New Real-World Evidence Tools for FDA Approvals

If you are a medical device manufacturer seeking premarket approval (PMA) for your device, your tool chest for evidence gathering just got a lot bigger.

The 21st Century Cures Act required the FDA to develop a framework and guidance for evaluating real-world evidence (RWE) and data (RWD) in approving new medical devices and medicines. In July 2016, FDA released a draft guidance on acceptable uses for RWE in the established medical device regulatory scheme.

On August 31, 2017, the agency released the final guidance.

This may turn out to be a big deal. The old rules regarding clinical studies still apply, but the guidance vastly expands the body of evidence the agency may now use in determining whether to approve medical devices. Traditional evidence has been from clinical studies conducted and provided to the FDA by the device sponsor. But the FDA knows there is a lot more out there. Sponsors can use the additional data to aid the FDA in reaching an approval decision.

Real-World Data

The guidance states that RWD includes data derived from electronic health records, claims and billing data, data from registries, patient-generated data including in home-use settings, and data gathered from other sources. The RWD sources can be used to support trial designs from randomized trials to observational studies. RWE can range from observational studies within an existing dataset to studies that incorporate planned interventions with or without randomization at the point of care.

“FDA recognizes that a wealth of data covering medical device experience exists and is routinely collected in the course of treatment and management of patients,” the agency said. Under certain circumstances RWD may constitute RWE, FDA says, “that may be of sufficient quality to help inform or augment FDA’s understanding of the benefit-risk profile of devices at various points in their life cycle, and could potentially be used to aid FDA in regulatory decision-making.”

The agency also noted that the guidance is a “cornerstone” of its strategic priority to build and implement the National Evaluation System for health Technology (NEST).

The FDA will use the guidance to evaluate whether RWD are of sufficient quality to support its regulatory decision-making. The agency may also use the aggregation of RWE to inform and implement postmarket control programs. However, the agency notes the new guidance “does not alter, or change in any way, the existing evidentiary standards use in regulatory decision-making.”

The bottom line. If you are planning a premarket approval application, consult with the FDA to see how RWE and RWD can be incorporated into your clinical design trials and evidence gathering process. Don’t leave an important new tool in the box.

To study the guidance, click here.