ALS Guidance Embraces Regulatory Flexibility to Speed Treatments

Senior FDA officials promised “flexibility” to help address the unmet medical need as the agency released final guidance for sponsors of treatments for amyotrophic lateral sclerosis (ALS) — Lou Gehrig’s disease.

The agency recognizes that scientific progress has been “particularly lagging” for complex neurological diseases such as ALS, said acting FDA Commissioner Ned Sharpless in a joint comment with CDER Director Janet Woodcock on the release of the final guidance. “[W]e stand ready to use the expedited development and approval programs available to help bring new treatments for ALS to patients as quickly as possible.”

Phase 1 studies can be conducted in healthy volunteers or in ALS patients. The agency also suggests decentralizing trials and broadening inclusion criteria to include as many different types of subjects as possible in trials.