Dear All (and with apologies … long note alert!):
Yesterday’s AMDAC was very productive. For reference, all the materials are here and a reasonably detailed set of notes are found below my signature. For further color, David Shlaes has also posted a summary on his Perfect Storm blog.
FDA nicely positioned the question for the day as “It will be easy to approval a new narrow-spectrum agent if clean non-inferiority or superiority data are possible, but we think it likely that this will be the exception rather than the rule. If we are presented instead with a product that seems to have all the right stuff preclinically but where the best available clinical data are unclear, how should we proceed?”
In response, my core takeaway is that the committee overall (some dissent, some nuances) recognized:
But, also coming through loudly and clearly was the message that this is really a pathway of last resort … every effort should be made to generate the best possible clinical data. Put in Tier A-B-C-D language (Lancet ID 13:269-75, 2013), approval could be based only Tier C-quality data if you truly have no choice … but, please, please, PLEASE get as close to the quality of data expected in Tier B as you can.
All of this makes good sense to me and I think this is a great outcome for the day – perhaps not a home run but certainly a triple if you’ll permit a baseball metaphor. I especially liked the balance around the idea that “you can rely more heavily on animal models + human PK if you really have no other options” vs. “If you bring a rock-bottom clinical package, the drug will be viewed skeptically … it is in everyone’s interest if you do more.”
Let me close by thanking our colleagues at FDA for the many hours of effort that went into bringing us to yesterday’s AMDAC. The two prior workshops have definitely moved the conversation forward and I can see how this could become a guidance document in short order. Exciting!
And finally, the topic of forward conversations brings up this list of events (in addition of course to ECCMID, etc.). I hope to see you at some or all of:
All best wishes and safe travels,
John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Chief Strategy Officer, CARB-X | Expert-in-Residence, Wellcome Trust. Follow me on Twitter: @JohnRex_NewAbx
== Detailed notes ==
The day begain with these presentations:
There were no votes taken but rather the meeting ended with the committee providing verbal commentary on two questions (paraphrased):
The discussion prior to commenting on the questions was wide-ranging and the committee need to do some exploratory thinking via its questions. This was perhaps not surprising --- this group is mostly new to the specific issues of antibacterial agents. When it came time to comment on the questions, these themes were dominant: