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.

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