Evidera, a leading provider of evidence-based solutions for the healthcare industry, announced that the Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration, has released its first draft qualification guidance document for a patient-reported outcome (PRO) measure in drug development: Qualification of Exacerbations of Chronic Pulmonary Disease Tool [EXACT] for Measurement of Symptoms of Acute Bacterial Exacerbation of Chronic Bronchitis in Patients With Chronic Obstructive Pulmonary Disease. This document proposes recognition of the EXACT, an electronically administered patient-reported outcome (PRO) daily diary, as a qualified measure for evaluating symptoms of acute bacterial exacerbations of chronic bronchitis in patients with chronic obstructive pulmonary disease (ABECB-COPD).
The EXACT (EXAcerbations of Chronic Pulmonary Disease Tool) was developed under the EXACT-PRO Initiative, a multi-year, multi-sponsor project initiated and led by Evidera. This project involved experts in pulmonary medicine, instrument development, and drug development regulatory issues dedicated to the task of developing a single, standardized instrument for evaluating the effects of treatment on acute exacerbations of COPD. Over 500 patients were involved in various stages of instrument development, including participation in focus groups, interviews, and a daily symptom diary study, to make certain the patient’s perspective was represented. Evidera was the first company to convene a consortium of industry experts and multiple pharmaceutical sponsors to develop a PRO instrument for use in medical product development trials. The resulting instrument and a derivative measure, the EXACT-Respiratory Symptoms scale (E-RS), are the first to undergo qualification review by the FDA. These instruments are also under qualification review by the European Medicines Agency (EMA). Evidera maintains copyrights to the EXACT and E-RS in English and all translations (50 to date).
“After years of development and testing, with contributions from a diverse team of scholars, practitioners, industry experts, and patients, the release of this draft guidance represents a major milestone for the EXACT,” said Nancy Kline Leidy, PhD, Senior Vice President, Scientific Affairs for Evidera and Principal Investigator and Director of EXACT-PRO Initiative. “Furthermore, as the first qualification guidance from the FDA addressing a specific PRO measure, it could well constitute a significant advancement for PROs in drug development.”
FDA qualification of drug development tools is designed to expedite the drug development process, in this case, research on the effects of new treatments on exacerbations of COPD. The EXACT draft guidance is the first for a PRO instrument, and follows on the heels of the FDA’s release of the final Guidance for Industry and FDA Staff – Qualification Process for Drug Development Tools. The draft qualification guidance document for the EXACT was posted on the Federal Register January 10, with public comments due to the FDA within 90 days. When finalized, the qualification guidance for the EXACT will represent the FDA’s current thinking on the use of this PRO instrument in drug development.
Chronic obstructive pulmonary disease (COPD), including emphysema and chronic bronchitis, is a progressive lung disease that causes breathlessness, coughing, mucus, and other symptoms. (For more information, visit http://www.nhlbi.nih.gov/health/health-topics/topics/copd/.) COPD affects over 24 million people in the US and over 64 million people worldwide. Exacerbations are an acute worsening of the disease that can lead to substantial morbidity, including lost work days, clinic or emergency room visits, and hospitalizations, and may cause death. Understanding these events and developing new treatments to reduce their frequency, severity, and duration, are important to patients and priorities for medical research. The EXACT was designed to advance the science through precise, standardized assessment of exacerbations to facilitate basic and applied research on exacerbations, including onset, acuity, recovery, relapse, and the effects of preventive and acute treatment. The EXACT is being used in multiple investigations, including pharmaceutical trials and natural history studies of COPD.
“EXACT qualification marks a major milestone in the partnership between FDA and developers of patient-focused clinical outcome assessments (COAs) targeted for clinical trial use,” said Laurie Burke, former Director of CDER’s Study Endpoints and Labeling Development Staff that leads the COA qualification process at FDA. “The EXACT development process enabled FDA to pave the way for more PRO measures to be qualified for drug development. Moreover, COPD patients in clinical trials now have a clear voice in the measurement of treatment benefit and the development of new treatments based on their symptom experience.”