Conflicting clinical results from Biogen and Eisai’s closely watched Alzheimer’s disease candidate aducanumab have already fueled plenty of controversies. Now a U.S. drug pricing watchdog has used that confusion to tag the med with a low price—one that Biogen itself probably won’t accept.
Thanks to “insufficient” evidence on aducanumab’s benefits, the drug should cost as low as $2,500 for a year of treatment to be considered cost-effective, the Institute for Clinical and Economic Review (ICER) said in a draft report (PDF) released Wednesday.
Even at the high end of the cost-effective threshold, ICER’s designated price of $8,300 still comes way below what a typical antibody drug would cost on the U.S. market. It’s far less than the $50,000 mark industry watchers have estimated for what could become the first treatment to change the course—rather than just treat the symptoms—of Alzheimer’s.
It also came in contrast with Biogen CEO Michel Vounatsos’ recent comment that his company worked closely with ICER ahead of the group's report. In determining a price for aducanumab, Biogen engaged with pharmacoeconomics bodies including ICER many times, Vounatsos said a few days ago during the company’s first-quarter earnings call, suggesting some kind of a mutual understanding.
In a statement Thursday, Biogen said the draft report “discounts the possible holistic value of aducanumab and does not accurately reflect the potential benefits of treating older adults.” The company stressed that an appropriate pricing assessment would consider “the full clinical benefits and long-term economic value for patients, families, caregivers and society.”
Still, opinions are split on whether aducanumab is indeed a breakthrough drug.
The debate stems from seemingly contradictory trial results from two trials that Biogen stopped after disappointing interim analyses. After picking apart the data, Biogen found that patients with longer exposure to the higher dose of aducanumab did indeed show an improvement in cognitive decline.
The results from the failed trial would have been positive had investigators followed the patients longer instead, Biogen argued. The company also pointed to an imbalance in a small number of patients with rapidly progressing Alzheimer’s to explain the inconsistent readouts.
For their part, ICER reviewers argued in the draft report that Biogen’s post-hoc analyses don’t “consistently explain” results from the low- and high-dose arms of the trials. Existing data couldn’t determine whether Biogen’s explanations are true, or whether the differences between the trial results happened simply by chance, they said.
If considering only the successful trial, ICER figured aducanumab’s annual price tag could rise to between $11,100 to $23,100. ICER is now soliciting public comments on the draft report and plans to post a final version by the end of June, after the FDA’s planned decision date for the drug.
While ICER is flagging concerns about aducanumab's data, the group has faced its share of controversy. ICER has often criticized the launch prices of new pharmaceuticals, but in recent years, it's brought more and more drugmakers to the table for its reviews, an official previously said.
Aducanumab's mixed data raised eyebrows at an external panel the FDA summoned in November to help assess aducanumab’s application. The experts voted “no” to key questions proposed by the FDA, essentially recommending against an approval.
Some of the advisers returned with their disapproval in a recent JAMA article, criticizing Biogen for “inadvertently selecting data precisely because those data were consistent with the outcomes that were hoped for.” They also questioned FDA’s relationship with Biogen.
After a three-month delay, the FDA is scheduled to deliver a verdict on the drug by June 7. Meanwhile, Biogen is launching a phase 3b study dubbed Embark to evaluate the long-term profile of aducanumab in treating patients with early Alzheimer’s who participated in the drug’s earlier trials, including the two controversial ones.
The decision is viewed as a make-or-break moment for Biogen. Sales for the company’s flagship multiple sclerosis therapy Tecfidera fell miserably in the first quarter thanks to generic erosion, and its follow-up drug Vumerity has yet to pick up sales in an increasingly crowded market. Its spinal muscular atrophy blockbuster Spinraza is also declining amid competition from Novartis’ gene therapy Zolgensma and Roche’s newly launched oral therapy Evrysdi.