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Leveraging RWE in regulatory submissions of medical devices: Three takeaways for medical device and diagnostic companies

Christian Howell
Vice President and General Manager of Medical Devices and Diagnostics, Aetion


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The U.S. Food and Drug Administration’s (FDA’s) Center for Devices and Radiological Health (CDRH) recently released a position statement on the use of real-world evidence (RWE) in regulatory decision-making for medical devices, as well as 90 examples of RWE supporting FDA approvals. This data both communicates the progress the FDA has made toward evaluating how RWE can inform decisions, and underscores the need for medical device and diagnostics manufacturers to continue to develop RWE strategies to strengthen FDA submissions. 

Building on the FDA’s 2017 guidance, “Use of RWE to support regulatory decision-making for medical devices,” this new information demonstrates how RWE can support decision-making across a range of submission types and data sources. According to the CDRH’s 90 published examples, data collected from devices, mobile apps, and registries all provided RWE to support approvals.   

In FDA Voices, authors Jeff Shuren, M.D., J.D., Director of the CDRH, and Daniel Caños, Ph.D., M.P.H., Director of the Office of Clinical Evidence and Analysis within the CDRH’s Office of Product Evaluation and Quality, shared how the use of RWE has grown within the medical device space. Read on to learn takeaways from the piece, and how medical device, in-vitro diagnostic, and digital therapeutic companies can select the right RWE partner to help prepare their evidence generation capabilities. 

  1. The time for RWE in the medical device and diagnostics space is now, and companies should be deploying robust RWE strategies to keep up with the pace of innovation.
    While data from randomized controlled trials (RCTs) provide useful information on product safety and efficacy, they’re not suitable in all situations—for example, they sometimes pose practical limitations depending on the intervention or condition in question. RWE can complement trial findings by providing needed flexibility in study design, and by more clearly portraying how a device performs in real-world patient populations who may not be able to participate in traditional clinical research.

    The demand for RWE is intensifying, and decision makers across the product lifecycle—regulators, payers, providers, and physicians—expect that the evidence needed to inform critical decisions can be found in real-world data (RWD).

    To better equip themselves for this shift, medical device companies should develop robust strategies to generate high-quality RWE. The examples provided by CDRH show the range of impact and value RWE can have across the product lifecycle, as well as the importance of transparent evidence that is auditable and replicable. It is essential that medical device companies seek RWE partners and platforms that can drive meaningful, validated, and expedited RWE generation through a traceable process—which regulators and other reviewers can use to pressure test the analyses and ensure the evidence is credible.
  1. Advances in RWD have made the increased use of RWE possible. When selecting RWE solutions, medical device manufacturers should choose partners and platforms that allow them to harness the full breadth of available data.
    In recent years, the amount, diversity, and quality of RWD sources available for analysis has increased, and now includes electronic health records, registries, medical claims, pharmacy data, and output from wearables and mobile technologies. As a result, the advancing data landscape has also allowed manufacturers to generate more—and better—RWE.  

    Understanding the RWD sources available, as well as their strengths and limitations, is key. When selecting an RWD source to address a research question, one must ensure it captures the necessary information, taking into account device identifiers, data provenance, population fit, analysis purpose, and access challenges. Leveraging an RWE platform that can transform disparate forms of RWD into decision-grade evidence can help address many of these questions and solve this—oftentimes confounding—RWD riddle.
  1. Medical device and diagnostics companies must make RWE models accessible to stakeholders across the product lifecycle to actualize the wide-ranging impact of RWE.  
    The spectrum of potential applications for RWE is broad: it can support premarket approvals (PMAs), inform label expansions, and evaluate products’ safety and effectiveness once they hit the market. These wide-ranging impacts are illustrated in the CDRH’s 90 examples of RWE-supported regulatory submissions: for instance, Drs. Shuren and Caños shared how two post-approval studies using data from the United Network for Organ Sharing Registry helped approve a PMA to expand the use of a portable normothermic perfusion system for donor lungs.

    Given this context, RWE has critical applications across device development. For instance, in early development, RWE supports understanding of the standard of care, unmet need, and the burden and natural history of a disease. Manufacturers can further leverage RWE to optimize clinical trial design, as well as to supplement RCT data in regulatory submissions. Within the realm of HEOR and market access, RWE can inform budget impact and cost-effectiveness models for relevant populations. And the postmarketing applications of RWE are numerous, spanning uses in safety (e.g., pharmacovigilance), commercial (e.g., head-to-head effectiveness and safety studies, and monitoring safe and effective off-label use), and beyond.

    To support this broad range of RWE applications, medical device and diagnostics manufacturers must design RWE models that can scale to a similarly broad range of stakeholders—and structure teams, select partners, and enlist technology solutions that can support this work.

Organizing for action
As the FDA continues to use RWE to inform its decision-making, medical device companies must be ready to meet the moment with a clear RWE strategy. This includes leveraging RWE platforms that can integrate an array of RWD sources, and that are optimized for stakeholders across the product lifecycle. With these tools at the ready, organizations can generate RWE that meets regulatory requirements, informs future RWE standards, and, ultimately, improves patient access to the interventions they need. 

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