New FDA Programs Full of Promise, but Pitfalls Loom

The following is re-published with permission form the author.

New FDA Programs Full of Promise, but Pitfalls Loom

By Mark McCarty, MedTech Scribe

FDA has rolled out several new programs recently that promise to cut red tape for medical product applications to improve patient access. Still, Nate Downing of Gardner Law told me in an interview that while these programs have much to offer, manufacturers shouldn’t see these programs as friction-free paths to market.

Downing, managing attorney at Gardner Law in Stillwater, Minn., said FDA’s breakthrough devices program is a good example of this dynamic. One benefit of the breakthrough devices program is that FDA reviewers prioritize the review of these devices, potentially shortening the time to market. This is music to the ears of patients and investors alike, but this program may also generate some dissonance.

One of the potential problems is that FDA reviewers may come up with an evidentiary standard for premarket review that is different or more onerous than the sponsor expected, Downing stated. This in turn could require a more expensive clinical trial than expected, but the hazards don’t end there.

Another possible tripwire is that the agency might demand more information in non-clinical testing than anticipated, something Downing said can be managed. One suggestion is that the sponsor be selective about the questions it poses to the FDA team during the early stages of interactions, but he also stated, “I don’t let clients ask open-ended questions” during these early interactions with FDA staff.

There are non-FDA incentives for taking part in the breakthrough devices program as well. A smaller device manufacturer that has little history with the agency might have a tough time selling investors on the notion that the company has the experience required to successfully traverse the FDA premarket gauntlet. However, a company that announces it has a product in the breakthrough devices program can alleviate any concerns about its ability to navigate the FDA premarket review mechanism, thus offering a value proposition “that’s pretty meaningful for an investor,” Downing said.

Drug Voucher Program a Complex Proposition

The Commissioner’s National Priority Voucher (CNPV) pilot program is another example of a leaner premarket review process, but this program requires that the drug be manufactured in the U.S. This might not be a showstopper for larger firms and firms that can do business with a domestic third party manufacturer, but smaller entities might have a problem with this mandate.

Downing said the answer to such concerns may depend on the company’s overall resources, including the skills needed for manufacturing site development. “If you are contracting or hiring talent on the go, or you don’t have that knowledge, you can see how that could add a lot of time” to market for that product despite the CNPV pilot, he remarked. He added that the availability of a contract manufacturer might be critical for some companies contemplating participation in this program.

The onshoring of drug manufacturing might do little to reduce a drug’s cost, a consideration for FDA for a number of years despite that CMS is the rate-setting agency for drugs where the Medicare and Medicaid programs are concerned. Downing said the CNPV pilot might not prompt a manufacturer to rethink any of its development programs despite the promise of a more rapid premarket review.

“You wouldn’t develop a program with this in mind,” Downing mused, even given the shorter premarket review time. CNPV is a pilot program and there’s no guarantee it will graduate into a permanent program. For a manufacturer with a development program in queue that might be a good fit for CNPV, “it’s not a question of when you go ahead, it’s a question of having multiple plans in place” to deal with the possibility that this pilot will have expired before the manufacturer can apply.

FDA stipulates that applicants have their chemistry, manufacturing and controls documented and in place at least 60 days prior to the start of the review clock under CNPV, which Downing said is not atypical. However, he speculated that making this a formal requirement might help thwart applications for development projects that simply are not far enough along to be serious candidates.

No Regulatory Template for FDA’s TEMPO Program

FDA’s Technology-Enabled Meaningful Patient Outcomes (TEMPO) program is another regulatory novelty and is directed toward digital health products that fit into the Home as a Healthcare Hub program at the Center for Devices and Radiological Health. This program poses a couple of rather unique questions, including the regulatory status of any participating products.

TEMPO allows a manufacturer to distribute digital health technologies that will be covered by Medicare, but FDA will exercise enforcement discretion regarding the lack of approval or clearance of the device. However, these candidate products might not need even an investigational device exemption (IDE), which would otherwise seem to obviate any anxiety over the lack of a marketing authorization.

Downing said FDA will grant entry into TEMPO with a sharp consideration of the risks inherent to the candidate products, but he acknowledged the possibility that an outside party will raise a stink about the use of enforcement discretion as a substitute for an IDE or a premarket review.

“It really is just about enforcement discretion, and there isn’t a regulation FDA can point to” that enables this road to market access, Downing mused. Some sponsors might be unfamiliar with the attendant postmarket surveillance requirements, which could leave FDA with additional work in tracking down adverse events. On the other hand, the agency can ensure that any participant products don’t present the type of risk that would lead to a class I recall, a fact which should tamp down on any anxiety agency staff might have.

There are several other recent FDA-related developments of interest to life science companies, including a report from Sen. Bill Cassidy (R-La.) on the FDA review process along with the ongoing negotiations over the next round of user fee schedules. Downing acknowledged that many of these accelerated market access programs place significant demands on the agency, demands that cannot be covered entirely by user fees.

Many who work at FDA are drawn to the agency due to intangibles that have nothing to do with money, but a lack of appropriations could make it difficult for the agency to draw the talent it needs. While most in Congress are attuned to the importance of what FDA does, “it takes meaningful conversations on Capitol Hill” to ensure that patients and investors alike continue to have confidence in FDA’s ability to manage its workload, Downing said.