FDA Finalizes Two Guidances To Speed Approvals For Innovative Medical Devices; Expedited Access Program Starts April 15

 

The Food and Drug Administration last week finalized two guidances intended to speed the process of getting innovative medical devices to patients in need. The Agency discussed its guidances in an article entitled “Providing Timely Patient Access To High-Quality, Safe and Effective Medical Devices.”

“We know that patients with life-threatening or irreversibly debilitating conditions lack treatment and diagnostic options,” states Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health. “For these patients, earlier access to promising new devices is critically important, and [a]t the same time, delayed access may mean the difference between life and death, or may result in irreversible disability.”

“In weighing the benefits and risks of new technologies for these patients, we understand the need to place greater weight on the benefit of earlier access, and to also account for the risks of delayed access,” Shuren states.

To this end,  FDA has developed the Expedited Access Program (EAP), a voluntary program for certain medical devices “that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions that are subject to premarket approval applications (PMA) or are eligible for de novo requests,” states FDA. The EAP relies on similar policy to the FDA’s accelerated approval program for pharmaceuticals, designed to fast-track breakthrough drug therapies.

Devices subject to premarket approval applications (PMAs) or requests for de novo designation are eligible for EAP designation if the following three criteria are met:

The device is intended to treat or diagnose a life-threatening or irreversibly debilitating disease or condition

The device meets at least one of the following: (1) No appropriate alternative treatment or means of diagnosis exists. (2) The device represents a breakthrough technology that provides a clinically meaningful advantage over existing legally marketed technology. (3) The device offers significant, clinically meaningful advantages over existing legally marketed alternatives. (4) The availability of the device is in the best interest of patients.

The sponsor submits an acceptable draft Data Development Plan.

Once an eligible device sponsor chooses to go down the EAP path, the FDA works with the sponsor to try to reduce the time and cost from development to marketing without changing the FDA’s PMA approval standard of reasonable assurance of safety and effectiveness, the standards for granting de novo requests, or the agency’s standard of valid scientific evidence.

Components of the program include priority review, more interactive review, senior management involvement, and assignment of a case manager.  The extent to which the FDA provides these features depends on the availability of resources.

“Starting April 15th, this program will be up and running and we will begin to accept requests for EAP designation,” states Shuren. 

Another important aspect of this program is FDA’s interest in shifting some of the premarket data responsibilities to the postmarket setting, when appropriate. “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,” states Shuren. “A few of the factors that can enter into this analysis include a low probability of serious harm, a high likelihood that postmarket surveillance can quickly identify instances of serious patient harm and a high likelihood that postmarket data collection will be completed in a timely manner.”

“We consider this balancing of premarket and postmarket data collection to be so important that we made it one of our three 2014-2015 strategic priorities, along with strengthening the clinical trial enterprise and providing excellent customer service,” Shuren concludes.

Guidances

Released April 8, 2015, FDA’s first guidance explains the the Agency’s Expanded Access Program at length. The second goes into more detail about when FDA will shift premarket data collection to postmarket in certain cases. “Getting the right balance between premarket and postmarket data collection – specifically, where appropriate, a greater reliance on postmarket collection, including real-world data collection, can reduce the extent of premarket data collection and directly impact when patients will have access to high-quality, safe and effective medical devices,” states the guidance. “But, greater reliance on postmarket data collection could undermine patient safety if the necessary and timely data collection does not occur.” FDA wrangles with this issue in the guidance.

• Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions (available here)

• Balancing Premarket Postmarket Data Collection for Devices Subject to Premarket Approval (available here)