New myeloma meds offer improvement, but are far too expensive: Report


The Institute for Clinical and Economic Review's new multiple myeloma drug assessment stirred up criticism even before it went public. Now, the not-for-profit group has unveiled its draft report, grading a half-dozen treatment regimens for cost-effectiveness.

The upshot: Newer drug cocktails that include Bristol-Myers Squibb's Empliciti, Amgen's Kyprolis and Takeda's Ninlaro were much less cost-effective than standard treatment with Celgene's Revlimid and dexamethasone, even though side effects tend to be fewer and patients live longer without their cancer progressing.

Amgen struck back at the ICER report last week before its publication, saying ICER's evaluation model did not include all the relevant data. The group hasn't shared enough information about its process to give it weight in decision-making, the drugmaker said, nor does it consider the "social value" of treatments.

The very idea of cost-effectiveness ratings is new in the United States, and ICER is a self-appointed judge rather than an official arbiter like the National Institute for Health and Care Excellence in the U.K. But as the only group applying cost-effectiveness measures to U.S.-marketed meds, ICER and its assessments are attracting quite a bit of attention.

In this report, the newer drugs are judged as significantly less cost-effective than standard Revlimid therapy. In patients who'd failed on one previous treatment, the premium for the newer cocktails over the Revlimid regimen ranged from $255,000 (Empiciti) to $391,000 (Ninlaro) per quality-adjusted life year (QALY), a common measure of cost-effectiveness. The group calculated similar differences in patients on their third round of therapy.

The only cocktail that won backing for increased effectiveness and more reasonable costs used Novartis' Farydak alongside Janssen and Takeda's Velcade. The lower cost--$46,925 per QALY--primarily depended on the fact that Velcade is less expensive than Revlimid. Treatment with Farydak-plus-Velcade alongside dexamethasone also was "time-limited" rather than open-ended as with other meds given until cancer progresses. But ICER also noted that the evidence backing this cocktail is less solid than the data behind the other treatment regimens.

"[T]he introduction of newer regimens for second- and third-line use in multiple myeloma appears to confer clinical benefits in terms of lengthening progression-free and overall survival as well as improved quality of life," ICER concluded. "However, at current wholesale acquisition costs, the estimated cost-effectiveness of these regimens exceeds commonly-cited thresholds."

Amgen says that ICER's conclusions about Kyprolis peg its cost-per-QALY far higher than Amgen's own economic model does, mostly because the nonprofit group's survival estimates were too low. Amgen says Kyprolis delivered total survival benefits of 7.83 life-years rather than ICER's range of 1.17 to 4.7.

The group uses "opaque methods" to combine data from various trials and makes assumptions that create "unrealistic 'worst case' scenarios," Amgen said in its statement.

ICER Communications Director Mitchell Stein told FiercePharma that the institute shares its methods   publicly. "We release all our drafts and final reports as public document, and we file our research plans on the Open Science site," Stein said via email. "Or to put it another way, we release reams of paper on our work."

Amgen isn't the only ICER critic. Robert Goldberg, founder of a think tank that's partly backed by pharma funding, agreed with Amgen. At his DrugWonks blog, Goldberg accused the group of cherry-picking data and using "a statistical magic trick" to turn different patients in different clinical trials "into carbon copies of each other," in a disease that's highly heterogeneous.

Then again, Goldberg also contended that NICE, a public U.K. agency that has been assessing drugs for many years, does much the same thing, a contention many health officials in the U.K. would dispute. "It accepts what it wants and rejects what it wants," Goldberg wrote.

Goldberg also accused ICER of failing to publish in academic journals. But ICER's Stein sent FiercePharma a list of journal publications, including a piece in JAMA Internal Medicine about the cost-effectiveness of hepatitis C drugs.

ICER's previous assessments haven't been all bad news for pharma. The group decided that Novartis had priced its new heart failure drug Entresto in line with its benefits at about $3,779 per year. And after assessing Novo Nordisk's new longer-acting insulin Tresiba, it called that med cost-effective at its current price.

GlaxoSmithKline's new asthma drug Nucala, along with PCSK9 drugs from Amgen and Sanofi/Regeneron, weren't so lucky. GSK's med should be discounted by more than 60% to reflect its value, ICER said, while Repatha and Praluent should cost up to 85% less.

GSK said it disagreed with ICER's conclusion at the time. "GSK believes Nucala offers value to healthcare systems and payers," spokesman Juan Carlos Molina told FiercePharma in a statement. "It has been fairly priced, balancing the innovation of the medicine and market value with patient access, while delivering real benefit to an underserved patient group."

- see the ICER report draft
- get Amgen's statement
- read the Center for Medicine in the Public Interest blog post

Editor's note: This story was updated with comments from ICER.