EMA Releases Draft Opinion on Two Patient-Reported Outcome (PRO) Measures for Evaluating New Treatments for COPD

Evidera, a leading provider of evidence‐based solutions for the healthcare industry, is pleased to announce the release of the European Medicines Agency’s (EMA) draft qualification opinion on the EXACT (Exacerbations of Chronic Pulmonary Tool) and E-RS (EXACT-Respiratory Symptoms), two patient-reported outcome (PRO) measures for evaluating treatment outcomes in COPD trials. The daily symptom diary that forms the basis for these instruments was developed as part of the EXACT-PRO Initiative, a multi‐year, multi‐sponsor project initiated and led by Evidera.

The EMA’s opinion, posted for public comment 13 April 2015, states that the evidence supports the use of the EXACT and E-RS as exploratory endpoints in COPD drug development trials. Referencing the EXACT, the opinion states “the suggested attempt to characterise COPD exacerbation events in terms of severity, duration and frequency in a highly-standardised and more symptom-driven manner can be considered a valuable contribution to the search for suitable efficacy endpoints in COPD trials.” EMA is accepting comments until 25 May 2015.

In 2014, the FDA released their draft qualification document for the EXACT, a first for PRO measures in the U.S., describing the EXACT as a “well-defined and reliable measure of symptoms of ABECB-COPD for use in phase 2 studies.” The E-RS is under review by the FDA.

“The release of qualification statements from the EMA and FDA are important milestones in patient-centered outcomes research, ” said Nancy Kline Leidy, Ph.D., Principal Investigator of the EXACT-PRO Initiative and Senior Vice President, Evidera. “PRO measures not only represent the patient’s voice, but are instrumental to scientific progress. The EXACT and E-RS are being used in clinical studies to further our understanding of COPD and the efficacy of interventions to alleviate exacerbations and provide symptomatic relief for people with this condition.”

The EXACT‐PRO Initiative was a consortium convened to develop a standardized PRO instrument for use in drug development trials. The diary has been translated into 55 languages and used in over 65 clinical studies, including 24 trials testing new treatments for COPD.