Duke-Margolis & FDA: Workshop on stats & methods for rare diseases

Dear All:

Duke-Margolis and FDA have now announced a 19 Mar 2018 workshop entitled Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Drug Development (FR notice). Here’s the teaser from the web: “Convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University and supported by a cooperative agreement with FDA, this public event will bring the stakeholder community together to discuss the challenges that rare disease settings pose for treatment development and regulatory decision-making, as well as the promising study designs and analytical methods that can help overcome them. The day’s discussion will center around novel approaches to trial design and analysis to improve efficiency and interpretability of results in rare disease trials.”

Note how neatly this fits with their previously announced 16 April 2018 1-day workshop on inclusion and exclusion criteria in clinical trials (corresponding FR notice): “The purpose of the public meeting is to bring the stakeholder community together to discuss a variety of topics related to eligibility criteria in clinical trials and their potential impact on patient access to investigational drugs, and how to facilitate the enrollment of a diverse patient population.” 

This is great to see! While neither of these two is specifically focused on AMR-related issues, the applicability to our trial design issues should be obvious. Unfortunately, I am probably going to miss both workshops but I hope that at least a few of you will get there, especially for the 19 Mar workshop on statistical design. If you do go, I’d love to share what you learned with the newsletter community … please let me know!

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:


Share

Lifeline, the Musical Story of Penicillin: A Review

Dear All, Last week I went to New York City to see one of the opening performances of Lifeline, The Musical, the world’s first musical telling the story of penicillin, Sir Alexander Fleming, and the rise of antibiotic resistance! This was my first time seeing the production in its entirety and it did not disappoint. The timing

WHO: AMR Policy Brief and Preclinical pipeline data call

Dear All, Supplementing and augmenting their review of the clinical and preclinical pipeline through 31 Dec 2023 (see the 15 June 2024 newsletter for details), WHO have now released: A call for data on preclinical antibacterial projects Scope: Traditional and non-traditional antibacterial candidates covering the WHO bacterial priority pathogens as well as other resistant pathogens of public health

UK Subscription model goes live! Value bands cover entire UK!

Dear All (with thanks to Kevin for leading on this newsletter and with a wonkish / long-read alert on this email … settle in for the ride!), Great news from TEAM UK! As of 12 August, the UK Subscription Program is no longer a pilot but is fully operational with an initial application deadline of

Needles and haystacks: Preclinical antibacterial pipeline review

Dear All, Just published in AAC is WHO’s updated review of the global preclinical pipeline. Here are the links you need to follow today’s discussion: The new WHO review: Gigante et al. “Multi-year analysis of the global preclinical antibacterial pipeline: trends and gaps”, https://doi.org/10.1128/aac.00535-24, AAC 2024. An excellent AAC podcast (overview webpage; YouTube video) discussing the WHO review. In

Scroll to Top