The US Food and Drug Administration (FDA) proposed a new program to expedite medical device approvals for patients whose serious medical needs are unmet by current technologies.
FDA has been criticized, occasionally by us, for slowing patients' access to innovative new devices with overly burdensome regulations. The "Expedited Access Premarket Approval Application" (EAP) program seeks to remedy that in part through a "collaborative approach to facilitate product development under the agency's existing regulatory authorities."
Thus, while the proposed program is "not a new pathway to market" for medical devices, EAP features "earlier and more interactive engagement with FDA staff." As part of the EAP program, FDA looks to provide more "interactive communications" during device development and more interactive review of Investigational Device Exemption applications and premarket approval applications. In addition, FDA states that it "intends to work interactively with the sponsor to create a data development plan specific to the device."
FDA notes that 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's plan hinges on the involvement of senior management and a collaboratively developed plan for collecting the scientific and clinical data to support approval. FDA's Safety and Innovation Act (FDASIA) allows FDA to collect substantial fees from pharmaceutical and device manufacturers to fund the regulatory review of new products. These user fees may have allowed FDA to beef up the resources necessary for their EAP proposal.
From a look at the proposal, devices must meet potentially challenging criteria to be eligible for "EAP Designation."
- First, the device must be intended to treat or diagnose a life-threatening or irreversibly debilitating disease or condition.
Second, the device must meet at least one of the following criteria for addressing an unmet need:
- (1) The device represents a "breakthrough technology" that provides a clinically meaningful advantage over existing technology; or
- (2) No approved alternative treatment or means of diagnosis exists; or
- (3) The device offers significant, clinically meaningful advantages over existing approved alternatives; or
- (4)The availability of the device is in the best interest of patients (e.g., addresses an unmet medical need).
- Third, the sponsor submits an acceptable draft Data Development Plan that has been approved by FDA.
In addition to the three prong test to qualify for the EAP program, device companies have to be prepared for some hands on collaboration with FDA. Health Data Management interviewed Bradley Merrill Thompson, a D.C.-based attorney, who questioned the value of EAP: "The possible speed of the approval--and there are no guarantees of course that it will in fact turn out to be quicker--comes at the cost of FDA looking over your shoulder the entire way," he stated.
Wide Variance in Performance Found Among FDA's Drug Reviews
The FDA proposal came out at the same time the Manhattan Institute for Policy Research (MIPR) published a research report entitled "An FDA Report Card: Wide Variance in Performance Found Among Agency's Drug Review Divisions." Interestingly, the MIPR found a wide gap in the approval rates of different drugs. RAPS notes that "[s]ome review divisions, such as the Center for Drug Evaluation and Research's (CDER) oncology and antiviral divisions, approved drugs with nearly twice the speed as the next-fastest divisions, and nearly four times as quickly as CDER's slowest division." Indeed, the Neurology division took nearly 600 days to approve a drug, and the two fastest units, Oncology and Anti-Viral, took under 200 days.
The authors of the study, Joseph A. DiMasi, Christopher-Paul Milne, and Alex Tabarrok, examined disparities in review and approval times across 12 review divisions within the FDA's CDER. The study concluded:
Our analysis of performance has revealed large differences among the FDA divisions. High-performing divisions are several-fold better on output measures than low-performing divisions, and they perform better without commensurately greater resources or less complexity of tasks or reduced safety. Inconsistent performance across divisions is thus a strong indication of inefficiency, but also of opportunity. A careful comparison of the performance of the agency's drug review divisions suggests that agency performance can be dramatically improved at little cost to taxpayers.
The authors note that more study is needed to identify best practices at work in the most productive divisions of the FDA. It will be interesting to see whether FDA's new EAP proposal utilizes inter-divisional best practice analyses in addition to its hands on approach to expedite drug approval.