August 21

Arlene Weintraub

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.”