FDA Used Real-World Evidence in Heart Valve Approval

Regulatory News

The US Food and Drug Administration (FDA) says it relied on real-world evidence in its decision to expand the use of Edwards Lifesciences' Sapien 3 transcatheter aortic valve replacement (TAVR) to valve-in-valve procedures.

In a blog post Wednesday, Center for Devices and Radiological Health (CDRH) Director Jeffrey Shuren and Bram Zuckerman, director of CDRH's Division of Cardiovascular Devices touted the agency's turnaround from 42nd in the world to approve a TAVR device, to being the first to clear the Sapien 3 for valve-in-valve use.

The approval comes two years after FDA cleared Medtronic's CoreValve and Edwards' Sapien XT for valve-in-valve TAVR procedures, both of which relied on data from clinical trials, and several years after those devices were CE marked for valve-in-valve use in Europe.

According to Shuren and Zuckerman, the decision to expand the use of Sapien 3 was based on data from the Transcatheter Valve Therapy (TVT) Registry, which contains records for TAVR procedures in more than 100,000 patients, 600 of which were for off-label valve-in-valve procedures.

"FDA relied on real-world evidence to evaluate the benefits and risks of this off-label use—such as the safety of the procedure, the function of the valve, and the improvement of patient symptoms—to approve the new indication for Sapien 3," Shuren and Zuckerman write.

According to Shuren and Zuckerman, leveraging the data from the TVT Registry made it possible "to accumulate more data faster, without the need for costly and time-consuming formal clinical trials."

The two officials said the approach could be used to expand the use of other devices with "robust" registries, and plays into FDA's plans to establish a National Evaluation System for health Technology (NEST) that uses registries, electronic health records and claims data to inform FDA's decision-making.

Shuren and Zuckerman also said that FDA's efforts to encourage device makers to study their devices in the US are starting to show results.

"US medical device companies have long been accustomed to going overseas to conduct early feasibility studies (including first-in-human studies) for new heart valve devices, securing marketing authorization in other countries, and then returning to the US for pivotal clinical trials before FDA approval," they said.

But, Shuren and Zuckerman said, "many heart valve companies have already responded" to the agency's efforts to boost the number of early feasibility studies conducted in the US.

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