April 22 — The Food and Drug Administration April 22 announced a new program for faster patient access to certain high-risk medical devices that demonstrate the potential to address unmet medical needs for life-threatening or irreversibly debilitating diseases or conditions.
The proposed Expedited Access Premarket Approval program (EAP) “will help patients have more timely access to these medical devices by expediting their development, assessment and review, while preserving the statutory standard of reasonable assurance of safety and effectiveness for premarket approval,” the agency said in a draft guidance document.
The document's title is “Expedited Access for Premarket Approval Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Disease or Conditions.” The draft guidance (Docket No. FDA-2014-D-0363) was announced in the April 23 Federal Register (79 Fed. Reg. 22,691). Comments are due July 22.
According to the agency, the 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—which, taken together, should provide these patients with earlier access to safe and effective medical devices.
“We are excited to offer a proposed program for expedited access for certain high-risk medical devices,” Jeffrey Shuren, director of the FDA's Center for Devices and Radiological Health, said in a statement. “The program allows manufacturers to engage early and often with the agency.” Shuren also said the agency expects most devices that enter the program will be in the pre-clinical trial phase.
The FDA has been working on the draft guidance for some time. In January, Shuren said a guidance was needed because there is no current framework for quickly getting devices to patients with unmet medical needs.
When utilizing the EAP program, the FDA said it will continue to apply the current approval standard of demonstrating a reasonable assurance of safety and efficacy.
The CDRH defined unmet medical needs for medical devices in its final 2013 guidance on priority premarket review. According to the guidance, an unmet medical need is demonstrated by the device being a breakthrough technology, having no approved alternative, offering a significant clinically meaningful advantage and being in the best interest of patients.
The proposed EAP program builds on the Innovation Pathway pilot, which the FDA launched in 2011.
The Innovation Pathway includes features designed to minimize delays that can occur when pioneering devices raise novel and challenging scientific questions. As a result, the FDA proposed a review time frame of 150 days—which is approximately half the time it takes to review most premarket approval applications (PMAs), the strictest form of approval for highest-risk devices.
The EAP program also builds on the FDA's experience with expedited review programs for pharmaceuticals, including Accelerated Approval and Breakthrough Therapies.
The FDA intends to work interactively with the sponsor to create a data development plan specific to the device.
The FDA also said it intends to work interactively with the sponsor to create a data development plan specific to the device. This plan should outline all data the sponsor intends to collect in support of device approval, including what data will be collected premarket and postmarket (after approval), the agency said. The data development plan “will help assure predictable, efficient, transparent, and timely device assessment and review,” the FDA said.
Participation in the EAP program is only at the request of the sponsor and with the FDA's agreement, the agency said. Sponsors will need to request the FDA designate the device as an EAP device, through the “EAP Designation” process.
Once a device is designated, the sponsor and the agency will need to work on the data development plan, which then leads to the submission and review of a PMA application, and eventually postmarket data collection if the PMA is improved.
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, and have an acceptable data development plan that has been approved by the FDA. In addition, the FDA said it must meet one of four criteria:
The agency said the EAP “is not a new pathway to market, but rather a collaborative approach to facilitate product development under the agency's existing regulatory authorities. While 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.”
The agency April 22 issued a separate, but related draft guidance, titled “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval.” That draft outlines the agency's current policy on balancing premarket and postmarket data collection during FDA review of PMA applications. The agency also published an April 23 notice about this document (Docket No. FDA-2014-D-0090) in the Federal Register (79 Fed. Reg. 22,690). Comments are due July 22.
The agency in its EAP guidance referenced the need to balance premarket and postmarket data. In the EAP draft guidance, the FDA noted that getting the right balance between premarket and postmarket data collection—specifically, a greater reliance on postmarket collection—can reduce the extent of premarket data submission and directly affect when patients will have access to high-quality, safe and effective medical devices.
The FDA said it “recognizes that device approvals are not made with absolute certainty because of significant obstacles, such as the time and cost involved to address possible rare adverse events or long-term safety issues, and because controlled clinical studies do not fully represent the benefit-risk profile of a device when used in real-world clinical practice.”
Since devices that qualify for the EAP program demonstrate the potential to address unmet medical needs for life-threatening or irreversibly debilitating diseases or conditions, the agency said it may accept less certainty regarding the benefit-risk profile of these devices at the time of premarket approval to get them on the market faster.
Janet Trunzo, senior executive vice president, technology and regulatory affairs at the Advanced Medical Technology Association, in an April 22 statement said AdvaMed is still reviewing the details of the drafts, and will be submitting formal comments.
Regarding the expedited process draft guidance, Trunzo said “it is important to remember that Congress required FDA to develop an expedited pathway for breakthrough devices under the FDA Modernization Act of 1997, and while the agency has developed guidance and procedures to fulfill this mandate, it has not succeeded in meeting Congress's intent. We will be looking to see if the new guidance truly creates an expedited pathway for PMA devices so that they are available to patients and physicians in a more timely manner.”
As for the premarket and postmarket balance draft guidance, Trunzo said the industry group will “need to make sure that any framework proposed by FDA has the support of all offices within the device center so that proposed programmatic changes can be implemented smoothly and effectively. In addition, we need to ensure that this new framework does not merely result in increased requirements for product sponsors.”
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The EAP draft guidance is at http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm393879.htm. The draft guidance on premarket and postmarket data is at http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm393882.htm.
The Federal Register notices are at http://www.gpo.gov/fdsys/pkg/FR-2014-04-23/html/2014-09189.htm (expedited access) and http://www.gpo.gov/fdsys/pkg/FR-2014-04-23/html/2014-09190.htm (premarket and postmarket data).
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