The FDA would require medtech companies seeking certain premarket approvals to submit timely postmarket study data or risk losing those approvals.
The requirement is part of a final guidance the agency released last week, detailing how it will consider uncertainty in making benefit-risk determinations on breakthrough device PMAs, de novo classifications and humanitarian device exemptions (HDEs).
The FDA acknowledged the difficulty of postmarket data collection and asked medtech companies to use real-world data on breakthrough devices to meet agency deadlines and provide reliable and relevant data. The agency said it would work with manufacturers to data submission timeframes with the “least burdensome approach” for the companies.
The FDA also said it might require manufacturers to label certain breakthrough devices with a description of the postmarket data collection and its purpose. If the postmarket data is sufficient, the company could change the label accordingly.
If the data raise safety and effectiveness questions, an FDA advisory committee would study it and recommend whether the FDA should withdraw the approval, restrict the device’s sale and distribution or narrow the indications for use.
The agency said it might apply the same conditions on a case-by-case basis to some devices that need PMAs for small patient populations.
Several factors would contribute to its decisions on uncertainty in benefit-risk determinations, including:
- The extent of the device’s probable benefits, taking into account the type, magnitude, probability, duration, and frequency of those benefits.
- The extent of the device’s probable risks, taking into account the severity, type, number, rates, probability, and duration of those risks.
- The extent of uncertainty regarding the benefit-risk profile of approved or cleared alternative treatments or diagnostics or the standard of care.
- Patients’ perspective on appropriate uncertainty about the probable benefits and risks of the device, if available.
- The extent of the public health need.
- The feasibility of generating extensive premarket clinical evidence based on considerations such as the prevalence of the disease or condition.
- The ability to reduce or resolve remaining uncertainty of a device’s benefit-risk profile postmarket.
- The likely effectiveness of mitigations such as labeling and other tools to provide a reasonable assurance of safety and effectiveness.
- The type of decision being made, such as for a PMA or an HDE.
- The probable benefits of earlier patient access to the device.
“The policies in the guidance further FDA’s mission to promote the public health by fostering medical device innovation and facilitating timely patient access to high quality, safe and effective medical devices,” the agency said. “In addition, the benefit-risk based framework described in this guidance aims to assure greater transparency, predictability, consistency, and efficiency, using least burdensome principles.”
