FDA committee tackles how to assess drugs that target serious infections but affect small populations

Last week’s meeting of the FDA’s Antimicrobial Drugs Advisory Committee was unusual. We didn’t address the merits of a single new drug the FDA is considering for approval but how to fairly assess drugs that target a single bacterial species causing very serious and deadly infections but that affect small populations. Getting sufficient numbers of appropriate patients for drug trials is challenging in many ways. Often there isn’t time to assess which species of bacteria is the problem, and not treating people as quickly as possible is not an option. As usual, there are no easy answers but the committee provided feedback on the options from diverse perspectives. Many members also thanked the FDA for being proactive in identifying a problem early, and working with companies to help them design meaningful but feasible studies of effectiveness and safety. More regulatory agencies, both federal and state, should take this constructive approach.