Seeking Clarity and Urgency in the FDA's Action Plan


This morning, we filed comments with the FDA asking for clarity and heightened urgency in the agency’s Action Plan for Rare Degenerative Diseases, including ALS. 

This plan, which is something ALS community worked to include in the ACT for ALS, is a requirement for the FDA to publish a report describing how it will foster the development of new drugs to treat ALS and other neurodegenerative diseases for the next 5 years. While we believe that the efforts described are largely beneficial and should be funded by Congress, the plan would benefit from significant refinement. 

In a letter accompanying our feedback, we laid out three guiding principles to help frame FDA’s work: 

  • Speed is essential. 
  • Accountability drives results. 
  •  Inclusivity builds momentum.

We are thankful the FDA has provided a plan and insights as to how they will support the development of new drugs for ALS and other rare neurodegenerative diseases. However, this 5-year plan does not sufficiently address the needs of someone currently living with ALS. The FDA should prioritize their activities to speed the development of new therapies for ALS. The Plan is also unclear as to who at the FDA is accountable for this effort at and how the FDA will update the public on their goals. 

We look forward to the FDA refining this plan and accelerating the search for treatments and cures for ALS. 

Click here to read our comments in their entirety.