Critics say U.K. decision on ALS drug could have ‘chilling effect’ on access to new genetic medicines Andrew Joseph By Andrew Joseph April 1, 2024

https://www.statnews.com/2024/04/01/als-nice-tofersen-biogen/?utm_campaign=morning_rounds&utm_medium=email&_hsmi=300634896&_hsenc=p2ANqtz-_CFTtBsNlBhaGXvCLi_9TKcDIy2aCEDgOJzSb4Sky4blO0nzJsxJtPZzdMoKf_LNFNAyZlILjVw0EDpNrSGHqfWJzRPQ&utm_content=300634896&utm_source=hs_email There’s a dispute in the U.K. right now over access to an ALS treatment available in the U.S. and headed that way in the European Union that has been stalled by the U.K.’s cost-effectiveness agency. The medicine is Biogen’s tofersen, which treats a rare genetic form of the neurodegenerative disease, one that affects about 100 people in the U.K. Instead of evaluating tofersen as a treatment for a rare disease, the National Institute for Health and Care Excellence, or NICE, is looking at it as a medicine for ALS as a whole. That worries not only neurologists and patient advocates, but also other critics who note that more and more new medicines target subgroups of patients with a disease based on underlying genetic mutations. “This just completely flies in the face of what the biomedical community — academics and industry — are moving towards,” said Brian Dickie of the Motor Neurone Disease Association. STAT’s Andrew Joseph explains.