As the 2017 Holiday Season [1] is upon us, here some concepts to ponder while in the mood. Certainly not strategic imperatives, just musings and thoughts for discussion:
#1: Consider developing your inhaled antibiotic in CF first. Pursuing a new indication is high risk, akin to playing Russian roulette but with a revolver that has 5 rounds and only 1 blank. You might get lucky but the odds are – hmm – against you.
#2: For therapeutic efficacy trials, do not commit to a superiority design unless you absolutely have to. There are a few exceptions to this rule but in most situations you stand to lose your shirt in the attempt.
#3: Stay away from therapeutic interventions, go instead first for prophylaxis . Again, we can think of a few exceptions to this rule, but therapeutic efficacy in non-CF indications is not something you want to establish unless you have a big NIH grant or don’t care about LoS.
#4: Don’t believe your aerosol is better than the concomitant systemic therapy – because it is not.
#5: Eliminate any and all kinds of background noise that could drown out a (probably weak) efficacy signal – and give your aerosol a chance to show the blip of efficacy enhancement you are trying to catch.
#6: Select your efficacy and infection markers carefully. When dealing with add-on therapy it is advisable to hone in on symptomatic patients with high bacterial load. Very high load is even better.
#7: Don’t study VAT: There is no price for improving colonization either qualitatively or quantitatively.
Footnotes:
[1] Sorry this blog did not make it in time for the 2016 Holiday Season – The Editors
Abbreviations:
LoS Likelihood of success
VAT ventilator-associated tracheitis
CF cystic fibrosis
