Posted by Michael Wonder on 20 Mar 2018
FDA, industry ponder changes to clinical trials for rare disease treatments
19 March 2018 - FDA statisticians pondered changing how the agency uses statistics to approve drugs for rare disease at a public workshop.
The challenge, regulators said, is that there are so few patients who suffer from certain rare diseases that it might not be possible or feasible to do many rounds of traditional clinical trials.
So, regulators and drug developers are looking at ways to import data collected in early studies — which are used to initially assess drug safety and efficacy — into the Phase 3 studies that the FDA uses to determine whether drugs should make it onto the market.
