Will the FDA’s new ALS guidance appease frustrated advocates?

The FDA released a final guidance yesterday on how drug developers should test drugs for treatment of ALS. While “disease specific” guidances, as the FDA calls them, rarely pique the interest of anyone beyond the most nerdy of FDA nerds, this one is a big deal: ALS advocates have been clashing with the FDA for years over the agency's safety standards, and this long-delayed guidance is at the center of that conflict. (In fact, it's this same clash that birthed the "right-to-try" movement and the ALS activist movement now known as "No More Excuses.")

The document is a pretty big win for advocates, many of whom had hoped that the new rules would give drug makers a more efficient path to drug approvals — and get rid of the requirements that some dying patients be given placebos.

The FDA significantly reworked the section about placebos in particular. It now reads: “All patients in ALS trials should receive the best standard of care, and no patient should be denied effective therapies in order to be randomized to a placebo-only arm.”

Stay tuned for what’s next: A group of prominent ALS activists, known as I Am ALS, are set to meet with the FDA later this week. They're the same group that delivered the FDA more than 20,000 petitions in August, urging it to release this guidance as quickly as possible.