After much delay, FDA and the device industry have agreed on MDUFA Performance Goals for the next five years, as memorialized in the MDUFA Commitment Letter. The agreement lays out the time to decision goals of the Agency, as well as a number of other points of interest. This outlines some of the concerns stakeholders should we aware of with respect to the agreement
Product Review Times are Shortened
While this is not, on its face, a problem, a shortened review timeframe, without any extensions of postmarket review or authorities, will be problematic to some public health and safety advocates. They will argue that device safety is already an issue. Less than two percent of FDA-reviewed medical devices are reviewed through the more stringent process PMA pathway which requires proof that the device is safe and effective. Some studies, like this one reviewing the strength of data submitted to support orthopedic PMAs and this one reviewing the strength of data submitted to support cardiovascular PMAs, show that the quality of data supporting PMAs is not universally high. The 510k pathway requires a showing that the device is substantially equivalent to one already on the market, and does not require a showing of safety and effectiveness. A recent study shows that PMAs are more likely to have recalls, including high-risk recalls, than 510ks. The current commitment letter could not address the PMA or 510k process, both of which are established by Congress. However, the letter could discuss industry and FDA’s commitment to postmarket monitoring, data gathering and recall processes.
As FDA has documented repeatedly, it needs to update and integrate its IT infrastructure. CDRH, too, has unique needs, as there are numerous approval pathways, databases and post marketing monitoring tools available. NEST is one of CDRH’s efforts to monitor the total product lifecycle through active surveillance. Advocates are likely to call for transparency of those databases as well as using post market surveillance tools to monitor for safety signals.
The use of and science of patient engagement tools has been a focus of the Center’s for years. The agreement calls for additional staff to work on patient reported outcomes, patient preference information and patient generated health data. CDRH will issue a guidance on “best practices on incorporating into premarket studies clinical outcome assessments including their use as primary or co-primary endpoints.” It will hold a public meeting about the use of patient generated health data and how it can be used for remote patient monitoring.
Real World Evidence/Real World Data
CDRH has, in some ways, pioneered the use of RWE/RWD through its registry programs. The agreement allows CDRH to use user fees for its RWE/RWD work, which includes NEST. The Agency will “update the 2017 guidance document Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” and hold two public meetings to update stakeholders on the RWE/RWD program over the next five years.
As the digital health market expands, FDA faces additional responsibilities. Digital health devices are not easily suited for existing premarket and postmarket authorities. FDA has piloted a framework to regulate digital health products, although it has not been entirely smooth sailing. FDA has committed to develop expertise and train staff on digital health. It will “finalize the Content of Premarket Submissions for Device Software Functions” draft guidance, and publish a guidance on change control plans, which was proposed in the AI/ML-Based SaMD Action Plan FDA published last year.
What to expect from Congress
Since this is an election year, Congress is hoping to get the entire user fee package, with its riders, enacted before August recess. That means the House and Senate both need to hold hearings, markups, pass legislation and ultimately, negotiate a final package in the next four months. Parties are interested in moving the package with all due haste. FDA will need additional authority from Congress to solidify its regulation of laboratory developed tests and create a tailored pathway for digital health products; as these proposals may be controversial and/or complicated, they may not be ironed out in time to be attached to the final bill.
Riders being considered include those on accelerated approval, Cures 2.0, ARPA-H, laboratory developed tests and medical device shortages.
A list of bills recently discussed by the Energy and Commerce committee can be found at the bottom of this page.