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FDA guidance on Substantial Effectiveness; EMA concept paper on Evaluation of Antifungal Agents): Comments sought!

Dear All: Both FDA and EMA have recently released important documents for comment. See below for links and my commentary on the two documents.

== FDA: The Substantial Effectiveness guidance, further updated: Comments due 22 Sep 2026 ==
FDA have released a further revision of their Dec 2019 draft guidance entitled “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.” Here are the links you need:

  • The new (June 2026, revision 1) draft guidance: FR notice, webpage, and direct link
    • Title: “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products”
  • The prior (Dec 2019) draft guidance: FR notice
  • The original (May 1998) guidance: FR notice, webpage, and direct link
    • Title (a little different): “Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products”
  • Useful companion documents
    • (May 2023) “Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence”
    • (Dec  2023) “Rare Diseases: Considerations for the Development of Drugs and Biological Products”
  • Brief background and comments:
    • The May 1998 guidance is the foundational document describing the requirements for approval based on data from a single trial (with confirmatory evidence) rather than multiple trials.
    • The key concept is that the 1962 Federal Food, Drug, and Cosmetic Act created a requirement for a demonstration of “substantial evidence … consisting of adequate and well-controlled investigations, including clinical investigations…”
    • Note that terminal “s” on “investigations”: The original idea was that more than one trial was needed.
    • This idea was gradually updated with the realization that a single trial could be adequate provided it was supported by strong confirmatory evidence. This concept was codified in the 1998 guidance.
    • The Dec 2019 and Jun 2026 draft revisions clarify this principle but do not change it.
      • Note as well the NEJM position paper discussed in 1 Mar 2026 newsletter (“FDA: One pivotal trial (plus confirmatory evidence) is default expectation for approval”) that further pushes this idea.
    • What’s new from Dec 2019? I’m still digesting but what I see is that things are moved around a bit but nothing is dramatically new. Here are a few things that caught my eye on an initial survey: 
      • There is a new section on Trial Conduct: Study quality matters!
      • The statistical analysis discussion hints at the idea that Bayesian methods might be useful (lines 220-221). Note here the FDA’s Jan 2026 draft guidance on use of Bayesian methods.
      • There is an interesting hint (lines 436-7) that requirements for approval for pediatric use might be simplified (on this, see also the 7 Apr 2021 newsletter entitled “Developing antibiotics for children: There are no easy answers”).

== EMA: Concept paper on evaluation of antifungal agents: Comments due 30 Sep 2026 ==
EMA have announced their intent to update their 2010 guidance on developing antifungal drugs. Here are the links you need:

  • Master webpage that provides links to:
    • The 2010 guidance on developing antifungal agents: “Guideline on the clinical evaluation of antifungal agents for the treatment and prophylaxis of invasive fungal disease” (CHMP/EWP/1343/01 Rev. 1)
    • The 29 Jun 2026 concept paper: “Concept paper on the revision of the guideline on the clinical evaluation of antifungal agents for the treatment and prophylaxis of invasive fungal disease” (EMA/133954/2026)
  • The planned update of the 2010 guidance is certainly welcome as much has evolved since 2010. The concept paper points out the need for an update that considers points such as these:
    • We’ve increasingly recognized the problems inherent in studying really rare infection subtypes
    • There has been work on updates to consensus response criteria for invasive fungal infections
    • Best approaches to developing data for children (especially neonates) have also evolved


Very glad to see both of these documents! Please review and submit your comments! 

With thanks to our colleagues at the agencies and with all best wishes, –jr

John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Operating Partner, Advent Life Sciences. Follow me on Twitter: @JohnRex_NewAbx. See past newsletters and subscribe for the future: https://amr.solutions/blog/. All opinions are my own.

John’s Top Recurring Meetings
Virtual meetings are easy to attend, but regular attendance at annual in-person events is the key to building your network and gaining deeper insight. My personal favorites for such in-person meetings are below. Of particular value for developers, the small meeting format of BEAM’s AMR Conference (March) and GAMRIC (September-October; formerly, the ESCMID-ASM conference series) creates excellent global networking. IDWeek (October) and ECCMID (April) are much larger meetings but also provide opportunities for networking with a substantial, focused audience via their Pipeline sessions. Hope to see you there!

