Marie Thibault

FDA has long combated stereotypes that its regulatory process is too slow, too cumbersome, and discourages medical innovation in the United States. While Congress has been focused on legislating away regulatory hurdles for devices and drugs with bills like the 21st Century Cures Act, the agency itself has taken a different tack.

Enter the Expedited Access Pathway (EAP), which CDRH director Jeffrey Shuren described in an FDA Voice blog post as a way “to speed qualifying devices to patients with life-threatening or irreversibly debilitating conditions without compromising FDA’s high standards for safety and effectiveness.”

More broadly, the program is an attempt by the agency to get innovative devices on the market faster.

“I see the EAP program as part of a continuum of several different programs that are aimed at moving important technologies toward the clinical environment as early and as quickly as possible where we think that there’s real promise,” says Owen Faris, PhD, acting clinical trials director at FDA.

The Details

The EAP program, which launched April 15, 2015, is available only to devices that tackle the toughest conditions and would file a PMA application or de novo request. For a device to be eligible, there must not be other good therapeutic or diagnostic options available or, if there are, the device must be a “breakthrough technology,” have important advantages over the alternatives, or be "in the best interest of patients," according to FDA’s description.

The EAP is supposed to speed up the regulatory process for these most-needed devices. Not only will the devices receive priority review, but FDA regulators will work closely with the device sponsor and consider using postmarket data whenever possible.

“Under the EAP, FDA may accept a greater degree of uncertainty if it is sufficiently balanced by other factors, including the probable benefits to having earlier access to the device," Shuren wrote. "If, after careful analysis, FDA determines that some data can be collected after the device is on the market, then patients in need will benefit sooner.”

That could be good news for companies with eligible technologies.

“In general, for companies with the right type of device, I think this could be an attractive program," says Yarmela Pavlovic, partner at Hogan Lovells. "How popular it will be remains to be seen.”

Importantly, the device sponsor needs to craft a Data Development Plan draft and submit it to FDA as part of a presubmission requesting EAP designation. The Data Development Plan is a thorough description of all studies that will be conducted, all premarket and postmarket data that will be gathered, intended labeling, and timelines. The presubmission should also include a cover letter, table of contents, description of the device, intended indications for use, and an explanation of the device’s EAP eligibility.

FDA plans to respond to an EAP presubmission within 30 days. If the agency requests more information, sponsors are expected to send in a supplement to the presubmission and will be notified of FDA's decision within 30 days of the additional info request. If the agency still needs more information, it will reject the EAP request.

FDA’s Faris wouldn’t divulge how many applications have been submitted so far but says the program has seen “substantial interest.”

What Came Before

The EAP program was proposed in April 2014 and formally launched in April 2015. The EAP program builds on the Innovation Pathway initiative, which began in 2011 and was updated in 2012 as “Innovation Pathway 2.0.” The Innovation Challenge was a pilot initiative of the Innovation Pathway 2.0 program with a focus on end-stage renal disease. Three device sponsors were selected as participants and worked together with regulators to create a data collection plan—a concept that is being carried forward with the EAP’s Data Development Plan.

CreatiVasc Medical Inc., maker of the Hemoaccess Valve System, was one of the Innovation Challenge participants, and Steven Johnson, CEO of CreatiVasc and president and chief operating officer of parent company Brookhaven Medical, speaks highly of the initiative.

“We felt as if we had a team of up to as many as 10–12 people involved with what we were doing . . . from biomechanical engineers to a clinical trial design specialist to labeling experts to a veterinarian. It was every major discipline within the agency . . .” Johnson says.

Sponsors and regulators kept in near constant contact. Johnson says there were monthly meetings as well as weekly interactions, and FDA regularly responded to questions within 24–48 hours. Many features from the Innovation Challenge, including close collaboration and input from a case manager and senior leadership, have been carried forward to the EAP program.

Victor Gura, MD, who participated in the Innovation Challenge with the Wearable Artificial Kidney he invented, reports a similar experience. Gura says he learned of IDE approval on a Friday night after receiving an e-mail from FDA at midnight Eastern Time—“that means the guy that sent the e-mail with the approval on Friday night was there working,” he says.

