FDA Proposes Expedited Access Program for Medical Devices That Address Unmet Medical Needs

April 22, 2014—The US Food and Drug Administration (FDA) announced a proposed new program to provide earlier access to medical devices that are intended to treat or diagnose patients with serious conditions whose medical needs are unmet by current technology.

The draft guidance document outlining the program is available on the FDA’s website and published in the Federal Register. The document, which is directed to industry and FDA staff, has been created by the FDA’s Center for Biologics Evaluation Research, which is part of the Center for Devices and Radiological Health.

According to the FDA, the proposed Expedited Access Premarket Approval Application for Unmet Medical Needs for Life Threatening or Irreversibly Debilitating Diseases or Conditions (“Expedited Access PMA” or “EAP”) program features earlier and more interactive engagement with FDA staff—including the involvement of senior management and a collaboratively developed plan for collecting the scientific and clinical data to support approval—features that, taken together, should provide these patients with earlier access to safe and effective medical devices.

The agency noted that EAP is not a new pathway to market, but rather a collaborative approach to facilitate product development under the agency’s existing regulatory authorities. Although other existing device programs have focused on reducing the time for the premarket review, EAP also seeks to reduce the time associated with product development.

In the agency’s press release, Jeffrey Shuren, MD, Director of the FDA Center for Devices and Radiological Health, commented, “We are excited to offer a proposed program for expedited access for certain high-risk medical devices. The program allows manufacturers to engage early and often with the agency. We expect most devices that enter this program will be in the preclinical trial phase.”

To be eligible for participation in the program, the medical device must be intended to treat or diagnose a life-threatening or irreversibly debilitating disease or condition. Additionally, it must meet one of the following criteria: no approved alternative treatment/diagnostic exists; be a breakthrough technology that provides a clinically meaningful advantage over existing technology; offer a significant, clinically meaningful advantage over existing approved alternatives; or its availability is in the patient’s best interest. The device must also have an acceptable data development plan that has been approved by the FDA.

The FDA advised that the EAP builds on the agency’s Innovation Pathway pilot initiative, which was launched in 2011, as well as its experience with expedited review programs for pharmaceuticals, including Accelerated Approval and Breakthrough Therapies. When utilizing the EAP program, the FDA will continue to apply the current approval standard of demonstrating a reasonable assurance of safety and efficacy.

In addition to the EAP draft guidance document, the FDA published a draft guidance–also available online for public comment—that outlines the agency’s current policy on when data can be collected after product approval and what actions are available to the FDA if approval conditions, such as postmarket data collection, are not met. Included in the guidance is advice on the use of surrogate or independent markers to support approval, similar to the data points used for accelerated approval of prescription drugs.  

Dr. Shuren stated, “To ensure that a device is safe and effective and provide timely patient access to breakthrough devices, it’s critical to get the right balance between premarket data collection and postmarket data collection.”

The FDA seeks public comment on both documents. Comments and suggestions regarding these draft documents may be submitted within 90 days of publication in the Federal Register of the notice announcing the availability of the draft guidance by electronic submission at www.regulations.gov or by mail to the FDA’s Division of Dockets Management. Further contact information and instructions are provided in the draft guidance document.