The Patient Reported Outcomes (PRO) Consortium
Public Health Context:
Traditionally, the assessment of a patient’s response to investigational medical products has been made by medical professionals. More and more, it has been recognized that some assessments might best be made by the patients themselves. A patient reported outcome (PRO) is any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. Well defined and reliable PRO instruments can be used to support a claim in medical product labeling if the claim is consistent with the instrument’s documented measurement capability. PROs currently used in clinical trials and incorporated in labeling usually are disease specific. However, there are multiple PRO tools to measure the same concept (e.g., asthma symptom severity). For some indications, reliable PROs have not been developed. The lack of consistent, validated PROs tools that are qualified for regulatory use has lead to inefficiencies in drug development and in FDA’s review processes. Also, this may result in inconsistencies in the types of outcome information contained in the labeling for drugs approved for the same indication.
Mission & Program Objectives:
The main objectives of the PRO Consortium are to develop, evaluate, and qualify PRO instruments with the FDA for use in clinical trials designed to evaluate the safety and effectiveness of medical products. Specific milestones include:
- Identifying the diseases and conditions for which industry and FDA agree that PRO instruments are needed.
- Establishing working groups to identify and evaluate existing PRO instruments and develop a single PRO measure for each disease and condition selected.
- Designing and implementing clinical studies for the development and evaluation of each PRO instrument indentified.
- Submitting development documentation to regulatory agencies (FDA and EMA) for qualification of PRO instruments for use as primary or secondary endpoints in clinical trials.
Accomplishments as of August 2010:
- Identification of 33 indications with a primary PRO endpoint and 8 symptoms applicable to multiple disease areas.
- Identification of the top 8 areas where PROs are needed: Asthma, Depression, Diabetes, Irritable Bowel Syndrome (IBS), Oncology, Pain, Cognition and Fatigue.
- Working groups organized for Asthma, Cognition, Depression, IBS, Non-Small Cell Lung Cancer, and Advanced Breast Cancer PROs.
- Scoping documents were developed and submitted to the FDA in December 2009 for Irritable Bowel Syndrome (IBS) and Cognition. Feedback from FDA was given on 3/15/10 and 4/6/10 for IBS and cognition respectively.
- Scoping documents for Asthma, Cystic Fibrosis, Depression and Non-Small Cell Lung Cancer were submitted to FDA between 3/2/10 and 5/19/10 and are under review.
- A Request for Proposals was released by the IBS working group to seek a contract research organization to conduct qualitative research for the IBS PRO.
- First Annual PRO Consortium Workshop was held March 23, 2010 in Bethesda, MD with over 150 attendees.
The PRO Consortium was formed by the non-profit Critical Path Institute (C-Path), in cooperation with the U.S. Food and Drug Administration (FDA) and the medical products industry. As a 501(c)(3), the Critical Path Institute serves as a recognized and respected neutral third party convener.
C-Path, with support from the FDA and the State of Arizona, provides a Director, Associate Director and staff who manage the Consortium, coordinate planning and implementation of all projects, provide financial oversight and establishment of a process for identification, prioritization, and development of potential PRO instruments. The twenty-six pharmaceutical and biotech company members provide expertise, information and funding for specific consortium research contracts for PRO development. The FDA and EMA utilize their respective qualification programs to review and approve PROs.
U.S. Food and Drug Administration
European Medical Agency (EMA)
National Institutes of Health (NIH)
Astellas Parma US, Inc.
AstraZeneca Pharmaceuticals LP
Boehringer Ingelheim Pharmaceuticals, Inc.
Bristol-Myers Squibb Company
Eli Lilly and Company
Forest Research Institute, Inc.
Johnson & Johnson, LLC
Merck Sharp & Dohme Corp.
Novartis Pharmaceuticals Corporation
Roche Palo Alto, LLC
sanofi-aventis U.S., Inc.