Biotech Values

[masterlongevity]

adaptive trial designs are a great idea and rare disease companies could learn a lot from oncology an some of the new AD trials in this area. The guidance on adaptive trials has been out for quite a long time now. Rare disease companies also need to focus more on surrogate biomarkers, and could also learn a lot from "big" disease drug development

but personally think its a bad idea to get away from PBO. I have heard from a lot of rare disease companies that PBO is unethical for >x amount of time. I think some are even pointing to 6 months, but there is no current tx, their drugs havent been proven to work, and there are many other Terminal disease where PBO is given for much longer periods (2-3 yrs). losing the PBO is just cutting corners and trying to find an easier way to win, however often disguised in only concerned for pts.

PBO helps determine true effect and leads to the greater good for all the future pts who will be on the drug