Dive Brief:

• FDA has finalized guidance on its approach to managing uncertainty when assessing the risks and benefits of medical devices submitted for approval.

• The document, published Thursday, hews closely to the draft FDA released for consultation last year. Trade group AdvaMed pushed for several changes to the draft, with mixed success. 

• While FDA tweaked parts of the document in line with the feedback, it maintained its plan to hold advisory committee meetings on postmarket data and kept other contested proposals.

Dive Insight:

The guidance exists because FDA must manage uncertainty when deciding whether to authorize a device, be it through premarket approval (PMA), De Novo classification or humanitarian device exemption (HDE). To ensure timely patient access to new devices, FDA weighs the probable benefits and risks they pose, rather than requiring companies to generate exhaustive data preapproval.

That is particularly true of breakthrough devices, which are therapeutically important enough that FDA may be willing to accept more uncertainty. To manage the uncertainty, FDA’s draft guidance said companies may have to gather postmarket data and discuss it at advisory committee meetings. Parts of that proposal met resistance from the industry. 

“We do not believe that advisory committee meetings should be the Agency’s default mechanism,” AdvaMed wrote in its feedback. “Advisory committee meetings may be needed in some cases, but in all cases they are massively time and resource intensive. It is not unusual for sponsors to commit thousands of hours and hundreds of thousands of dollars (or more) to preparing for these meetings.”

FDA softened its language following the feedback but advisory committee meetings remain central to its plans. In the draft, FDA said it “intends” to hold advisory committee meetings. In finalizing the test, FDA replaced “intends” with “generally expects.” 

The change is one of several revisions that partly address issues raised by AdvaMed. The trade group successfully called for FDA to expand on its approach to the development of devices for small patient populations by stating it will make decisions on a case-by-case basis using certain criteria. 

However, FDA largely resisted a request to throughout the text replace the term 'appropriate,' which AdvaMed called ambiguous, with 'reasonable,' which the group said is a well-established term in law and regulation that is familiar to FDA and its stakeholders. The agency also seemed to mostly disregard a call for more information about routine PMAs, De Novos and HDEs, in light of heavier focus on breakthrough devices and small patient populations called out by AdvaMed.

FDA also received feedback from consumer advocacy group the Center for Science in the Public Interest, which raised a different set of concerns. As CSPI sees it, the examples cited in the guidance provide “clear stepping stones to reduced regulatory standards” that will put patients at risk.

“Companies will undoubtedly seize upon these scenarios to craft an argument about how a particular device should be the recipient of an even greater degree of uncertainty than the agency might have contemplated, and thus a shift in data requirements to the postmarket period, resulting in reduced premarket sample size,” CSPI wrote.

FDA made no major changes to the examples in finalizing the document.