—Mitchell walked away disappointed.

Founder of the advocacy group Patients for Affordable Drugs, Mitchell went to the Swiss drugmaker's New Jersey offices with his organization’s executive director, Ben Wakana, and a representative from the Boston-based Institute for Clinical and Economic Review (ICER). Another cancer patient joined by phone. After an hour-long discussion with Hinshaw and four other Novartis executives, Mitchell said the company blamed the healthcare system for patients' rising drug bills.

"Novartis politely explained that drug prices are ‘complex,’ and patient harm from high prices is the fault of the overall health care system, not drug prices." Mitchell told Fierce. 

A spokeswoman for Novartis said the company shares Mitchell's approach to responsible pricing. "We have carefully considered the appropriate price for CTL019, based on the potential value that this treatment represents for patients and the healthcare system, as well as input from external health economic experts."

After the treatment was approved, the company announced its price would be $475,000. Novartis is also planning a performance-based arrangement with the Centers for Medicare and Medicaid Services, and a patient-access program for those who can't get insurance coverage.

Mitchell said Wednesday that he was planning to reach out to Gilead Sciences, which jumped into the CAR-T race when it agreed to buy Kite for $11.9 billion earlier this week. Ultimately, he plans to engage in discussions with Juno Therapeutics, too, which is in late-stage testing of its own CAR-T technology. "We’re focusing on Novartis because it’s first, therefore the pricing decision is imminent," Mitchell says.

Mitchell’s campaign for fair pricing of tisagenlecleucel started on Aug. 17, when he sent a letter to Novartis outlining his group’s findings that taxpayers laid out $200 million starting in 1993 to fund research performed by Novartis’ CAR-T development partner, the University of Pennsylvania, as well as the National Cancer Institute and about 60 other academic research sites and biotech companies.

In addition to reaching out directly to Novartis, his group planned to place ads on cancer websites, and it sent an e-mail blast to 15,000 people—3,455 of whom signed a petition demanding that Novartis set the prices of its cancer drugs fairly. Mitchell brought that petition with him to the company.

The $475,000 price may be viewed by some as conservative, considering that R&D chief Vas Narasimhan did refer recently to stem cell transplants as a model. Those procedures can cost up to $800,000. The Novartis spokeswoman notes that advancing the CAR-T product to market required "substantial, ongoing investment" in clinical trials, as well as "a sophisticated manufacturing site and process for this personalized therapy."

Gilead is also staying mum on its pricing plans for Kite’s lead CAR-T treatment Axi-Cel, which is expected be approved to treat aggressive non-Hodgkin lymphoma at the end of November. During a conference call after the announcement of the acquisition, Gilead COO Kevin Young declined to answer questions about expected pricing, saying only that the company expects "healthy reimbursement" talks with insurers.

Analysts who cover Gilead have put out estimates ranging from $400,000 to $750,000 per patient for Kite’s treatment.

Mitchell says he's concerned about Gilead’s pricing plans because of Sovaldi, its curative hepatitis C treatment, which hit the market at $84,000, only to be met with extreme pushback from pharmacy benefits managers and other payers. "In light of Gilead’s behavior with its hepatitis C drugs, we will definitely reach out to them" on CAR-T, Mitchell says. "An appropriate price should be set in light of taxpayers’ investments."

As for Novartis' $475,000 price tag, Mitchell isn't satisfied. "While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive," he said in an e-mailed statement after the approval.

Mitchell himself is not eligible for CAR-T—the technology has not yet advanced for multiple myeloma—but he’s thrilled to be part of the discussion about how the engineered cells should be priced. In fact, after Novartis opened its doors to him, he got a second invitation from the organizers of next week’s CAR-TCR Summit in Boston, a who’s who of scientists and executives working in the field of cell therapies for cancer. The conference added Mitchell as a speaker.

"Maybe nobody will come to my talk," he says, "but the fact that they put me on the program at the last second is an indication that attention is being paid to the matter of pricing."