How would you change trial design to enhance this power where you have a disease state that affects 1/10,000 people so enrolment numbers are never going to be big
Plus in a disease state where no treatment options are available whatsoever
I look at it a different way. Primary endpoint like u said is safety which is no issue
Secondary efficacy endpoints are as you mentioned and we don't need home runs with amazing results on them - we just need some change , big or small because there is no treatment - any change is better
And I think the FDA would agree
Very different scenario to mainstream /non orphan indications .
For me any win is a win and it will get to market whether efficacy outcomes are big/small or poorly powered
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