Both Johnson and Gura say they saw major benefits from their involvement in the Innovation Challenge. Gura says FDA “really shepherded” the process of getting the first IDE approved.

CreatiVasc’s Hemoaccess Valve System didn’t have a predicate device and might have otherwise been considered a Class III PMA device, Johnson explains. But FDA decided to reclassify it from a Class III PMA device to a 510(k) device because it could be considered an accessory to the approved arteriovenous graft.

“When [FDA] said they wanted to think outside the box, they really did," Johnson says. "They really did think of ways for simplifying the review and approval process."

Is EAP Right for Your Company?

Despite the positive experiences reported by the Innovation Challenge participants, companies may still be skeptical of the EAP program. Pavlovic notes that with new programs there is always some nervousness, as no company wants to be the first to “work out all the kinks.”

Another reason for trepidation may be the lack of actual rulemaking around the EAP program. The EAP program is not backed by a final rule from FDA but is instead supported by two guidances—“Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions” and “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval.”

Beverly Lorell, MD, senior medical and policy advisor with King & Spalding’s FDA & Life Sciences Practice Group, says that on the drug side, there is both rulemaking and precedent to make companies more comfortable with the Accelerated Approval program, which is similar to the EAP program for devices.

“. . . [T]he rules of the road for the Accelerated Approval have really been in place and well trod for over two decades . . . " Lorell says. "It’s based on rulemaking . . . with roughly between three and four new drugs a year being approved on that pathway . . .[There’s] a lot of experience as to what FDA is going to expect . . . For a device company, there’s not yet that experience on interacting with CDRH.”

Lorell’s colleague, David Farber, a partner at King & Spalding, points out that since one requirement for EAP eligibility is a draft Data Development Plan, there may be some concern that FDA could agree to a sponsor’s plan, then backtrack on it later. Still, he admits this is probably unlikely. “[FDA] has an interest in making the EAP program successful. . . it’ll stick to its commitment, is my prognosis,” Farber says.

FDA’s Faris adds that as much as possible, the agency will stick to its word. “When you look at our presubmission guidance, we have specific language in there that talks about how we want to document a commitment to any advice we provide," he says. "However, there are certain things that could make that advice no longer applicable . . . we’re giving you our best advice, and we’re not going to change that advice unless there are very strong reasons that have to do with protecting the public. . .”

Innovation Challenge participants spoke highly of FDA’s close involvement with the companies, which, of course, required financial and personnel resources. Will the agency also do what it takes to support this new effort?

“I think we do have the resources to make a real meaningful difference in the space under the EAP program,” Faris says.

But he adds that the agency did learn one important lesson from the Innovation Pathway: “Because of the resource intensiveness of this program, we really do want to see companies coming to us as prepared as possible, so that we’re not really starting at absolute ground zero.”

Thinking of Diving In?

So what should prospective EAP designees do to get started?

“I think the biggest point of guidance is to come talk to us early," Faris says. "The earlier we engage with the sponsors, the more flexibility and opportunity there is to think about how to do things differently and to engage at a very high level and speed things up.”

When asked how best to interact with FDA, Johnson recommends a collaborative approach. “My advice would be, don’t go in there with two lawyers and two consultants," he says. "Go in with your team.”

He makes an apt analogy: “FDA is much like a swimming pool. If you want to do a bellyflop and fight your way into the water, then it’s going to hurt . . . But if you ease into the water and take it a step at the time, work with them, you’re all of a sudden in the middle of the pool without even knowing it.”

The impact of the EAP program is yet to be seen, but perhaps the best indication that FDA’s programs are having their intended effect comes from Gura, who says if he hadn’t taken part in the Innovation Challenge, “I’d probably be working outside the U.S."

Marie Thibault is the associate editor at MD+DI. Reach her at [email protected] and on Twitter @medtechmarie. 

 [Image courtesy of JESADAPHORN/FREEDIGITALPHOTOS.NET]