(Another!) FDA trial design workshop: Complex innovative designs

Dear All:

Adding to the 19 Mar 2018 (Designs for Rare Diseases) and 16 Apr 2018 (Inclusion/Exclusion criteria) workshops already announced (see list below), FDA has now announced a 20 Mar 2018 workshop on complex innovative designs (CID) in clinical drug trials. From the Federal Register notice, we are told that the workshop seeks to (1) facilitate discussion and information sharing about the use of CID in drug development and regulatory decision making and (2) obtain input from stakeholders about a CID pilot program. Ideas to be discussed include complex adaptive designs, use of external/historical control subjects, Bayesian designs, master protocols, clinical trial simulations, and the proposed CID pilot program.

Why is this relevant to those of us working on new approaches to antimicrobial resistance (AMR)? Although AMR does not appear in the titles of any of these workshops, some of the ideas could be (or could become) very relevant for the AMR community.

In particular, the mathematics of rare events make it hard to develop narrow-spectrum agents outside of a few special situations. As non-traditional alternative therapies are often narrow-spectrum (e.g., most monoclonal antibody approaches), we simply must find ways forward lest we never develop such products. 

We’ve had several public discussions on this problem (see this note for further details) and we’re definitely not done with the conversation. As the ideas to be discussed in the upcoming workshops might help us with trial designs for narrow-spectrum products, I’m delighted to see them on the calendar! Also, the mention of a pilot program for complex innovative designs is new and of interest. 

All best wishes, –jr

John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Expert-in-Residence, Wellcome Trust. Follow me on Twitter: @JohnRex_NewAbx. See past newsletters and subscribe for the future: http://amr.solutions/blog/

Upcoming meetings of interest to the AMR community:


Leaky pipe(line)s, Part 2 / CARB-X reboot / WHO 2021 pipeline review

Dear All, We have a 3-part discussion today on the theme of “I want a new drug … so how do I find it?” Off we go! First, the 14 June 2022 newsletter entitled “Leaky Pipe(lines) / When Is A Molecule A Drug” generated further discussions that are worth sharing: It was noted the microdosing

Antibiotic procurement models for LMICs / G7 Leaders call for Pull!

Dear All, Two stops on our tour today: (i) an excellent survey of incentive models and (ii) a final communique from the recent G7 meetings. First up, CGD (Center for Global Development) have released a report (link) entitled “Leveraging Purchasing Systems to Ensure Access, Stewardship, and Innovation: A Landscape Review of Current and Potential Market Structures

Leaky pipe(lines) / When is a molecule a drug? (Part 1 of 2)

Dear All, I was fascinated by this recent paper in AAC: Neha K. Prasad, Ian B. Seiple, Ryan T. Cirz, and Oren S. Rosenberg. Leaks in the Pipeline: a Failure Analysis of Gram-Negative Antibiotic Development from 2010 to 2020. Antimicrob Agents Chemother. 2022 May 17;66(5):e0005422. doi:10.1128/aac.00054-22. (Addendum: This newsletter has a follow-up newsletter.) In brief,

FDA/CVM: Antimicrobial use in companion animals

Dear All, Post-newsletter addendum: I’ve learned that USDA will host a 10 Aug 2022 (virtual, 10a-4.30p ET) workshop on AMR in food agriculture. See the meetings calendar for more details; go here to register. I’ll confess to having missed entirely the request back in February 2022 from FDA’s Center for Veterinary Medicine (CVM) for comments on antimicrobial

Scroll to Top