PRO Development

A patient reported outcome measure, or PRO, may be defined as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” These measures are widely used to study chronic diseases and are considered by regulatory agencies such as the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA), to be the gold standard for quantifying patient experience. For the inflammatory bowel diseases (IBD), this view is reflected in regulatory guidance for the design of future registration trials that requires a primary endpoint consisting of both a PRO measure and an endoscopic outcome.

Although treatment options for IBD have expanded and improved over the past two decades, many patients nevertheless do not respond, or eventually lose response, to currently available therapies. A significant unmet need for novel therapies for IBD therefore remains, and multiple compounds are in early clinical development. Until recently, however, the lack of a valid and reliable PRO has been viewed as an important barrier to the drug development process for IBD given regulatory guidance on requirements for registration trial endpoints. Furthermore, the process for development and validation of a PRO instrument is both rigorous and resource-intensive and frequently requires many years to complete.

At Alimentiv, we strive to break down the barriers that impede our mission to transform human health. We invested significant research efforts to develop and validate the Symptoms and Impacts Questionnaire for Crohn’s Disease and Ulcerative Colitis (SIQ-CD and SIQ-UC, respectively) with the ultimate goal of ensuring that future therapies target not only the underlying disease process but also provide meaningful improvements to patient well-being and function. Unlike prior PRO instruments, the SIQ-CD and SIQ-UC were developed using centrally-read endoscopy to qualify patients based upon a range of disease activity. The draft questionnaires are now available for use in Crohn’s disease and ulcerative colitis clinical trials, and the manuscript describing our study can be accessed here.