OBJECTIVES : Development of reliable measures for capturing treatment benefit in ulcerative colitis (UC), gastroesophageal reflux disease, and irritable bowel syndrome (IBS) can be challenging. Drug developers may rely on PRO measures as clinical trial endpoints to determine therapeutic benefit. Therefore, the EMA and FDA provide guidelines to assist with developing therapies for these conditions. This study aimed to compare EMA and FDA recommendations for UC, reflux, and IBS specific to PRO measures.

METHODS : Searches of EMA and FDA guidances for these three conditions were performed and data were extracted and compared.

RESULTS : Evaluation of outcomes is difficult with IBS clinical trials and PRO measures may be the only currently available method to evaluate treatment effects. The EMA notes that a rigorously validated instrument for IBS is currently not available while the FDA states they are actively participating in development of qualified PRO measures. Within UC, the available PRO measures may not be reliable and valid; thus, they require further development. The EMA recommends the Simple Clinical Colitis Activity Index (SCCAI), which mainly includes PROs, while the FDA recommends the Mayo Score and Ulcerative Colitis Disease Activity Index (UCDAI) which incorporate PROs as well as physician assessment of disease activity. Within reflux, both guidances recognize the need for development of PRO and observer-reported outcome (ObsRO) measures. Neither guidance provides specific information on treatment goals and potential labeling claims for IBS. Both guidances note that labeling claims for reflux may include erosive or symptomatic reflux while the EMA provides additional indications suitable for labeling claims. The EMA provides more specific details regarding UC labeling claims and outcomes.

CONCLUSIONS : While many similarities and differences exist between EMA and FDA guidances for these GI disorders, both regulatory agencies note the need for future development of PRO measures.