7 Nov 2017 FDA VRBPAC: Clinical development plan for a S. aureus vaccine

Addendum: This is the first in a 3-newsletters series on this topic. Go here and here for the next two newsletters.

Dear All: 

I just learned that FDA’s Vaccines and Related Biological Products Committee (VRBPAC) will meet at White Oak in an open session on 7 Nov 2017 to discuss and make recommendations on the clinical development plan for Pfizer’s investigational Staphylococcus aureus vaccine intended for pre-surgical prophylaxis in elective orthopedic surgical populations. The only materials available are the announcement at this link — more materials are promised no less than 2 business days in advance.

This is really interesting to see. We’ve had prior debates on this topic that have revolved around these questions:

  1. Can you use eradication of carriage as a measure of the value of prophylactic agent?
  2. How big does the study have to be if you must show reduction in a serious (non-trivial) clinical infection?
  3. In what population can you do this?

In general the answers to date have been NO, VERY LARGE, and NOT SURE. The answer of NO for Q1 is well stated in the 23 Oct 2013 EMA Addendum (Section 3.5.3): Indications that relate to the reduction or eradication of a pathogen from a specified body site are not acceptable unless the microbiological effect of active treatment has been shown to result in a measurable clinical benefit. In the absence of adequate data to support such a link, the clinical benefit associated with the effect of treatment on carriage should be demonstrated in a placebo-controlled study with a primary clinical endpoint.

The final NOT SURE answer follows in part because the moment you identify a group at real risk, there is an ethical requirement to do everything you can to reduce the rate of infection (e.g., use really good peri-operative antibiotic prophylaxis). And the VERY LARGE follows from the need to then limit studies to a setting where the risk of infection is low!

Although it is important that we have tools of this type, the difficulties implied by these answers have stymied work to date. I am delighted to see this VRBPAC announcement and look forward to hearing the discussion!

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:


Impact of PASTEUR: 9.9m lives saved, ROI of 125:1

Impact of PASTEUR: 9.9m lives saved, ROI of 125:1 Dear All (and with thanks to Kevin Outterson for being lead author on this newsletter), (wonkish alert on this one … refresh your coffee and dig in!) The Center for Global Development have released a blog post and a paper estimating the potential impact of the PASTEUR Act.

Five FDA RFPs! Antifungal animal models, MIC breakpoints, and more!

Dear All, I just today learned that FDA have posted their FY 2023 funding opportunities. All the details are on this webpage and I’m just going to reproduce the text below my signature for your reference. I’ll also add a few details in italics. Lots of good opportunities here! –jr John H. Rex, MD |

WAAW! Must-Read Article, A Quiz, A Video, A Call to Action

Notes: Newsflash … BARDA have opened their long-running BAA-18-100-SOL-00003 to include support for antifungal agents. Search for “antifungal” in the posted .pdf to find the text. The first deadline for proposals is 15 Dec 2022 and further deadlines will doubtless follow: as shown on page 9, this BAA has offered 4 deadlines/year since 2018!  Dear All, In

Fireside Chat with AMR Action Fund CIO Martin Heidecker

Dear All, Ahead of World Antimicrobial Awareness Week, I sat down with AMR Action Fund Chief Investment Officer Martin Heidecker for a Fireside Chat. It was a fabulous conversation that covered everything from how the Fund’s investment process works to what it’s looking for in portfolio companies to broader investment trends in the AMR space.  As

Scroll to Top