This Is What The FDA Heard Yesterday

Below are my comments delivered at yesterday's FDA meeting on their Patient-Focused Drug Development disease areas --

Thank you for the opportunity to comment today. 

We lost Mom to ALS over 15 years ago.

I know that the FDA doesn't need an ALS impact statement. You get it. We are grateful for that.

You also know about the ticking clock that ALS patients hear. It is fast and relentless. Those patients with their terrible ticking clocks can be a special asset to the FDA in patient-focused drug development discussions.

Whether you discuss risks or endpoints or measures directly with people with ALS, the ticking clock is always on the table. That can raise your game, just as speed training strengthens world-class athletes.

People with ALS ask good questions. They challenge us all (to borrow an expression from Apple) to "think different."

• Some may talk of the frustrations of inconsistent FVC measurement.
• Some may speak of those "last times" dates that they use to index progression - The last time one could turn the key in a lock, button a button, blow out the birthday candles.
• Some may question today's gold-standard -- a functional rating scale that buries possible good outcomes among the bad.
• Some may ask if you are sitting on top of an untapped gold mine of control data… if only they were gathered at clinic visits.

Just the FDA, ALS patients, and the ticking clock in the room, without the background noise of all of the rest of us stakeholders. Please consider the value that they will add to your insights. Please include their "speed training" as just five percent of your disease area discussions.

   

I leave you with a quote from Mom -  

"If you can't cure me, then at least learn from me."

Thank you very much.