FDA-approved surrogate endpoints for developers

Dear All:

As requested by Congress as part of the 21st Century Cures Act, the US FDA has now released for public comment a table of surrogate endpoints that can be considered for use by developers:

  • The public docket soliciting comments is here.
  • The web page providing an overview of surrogate endpoints and biomarkers is here.
  • The surrogate endpoint table itself can be found online here.
  • An instructive commentary on the overall FDA approval process is also provided.

The surrogate endpoint tables (one each for adults and pediatrics) are absolutely fascinating. To assist you I have created a version of the .xlsx file (get my summary file here) that combines the pediatric and adult surrogates and focuses on just the Infection-related endpoints. 

What are surrogate endpoints? The overview page provides this summary:

  • Surrogate endpoints may be used instead of clinical outcomes in some clinical trials.
  • For example, surrogate endpoints are used when the clinical outcomes, like strokes, might take a very long time to study, or in cases where the clinical benefit of improving the surrogate endpoint, such as controlling blood pressure, is well understood.
  • They are also used in cases where conducting a clinical endpoint study would be unethical.
  • Before a surrogate endpoint can be accepted in place of a clinical outcome, extensive evidence must accumulate, including evidence from epidemiological studies and clinical trials.

Almost all of the infection-related surrogates fall into two categories: (a) antibody response to vaccines and (b) direct measures of viral burden (e.g., plasma CMV DNA or plasma HIV RNA). The notable exceptions are Chagas Disease (IgG to T. cruzi antigens) and M. tuberculosis (time to sputum conversion to negative). 

Consistent with this, and for those of you interested in bacteria or fungi, it will be no surprise that you won’t see related surrogate endpoints for these types of products. In general, these infections progress so quickly that it’s really better to just look at the disease itself. 

Happy reading! 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:

  • 7-9 Nov 2018 (Seville, Spain): Better Methods for Clinical Studies in Infectious Diseases and Clinical Microbiology: A Hands-on Workshop
  • 8 Nov 2018 (Alderley Park, UK): Bionow’s 1-day Bioinfect conference
  • 12-18 Nov 2018 (everywhere): WHO (and US CDC) Antibiotic Awareness Week. Events now being planned … see the WHO and CDC links for ideas, fact sheets, and graphical materials.
  • 12-13 Nov 2018 (London): Joint SCI– and Royal Society of Chemistry-sponsored 2nd annual Symposium on Antimicrobial Discovery. Online materials here.
  • 13 Nov 2018 (London): All-Parties Parliamentary Working Group on AMR meeting. Online materials here
  • [NEW] 15-17 Nov 2018 (Santiago de Cali, Columbia): 16th Infocus Forum on Fungal Infection in Clinical Practice. Online materials here.
  • 16 Nov 2018 (Berlin): Max Planck Institute for Innovation and Competition is organizing a conference on life sciences and innovation. Online materials here.
  • 19-20 Nov 2018 (Accra, Ghana): Call to Action on Antimicrobial Resistance. Latest agenda here. To attend, contact AMRCalltoAction@wellcome.ac.uk.
  • 21 Nov 2018 (London): NICE- & APBI-sponsored masterclass: “Using non-randomised data to estimate treatment effects in NICE submissions”. Details here.
  • 29-30 Nov 2018 (Birmingham, UK): BSAC (British Society Antimicrobial Chemotherapy): Antibiotic Resistance Mechanisms Workshop for Researchers
  • [NEW LINK] 7 Dec 2018 (Boston, MA): BAARN, Boston Area Antimicrobial Resistance Network 2018 symposium, 8:30am to 7pm at The Starr Center (185 Cambridge Street, Boston, MA). This is an excellent networking opportunity, especially for those based in the Boston area. Register here.
  • 15 Jan 2018 (London): BSAC’s Antimicrobial Chemotherapy Conference 2019: “An ABC for everyone involved in developing new antimicrobials.” Details here.
  • 14-15 Mar 2019 (Berlin): BEAM– and ND4BB-ENABLE-sponsored Berlin Conference on Novel Antimicrobials and AMR Diagnostics. Details here.
  • 21-22 Mar 2019 (Birmingham, UK): BSAC Spring Conference.

 

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