Regulatory Guidance / Compliance Management

Building a bridge to tomorrow's medicine

Advancing FDA's Real-World Evidence Framework for evaluation, monitoring and regulation

By Jacqueline Corrigan-Curay, J.D., M.D. Director, Office of Medical Policy, CDER, U.S. FDA

Screen Shot 2019 04 22 at 12.23.10 PMBig data has the potential to be a powerful tool for accelerating the development of therapeutics along with ensuring the delivery of the right medical care to the right patient at the right time. Continual advances in data acquisition from clinical trials and clinical care should lead to new opportunities to optimize the assessment of product safety and efficacy throughout product lifecycle.

At the U.S. Food and Drug Administration (FDA), our continued exploration of how to use real-world data (RWD) to develop real-world evidence (RWE) is part of a larger commitment to employ innovative tools and approaches to streamline product development.

RWD are data relating to patient health status and/or the delivery of healthcare. It is routinely collected from a variety of sources, including electronic health records, insurance claims, product and disease registries, and wearable devices. RWE is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. Optimizing the use of RWE — including within clinical trials —will inform product research, development, regulation, and post-market monitoring.

FDA believes that a well-designed and executed RWE initiative, rigorously developed through strategic, interdisciplinary collaboration, can ultimately improve treatment decisions and enhance patient care.

By breaking down the barriers between data derived from clinical trials and data generated in clinical care, we build a bridge toward a future of seamless therapeutic development and responsive, patient-centered clinical care powered by a variety of relevant evidence and data, including RWE.

In December 2018, the FDA released a strategic Framework designed to evaluate the use of RWE for regulatory decisions. We outlined key actions that we will take including sharing new tools and collaborative opportunities to advance the science.

Braced for transformative change and focused on addressing complex scientific and technical challenges, FDA has set out to work with all involved stakeholders including pharma manufacturers, agency scientists and managers, and patient-advocacy groups. Together, we are working to conceptualize new platforms, methodologies, templates, and strategies for optimizing the potential use of RWE to support regulatory decisions. Here are a few examples of our ongoing activities:

A digital tool for RWE: In November 2018, FDA launched the MyStudies app, which is designed to help capture RWD from patients. This tool will facilitate data acquisition and analyses to support regulatory decision-making. Based on a study demonstrating the functionality and engagement of the mobile app, FDA made the source code publicly available to allow other stakeholders to customize the tool for RWE.

Community practice and adoption of labeling changes: Drawing from RWD gathered through the FDA’s Information Exchange and Data Transformation (INFORMED) infrastructure, FDA is exploring how community practices are adopting label changes for two approved products from weight-based to flat-dosing of immune checkpoint inhibitors.

Standards, methods and guidance: FDA is developing guidance on assessment of standards and reliable methods for collecting and analyzing RWE.

A Sentinel demonstration: FDA launched the first randomized, controlled trial within the Sentinel system that is generating data about the effectiveness of an educational intervention designed to improve the health of patients with atrial fibrillation.

The agency will continue to create shared learning opportunities across divisions and develop new guidance documents to inform our stakeholders. FDA will ensure that lessons learned from supported demonstration projects are used to inform future steps.

FDA recognizes that if RWD can reliably generate high-quality RWE, it could provide significant benefits for all stakeholders, including industry, scientists, and the public. We will continue to work diligently across public and private sectors to bring the scientific, technological, and regulatory changes needed to drive this transformation.

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