Biogen’s spinal muscular atrophy drug Spinraza is picking up steam in the U.S. and beyond, but in the U.K., cost-effectiveness watchdogs have found it too expensive to endorse.
In just-released draft guidance, NICE experts determined that Spinraza provides a “substantial benefit” for patients with SMA. But they decided not to back the drug for routine use because of its high price and uncertainties about its long-term effectiveness.
In the U.K., the drug would cost £75,000 per vial, according to NICE documents. Biogen proposed a confidential discount, but NICE still couldn’t sign off, the agency said.
NICE is “actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee,” Meindert Boysen, NICE's centre for health technology evaluation director, said in a statement.
After the decision, Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said in a statement the "news will be heartbreaking for the families of those living with the condition."
"Once again we are seeing families suffer due to the appraisal process being too limited to assess costly but life-changing rare disease drugs," he said. "The one glimmer of hope is for a temporary scheme that ensures access."
As of July, Biogen had won reimbursement for Spinraza in 24 countries, according to a recent company presentation. In the second quarter, sales for the rare disease med came in at $423 million; U.S. sales represented $206 million of the total.
All told, more than 5,000 patients were using the drug as of June, including those in an expanded access program, clinical trials, and a postmarketing study.
Spinraza won its first approval back at the end of 2016, and Biogen launched the drug in early 2017 in the U.S. at a cost of $750,000 for the first year of treatment and $375,000 for subsequent years. By the end of the year, Spinraza had reeled in $884 million in global sales.
While the NICE rejection is a setback to Biogen’s Spinraza ambitions in the U.K. and to patients there, the company can take comfort that NICE has historically turned away new, expensive drugs from a range of pharma companies. The agency often rejects a drug on initial review and then endorses it after negotiating bigger discounts or pay-for-performance deals. Roche, Eisai, AstraZeneca and others are among the drugmakers that recently have seen their meds rejected at NICE.
Editor's note: This story was updated with a statement from Muscular Dystrophy UK chief executive Robert Meadowcroft.