Why I believe $SRPT (Eteplirsen) will be approved w/ positive 48 week data despite small N:

If you're the FDA, you have to ask yourself what are the risks of taking a drug vs the risks of not taking a drug. In this case, 100% of the boys progress and 100% die. They simply can't wait for a larger phase 3 trial to be completed. Eteplirsen has proven to be safe thus far and the risks of not allowing patients to have access to the drug far outweighs the risks of the drug having a safety issue in the future.

I also believe efficacy is not an issue, even with a small N. There can be no placebo effect here, nor can it be random. With this disease, 100% progress. By definition, if 1 person (let alone 4) stop progressing and actually show RECOVERY, this can't be attributed to anything other than Eteplirsen. I don't think you even need to show that all 4 responded.

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