JMIR Publications

ABSTRACT

Background:

The Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) established the Biologics Effectiveness and Safety (BEST) Initiative with several objectives, including to expand and enhance CBER’s access to fit-for-purpose data sources, analytics, tools, and infrastructure to improve the understanding of patient experiences with conditions related to CBER-regulated products. Due to existing challenges in data collection, especially for rare disease research, CBER recognized the need for a comprehensive platform where study coordinators can engage with study participants and design and deploy studies, while patients and/or caregivers could also enroll, consent, and securely participate in those studies. This would support data capture for clinical trials, pragmatic trials, observational studies, disease registries, and more.

Objective:

To describe the design and development of the Survey of Health and Patient Experience (SHAPE) Platform, and its data collection support for health research and patient experience studies.

Methods:

We used an Agile-based process that engaged multiple stakeholders in SHAPE’s design and development. SHAPE is hosted in a Google Cloud environment and consists of three parts: the administrator application, participant application, and application programming interface. The administrator can build a study consisting of a set of questionnaires and self-report entries through the application. Once the study is deployed, the participant can access their app, consent to the study, and complete its components. To build SHAPE to be scalable and flexible, we leveraged the open-source software development kit, Ionic Framework. This enabled building and deploying applications across platforms, including iOS, Android, and Progressive Web Apps from a single codebase, using standardized web technologies. SHAPE has integrated with a leading Fast Healthcare Interoperability Resources (FHIR®) API platform, 1upHealth, which allows participants to consent to a one-time data pull of their electronic health records (EHRs).

Results:

SHAPE allows study coordinators to plan, develop, and deploy their questionnaires to obtain important endpoints directly from patients or caregivers. The EHR integration enables access to the patient’s health records, which can validate and enhance the accuracy of data capture methods. The administrator can then download the study data in FHIR®-formatted JSON files. In this paper, we illustrate how study coordinators can use SHAPE to design patient-centered studies. We demonstrate its broad applicability through a hypothetical type 1 diabetes cohort study and an ongoing pilot study on metachromatic leukodystrophy to implement best practices for designing a regulatory-grade natural history study for rare diseases.

Conclusions:

SHAPE is an intuitive and comprehensive data collection tool for the full variety of clinical studies. Further customization of this versatile and scalable platform allows for multiple use cases. SHAPE can capture patient perspectives and clinical data, providing regulators, clinicians, researchers, and patient advocacy organizations with data to inform drug development and improve patient outcomes.