People With ALS Deserve Access AND Proof of Efficacy

Dear Advisory Committee Advisors, FDA Staff, and, yes, Brainstorm Cell Therapeutics Management,

As you know from hundreds of other comments, ALS is an awful disease. It's just awful. We lived the nightmare with Mom in 1997. It's not substantially better today.  People with ALS deserve more options than the few they have.

They deserve access to investigational therapies. People with ALS should not have to wait years (literally lifetimes) for successful clinical trials and FDA approvals before they can try experimental treatments that have been proven safe.

Many have recently been convinced that the answer to access is to simply change the adjective from "investigational" therapy to "approved" therapy and let the company sell the therapy as if it had met the evidentiary standards for an approval.  I respectfully disagree.  Companies should provide access, and they have Expanded Access tools that the FDA provides until they establish efficacy.

We run Phase 3 trials for a reason.  If we are not going to believe them, then let's not waste the tremendous amount of time and money and human commitment spent on them. 

The NurOwn (debamestrocel) trial took three years, and it seems the company has then spent the next three years telling us not to pay attention to the trial. Six years. They could have run another Phase 3 trial in the last three years that might have focused on one of their suspected responder groups. Had they done so, we wouldn't be in the pickle that we are all in today.

If the FDA finds that the existing evidence supports approval, fine.

But if we're lowering the bar for Phase 3 evidence so far that we're completely ignoring the facts from that trial, then I think we're taking the idea of regulatory flexibility past its elastic limit. Springs that have been stretched too far don't just bounce right back.

We can't afford to make FDA approvals a charade when it comes to efficacy. 

Access and evidence are both critical, and I appreciate that we have a regulatory system that can and should support both. 

Thank you for listening.