Progress on developing endpoints for registrational clinical trials of community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections: update from the Biomarkers Consortium of the Foundation for the National Institutes of Health

Clin Infect Dis. 2012 Oct;55(8):1114-21. doi: 10.1093/cid/cis566. Epub 2012 Jun 28.

Abstract

Efficacy endpoints for previous registrational trials of antimicrobials for acute bacterial skin and skin structure infections (ABSSSIs) and community-acquired bacterial pneumonia (CABP) were based on nonstandardized, clinician-based observations and decisions, as well as on patient reports. More quantifiable, reproducible, and externally verifiable endpoints could improve the design of future noninferiority trials. At the request of the Food and Drug Administration, the Foundation for the National Institutes of Health convened a broadly representative scientific project team to evaluate potential endpoints for such registrational trials. Review of historical and modern data led to the conclusion that antimicrobial treatment effects are most apparent early in therapy; later outcomes provide important supportive information. Although evidence is incomplete, early response endpoints can anchor noninferiority hypotheses in ABSSSI and CABP registrational trials, thereby allowing evidence-based drug development to continue. Further research is underway to establish which short- and long-term outcomes are well-defined, reliable, and reflective of how patients feel, function, or survive.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Anti-Bacterial Agents / therapeutic use*
  • Biomarkers / metabolism
  • Clinical Trials as Topic / methods*
  • Community-Acquired Infections / drug therapy*
  • Community-Acquired Infections / metabolism
  • Endpoint Determination / methods*
  • Humans
  • National Institutes of Health (U.S.)
  • Pneumonia, Bacterial / drug therapy*
  • Pneumonia, Bacterial / metabolism
  • Skin Diseases, Bacterial / drug therapy*
  • Skin Diseases, Bacterial / metabolism
  • Vereinigte Staaten

Substances

  • Anti-Bacterial Agents
  • Biomarkers