Regulators have been steadily increasing their use of real world evidence (RWE) to support decision-making. Simultaneously, access to real world data (RWD) continues to increase, and there’s growing clarity on how RWD can be used to generate relevant and reliable RWE. RWD can come from multiple sources including electronic health records (EHRs), claims data, and registries, and can be collected directly from the patient via connected devices such as wearables. Additionally, with more mature digital analytics, we now have the ability to draw insights from health-related data.
Against this backdrop, RWD and RWE are being used in even more applications across the product lifecycle, from proof of concept to patient care. In this three-part series, we’ll explore how RWE is being used to support regulatory decision-making for medical devices, describe some of the RWD sources available to medical device companies, and share some real-life case studies in which medical device companies are demonstrating value of their products through the use of RWD.
Increasing adoption of RWE to support regulatory decision-making
Over the last few years, regulators have been increasingly accepting the use of RWE in regulatory decision-making. In general, regulators are increasingly looking at how RWE can be used to support monitoring the safety, effectiveness, and performance of medical devices, as well as the long-term benefit/risk profile of products.
Bridging the evidence gap with RWE
U.S. regulators have stated that “By unleashing the power of RWE, we can accelerate medical product development and bring new innovations and advances faster and more efficiently to the patients who need them, without compromising patient safety."1
The following are examples of guidance documents that are supporting the industry in its ability to ‘unleash the power of RWE.’
- In 2017, the FDA’s Center for Devices and Radiological Health (CDRH) issued the Use of Real World Evidence to Support Regulatory Decision Making2 for Medical Devices. In it, the CDRH detailed the use of RWE for regulatory decision-making across the total product lifecycle (TPLC) for medical devices. The FDA explained that it would determine the suitability of RWD for use in its decisions based on the relevance and reliability of the source and its specific elements. The guidance also provides real examples of sources of RWD that were used to support regulatory decision-making. These include labeling expansion, post-approval surveillance, control groups, and supplementary data.
- The External Evidence Methods Working Group of the Medical Device Innovation Consortium (MDIC) published a draft framework on external evidence methods in January, 2021. “This program aims to establish a more predictable pathway for use of external evidence methods (EEM), such as new, innovative (Frequentist/Bayesian) methods and the cataloging of existing methods for evidence fusion from data external to a clinical trial. External trial data includes but is not limited to real-world data (RWD), real-world evidence (RWE), engineering modeling and simulation, similar device clinical trial data, to support regulatory medical device decisions and other stakeholder decisions.”3 Although this is a draft framework, it is already highlighting the interest in leveraging secondary data sources.
- Most recently, the FDA CDRH released a new report of their experience and guidance on how RWE sources from registries to claims data can be used to support marketing applications for medical devices. In the report, they give 90 case studies4 in which RWE was used to support final premarket or post-market regulatory decisions from 2012 through to 2019. These examples include premarket notification 510k submissions, De Novo classification requests, humanitarian device exemption (HDE) applications, premarket approvals, and continued access programs.
Regulators around the world are more accepting of RWE than ever before. With the increase in guidance frameworks, regulations, and publications, this is paving the way for multiple stakeholders to embrace it. Payers, for instance, can leverage RWE to assess the value of products and to monitor their use, physicians can use it to support and improve clinical decision making, and, ultimately, patients can better understand their treatment options and be more involved in their treatment decision making.
The next installment in our series details several datasets that may be useful to medical device developers in producing evidence for regulators and payers.
1 Shuren, Jeff, MD, JD, Director, Center for Devices and Radiological Health and Caños, Daniel, P:hD, MPH, Director, Office of Clinical Evidence and Analysis, Office of Product Evaluation and Quality
2 U.S. Food and Drug Administration (FDA), Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices: Guidance for Industry and Food and Drug Administration Staff
3 Medical Device Innovation Consortium (MDIC), External Evidence Methods Framework
4 FDA, Examples of Real-World Evidence (RWE) Used in Medical Device Regulatory Decisions
