The Crohn’s & Colitis Foundation was selected as one of two patient organizations to participate in an U.S. Food & Drug Administration (FDA) Real-World Evidence Program demonstration project using the FDA MyStudies App through the FDA-Catalyst system. The FDA Real World Evidence Program will inform the assessment of the fitness for use of real-world data to support real-world evidence generation for regulatory decisions. Through the FDA MyStudies App, the Foundation will expand IBD Plexus®- a first-of-its-kind research information exchange platform and registry that centralizes data and biosamples from diverse research cohorts -to incorporate the use of a mobile application designed to fill in important real-world data gaps and capture patient experience data beyond the clinical care system.
Using the FDA MyStudies App technology, an open source mobile device and data storage system, the Foundation will launch IBD PROdigy, a digital IBD tracker tool that facilitates collection of longitudinal patient-reported data at critical points throughout a patient’s disease course. Through the IBD Plexus platform, the Foundation will then be able to link patient-reported outcomes data collected through IBD PROdigy to robust clinical data, biosamples, and their derived molecular (genetic and ‘omics) data. IBD PROdigy will be initially rolled out to the SPARC IBD cohort – a Foundation-supported, multi-centered longitudinal study of adult IBD patients focused on identifying predictors to help find the right therapy for the individual patient. The goal would then be to release IBD PROdigy to additional IBD Plexus study cohorts.
“IBD PROdigy will become a key link between patients’ experiences and scientific advancements in IBD, enabling deeper patient participation in research. We cannot gather real- world data efficiently and effectively from patients without engaging with user-friendly tools such as IBD PROdigy,” said Angela Dobes, the Foundation’s senior director, IBD Plexus. “The more the app improves our interaction with patients, the more valuable and enriched data we will get. This app will engage patients and enable us to follow and learn from them throughout their disease journey.”
The FDA Center for Drug Evaluation and Research Office of Medical Policy is currently leading Real-World Evidence demonstration project efforts, which will evaluate the potential use of real-world evidence to help support the approval of new indications for approved drugs, consistent with the expectations outlined in the 21st Century Cures Act (21CC). The 21CC Act is designed to help accelerate medical product development and bring new innovations and advances to patients who need them faster and more efficiently. The FDA’s Real-World Evidence framework describes the sources of real-world evidence, gaps in data collection activities, standards, and methodologies for collection of the real-world evidence, and priority areas.
The MyStudies mobile device application and platform were developed to facilitate the input of real-world data directly by patients in a secure environment and to link patient-reported / generated data to electronic health data supporting traditional clinical trials, pragmatic trials, observational studies, and registries.
“FDA involvement with IBD Plexus provides our Real-World Evidence Program important experience with an exchange platform that uses multiple sources of health data, including real-world data,” said David Martin, MD, associate director for Real-World Evidence Analytics, Office of Medical Policy, FDA CDER. “In addition, collaborating with the Crohn’s & Colitis Foundation has allowed for improvements to the FDA MyStudies App that otherwise might not have occurred. The public will certainly benefit from these advancements as we evolve the field of real-world evidence through demonstration projects like this one.”
“IBD PROdigy will provide IBD Plexus cohorts the opportunity to fill in important information gaps critical to advance research,”Dobes added. “Periodic data collection that happens within the clinical delivery system only offers a snapshot into a patient’s disease. The app will allow us to fill in known missing care events, such as ER visits, and also incorporate patient’s symptoms and experiences. Linkage of this patient-reported data to clinical and molecular data then acts as a catalyst to augment research. These robust, multi-dimensional datasets will lead to stronger evidence to support and help expedite regulatory decisions.”
IBD PROdigy will launch in early 2020, making the Foundation the first patient organization to leverage the technology for research. Ultimately, the IBD PROdigy app and IBD Plexus will give:
- Researchers the ability to leverage real-world data to enhance clinical research efficiencies, including, but not limited to: reducing time and cost of study startup, such as patient recruitment; help support evidence development associated with regulatory decisions, including post-marketing safety studies; and opportunities for leveraging real world data to better understand hard-to-study sub-populations.
- Clinicians the ability to leverage data to create more holistic interventions based on findings from both clinical trials and real-world evidence in order to improve overall health of patients and inform decision-making to provide better care.
- Technologists the ability to leverage open source code developed during the implementation phase of the demonstration project, such as triggering of notifications and questionnaires based on a study event reported the electronic data capture system for a trial or registry, available at the FDA My Studies Mobile application Git Hub page.