- A new cell therapy for multiple myeloma likely provides "small to substantial" health benefits over commonly used drugs for patients sick with the blood cancer, but is priced too high to be cost-effective, according to the Institute for Clinical and Economic Review, an influential non-profit that reviews new drugs.
- The therapy, approved last month by the Food and Drug Administration and sold as Abecma, was developed by Bristol Myers Squibb and Bluebird bio. The companies set a list price of $419,500 for an infusion of the treatment, which is made from a patient's own immune cells and can be powerfully effective in some.
- ICER, which has become an important voice in the debate over the cost of drugs, also reviewed a similar cell therapy from Johnson & Johnson and Legend Biotech that's now under review at the FDA, as well as an antibody drug from GlaxoSmithKline. All three are part of a new class of multiple myeloma treatments that target a protein called BCMA.
ICER's report offers a relatively strong endorsement for CAR-T therapy in multiple myeloma, a disease that can be readily treated with existing drugs but remains incurable in the long term.
The group rated both Abecma as well as J&J and Legend's experimental cilta-cel treatment with a "B+" grade, meaning there is "high certainty" that both "provide at least a small net health benefit compared to usual care, with the possibility of a substantial benefit."
In clinical trials, most patients had some response to treatment, with between a third to half of study participants put into remission. Follow-up is relatively short in each case, but preliminary data indicate treatment held patients' disease in check for a median of two to three times as long as would be expected with usual care, according to ICER's review.
"Data are extremely limited for the two CAR-Ts, but those data suggest the potential for patients to live longer with manageable side effects," said ICER President Steve Pearson in a statement on the report.
In theory, CAR-T treatment may in some cases be considered a one-time infusion. The therapies consist of immune cells extracted from each patient, genetically engineered in a lab to target a specific cancer-linked protein, and then re-infused into the body, where they multiply and seek out tumors.
That potential for definitive benefit is one factor CAR-T developers have cited in setting high prices for the therapies, which, due to their personalized nature, are also costly and time-consuming to manufacture.
But in reality, treatment doesn't work equally well for everyone and, in some cases, patients have received a second dose of CAR-T therapy. In its report, ICER noted how about a fifth of patients treated in Bristol Myers and Bluebird's study received a second infusion after their disease progressed.
It was not clear to ICER whether a second dose would cost the same amount as the first, a variable that could have "significant impact" on the price that could be charged and still meet the group's benchmarks for cost-effectiveness.
At the current price of $419,500 for a single infusion of Abecma, and assuming a second charge, ICER recommended a discount of between 37% and 54% to bring its cost under the groups' common cost-effectiveness thresholds.
In a statement, Bristol Myers said it takes "great care" in pricing its medicines based on their value, the scientific research and investment needed to develop them as well as other factors affecting patient access.
"ICER's assessment of ide-cel was, in our view, limited by criteria that disregarded these broader dynamics and their impacts on price," the company added, using a short-hand name for Abecma.
For J&J and Legend's treatment, called cilta-cel, ICER calculated a higher preliminary cost-effective price, but noted that as data are still maturing its finding is likely "optimistic."
ICER gave a more lukewarm review to GlaxoSmithKline's antibody treatment Blenrep, concluding the available evidence was "promising but inconclusive," despite objections from the British drugmaker to its methods. ICER did find the drug's price of $8,277 per vial was cost-effective without any further discount.
Multiple myeloma is relatively rare, with an estimate 140,000 Americans living with the disease. All three treatments are for people who received several previous drugs for their cancer. ICER therefore calculated roughly 10,500 multiple myeloma patients per year would be eligible for CAR-T treatment in the U.S., and about 5,000 for Blenrep.
ICER's report is the result of an assessment process that began in August last year and produced a draft version in February. Bristol Myers and GlaxoSmithKline both submitted feedback on the draft, as did Sanofi and Amgen, which both have currently approved multiple myeloma therapies.
An independent review committee set up by ICER will discuss the report at an April meeting, after which ICER will issue a final report.
Note: This story has been updated to include comment from Bristol Myers Squibb.