Pfizer lashes out at ‘frustrating’ NICE decision on ALL drug Besponsa

Pfizer’s new targeted drug for the treatment of the rare but deadly acute lymphoblastic leukemia (ALL) won FDA approval last week after already getting a nod by the EU. But U.K. cost watchdog today snubbed Besponsa, eliciting a quick and heated response from the U.S. drugmaker.

The National Institute of Health and Care Excellence (NICE)  in final guidance today said the drug was not cost effective because it didn’t show an overall survival benefit compared to standard chemotherapy. The panel did acknowledge more patients taking Besponsa were able to have a stem cell transplant for their condition than those getting chemo.

"Today’s frustrating decision for inotuzumab ozogamicin is another example of how NICE is not appropriately assessing the value of modern cancer medicines, leaving patients without access to new treatments that could transform their lives," Pfizer said in a statement. "We strongly believe this is an important and effective medicine which should be routinely available to patients and we will be appealing this decision."

While Pfizer estimated that the drug would cost around £46,000 (about $59,372) per Quality Adjusted Life Year (QALY) gained, NICE said its calculations found it would be "substantially higher" than £50,000 QALY threshold that would make it acceptable, even after Pfizer discounted the list price of £8,048 per 1 mg vial.

In the U.S., Pfizer has said that based on the typical duration of treatment, the total cost of the drug will be $168,300, before discounts.

Besponsa advocate Professor David Marks, who was also quoted in the Pfizer statement, said he was particularly offended by NICE’s cost determination. That was because NICE relied on data from independent drug cost evaluator the Institute for Clinical and Economic Review (ICER). Marks is with the department of hematology and BMT at University Hospitals Bristol NHS Foundation Trust.

"Of specific concern in the final appraisal determination for inotuzumab ozogamicin is a gross misrepresentation of clinical practice in the U.K.," Marks declared. " The ICER has been calculated incorrectly, based on clinicians using six courses of the drug. This is totally wrong. No patient in the U.K. will require more than two to three courses of the drug before a potentially curative transplant. Despite NICE being informed about this, it appears not to have been taken into account."

NICE often turns down new pricey drugs, approving them only after drugmakers come back with deeper discounts. But its practices have led to some drugmakers complaining loudly about the cost overlord’s treatment of patients. Last year Eisai even threatened litigation when NICE left its thyroid cancer drug Lenvima off its revamped Cancer Drugs Fund (CDF) and delayed reconsideration for a year.