FDA Credits Device Trial Redesign for First-in-the-World Valve Approval
A more practical, streamlined approach to device regulations by the FDA is credited with helping make the agency the first in the world to approve a new heart valve.
When the agency approved the first transcatheter heart valve, the Edwards Sapien THV, nearly six years ago, the United States was the 42nd nation in the world to do so, according to Jeffrey Shuren, M.D., Director, CDRH and Bram Zuckerman, M.D., Director, Division of Cardiovascular Devices, CDRH.
Since then, the FDA has refocused its device priorities, emphasizing patient access, real-world evidence and transparency in agency expectations. Shuren and Zuckerman credit these improvements for the FDA’s recent approval of the latest Sapien valve, the Sapien 3—this time, the first national regulatory agency to do so.
The first step in the process was rethinking the agency’s non-clinical testing requirements. The FDA improved transparency, predictability and consistency during the process and as a result, trimmed the time before devicemakers could initiate clinical studies. The agency also stepped up its collaboration with industry stakeholders on trial design to ensure the design produced data that applied to the device’s intended patient population.
Since the first valve’s FDA approval in 2011, more than 600 patients have engaged in off-label, valve-in-valve use, which allows for use of the device without open heart surgery through either a small cut in the patient’s chest or a blood vessel; during the approval process for the Sapien 3, the FDA incorporated real-world evidence to evaluate the safety and effectiveness of this use.
Cause for Optimism
This methodology is cause for optimism in the device industry, Zuckerman and Shuren write, and could lead to further expansion of approved uses for other devices, but only if “robust registries” are available.
The agency’s creation of the National Evaluation System for Health Technology (NEST) will also be key to creating more opportunities to incorporate real-world evidence into the device approval process, according to the post; NEST will weigh device safety and effectiveness based on a combination of clinical registry, electronic health record and medical bill data.
CDRH has also launched a program to incentivize early feasibility studies for new devices, according to the blog post. Through these efforts, the FDA hopes to reverse the trend of outsourcing during the trial and feasibility study process. Device manufacturers have increasingly conducted feasibility studies overseas in recent years, according to Zuckerman and Shuren, securing their marketing authorizations overseas first and returning to the United States for clinical trials and final FDA approval.
However, many manufacturers have already committed to remaining in the United States as a result of the program, which will give device makers the option to conduct early clinical testing of devices to provide initial clinical safety data, an important option in scenarios where nonclinical testing methods are not available or appropriate.
By giving device makers an alternative to outsourcing, the program will save travel costs, streamline communications and allow for more early interactions with FDA officials, according to the blog post.