FDA Approvals Are a Matter of Life and Death

Today the medical-device industry flourishes overseas, even as it struggles under unnecessary regulatory burdens in the U.S.

In November, the U.S. Food and Drug Administration (FDA) approved an innovative product called the Sapien Transcatheter Heart Valve, for the treatment of severe aortic valve stenosis. The Sapien valve can be implanted endoscopically, making it a boon for patients who are too sick to endure open-heart surgery. Clinical trials found that almost 70% of patients receiving Sapien valves were alive one year after treatment, while only 50% of those receiving alternative treatments survived that long.

This would be a great story for American patients, but for one frustrating detail: The Sapien valve has been available in Europe since 2007, saving lives there but not here.

Unfortunately, this delay was not exceptional. American patients used to be the first to benefit from their country’s enormous investments in basic medical research. Today, Americans wait as much as 60% longer than they did in 2005 for new lifesaving and life-enhancing medical devices—such as stents that keep arteries open and defibrillators—to reach the market, according to a recent Government Accountability Office report. During that wait, many remain sick or disabled. Some die.

One significant cause of this delay is the FDA and a decades-old regulatory process that is outmoded and needlessly long. Advances in science and new technologies offer opportunities to update FDA regulations in need of reform. If the agency modernized its premarket approval process, for instance, it could grant health-care professionals faster access to many medical devices with proven benefits and acceptable safety profiles, and then monitor the performance of those devices once they are in use.

This process could start with a better determination of which benefit-and-risk questions can be answered best by faster and lower-cost nonhuman studies—and which require human testing. The FDA can also provide faster reviews of clinical-trial protocols that focus on ensuring patient choice. There is a compelling argument for letting patients and their doctors decide what risks to take. Given the tremendous gains in quality of life and life expectancy associated with medical innovation, it’s easy to predict what many patients would choose.

Yet these approaches won’t work until the FDA’s premarket approval process is modernized in three areas:

• Developing alternatives to clinical trials. Clinical trials have been a traditional part of medical-product regulation, but they are of less value for evaluating medical devices than they are for drugs. The mechanical, electrical or physical properties of devices can often be more robustly tested in the lab than on people.

A small number of recent, high-profile recalls of medical devices have garnered tremendous media attention and led to calls for stricter FDA regulations. But a close analysis of the data demonstrates that serious device problems are rare. According to a six-year study by Ralph Hall, more than 99.5% of medium-risk and high-risk devices—such as heart valves and orthopedic implants—are never subject to Class I recalls, which involve the highest risk of injury or death. Of the very few Class I recalls that do occur, fewer than 9% are for problems that more clinical testing could have discovered.

In the future, instead of implementing more clinical trials—with their long time frames, high cost and human risk—the FDA should enhance its "quality system" regulation process, and make more use of bench testing and computer modeling to identify potential clinical problems. The FDA should also expand its recently-announced Innovation Pathway 2.0, which seeks to develop a faster approval process for breakthrough devices—for kidney-failure patients, for instance—by encouraging collaboration between product developers and FDA experts throughout the development process.

• More and better postmarket surveillance. When devices are recalled, it’s usually because of problems in the quality system, such as manufacturing or labeling errors, or long-term product-performance issues. Yet the FDA’s system for tracking these failures is currently largely passive, based on reports the FDA receives from manufacturers, which in turn received them from providers and patients.

With advances in wireless communications, sophisticated information-sharing systems, and electronic medical databases, the FDA can create a proactive system of surveillance—in real time in some cases—that would greatly enhance safety by identifying patients who benefit the most from the device, detect early signs of device failure, and allow rapid mitigation of potential harm to patients.

• Collaboration. Finally, both before and after approval, the FDA, industry and other stakeholders need to work together more. The reauthorization of the Medical Device User Fee and Modernization Act—which will provide better and more transparent metrics for evaluating the FDA’s performance, and some limited reforms to premarket review—is only a start. We need a public-private partnership in which regulators, researchers and industry experts can learn from each other and develop patient-centered regulatory standards, focus on the most-serious safety challenges, and identify the greatest opportunities for medical-device innovation.

The consequences of not doing so are clear. Today the medical-device industry flourishes overseas, even as it struggles under unnecessary regulatory burdens in the U.S. A 2010 study found that European patients gain access to "new U.S.-created medical technologies" more than two years earlier, on average, than American patients. Not surprisingly, the device industry is leaving. According to a summer 2011 survey by the National Venture Capital Association, in the next three years, 85% of venture-backed health-care companies expect to seek regulatory approval for their new products outside the U.S. first.

With assets like the National Institutes of Health, cutting-edge academic medical centers, vibrant venture-capital networks, and a vast industrial infrastructure, the U.S. has no excuse not to lead the world in providing new medical devices. American workers should be employed in this vital industry; American investors should profit from it; and most of all, American patients should benefit from it, in the form of better health and longer life. But that won’t happen if we saddle the industry with regulations that are slow, costly and ineffective.

The medical-device industry is valuable. If the U.S. doesn’t want it, someone else will. For patients, meanwhile, the only alternative to improving the regulatory process is the current system of withholding products, when delay can mean death, disability or "just" a diminished quality of life.

This piece originally appeared in Wall Street Journal

This piece originally appeared in The Wall Street Journal