TradePunk...interesting question.
Indeed while there may not be a formal definition and criteria, read endpoints, stipulated by the FDA, there are certainly bodies of proof that will satisfy them.
Some that I think would have to be met include, but may not be limited to:
1) Hard Cord Clinical observations of the standard...eg Pain and Function WOMAC score improvement to baseline and subsequent comparison to placebo.
2) Some sorta prognostic biomarker, that acts as a surrogate. We know OA typically progress over lengthy period of time, ie 10 years generally. Show us some proof of reducing, arresting or even reversing of this biomarker and it will go a long way to getting that label where we need it!
3) All of the above but ideally we want an immaculate safety record please.
Point 2) could be as Dr V Kraus suggests, CTX II, but there are others on the hit list, namely HA as well as COMP as examples.
All of the above would be fairly much base level requirement. Now add any clinically significant structural biomarkers going the right way, and it may only be in smaller numbers and I think you'd have a drug safely over the line in terms of label making where DMOAD is concerned. At the end of the day what do the FDA want?
They want a safe drug that works and ideally at least in a good amount proportion of patients, then there is a good chance of DMOAD being accredited to the all important label.
Faster pathways are possible. I also think some sorta study would need to be set up in a formalised clinical setting but Accelerated utilising the surrogate endpoint(s) is a rapid way to get this done...and in our case I do rate it as a chance. Don't also forget, ordinarily you would want to see numbers...ie larger patient numbers, the whole purpose for a P3 for instance. But having achieved Statistical Significance already in such an incredibly tiny cohort is gold for us. This should in theory allow us to conduct a future trial with manageable numbers based on powering of the trial and drug effect size.
Get that (AA) and of course we will still want an NDA eventually and thus full licence but in the interim, this will be a very very big turning point for us.
The SP would no doubt agree at this juncture.
My thoughts
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