FDA Editorial Justifies Approving Some High-Risk Medical Devices Without Clinical Trials

Plans to streamline the regulatory process for approving medical devices could involve stepping away from randomized double-blind clinical trials, which are considered a “gold standard” for medical research. However, the move by the FDA has come under fire from consumer watchdog groups and some health experts. 

In an editorial published in the New England Journal of Medicine on April 6, officials with the FDA defended the controversial new direction of the agency, indicating that it needs to allow flexibility in clinical data gathering in certain cases.

The editorial comes just days after the FDA published a notice in the Federal Register, indicating that it intends to allow certain new Class II medical devices to be approved without having to undergo premarket notification requirements, in accordance with the recently passed “21st Century Cures Act.”

The proposal has raised concerns that medical devices may hit the market without even a modicum of review, allowing many more products than before to be approved through an even less restrictive version of the FDA’s controversial 510(k) program, meaning there would be no clinical trials or review, and approval would be based on whether they are substantially equivalent to a device already on the market.

Many critics have urged the FDA to maintain clinical trials as the benchmark for medical device approval, and the FDA acknowledged the strength of randomized clinical trials in its comments.

“For many devices, however, practical limitations related to the device or disease condition require alternative approaches to conducting large, randomized, controlled, double-blind studies and increased flexibility in trial design and statistical analysis,” the editorial argues. “For the majority of devices, the benefits and risks are expected to be manifest through registries and evolve as clinical techniques are refined and the technologies themselves are rapidly modified and improved. Such a continuous improvement cycle would be impossible if every device iteration required a full trial to test its safety and efficacy.”

Consumer and patient safety advocates have already raised serious concerns about steps the FDA has taken that make it too easy for untested medical devices to reach the market in the U.S.

Many devices that are approved based on claims that they are “substantially equivalent” to existing devices, are marketed as featuring superior features, new manufacturing processes, materials, and surgical techniques that have never been seen or used before. Critics warn that the “21st Century Cures Act” will make it even easier for dangerous and defective medical devices to reach the market.

Rolling back regulatory oversight may further erode the ability of the FDA to protect patients from dangerous products, according to consumer advocates. A number of examples in recent years have highlighted the risks consumers face when untested devices are widely adopted in the medical field before dangerous defects are discovered based on post-marketing adverse event reports, essentially making U.S. patients unwilling test subjects.

Despite questions about the latitude already provided to manufacturers, the 21st Century Cures Act loosens the approval process further, allowing the agency to approve drugs and medical devices that have not had adequate clinical trials to ensure they are safe and effective, critics warn.

However, the FDA editorial says that the agency is not abandoning clinical trials, but is seeking more flexibility in approving new devices.

“The FDA works with sponsors to develop a clinical trial design and statistical analysis approach that is best tailored to the technology, the medical need being addressed, the feasibility of data collection, and the benefits and risks to affected patients,” the agency noted. “In some cases, the FDA expects and is provided with clinical data from trials that are similar in design to a ‘gold standard’ drug trial — large, blinded, randomized, controlled trials. For many devices, however, such designs are impractical or unnecessary.”

Some say the FDA is already struggling to adequately confirm the safety and efficiency of medical devices on the market today or under development. A study published in the Journal of the American Medical Association in August 2015 found that only a fraction of post-market clinical trials required by the FDA for high-risk medical devices are actually being completed.

Another analysis, published earlier in 2015 in The BMJ, warned that researchers found numerous discrepancies in data submitted to the FDA by medical device manufacturers seeking premarket approval for cardiovascular devices. Those discrepancies often included the number of participants varying in the actual study from the number reported to the FDA, substantially different results from similar FDA studies, and many of which were never peer-reviewed.