PRS61 A COMPARISON OF EUROPEAN MEDICINES AGENCY (EMA) AND FOOD AND DRUG ADMINISTRATION (FDA) PATIENT-REPORTED OUTCOME (PRO) GUIDANCES FOR CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD)

Within COPD, development of reliable measures for capturing treatment benefit is challenging. Utilizing reliable and valid PRO measures is crucial, and both the EMA and FDA provide guidances to assist with selection of these endpoints in COPD. This study aims to compare EMA and FDA recommendations on COPD PRO measures. Searches were performed for EMA and FDA guidances on COPD published between 2009 and 2019. Data from guidances were extracted and compared. EMA guidance recommends disease-specific PRO measures to characterize treatment response. While, it is nonspecific when recommending measures, there are references to the Chronic Respiratory Questionnaire and the St. George’s Respiratory Questionnaire (SGRQ) as measures that are sensitive to change and potentially suited to measure treatment effects. The EMA guidance mentions additional PRO measures: the COPD Assessment Test; COPD symptom scales; general questionnaires; and diary cards to capture unreported, mild exacerbations. Conversely, the FDA has a SGRQ-specific guidance, recommending its use in interventional clinical trials. Responder analysis is the preferred primary method for reporting SGRQ results, frequent measurements using the SGRQ tool are important, and the SGRQ can be implemented as a co-primary or secondary endpoint to support efficacy statements. Two other guidances released by the FDA – one for an instrument Evaluating Respiratory Symptoms in COPD, pointing to its use in stable COPD patients but stating that additional research is needed, and another guidance on drug development for COPD patients with acute bacterial exacerbations of chronic bronchitis, calling out the Exacerbation of Chronic Pulmonary Disease Tool as an acceptable electronically administered PRO measure. There is a need to utilize validated PRO measurement tools in COPD, since there is variation between regulatory guidances on proper utilization of such measures. Additional research is needed to interpret PRO measurement changes as clinically meaningful efficacy statements.