(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: https://13.43.35.2/blog/

Upcoming meetings of interest to the AMR community:

• 28 Feb 2018 (Brussels): Deadline for responses to IMI2 Call 13 (Topic 3) on AMR diagnostics
• 2 Mar 2016 (Berlin): BEAM-sponsored 1-day “Big Drugs for Bad Bugs” conference
• 11-16 Mar 2018 (Ventura Beach): Gordon Research Conference on Antibacterial Discovery
• 12-13 Mar 2018 (London): BSAC’s Spring Conference
• 19 Mar 2018 (Washington: Duke-Margolis & FDA event: “Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings”
• [NEW] 20 Mar 2018 (Washington): FDA workshop on complex, innovative trial designs. See also this FR notice.
• 27-29 Mar 2018 (Cardiff, UK): BSAC-sponsored 3-day residential Workshop on Susceptibility Testing
• 16 Apr 2018 (Washington): Duke-Margolis & FDA event: “Evaluating Inclusion and Exclusion Criteria in Clinical Trials”
• 21-24 Apr 2018 (Madrid): ECCMID
• [NEW] 4-7 Jun 2018 (Boston): BIO (multiple AMR-focused sessions are expected)
• 7-11 Jun 2018 (Atlanta): ASM Microbe
• 22-27 Jul 2018 (Bryant University, Smithfield, RI): Gordon Research Conference on Drug Resistance for Cancer, Infectious Disease and Agriculture
• [Don’t miss this one] 4-7 Sep 2018 ESCMID-ASM Conference (#3) on Drug Development for AMR (Lisbon, Portugal) (initial flyer)
• 6-14 Oct 2018 International Course on Antibiotics and Resistance (ICARe, Les Pensières, Annecy, France)