On October 24, 2017, FDA Commissioner Scott Gottlieb, M.D., issued a statement regarding additional steps the FDA is taking as part of the Medical Innovation Access Plan he announced earlier in 2017. These steps include the first completion of the validation of a medical device development tool and the release of three new guidance documents related to medical devices.
The FDA completed the validation of a medical device development tool (MDDT) to provide a more objective platform for developing devices related to cardiovascular health. The 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ), a Clinical Outcome Assessment, measures patient-reported outcomes from patients with congestive heart failure or weakened heart muscle due to prior heart attacks, heart valve problems, viral infections, or other causes. Patients are asked about their symptoms, physical and social limitations, and their views on whether these symptoms have an impact on their quality of life. By measuring and evaluating this information, the KCCQ can assess whether a patient perceives that the patient’s heart failure symptoms have improved following an intervention. These results can then be used as part of the benefit/risk assessment to help determine a device’s safety and effectiveness.
Such a tool has the potential to help engineers designing heart failure devices to more efficiently and accurately quantify how much the device could actually improve a patient’s quality of life. By qualifying the tool under the FDA’s new, voluntary program, the FDA made it easier for product developers to rely on the outputs of this newly qualified tool as part of development plans. MDDTs qualified by FDA can be used in clinical trials by the medical device industry to support both device submissions and post-approval studies. Innovators can trust in advance that the FDA has already found the outputs of these measures to be reliable.
Under the FDA’s voluntary MDDT program, the FDA can evaluate a tool and decide whether to “qualify” it—which means the FDA can determine if the tool measures what it is supposed to measure, and does so reliably. The FDA only intends to qualify tools where it can make certain high level information about the tools publicly available, so that the device development community can benefit from using tools that they know work. MDDTs can be used to provide more efficient and accurate ways to measure risk and benefit, as part of the medical product development process.
To make the development process more efficient and predictable, on October 24, 2017, the FDA also released three new guidance documents related to medical devices. The first is draft guidance delineating the FDA’s new “Breakthrough Devices Program,” which was created by the 21st Century Cures Act passed by Congress last year. Building on the Expedited Access Pathway (EAP) program, this new program is intended to help patients more quickly access certain devices that more effectively diagnose or treat life-threatening or irreversibly debilitating diseases or conditions, such as technologies that have no alternative or that offer a significant advantage over FDA-cleared or approved alternatives. Breakthrough device innovations that are highly novel can also be more complex to assess. Thus, earlier and more frequent interaction between the FDA and manufacturers – as outlined in the FDA’s draft guidance on the Breakthrough Device Program – should allow manufacturers to make the best use of resources to bring state-of-the-art medical technologies to the market faster.
Two other final guidance documents will help innovators determine when they need to submit a new 510(k) prior to making a change to a legally-marketed device subject to premarket notification requirements. These policies will help innovators introduce iterative improvements that can improve a product’s safety and performance by establishing a brighter line regarding when the FDA needs to review and clear these changes in advance.
These new guidance documents do not change the FDA’s review standard: a new 510(k) is required when a marketed device has changes, including changes to software, that could significantly affect the safety or effectiveness of the device or when there are major changes in the intended use of the device. Instead, the new policies enhance predictability and consistency for innovators deciding when to submit new 510(k)s by better describing the regulatory framework, policies, and practices underlying such a decision.
This improved clarity will help lower the barriers to innovation and improve patient care by reducing unnecessary submissions to the FDA for changes that could not significantly affect device safety or effectiveness, so patients can benefit from upgraded products more quickly. Many devices function as tools in the hands of physicians. They benefit from feedback from doctors that helps innovators make small adaptations to improve a device’s performance. A regulatory framework that fosters this kind of useful revision is a key to improving their safety and performance.
See the FDA Announcement
See the FDA Blog Post
See also Medical Law Perspectives Report: Using Medical Devices Off-Label: False Claims, Overpromotion, Malpractice, and Other Dangerous Territory