EXACT-PRO UBC

 

The EXACT

The EXAcerbations of Chronic Pulmonary Disease Tool (EXACT) is a patient-reported outcome (PRO) measure designed to standardize the method for evaluating the frequency, severity, and duration of acute exacerbations of COPD and chronic bronchitis (AECB) in international studies and clinical trials. The instrument was developed using qualitative methods, including focus groups, 1:1 interviews, and cognitive debriefing interviews involving over 80 patients with COPD, with input from a team of experts in pulmonary medicine, clinical research, instrument development and PRO translation methodology.

The EXACT is a 14-item daily diary to be completed by the patient on a personal digital assistant (PDA) each evening before bedtime, taking less than 3 minutes to complete. A naturalistic, prospective study of 410 stable and acute patients showed that the EXACT scores are internally consistent and reproducible in stable patients, differentiate acute and stable patients, change over time in acute patients, differentiate physician and patient-rated severity of exacerbation, and are correlated with clinical variables (e.g., SGRQ-C). To date, the instrument has been translated into 28 languages following accepted PRO translation protocol, with additional translations in progress.

An overview of the EXACT-PRO Initiative, the background for the work, phases of development, and a list of upcoming and previous presentations are provided below.

For additional information about the EXACT, please free to e-mail exactpro@unitedbiosource.com

The EXACT-PRO Initiative - A Consortium Approach to Instrument Development

  • The EXACT-PRO (EXAcerbations of Chronic pulmonary disease Tool – Patient Reported Outcome) initiative is a unique, multi-sponsor project bringing together experts in instrument development and validation, international specialists in clinical practice and research in COPD, and experts from the U.S. Food and Drug Administration (FDA) (Pulmonary, Anti-infective, and Study Endpoints and Labeling Divisions) to develop a single, validated, patient-reported outcome (PRO) measure to evaluate exacerbation-related outcomes of treatment in drug development trials of chronic obstructive pulmonary disease. A key element of the project has been the interest and enthusiasm of the pharmaceutical sponsors, the FDA, and the content experts committed to improving PRO evaluation in chronic pulmonary disease through this innovative, cooperative program.
  • The project is being conducted under the direction of Dr. Nancy Kline Leidy, Senior Vice President of Scientific Affairs for United BioSource Corporation, with Dr. Terry Wilcox, Senior Research Scientist, and Senior Clinical Consultants, Drs. Paul Jones and Sanjay Sethi.

Background

  • Exacerbations are an important feature of chronic obstructive pulmonary disease (COPD), leading to significant morbidity and mortality. Reducing the frequency, severity and duration of acute exacerbations is of great interest to patients, providers, and payers. These same parameters are often used as primary or key secondary endpoints in clinical trials, including pre- and post-approval pharmaceutical trials evaluating the efficacy and safety of anti-infective or maintenance therapies for COPD.
  • Despite widespread commitment to understanding the effect of treatment on exacerbations in COPD, prior to development of the EXACT, there was no standardized PRO instrument for evaluating this outcome. A variety of symptom diaries, often with areas of overlapping content and/or different structure, have been used in clinical trials, with no single, validated, accepted "gold" standard. This has created problems for the industry, regulatory agencies, and clinical decision makers as they attempt to evaluate the absolute and relative efficacy of new treatments for this important health problem. In an effort to meet this need, UBC's Outcomes Research began the EXACT-PRO Initiative to develop a patient-reported outcome tool to measure acute exacerbation of COPD and chronic bronchitis (AECB).

Development Overview

  The EXACT-PRO Initiative is being conducted in phases, as follows:  

  • Phase I, completed in the fall of 2006, included a literature review complete with tabled summaries and analyses based on content; patient focus groups and one-on-one interviews; two expert panel meetings with experts to address the patient's perspective of exacerbations, development of the item pool and response options, and the draft questionnaire; cognitive debriefing interviews; and the development of a draft validation protocol.
  • Phase II, completed in winter of 2007, included finalizing the measure, an additional round of cognitive debriefing interviews, implementation of the validation protocol, data collection, psychometric analysis of the data, and a third expert panel meeting to discuss the final measure and its empirical performance properties.
  • Phase III and Phase IV includes dissemination of information about the tool, increased dialogue with experts and organizations with interest in the EXACT, translation of the EXACT for international use, and completion of a comprehensive evidence dossier for FDA and EMA submission, among other activities.
  • In addition to the activities listed above, work has begun on the E-RS for use as a respiratory symptoms outcome measure in clinical trials of COPD. Work is also underway to develop the EXACT-U, an algorithm that allows utilities to be reported from EXACT scores for cost-effectiveness analyses

Publications

Leidy NK, Wilcox T, Jones PW, Roberts L, Powers J, Sethi S. and the EXACT-PRO Study Group (In press). Standardizing measurement of COPD exacerbations: Reliability and validity of a patient-reported diary.  American Journal of Respiratory and Critical Care Medicine. 2010 Sep 2. [Epub]

Jones, PW, Chen WH, Wilcox T, Sethi S, Leidy NK for the EXACT-PRO Study Group (In press). Characterizing and quantifying the symptomatic features of COPD exacerbations. Chest 2010. Nov 11 [Epub ahead of print].

Leidy, NK, Wilcox T, Jones PW, Murray L, Winnette, R, Howard K, Petrillo J, Powers J, Sethi S and the EXACT-PRO Study Group.  Development of the EXAcerbations of Chronic Obstructive Pulmonary Disease Tool (EXACT): A Patient-Reported Outcome Measure.  Value in Health. 2010 13 (8): 965-975.  [Epub 2010 Jul 23]

 
EXACT-PRO FOLDERS (Expand all)
 
EXACT-PRO Presentations 2008-2010
 
EXACT-PRO EXACT eDiary Certification Program
 
EXACT-PRO Selecting an eDiary Device
 
EXACT-PRO EXACT Translations
 
 

 

ePRO Partners:

EXACT-PRO

 


 

       

 

  

Current Project Sponsors:

Additional Support has been provided by:

 

Adams Respiratory
Almirall
Altana (Nycomed)
Bayer
Boehringer Ingelheim
Dey
Forest Laboratories
Merck
Ortho-McNeil
Sepracor

For information on the EXACT or sponsorship opportunities, contact us:

Email:

exactpro@unitedbiosource.com

Phone: 1 (877) 954-4252

  

 
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