‘Jerking families around’: Canceled Roche rare disease trial devastates parents, angers researchers By Elizabeth Cohen Aug. 6, 2024

https://www.statnews.com/2024/08/06/roche-basmisanil-rare-disease-dup15q-syndrome-trial-canceled/?utm_campaign=morning_rounds&utm_medium=email&_hsenc=p2ANqtz--U_ZjNYU_PDz2_9LPdAA_LJYr3bji9DlneTbvGOJZjAZBj1LZzqatGE97CzJlljaPJby0HKhGLHLTkyn47e18XJgFSHQ&_hsmi=318823117&utm_content=318823117&utm_source=hs_email Logan Sadecki received his first dose of a potential treatment for dup15q syndrome on Nov. 1. At 10 years old, he’s nonverbal and has autism. His mother, Shannon, remembers how he was poked and prodded and often had to be held down to get his blood drawn during the clinical trial, run by the pharmaceutical company Roche. It would have been worth it, she said, to learn if the new drug could help her son. When she found out the trial was canceled, she felt like she’d been “hit by a bus.” Pharmaceutical companies are businesses, and it’s normal for them to shift priorities in the name of profit. Drugs in development for rare diseases are particularly vulnerable to these cuts, STAT contributor Elizabeth Cohen writes. So it was perfectly legal for Roche to end the trial that Logan and a handful of other children were participating in, but it was unusual “bad behavior,” as one bioethicist put it. Read the STAT+ exclusive story on what happened and how parents and researchers dealt with the fallout.