David Mitchell had just finished a five-hour infusion of drugs to keep his multiple myeloma under control when he decided to dash off a letter to Joe Jimenez, CEO of Novartis, requesting a meeting about how the company plans to price tisagenlecleucel (CTL019), its CAR-T leukemia treatment expected to win approval from the FDA in October.
Because the treatment is personalized—immune cells are extracted from patients and engineered to recognize and kill their cancer—its impending approval has sparked concerns about just how expensive it might be.
"I write today to urge you in the strongest possible terms to price your CAR-T drug fairly in light of the fact that U.S. taxpayers invested hundreds of millions of dollars to develop CAR-T before your company became seriously involved," Mitchell wrote in the letter, which was co-signed by two other patients suffering from blood cancers. Mitchell requested a meeting with Jimenez, even offering to bring along two experts in drug pricing: Steven Pearson, president of the Institute for Clinical and Economic Review (ICER), and Aaron Kesselheim, professor at Harvard Medical School and head of its program on regulation, therapeutics and law.
It worked. On Friday afternoon, Mitchell received a note from Jimenez stating he had asked Bill Hinshaw, Novartis’ head of oncology, to schedule the proposed meeting.
In an interview with FiercePharma, Mitchell emphasized that his goal for meeting with Novartis is not to give the company’s executives a hard time for developing a treatment that’s inherently costly to make. "I’m very excited about this new treatment. I have an incurable blood disease," Mitchell says. "This CAR-T will initially be approved for leukemia, but other blood diseases are not far behind. It’s really important. But the question is, what’s the appropriate price? Should the price reflect the fact that taxpayers laid the groundwork?"
Here’s the crux of Mitchell’s argument: Taxpayers invested $200 million in early-stage research on CAR-T science before 2012, when Novartis gained exclusive rights to the treatment that would become tisagenlecleucel. "Novartis only came to the party big time after the promise of the CAR-T treatment had been demonstrated," Mitchell says. "Taxpayers made the long-term investment starting in 1993 and took all the risk until Novartis stepped up."
A spokeswoman for Novartis said in an e-mail to Fierce that the company is working hard to establish the appropriate price for the CAR-T product, "taking into consideration the value that this treatment represents for patients, society and the healthcare system, both near-term and long-term, as well as input from external health economic experts. We recognize our responsibility in bringing this innovative treatment to patients and are committed to doing everything we can to help those who can benefit from CTL019 have access to the therapy."
Novartis has only offered fleeting references to how it might price tisagenlecleucel: In June, R&D head Vas Narasimhan said the company was looking at stem cell transplants—which can cost $800,000—as a model.
Mitchell’s estimate that taxpayers funded $200 million worth of CAR-T research is based on a keyword search on the National Institutes of Health (NIH) website. Novartis’ academic partner, the University of Pennsylvania, got about $17 million of that. The rest went to the National Cancer Institute and more than 60 academic institutions and biotech companies.
Mitchell is well aware that the fruits of a lot of that research went to other companies that are also advancing CAR-T treatments, like Juno Therapeutics and Kite Pharma, but he believes all of the early-stage science that taxpayer money funded fed into the overall success of the new technology, and that all pricing by any company that commercializes it should reflect that, he says.
In addition to the planned meeting at Novartis, Mitchell’s group is placing ads on cancer-related websites, speaking out on social media and running an e-mail campaign directed at the 15,000 people who have signed up on his organization’s website.
The value of Novartis' original deal with Penn was not disclosed, and the company invested an additional $20 million towards a joint CAR-T research center, the spokeswoman says. But Novartis u...
(All Rights Reserved)