Biogen’s big breakthrough drug Spinraza runs into a barrier as NICE turns thumbs down on reimbursement

Once again, UK regulators have proven that they are unwilling to pay a heavy price for the breakthrough drugs that the FDA enjoys hustling through to quick approvals.

In this case, the UK’s NICE is at least temporarily closing the door on Spinraza, Biogen’s landmark new therapy for rare cases of SMA, despite discounting the $750,000 US list price for the first year of therapy.

NICE acknowledged that it would only have to foot the bill for a relatively small group of patients and that the drug, nusinersen, had passed muster as a new therapy for a severe unmet medical need. But without a better idea of the longterm impact of the drug on patients, some of whom face a death sentence at the age of 2, it wasn’t possible to conclude that the price was justified.

Meindert Boysen

“The committee was willing to be flexible because of the nature of the condition and the paucity of the evidence, but the very high cost of nusinersen meant it could not recommend the drug as a cost effective use of NHS resources,” noted Meindert Boysen, director of the centre for health technology evaluation at NICE.

But that doesn’t mean the talks are over.

“Nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients. We are actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee, while demonstrating the potential for nusinersen to be considered cost effective and managing the risk to the NHS of allowing access to this treatment.”

Disagreements between biopharma companies and NICE are nothing new. Vertex has employed some of the toughest tactics, singling out the Prime Minister for harsh criticism while castigating NICE’s outmoded “single technology appraisal.”

But NICE seems immune to such confrontations.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
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Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
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Recursion Pharmaceuticals Salt Lake City, UT

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