Wikipedia -WSS

Wikipedia

author：focus 2025-10-01 22:48:17 - browse：54144

A vial of Tofersen injection on the left of its box — The ALS drug tofersen is marketed as Qalsody in the U.S. Biogen

LONDON — Neurologists and patient advocates are up in arms over a policy decision by a U.K. health agency that they say will imperil access to an ALS treatment that’s available in the U.S. and on its way to approval in the European Union.

The medicine in question, Biogen’s tofersen, is designed to treat a rare genetic form of the neurodegenerative disease, accounting for some 2% of all ALS cases. And the issue is how the U.K.’s cost-effectiveness agency has said it would assess the costs and benefits of the therapy.

The agency, the National Institute for Health and Care Excellence, or NICE, has a special pathway for drugs for very rare diseases, which advocates and doctors argue is the route through which tofersen should be reviewed. But NICE has indicated it would review the drug through its standard process, saying it’s because ALS as a whole does not count as a “very rare” disease. That sets a higher bar to clear.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： How to save PrEP access — and even expand it
Next article： Abridge raises $150m as clinical notes automation market heats up

focus

WSS

Bipartisan group of lawmakers warns of a rising Chinese threat to U.S. biotech

Reporting on presidential health from Reagan to Trump and Biden

A new drug could help frostbite victims avoid its ravages

AbbVie CEO Gonzalez to step down after a decade fiercely defending Humira

With trial win, GSK could get blood cancer drug back on market

Medicare Advantage enrollment hits roughly 33.4 million

knowledge

Wikipedia

focus

Medical leaders decry Supreme Court decision on affirmative action

AbbVie CEO Gonzalez to step down after a decade fiercely defending Humira

New air purifier tech could be key to improving indoor air quality

Hospitals are piloting generative AI tools as regulators play catch

In age of alternative facts, a scholarly course on calling out crap

Medicare Advantage enrollment hits roughly 33.4 million

Wildfire smoke exposes gaps in outdoor worker protections

Change Healthcare can notify patients about the breach, HHS says

Medical leaders decry Supreme Court decision on affirmative action

ASCO: Palliative care found as helpful given virtually as in person

explore

ASCO Daily Recap: Safety data from Novartis, survival from Pfizer

Change Healthcare can notify patients about the breach, HHS says

At ASCO, AstraZeneca reports strong data for breast cancer drug

Weight loss drugs aren’t covered by Obamacare. That may change

A new drug could help frostbite victims avoid its ravages

Flagship Pioneering startup Sonata Therapeutics lays off 21

entertainment

focus

hotspot

explore