On November 7, the U.S. Food and Drug Administration (FDA) announced the launch of the MyStudies app, the latest in a series of concrete activities to drive the integration of the patient voice in clinical trials and the drug development process. The new app is intended to gather data directly from patients through their mobile devices for clinical trials and research.
FDA Commissioner Dr. Scott Gottlieb said in a statement, “our hope is that the collection of more real world data directly from patients, using a secure app, will lead to more efficient product development and assist with safety monitoring."
Coming on the heels of the October 15-16 two-day FDA Patient-Focused Drug Development (PFDD) Workshop on Draft PFDD Guidances 2 and 3, during which a panel discussed the use and importance of digital health technology, the FDA has sent a clear signal that the agency is not only open to the use of digital apps to collect clinical trial data, but is promoting its own.
As described in the FDA report, the MyStudies App is “designed to facilitate the input of real world data directly by patients which can be linked to electronic health data supporting traditional clinical trials, pragmatic trials, observational studies and registries.” The FDA also recognized the importance of capturing patient input, data that is lacking through traditional data sources such as claims data. By releasing the tool, FDA aims to make it easier for stakeholders to collect patient input in a way that is both meaningful to inform FDA decision making and compliant with clinical research and regulatory requirements.
The FDA underscores the importance of the patient perspective in the design on this tool and the utility to drug development in saying:
A patient-centered app-based electronic method suitable for capturing the patient perspective and linking it to existing electronic health data that is scalable, secure, configurable, reusable, compliant with clinical research and regulatory needs, and capable of supporting defined research cohorts is necessary to expand the capacity of comparative effectiveness and drug safety research.
While Center for Drug Evaluation and Research’s (CDER) Office of the Director has typically led the charge in regards to PFDD activities, this new tool stemmed from the FDA’s Sentinel System, a national electronic system for monitoring the safety of products that are FDA approved, highlighting the Agency-wide dedication to advancing PFDD. The utilization of Sentinel in clinical trials of investigational drugs represents a new use for the program which proactively monitors the safety of FDA-regulated medical products.
As part of the launch, the FDA also released the source code and documentation of the MyStudies app so that it can be reconfigured and rebranded by organizations conducting clinical research. The FDA noted the importance of designing the app in a way that could be adapted for use in multiple therapeutic areas and a broad range of health outcomes research, and reduce the burden of software development for patient communities and non-FDA users seeking to gather patient experience data.
In recognition of the need for multi-purpose tools like the MyStudies app, the report notes that most of the survey instruments and tools that exist today are “typically purpose-built for one study and require significant programming expertise to reconfigure for new use... This project developed the first platform that enables research organizations to manage nearly all aspects of multiple smartphone-based multi-site clinical studies, including data collection and storage, via a point and click web-based configuration portal and a single mobile app.”
While the release of the source code will promote the use and uptake of the MyStudies app, the report adds that the “sponsoring organization and its vendors will need a variety of clinical and technical skills” and recommends that patient advisors, clinical experts, software developers and staff dedicated to central coordination be involved in implementation, project management, configuration and testing.
It is clear that the FDA sees enormous potential in this new app and states in the report, “given the scope of information provided by participants, the app appears capable of expanding the depth and diversity of “big” electronic health data for clinical research purposes.” The agency also aims to continue to improve the MyStudies app. “Over time, continued app development using information in the public domain will likely increase usability, enable integration of other real world data sources, and enhance the overall ease with which the patient perspective can be included in clinical research.”
The launch of the MyStudies app provides an exciting new resource and tool for patient organizations and drug developers gathering perspectives from patients and caregivers and is a sign that Patient Focused Drug Development remains a paramount FDA priority.