As FDA offers breakthrough designation to device makers, patients and providers are left with questions

Since launching in 2017, an ambitious and secretive Food and Drug Administration program to speed up medical device development has helped at least 44 authorized devices hit the market.

There are now more than 650 devices that have earned “breakthrough designation,” a signal that the agency believes — based on early data and the options currently available to patients — that a product could one day be “more effective” at treating or catching serious or even fatal conditions. STAT has identified details on more than two-thirds of those products in an authoritative new database, the Breakthrough Device Tracker.

The database sheds light on what happens after the FDA designates a new device a “breakthrough,” a process that has remained largely out of sight for the patients and providers the products are supposed to help. For the first five years of the program, the FDA did not publish the list of devices used in patient care that benefited from breakthrough status. On Friday, the agency released a list of those products for the first time, following months of requests from STAT as part of an investigation into how financial motivations have warped the goals of the agency’s “breakthrough devices” program.

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The agency still does not publicly disclose how and why a device earned the designation. Experts say that information is critical to help make decisions about patient care and reimbursement for new technologies, especially because the program allows some devices to go to market with more uncertainty about risks and benefits.

“The [program’s] intentions are terrific, they’re noble. They’re exactly what everyone should want to see: That we get innovations to patients if they help to improve outcomes,” said Sanket Dhruva, a cardiologist and health policy researcher at the University of California, San Francisco. “It still remains to be seen if the reality is meeting that goal.”

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Companies with breakthrough status still have to jump through regulatory hoops if they want to make it to market. But they get extra help navigating that process, including the option to talk more often with FDA officials, have more flexibility in how they design clinical trials, and receive a speedier review when they file for FDA authorization.

“The whole focus of the program is to provide a high-level engagement between the FDA and device innovators, with the goal of speeding the development and evaluation of products that can make a real difference where there is no good alternative for patients,” said Owen Faris, principal deputy director of the Center for Devices and Radiological Health’s Office of Product Evaluation and Quality, the FDA branch tasked with medical device oversight.

But because those conversations between regulators and companies often happen behind closed doors, and because the FDA does not make a practice of publicizing information on the breakthrough process, experts say it can be difficult to know the true risks and benefits of devices.

“The two sides of the coin for caring about patients is wanting access to new technologies and then making sure that they’re actually vetted appropriately and that we’re confident that we’re giving patients access to technologies that are actually to make them healthier,” said Ariel Stern, a health economics and technology researcher at Harvard Business School.

To be authorized, devices have to show the FDA that they will be safe and effective. For high-risk devices like cardiac implants, that often requires studies of hundreds of patients who are followed over time. But for breakthrough devices, there’s one significant difference: Because solutions are desperately needed by patients, the FDA will sometimes accept more uncertainty than usual about those results, with a commitment from companies to continue researching the devices after they’re authorized. But those studies may be far from done when products hit the market — meaning patients and their providers, in some cases, are making decisions about whether to use a device without complete data. And they’re not designed to answer the question of whether a breakthrough device delivers on its promise to be “more effective.”

“Is there any benefit to these products? How can we know whether they actually work, and work better, to improve the lives of patients?” said Joseph Ross, a physician and health policy researcher at the Yale School of Medicine. “There’s a lot of products that we just don’t even know.”

Health policy experts are calling for more transparency into how the breakthrough program runs, both to inform patients’ decisions and drive more effective regulation. By learning not only which devices get breakthrough status, but also how they won the label, policy researchers can better examine whether the FDA’s bets paid off in the way they intended.

With only limited information available about breakthrough devices, though, some experts worry that patients and providers may assume device makers have definitively proven their products are “more effective” than alternatives.

Real answers to that question could take years, said John Doucet, a regulatory consultant at MCRA who previously led policy for the breakthrough device program at FDA. “I think it could take quite a long time for us to really understand whether a breakthrough device that is designated is truly more effective than standard of care,” he said, through research directed by physicians and payers. The FDA can only require studies directly comparing products if a breakthrough intends to market itself as a better solution. “The FDA will not be doing that science, almost certainly,” said Doucet.

“It would be helpful if we were able to understand what the comparative benefit is compared to the standard of care,” said Dhruva. “Are we getting innovation that’s going to help patients? And I think it’s just too early to say.”

Source: STAT