When Amgen rolled out its Blincyto treatment for an ultra-rare form of leukemia, the California biotech set what seemed to be an eye-watering price--$178,000 for a typical patient. But some number-crunching of clinical trial data, coupled with documents from the Centers for Medicare and Medicaid Services, suggests that the real-world cost will be much lower.
About 60% lower, in fact, according to the analysis from VOI Consulting. The life sciences advisory firm says Blincyto treatment should run about $71,000, on average.
That's because patients aren't likely to get two full treatment courses of 28 days apiece, VOI Consulting President Todd Clark told FiercePharma. In clinical trials, the average time on therapy was 21 days for the first cycle and 10 days for the second--and many patients may not need more than one cycle, Clark said.
|VOI Consulting President Todd Clark|
Some three-fourths of patients in trials received a shorter course of therapy than the 56 necessary to hit that $178,000 price tag, the analysis showed--"even in the clinical trial environment where payment was not an issue and patients could be treated for an extended period of time," Clark noted.
Coupled with information gleaned from Amgen's ($AMGN) interactions with CMS when negotiating for additional Blincyto funding, the days-of-treatment analysis showed that "very few patients will actually be treated" for the full 56 days, Clark said.
Amgen couldn't comment on the VOI Consulting report but said that in clinical trials, the median duration of treatment for patients who responded to Blincyto was two cycles, and 41.6% saw remission within two cycles.
"The price of Blincyto reflects the significant clinical, economic and humanistic value of the product to patients and the healthcare system, for an ultra-orphan population with a serious illness," the company said in an emailed statement. "The price also reflects the complexity of developing, manufacturing and reliably supplying innovative biologic medicines, ongoing investments in additional research in existing and new indications as well continued innovation in the formulations and delivery of our products."
In further digging into Blincyto's costs, Clark said, its list price assumes one vial per day of therapy. But in practice, Medicare contractors are recommending that clinics use just 0.89 vial per day, VOI Consulting found. "That's a relatively small difference, but when a drug is this costly, it adds up quickly," Clark said.
At a time when cancer drugs are increasingly costly, and expensive treatments in other categories are stacking up, payers are likely to seize on any opportunity to work data to their advantage, Clark points out.
And payers' desire to dig into the nitty-gritty on Blincyto spending could soon grow. The drug is now approved specifically for Philadelphia chromosome-negative acute lymphoblastic leukemia (ALL), a rare form of the blood cancer, so its patient population is necessarily limited. About 900 patients in the U.S. would be eligible for the treatment under its current approval, Amgen says. That makes it easier for payers to swallow a big price tag.
But in a recent trial, the drug put a significant number of patients with Philadelphia-positive ALL into complete remission, helping to set the stage for another FDA approval. Blincyto is also under study in chronic lymphocytic leukemia (CLL), B-cell lymphoma, and other blood and marrow cancers. All of that could grow Blincyto's target market significantly, putting a bigger burden on payers at the same time as it puts more oomph in the drug's sales prospects.
- get the full VOI Consulting report
Special Reports: The new drug approvals of 2014 - Blincyto | The top 15 pharma companies by 2014 revenue - Amgen