The Expedited Access Pathway (EAP) became effective on April 15th this year with the issue of the Food and Drug Administration (FDA or the Agency) guidance entitled  “Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions.”

The EAP is a promising resource for timely approval and access to devices for patients with life-threatening or irreversibly debilitating conditions without comprising safety and effectiveness. In order to be successful, sponsors should prepare to bring FDA into their design development process early and plan to work collaboratively with FDA throughout the EAP program.

This guidance outlines FDA’s new, voluntary program for certain medical devices that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions and that are subject to premarket approval (PMA) applications or de novo classifications. Participation in the Expedited Access Pathway (EAP) program must be requested by the sponsor and agreed to by the FDA.

The EAP program 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, consistent with the Agency’s mission to protect and promote public health.

It is also an opportunity for the FDA to work closely with device sponsors. The guidance reviews the key points to obtain expedited access to market for premarket approval (PMA) applications and De Novo devices, how to obtain Expedited Access Pathway (EAP) designation, the importance of the Data Development Plan (DDP) and factors to support the Benefit – Risk determinations. The document also discusses how the EAP program approaches the balance of premarket and post market data collection.

Device sponsors should bring the Agency into the discussion as early into the design development process as possible and this program will provide to the sponsor, as appropriate, FDA interactive communication, FDA senior management involvement, a case manager, and priority review. The guidance provides a thorough review and examples of the eligibility requirements to participate in the EAP program, the process to follow, and the content to provide FDA for consideration to obtain designation.

In order to obtain designation, the device should meet a set of very specific criteria outlined in the guidance:

1. Intended to treat or diagnose a life-threatening or irreversibly debilitating disease or condition.
2. Meets at least one of the following criteria for addressing an unmet need.

o No appropriate alternative treatment or means of diagnosis exists.
o Represents a breakthrough technology that provides a clinically meaningful advantage over existing legally marketed technology.
o Offers significant, clinically meaningful advantages over existing legally marketed alternatives.
o Availability is in the best interest of patients (e.g., addresses an unmet medical need).

3. Submission of an acceptable draft Data Development Plan.

If a device meets these requirements, the device sponsor can proceed to the next step, the Pre-Sub, to request an EAP designation. The sponsor should follow the Pre-Sub Guidance (discussed in my previous blog, insert link to previous blog) to provide a rationale explaining why it meets the EAP criteria, a proposed draft of the Data Development Plan and a Benefit-Risk analysis. These three items will be key considerations in determining whether or not a designation is assigned. The FDA then has 30 days, once the Pre-Sub is received, to provide a designation, request more information or issue a denial.

During the EAP process, the FDA will collaborate with a sponsor to further develop the Data Development Plan, a key program feature evolving into an agreed upon plan prior to submission of a PMA, IDE, or de novo application. The plan outlines all clinical and non-clinical data (including premarket and postmarket data) the sponsor is required to collect in support of device approval. A significant component of this collaboration is to develop a clinical study plan that weighs the amount of premarket data needed to be collected against clinical data that can be collected post-approval. The clinical study plan is based on the benefit-risk framework described in the FDA’s 2012 guidance, Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approvals and De Novo Classifications.

The following new guidance, Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval, is a companion guidance to the EAP, and a resource for both industry and the FDA staff to understand when it is appropriate for a PMA sponsor to collect some data post- PMA approval. The FDA believes the guidance will improve predictability, consistency, transparency and efficiency during the review process, and improve patient access to safe and effective medical devices.