Your Guide to 2013's Must-Read FDA Documents

This year saw FDA hand down a long-awaited rule and a slew of guidances—including a few that show signs of a more flexible agency.

December 9, 2013

8 Min Read
Your Guide to 2013's Must-Read FDA Documents

By Jamie Hartford 

The medical device industry has long called for clarity, consistency, and common sense from FDA, and 2013 saw the agency issue a few guidance documents that represent progress—however slow—toward achieving those objectives.

Stay up to date on the goings-on at FDA by attending the FDA and Global Regulations in Practice conference track at MD&M West in Anaheim, CA, February 10–13, 2014.

While many of the guidances released this year weren’t earth-shattering and came long overdue, a few showed signs that the agency is perhaps beginning to evolve.

Vicki S. Anastasi, senior vice president for medical devices at contract research organization Aptiv Solutions, walks us through some of the important developments at CDRH this year.

Priority Review of Premarket Submissions

FDA’s priority review pathway can be used to speed review of PMA applications and 510(k) submissions for certain devices, but it has long been unclear which devices qualify for the expedited process and what companies must provide to prove their devices are eligible. The agency sought to clear that up in a long-awaited guidance released in May.

According to the document, for a device to qualify for priority review, the manufacturer must prove it is intended to “treat or diagnose a life-threatening or irreversibly debilitating disease or condition” and meet at least one of the following criteria:

  • The device represents a breakthrough technology that provides a clinically meaningful advantage over existing technology.

  • No approved alternative treatment or means of diagnosis exists.

  • The device offers significant, clinically meaningful advantages over existing approved alternatives.

  • The availability of the device is in the best interest of patients.

The priority review guidance is one example of the agency’s willingness to be more proactive and flexible in its approach to regulating medical devices, according to Anastasi. “They’re looking out into the industry and realizing that some of these [devices] are getting really high tech and that they can change the course of disease management for patients who need remediation quickly,” she says. “It shows great external focus on the part of FDA.”

Radio-Frequency Wireless Technology in Medical Devices

Wireless technology has opened up a world of opportunity in the medical device space, enabling everything from better patient monitoring in hospitals to healthcare inside the home. But with these rewards come risks. In August, FDA released a guidance highlighting considerations for the design, testing, and use of wireless medical devices in an attempt to help developers minimize those risks.

The document called out six factors that should be taken into account: selection and performance of wireless technology, wireless quality of service, wireless coexistence, security of wireless signals and data, electromagnetic compatibility of wireless technology, and information for proper set-up and operation. It also outlined recommendations for premarket submissions for devices that incorporate wireless technology.

That information mostly confirms what many in the industry already knew. “I think it just clarified things,” Anastasi says. “I honestly couldn’t tell you anyone who had more than a passing concern about it.”

But that’s not necessarily a bad thing. “It’s always good to put the rules of the road down in writing so you don’t have to build consensus,” Anastasi says.

Risk-Based Monitoring and Adaptive Design

Among the most important documents released by FDA this year was the final guidance on risk-based monitoring. The document emphasizes a flexible approach to monitoring of clinical trials, while laying out a few guidelines for sponsors.

"This is a significant step forward to enhancing clinical trial efficiency while improving data quality," Anastasi says.

According to the guidance:

Sponsors should prospectively identify critical data and processes, then perform a risk assessment to identify and understand the risks that could affect the collection of critical data or the performance of critical processes, and then develop a monitoring plan that focuses on the important and likely risks to critical data and processes."

In addition to risk-based monitoring, device companies are also becoming interested in adaptive design, she says. This gives device companies a license to be more creative in their clinical trial design. It enables them to move away from the traditional sequential standard toward a more adaptive, risk-based approach in which trial assumptions can be assessed early and preplanned adaptations made if those assumptions need changing, Anastasi says. “It’s a big deal.”

