Biotech Values

[iwfal]

Perhaps iwfal's skepticism is well founded and only those companies already good at running trials would navigate Adaptive Trials efficiently.

Just as a thought experiment - BMRN's 701 in Pompe. It has taken them 1 year from determining the somewhat surprising results of the ph 2, to starting enrollment of the ph 3. And then, of course, it will have taken some time to get the sites going etc - but I wouldn't expect too much time to spool up since we know Biomarin is very good at keeping a registry.

Thus, perhaps, in a perfect world if Biomarin could have moved instantly from the ph ii results to the ph iii they could have saved 15 months.

BUT - the results were somewhat surprising. Undoubtedly even in the best of circumstances they would have to retool the protocol (e.g. they tuned the age for the ph 3, and I believe they narrowed the entry criteria for MIP/MEP). And they would have had to engage with the FDA either after the ph ii or while setting up the ph ii on what MIP/MEP endpoints were acceptable. Call that a dead minimum of 4 months. Then you'd need a longer ph 1 (or additional animal studies or both) to further pin down which options you thought were the most probable outcomes of the ph 2 (MIP or MEP or FVC of 6MWT and Responders or Mean or ...). Call that a dead minimum of 6 additional months before getting to the ph ii negotiations with the FDA. That is now a total of 10 months expended to save 15 months. Net 5 months savings (at best IMO). And there is a risk it is for naught - e.g. if the ph 2 outcomes look too much different than predicted then all the extra time to set up the adaptive is largely wasted.

This is an example of my point when I say that under most circumstances it doesn't save much (if anything). And with an inept company the risk goes up, and the savings goes down. I'd suggest that for most small biotech trying to do an Adaptive is a net negative. Don't try basejumping until you've done multiple smooth solo plane jumps first. That said - when you already have strong data and good trial practices and all you have left to do is tweaking - then I'd agree completely that Adaptive could save some time with little risk.