David Martin

	One of the most powerful and promising tenets of modern medicine is the idea that the patient experience is at the heart of clinical care. At the U.S. Food and Drug Administration (FDA), we are committed to listening to patients and to advancing evidence-based tools that support patient-centered medicine.

	Last year, the FDA introduced MyStudies, a digital platform for capturing the patient experience and bringing it -front and center- to drug development and regulation. The new mobile technology is designed to facilitate the collection of real-world data (RWD) directly from patients in conjunction with a variety of data sources, such as clinical trial data capture systems, electronic health records, insurance claims, product and disease registries, and potentially patients’ other digital health technology such as wearables. While serving as a patient source of RWD, these valuable data streams can inform clinical trial development and real-world evidence (RWE) studies. RWD can also lead to new medical product development and inform regulatory decision-making.

	A well-developed evidence base that uses patient experience can make product development more efficient and innovative. Patient experience data can also contribute to monitoring the safety profile of drugs after they have been marketed. Ultimately, better incorporation and enhanced understanding of the patient experience can improve drug development.

, which was first demonstrated at the American Medical Informatics Association Annual Symposium in November 2018, has important features supporting end-user accessibility, patient privacy, and configurability.

		Two versions of the app are available. One version is built on Apple’s ResearchKit framework, which runs on iOS. The Android version is built on the open source ResearchStack framework.

		The secure patient data storage environment supports auditing requirements for compliance with 21 CFR Part 11 and the Federal Information Security Management Act, allowing the app to be used for trials under Investigational New Drug (IND) oversight.

		The data storage environment is partitioned ensuring that each site can only access data from patients enrolled at that site. This supports multi-site trials or “distributed database” studies.

		To abate software development hurdles for non-FDA users, the app is configurable for different therapeutic areas and health outcomes.

	Paving the way for redesign and rebranding, the FDA released the app’s open source code and technical specifications on GitHub, a resource for software developers. After re-branding, healthcare research organizations can freely tailor the app to fit their needs. Health IT developers can also use the existing code as a framework for new development work that could expand the capabilities and functionality of the app.

	The MyStudies project was born from a directive of the Affordable Care Act of 2010 (ACA) requiring that the U.S. Department of Health and Human Services (HHS) build data capacity for patient-centered outcomes research. The project was supported by an investigator-initiated grant from HHS’s Patient Centered Outcomes Research Trust Fund.

	The app’s open source code and technical specifications were developed in collaboration with Harvard Pilgrim Health Care Institute, LabKey, and Boston Technology Corporation.

	Conceptualized with multisystem linkage in mind, the MyStudies project was nested within the 

 program, which leverages the FDA’s Sentinel infrastructure and includes interactions and/or interventions with patients and/or medical providers. The data storage environment includes appropriate partitions that support privacy-sparing approaches to data linkage whether there are one or many sites or data sources.

	In partnership with the Kaiser Permanente Washington Health Research Institute, the app’s functionality and engagement were successfully tested in a

 exploring medication use patterns and health outcomes among pregnant women. Participants reported that they were taking fewer prescription drugs and more over-the-counter drugs than the existing electronic health data sources captured. They also reported on sensitive topics such as alcohol use prior to and during pregnancy. Suggestions for improvement were also collected from a subset of the participants.

	Listening and responding to the patient experience

	The MyStudies project is an example of the FDA developing technology that can collect patient-reported outcomes and support patient-focused drug development.

	The MyStudies platform is the mobile technology accompaniment to other FDA efforts aimed at incorporating patient voice and experience into product development and regulation. For example, in 2015, the agency established the Patient Engagement Advisory Committee (PEAC) to help assure the needs and experiences of patients are included as part of the FDA’s deliberations on complex issues involving the regulation of medical devices and their use by patients. PEAC is the FDA’s first and only advisory committee whose members are all patients, caregivers, and representatives of patient organizations.

	The FDA has also conducted Patient-Focused Drug Development (PFDD) meetings in more than 20 disease areas, including opioid use disorder, autism, HIV, Parkinson’s disease, and various conditions involving pain. Building on lessons learned from the PFDD meetings, the FDA has released several 

 addressing how stakeholders can collect and submit patient experience data and other relevant information to support product development and regulatory decision-making.

	The MyStudies platform provides a technology tool that could support these patient listening efforts and help actualize the modern tenet of patient-centered care. In addition, this mobile technology has the potential to support RWE studies by capturing patient-reported outcomes that are not available in other RWD sources and expanding the range of endpoints available for study. Finally, FDA MyStudies can enhance convenience for clinical trial participants.

	Since the code for the FDA MyStudies app and its associated web-based configuration portal were made publicly available on GitHub, 22 groups have cloned the code for their own purposes. In addition, a five-hour webinar coordinated by FDA’s Small Business initiative had 1,956 registrants from 60 countries on six continents, demonstrating broad interest in this new public resource. Recently, the FDA MyStudies app was rated #1 by both the audience and the “sharks” at an Emerging Technologies “Shark Tank” session during a Health Innovation Forum, an event sponsored by the American Council for Technology and Industry Advisory Council (ACT-IAC). ACT-IAC standardizes and promotes best practices for information technology across the entire federal government and now includes industry partners. Apple Inc. also highlighted the app at its 2019 annual worldwide developer conference.

	The FDA MyStudies app is currently being re-branded for two projects: one in collaboration with the Patient Centered Outcomes Research Institute (PCORI), and the other in collaboration with the Crohn’s and Colitis Foundation (CCF). The PCORI-funded Limit JIA randomized controlled trial will enroll 306 patients with Juvenile Idiopathic Arthritis, and the CCF-funded SPARC Registry will enroll approximately 900 patients per year over a multi-year period. These projects will advance public health and further our knowledge and confidence in use of mobile technologies in clinical trials and registries.

	As the MyStudies platform continues to evolve in the public and private sectors, and as we continue to bridge streams of RWD to inform RWE - informing product development, regulation, safety monitoring and clinical care - we believe that this emerging technology will move the nation closer to realizing the possibilities of patient-centered medicine.

 is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, Center for Drug Evaluation and Research, for the FDA

Articles

MyStudies, introduced by the FDA, bridges data streams to real world evidence by facilitating the collection of real-world data directly from patients in conjunction with a variety of data sources. 

David Martin

Articles

MyStudies Platform Brings Patient Experience to Drug Development