If you want to call me a geek, that’s fine but you’re at the end of a long, long queue. I worked out the math for how many pALS would need to participate in a clinical trial to have a valid random sample group. Spoiler alert: Lots.
I won’t bore anyone with the formula, but I’ll come clean on assumptions. I used a rough estimate that 50% of the total pALS wouldn’t qualify for some reason. That leaves about 17,500 people for the total population of people from which to select a sample.
If we want to be 95% confident of our results, +-2.5%, about 1400 people need to be enrolled in a study. That number swells to 2400 if we want to be 99% confident.
So, FDA, what was the last ALS trial to enroll that number of patients? Let me answer that for you: it’s never happened.
Be real scientists. There’s only one way to get this many people to submit data: Grant conditional approval for Genervon GM6. Now.