Essentially, this paves the way for a clinical trial that was slated to last, say, two years to be stopped at six months if the data show that the endpoints won’t be proven. That saves patients from being enrolled, FDA from criticizing the results, and, ultimately, companies’ money, Anastasi says. On the other hand, if a trial proves efficacy in a certain population at six months, the sponsor can go to FDA with the data, potentially bringing a vital technology to patients who need it a year-and-a-half earlier.

“This means that innovative or breakthrough products will be out on the market faster, which means more of them will go out,” Anastasi says.

Unique Device Identification

On September 20, 2013—nearly six years after Congress authorized the agency to establish a system for tracking individual medical devices—FDA issued its final rule on unique device identification (UDI).

In many ways, the final rule was similar to the draft guidance the agency issued more than a year earlier. FDA did, however, make several concessions to the industry, including scrapping the requirement that implantable devices be directly marked with an identifier, enabling single-use devices sold in multipacks to carry an identifier on the package as opposed to each discrete device, and allowing manufacturers three years to deplete their inventories.

“I think very little of the UDI guidance is new or surprising,” Anastasi says. “It took a long time to come out, but in this situation, that time was probably needed because there were a lot of conversations and collaborations that took place. If that hadn’t happened, we would have had something that could have negatively affected the industry versus helping the industry to be better and stronger.”

Mobile Medical Apps

FDA attempted to establish some ground rules for the burgeoning mobile medical apps marketplace with a draft guidance back in 2011, but it wasn’t until this past September that it solidified its ideas on how to regulate this new and innovative arm of the medical device industry. In the end, the agency opted for a tailored, risk-based approach to apps regulation, targeting only apps used as accessories to regulated medical devices and apps that transform a mobile platform into a regulated medical device.

“I think that the FDA responded to mHealth with a consistent, predictable approach to the regulation of mobile medical apps,” Anastasi says. “They’ve put together something that is a good framework and a scientifically rigorous approach to mobile medical apps, and they’re saying here’s how they’re going to be regulated.”

But does that mean the agency has answered every question on the subject of mHealth? Not quite. Anastasi says the next mHealth guidance FDA issues should cover clinical trials for mobile health devices.

Medical Device Development Tools

Clinical trials can be lengthy and costly, so it’s particularly frustrating to medical device makers when regulators find fault with their methods. A draft guidance released this past November proposes a new process by which companies can obtain qualification of medical device development tools such as clinical outcome assessments, biomarker tests, and nonclinical assessment models.

The new process would enable tools to be qualified for a certain context of use, whereas in the past tools were required to be evaluated for each product or setting discretely. The draft guidance also lays out a few of the common questions companies will have to address for medical device development tools.

“I like this one,” Anastasi says of the guidance, adding that it’s especially important because of the speed with which medical device technology is evolving. Some newer technologies don’t fit neatly into product classes, and companies are forced to “shove square pegs into a round hole” when it comes to clinical trials, she says. “If we can use this early and go to FDA and say we’re going to codevelop this tool or assay or software and it’s going to be able to show the performance, I think that’s a great opportunity.”

What’s in Store for 2014?

No one can predict the future—and which way FDA will swing is always anybody’s guess—but Anastasi says she expects next year will bring more of the same from the agency.

“I personally think we’ll see a repeat of 2013, with some long-awaited confirmations of information that is understood but that has not put down in writing,” she says. “Also, I think we’ll have more like the UDI rule, which have a need for consensus building and collaboration, and therefore took a while.”

In any case, Anastasi says next year will demand that both the industry and FDA look beyond tradition to find new and innovative ways to get necessary products to patients faster.

Did we miss any must-read guidances from 2013? Let us know which documents you would add to this list in the comments below.

Stay up to date on the goings-on at FDA by attending the FDA and Global Regulations in Practice conference track at MD&M West in Anaheim, CA, February 10–13, 2014.

Jamie Hartford is the managing editor of MD+DI. Reach her at [email protected]


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