  • [LATE-BREAKER ABSTRACTS open soon] 22-24 Sep 2026 (Lisbon, Portugal): The 2nd GAMRIC, the Global AMR Innovators Conference (London, UK). Formerly the ESCMID-ASM (or ASM-ESCMID depending on location) Joint Conference on Drug Development for AMR, 2026 will be the 11th year for this series that is now under the joint sponsorship of CARB-X, ESCMID, BEAM Alliance, GARDP, LifeArc, Boston University, and AMR.Solutions. The ongoing series employs the successful format of prior meetings with a single-track meeting and substantial networking time. The 2025 meeting was a sell-out success: a written summary is here and the videos are here. Registration for the 2026 meeting is now open and the near-final program is here: in addition to a keynote lecture by Karen Bush on the threat to beta-lactams due to the emergence of PBP3 mutations, there are sessions on funding, access, program design for narrow-spectrum agents such as phage, development for pediatrics, and more. Registration is ongoing — register now as early-bird rates end on 15 July! The main abstract submission window is closed but late-breaker abstracts will be accepted 8-22 July 2026.
  • 21-24 Oct 2026 (Washington, DC, USA): IDWeek 2026, the annual meeting of the Infectious Diseases Society of America. I would expect the program to continue to provide a substantial opportunity to present a product to a large audience (see also adjacent note about ESCMID) as well as opportunities to present at an IDWeek Pipeline Session. Go here to register.
  • 23-24 Mar 2027 (Basel, Switzerland): The 10th AMR Conference (3-4 Mar 2026) is now over and offered a rich program that included a 10-year retrospective (we’ve done a lot!), regulatory updates, discussions of how to pursue development in China, and much more … in addition to being a superb opportunity for networking! I am told the session videos will soon be available on the conference website.
  • 9-13 April 2027 (Stockholm, Sweden): ESCMID Global 2027, the annual meeting of the European Society for Clinical Microbiology and Infectious Diseases. Details won’t be out for some months, but the website is here. I do know that the meeting schedule will again include a Science Policy Forum on Friday 9 April 2027 (see here the newsletter about the 2026 science policy forum) and all who are interested in Pull incentives and the antibiotic ecosystem should plan to be there!)..

  Upcoming meetings of interest to the AMR community:

  • [NEW] 22 July (virtual, 2-3.30p CET): The WHO AMR Department and the WHO GAP-r (Global Accelerator for Pediatric Formulations) are hosting an industry dialogue on pediatric and neonatal antibiotic development. The discussion will cover timing of pediatric and neonatal studies, extrapolation from adult data, feasibility of trial designs in children and neonates, and variability in regulatory expectations across jurisdictions. This topic is a long-standing source of frustration for developers (see the 7 Apr 2021 newsletter: “Developing antibiotics for children: There are no easy answers”) and I am glad to see WHO convening discussions on ways to facilitate pediatric work! Go here to register.
  • [NEW] 27 July (virtual, 2-3.30p CET): Inaugural webinar of the WHO Bacteriophage Community of Practice (CoP), a global platform for knowledge exchange across the bacteriophage ecosystem. You can register for the webinar here. You will also want to join the CoP itself — for this, you need a free account WHO Integrated Health Services (IHS) Hub account; look for  “WHO AMR Community Exchange” and then for the “Bacteriophage group” under “Explore our groups.”
  • 22-24 Sep 2026 GAMRIC (Lisbon, Portugal), the Global AMR Innovators Conference (London, UK; formerly the ESCMID-ASM Joint Conference on Drug Development for AMR). See list of Top Recurring meetings, above..
  • 10-18 Oct 2026 (Annecy, France, residential in-person program): ICARe (Interdisciplinary Course on Antibiotics and Resistance) … and 2026 will be the 10th year for this program. Patrice Courvalin orchestrates content with the support of an all-star scientific committee and faculty. The resulting soup-to-nuts training covers all aspects of antimicrobials, is very intense, and routinely gets rave reviews! Registration for 2026 is now open and runs through June 21, 2026. Go here to register!
  • 21-24 Oct 2026 (Washington, DC, USA): IDWeek 2026. See list of Top Recurring meetings, above.
  • 10-13 November 2026 (Madrid, Spain): The International Society for Infectious Diseases (ISID) has announced its 21st International Congress on Infectious Diseases (ICID). Register and view the preliminary program here (Early bird closes 30 July 2026); abstract deadline is 28 April 2026.
  • [NEW] 27-29 Jan 2027 (Las Vegas, USA): IMARI (Interdisciplinary Meeting on Antimicrobial Resistance and Innovation) sponsored by IDSA and ASM. Abstract submissions are open through 19 Aug; registration and housing will open later this summer.
  • 23-24 Mar 2027 (Basel, Switzerland): The 11th AMR Conference sponsored by the BEAM Alliance. See list of Top Recurring meetings, above.
  • 9-13 April 2027 (Stockholm, Sweden): ESCMID Global 2026, the annual meeting of the European Society for Clinical Microbiology and Infectious Diseases. See Recurring Meetings list, above.
  • ??? Mar 2028 (yes, that’s 2028, with location TBD): The 2028 Gordon Research Conference (GRC, https://www.grc.org/) entitled “Antibacterials of Tomorrow to Combat the Global Threat of Antimicrobial Resistance” and its related Gordon Research Seminar (GRS) for young doctoral and post-doctoral researchers will be sometime in March 2028. The organizers hope to coordinate dates and location with the 2028 BEAM-AMR meeting. Details to follow — mark your calendar!

