Biomarkers Consortium - Developing Endpoints for Clinical Trials in Community Acquired Bacterial Pneumonia (CABP) and Acute Skin and Skin Infections (ABSSSI)

Advancing the development of effective treatments for serious bacterial infections

The Problem
To develop effective treatments for community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections, researchers must first establish reliable measures to confirm whether a treatment is indeed working.
The Solution
This project helped establish reliable measures of how patients enrolled in clinical trials of antibacterial treatments feel, function and survive, ultimately enabling the development of effective treatments for bacterial infections.

Overview

The Biomarkers Consortium - Developing Endpoints for Clinical Trials in CABP and Skin Infections developed a set of recommendations to help FDA establish efficacy outcome measures (i.e., endpoints) for clinical trials of investigational agents to treat community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI). The endpoint recommendations are tied to historical data in each indication, thereby providing the basis for sound non-inferiority (NI) trial design and NI margin justification. A second key deliverable of the project was to create a Patient-Reported Outcome (PRO) instrument for CABP and ABSSSI that accurately measures the symptoms of each indication. This instrument will enable clinical trialists to assess how a patient feels, functions and survives in anti-infective clinical trials and studies.

Goals

  • Develop reliable and well-defined clinically relevant endpoints for clinical trials of antibiotics for treatment of CABP and ABSSSI, two common and serious bacterial infections.
  • Complete the content validity phase of a PRO measure for each of the ABSSI and CABP indications.

Results & Accomplishments

Developed Novel Outcome Measures

The FNIH CABP/ABSSSI Project Team developed relevant outcome measures for CABP and ABSSSI via retrospective analyses and synthesis of data from clinical trials. The results of the project were submitted to the FDA and subsequently incorporated into FDA guidance to include symptomatic endpoints for CABP and ABSSSI. Six antibiotic drugs have been approved using the updated FDA guidance.

Partners

Public-Sector Partners

  • National Institute of Allergy and Infectious Diseases (NIAID)
  • U.S. Food and Drug Administration (FDA)

Private-Sector Partners

  • Actelion Pharmaceuticals Ltd.*
  • American Thoracic Society
  • AstraZeneca Pharmaceuticals LP*
  • Basilea Pharmaceutica International Ltd.*
  • Cempra Pharmaceuticals*
  • Cerexa, Inc.*
  • Cubist Pharmaceuticals, Inc.*
  • Durata Therapeutics, Inc.*
  • ICON plc.
  • Infectious Diseases Society of America
  • Janssen, L.P.*
  • The Medicines Company*
  • Merck Sharp & Dohme Corp.*
  • Nabriva Therapeutics AG*
  • Trius Therapeutics, Inc.*
  • Pfizer Inc*
  • Pharmaceutical Research and Manufacturers of America*
  • Theravance BioPharma*

*Provided financial or in-kind support for this program.

FNIH Contacts

Steve Hoffmann, Director, Inflammation and Immunity, shoffmann@fnih.org

Stephanie Cush, Ph.D., Senior Project Manager, scush@fnih.org

 

Developed Patient-Reported Outcomes (PRO)

Poster Presentations

Resulting FDA Guidance Documents

Scientific Publications

Media