The two FDA-approved CAR-T cell therapies to treat blood cancer, Novartis’ Kymriah and Gilead’s Yescarta, were both cleared for marketing in Europe last Monday—but Novartis is having a much easier time negotiating a price palatable to the tough cost watchdogs at England's National Institute for Health and Care Excellence (NICE).
NICE last week rejected Yescarta as a treatment of adults with relapsed large B-cell lymphoma, citing a price that’s too high to be cost-effective for the National Health Service (NHS), given a lack of data on the long-term benefits of CAR-T treatments. The agency didn’t disclose the price Gilead was requesting, but in the U.S. Yescarta sells for $373,000.
On Wednesday, NICE and the NHS gave Novartis a very different verdict, greenlighting Kymriah at a list price of £282,000 ($361,000).
Kymriah, a one-time cell therapy that’s made from individual patients’ own immune cells, was recommended in the U.K. to treat children and young adults with B-cell acute lymphoblastic leukemia (ALL) who haven’t responded to other treatments. The negotiated price tag came in below Kymriah’s cost in the U.S., where it’s listed at $475,000 to treat leukemia and at a Gilead-matching $373,000 for adult patients with relapsed diffuse large B cell lymphoma.
Novartis said in a statement that the NICE decision resulted from a “close collaboration” with the agency and the National Health Service of England—a collaboration that's continuing as the company seeks the agency’s approval for lymphoma.
“We trust that the NHS will continue this collaboration and flexibility in granting [Kymriah] access to adult patients with diffuse large B cell lymphoma (DLBCL), the most common non-Hodgkin’s lymphoma, where there is also a high unmet need for patients who have relapsed and have no other treatment options,” the company said.
The NHS said that the deal, just 10 days after Kymriah was approved by the European Medicines Agency, was one of the quickest in NHS’ 70-year history. The “fast-track” decision “shows how responsible and flexible life sciences companies can succeed—in partnership with the NHS—to make revolutionary treatments available to patients,” said Simon Stevens, chief executive of NHS England in an announcement.
If Novartis succeeds in getting its CAR-T into the NHS as a DLBCL treatment, it would pull ahead of Gilead in a key market. Gilead’s Yescarta was approved by the FDA to treat patients with DLBCL in October 2017, while Novartis got the FDA nod for Kymriah in that disease the following May. Reimbursement hurdles have made for a slow launch in both cases, however: Yescarta brought in $68 million in the most recent quarter, and Kymriah sales were just $16 million.
Gilead said it is doing everything it can to reverse NICE’s decision on Yescarta, though. The agency declined even a short-term funding agreement through the U.K.’s Cancer Drugs Fund, but the company continues to negotiate for some sort of payment agreement, a spokesman told FiercePharma last week.
"Cell therapy is a new and advanced technology and, as NICE has identified, there is little available data about the salvage treatment of patients with aggressive forms of non-Hodgkin lymphoma (NHL) in England," the spokesman said. "We’re therefore in ongoing discussions with NICE to identify appropriate treatment comparators which can clarify how cell therapy may be made available to patients in the U.K."
NICE has made a string of decisions lately on pricey new immuno-oncology treatments, all of which point back to a single tough question: Do the long-term benefits of these therapies outweigh the costs? It’s not always easy for pharma companies to provide the data NICE needs to answer the question.
Witness Merck, which failed earlier this week to persuade the agency that the NHS should pay for its PD-1 checkpoint inhibitor, Keytruda, to be used in patients with Hodgkin lymphoma who've failed to respond to a stem cell transplant.
The reason? Merck didn’t adequately explain why Keytruda is more cost-effective than Bristol Myers-Squibb’s PD-1 inhibitor, Opdivo, in that population of patients, NICE determined.