Self-paced courses, online training materials, and other reference materials:

Current funding opportunities

  • Novo Nordisk Foundation (NNF) have an EUR 20m call for development of rapid, point-of-care diagnostics. See the 28 May 2026 newsletter for details; the deadline for expressions of interest is 7 Oct 2026.
  • CARB-X have had one funding round (8-22 April 2026); a further round is expected during 4Q 2026. There are 4 funding themes for these rounds as below.
    • Direct-acting therapeutics for infections caused by Gram-negative bacteria
    • Novel Chemistry for AMR Challenge – target-based therapeutics.
    • Non-vaccine approaches to prevent neonatal sepsis
    • Diagnostics for neonatal sepsis
  • The Horizon Europe Work Programme 2026-2027 includes at least 3 calls of interest within its Cluster 1 — see the list below.  The application window starts 10 Feb 2026 and closes on 16 Apr 2026. See also the 12 Dec 2025 newsletter about the call. Note as well that there calls for agents to prevent and/or treat viral infections.
    • HORIZON-HLTH-2027-01-DISEASE-08: Development of innovative antimicrobials against pathogens resistant to antimicrobials
    • HORIZON-HLTH-2027-02-IND-02: Portable point-of-care diagnostics
    • HORIZON-HLTH-2026-01-DISEASE-03:Advancing research on the prevention, diagnosis, and management of post-infection long-term conditions. 
  • ENABLE-2 has continuously open calls for both its Hit-to-Lead program as well as its Hit Identification/Validation incubator. Applicants must be academics and non-profits in Europe due to restrictions from the funders. Applications are evaluated in cycles … see the website for details on current timing for reviews. 
  • BARDA’s long-running BAA (Broad Agency Announcement) for medical countermeasures (MCMs) for chemical, biological, radiological, and nuclear (CBRN) threats, pandemic influenza, and emerging infectious diseases is now BAA-23-100-SOL-00004 and offers support for both antibacterial and antifungal agents (as well as antivirals, antitoxins, diagnostics, and more). Note especially these Areas of Interest: Area 3.1 (MDR Bacteria and Biothreat Pathogens), Area 3.2 (MDR Fungal Infections), and Area 7.2 (Antibiotic Resistance Diagnostics for Priority Bacterial Pathogens). Although prior BAAs used a rolling cycle of 4 deadlines/year, the updated BAA released 26 Sep 2023 has a 5-year application period that ends 25 Sep 2028 and is open to applicants regardless of location: BARDA seeks the best science from anywhere in the world! See also this newsletter for further comments on the BAA and its areas of interest.
  • HERA Invest was launched August 2023 with €100 million to support innovative EU-based SMEs in the early and late phases of clinical trials. Part of the InvestEU program supporting sustainable investment, innovation, and job creation in Europe, HERA Invest is open for application to companies developing medical countermeasures that address one of the following cross-border health threats: (i) Pathogens with pandemic or epidemic potential, (ii) Chemical, biological, radiological and nuclear (CBRN) threats originating from accidental or deliberate release, and (iii) Antimicrobial resistance (AMR). Non-dilutive venture loans covering up to 50% of investment costs are available. A closing date is not posted insofar as I can see — applications are accepted on a rolling basis; go here for more details.
  • The AMR Action Fund is open on an ongoing basis to proposals for funding of Phase 2 / Phase 3 antibacterial therapeutics. Per its charter, the fund prioritizes investment in treatments that address a pathogen prioritized by the WHO, the CDC and/or other public health entities that: (i) are novel (e.g., absence of known cross-resistance, novel targets, new chemical classes, or new mechanisms of action); and/or (ii) have significant differentiated clinical utility (e.g., differentiated innovation that provides clinical value versus standard of care to prescribers and patients, such as safety/tolerability, oral formulation, different spectrum of activity); and (iii) reduce patient mortality. It is also expected that such agents would have the potential to strongly address the likely requirements for delinked Pull incentives such as the UK (NHS England) subscription pilot and the PASTEUR Act in the US. Submit queries to contact@amractionfund.com.
  • INCATE (Incubator for Antibacterial Therapies in Europe) is an early-stage funding vehicle supporting innovation vs. drug-resistant bacterial infections. The fund provides advice, community, and non-dilutive funding (€10k in Stage I and up to €250k in Stage II) to support early-stage ventures in creating the evidence and building the team needed to get next-level funding. Details and contacts on their website (https://www.incate.net/).
  • These things aren’t sources of funds but would help you develop funding applications
    • The Global AMR R&D Hub’s dynamic dashboard (link) summarizes the global clinical development pipeline, incentives for AMR R&D, and investors/investments in AMR R&D. See also the 7 Feb 2026 newsletter (“The global funding pipeline, 2017-2023: A review”) about an excellent deep dive by the Hub team into patterns of funding over time.
    • Antimicrobial Resistance Research and Innovation in Australia is an actively updated summary that covers Australia’s AMR research and patent landscape. It is provided via collaboration between The Lens (an ambitious project seeking to discover, analyse, and map global innovation knowledge) and CSIRO (Commonwealth Scientific and Industrial Research Organisation, an Australian Government agency responsible for scientific research). Lots to explore here!
    • Diagnostic developers would find valuable guidance in this 6-part series on in vitro diagnostic (IVD) development. Sponsored by CARB-X, C-CAMP, and FIND, it pulls together real-life insights into a succinct set of tutorials.
  • In addition to the lists provided by the Global AMR R&D Hub, you might also be interested in my most current lists of R&D incentives (link) and priority pathogens (link).
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