Comments Opposing FDA’s Draft Guidance on Speeding Access to High Risk Medical Devices

Division of Dockets Management (HFA-305)

Food and Drug Administration

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Comments of Members of the Patient, Consumer, and Public Health Coalition

on the Draft Guidance

“Expedited Access for Premarket Approval Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions”

Docket No. FDA-2014-D-0363

As members of the Patient, Consumer and Public Health Coalition, we do not support the draft guidance for the “Expedited Access for Premarket Approval Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions.”

The standards for PMA are already substantially lower than for prescription drugs.  Most PMA devices are approved through supplements that do not require clinical trials.  Original PMA clinical trials are often small and short-term with limited use of control groups.  And yet, the draft guidance lowers the bar for certain PMA medical devices even further.

The 1% of devices that go through the PMA process are the most risky of the devices that the FDA defines as “those that support or sustain human life, are of substantial importance in preventing impairment of human health, or which present a potential, unreasonable risk of illness or injury.”[i]

The purpose of the proposed Expedited Access for Premarket Approval (EAP) program “is earlier access to devices that address an unmet medical need.”[ii]  Although we sympathize with patients who suffer from life threatening or irreversibly debilitating disease, the PMA standards are already dangerously low.  And, the proposed guidance defines “urgent, unmet need” much too loosely.  The proposed guidance would lower existing standards further for devices intended to treat serious illnesses for which effective treatments are already available.  As a result, there is no guarantee that the benefits would outweigh the risks for patients.  Accelerated access to unproven devices could lead to unnecessary deaths and injuries.

The FDA states that it intends to work with sponsors of EAP devices to develop a Data Development Plan “with the goal of significantly reducing the time and cost from device development to FDA marketing decisions.”  We support the emphasis on earlier interactive communication between sponsors and the FDA.  However, the guidance gives undue emphasis on postmarket studies that are poorly monitored, have high loss to follow-up, and are rarely conducted in a timely manner that provides useful results.  The guidance would lower the standards to the point where they could not provide reasonable assurance of safety or effectiveness.

Below are our specific concerns and questions regarding the draft guidance:

The draft guidance proposes to speed approval of PMA devices by increasing interactive communication between sponsors and the FDA; relying on postmarket data; accepting more uncertainty; using less rigorous evidence; and forgoing inspections of manufacturing sites.  All of these proposals lower the evidence threshold for FDA’s review of high-risk Class III PMA devices.  They decrease the likelihood that the devices will have what patients, consumers, and public health experts would consider a “reasonable assurance of safety and effectiveness.”[iii]

Lack of resources for Earlier Interactive Communications

The draft guidance encourages the FDA and sponsors to have earlier interactions but there are no additional resources dedicated to these meetings. The draft guidance recognizes this and states that FDA will work with the device’s sponsor “resources permitting.” If the FDA is relying on resources that do not exist, it is setting itself up for failure.

Urgent Unmet Need

The lack of adequate resources is even more acute because the guidance defines “urgent, unmet need” much too broadly.  For example, it includes diseases like diabetes where the FDA’s example of an eligible device provides more frequent glucose readings that may not be as accurate as current devices offer.  There is no scientific evidence that more frequent glucose readings is an urgent, unmet need, especially if accuracy is reduced.  Similarly, devices with a new mechanism of action would not necessarily meet an urgent, unmet need if other treatments are proven effective for most patients.  This loose definition of urgent, unmet need is not surprising, since the guidance is modeled after similar new pathways developed for drugs – pathways that have similar shortcomings.  For example, new research indicate that companies are submitting dozens of new applications to FDA pathways intended for urgent, unmet needs, and most of those drugs do not qualify.[iv]  Using such vague terminology greatly adds to FDA’s workload, does not benefit patients, and slows down the process for companies that play by the rules.

Uncertainty about Benefits and Risks

The draft guidance states that the FDA may accept less certainty regarding the benefit-risk profile of EAP Devices.  However, the FDA already accepts very small, short-term studies as the basis for approval through the PMA process, and this is already putting patients at risk. Lowering the standards further would put patient safety in even greater jeopardy.

CDRH’s experience with the Wingspan stent is informative.  After the device was approved, it became clear that there is no evidence of benefit with use of the system in most patients with stroke.  Despite those findings, the company and the FDA continue to make the device available, positing a benefit in a subpopulation, based on a small, poorly defined study.[v]

Less Rigorous Evidence

The draft guidance notes that surrogate endpoints “allow for smaller trials or shorter follow-up…than traditional clinical outcomes of a disease or condition.”  A major problem with surrogate endpoints is that even the most reasonable surrogate endpoints often do not predict better health outcomes.  We agree with the statement in the draft guidance that, “The principle risk of accepting surrogate endpoints as the basis for approval is the possibility that patients will be exposed to a device that may ultimately be shown to have an unfavorable benefit-risk profile.”

Relying on Postmarket Data

In order to speed approval of PMAs, the draft guidance relies on postmarket data, stating that this may reduce the amount of premarket data needed and “directly impact when patients will have access” to medical devices.  However, the PMA process already has much lower standards in clinical trials than does the approval process for prescription drugs and already relies heavily on postmarket studies to provide information based on larger number of patients studied for a longer period of time.  For those reasons, we strongly oppose even greater reliance on postmarket studies, since, as the draft guidance cautions, “Greater reliance on postmarket data collection could undermine patient safety if the necessary and timely data collection does not occur.” 

In addition to concerns about lowering pre-market standards and emphasizing post-market studies, there is reason to be concerned about when postmarket studies would be completed and whether they would gather the intended safety and effectiveness data.  The draft guidance states that postmarket data will be “completed in a timely manner,” but it does not define the term “timely manner.”  The draft guidance further undermines the likelihood that postmarket data will be available in a timely manner when it states that the requirement will merely be a “best estimate and that circumstances may arise…that may adversely affect the ability of the sponsor to complete the post-approval study on time.”

FDA has improved its statistics for the required completion of post-approval studies, but it has not analyzed the quality of postmarket studies.  There are too many examples of postmarket studies that are started years after the requirement goes into effect, and then loses half or more of the patients to long-term follow-up.  If a sponsor fails to comply with post-approval requirements, the FDA could declare that the device is misbranded, “which could result in seizure, injunction, or civil money penalties.”  However, FDA has rarely used these powers in the past.

Conditional approval

The draft guidance states, “FDA would generally require postmarket confirmatory data as a condition of approval” when surrogate endpoints are used for approval for EAP Devices.  The word “generally” opens a loophole.  The use of surrogate endpoints already puts patients at risk; at the very least, the agency should always require postmarket confirmatory data for all such approved devices.

Non-implantable devices

The guidance states that EAPs should be for devices that are “non-implantable.” Over the last five years (2009 – 2013), FDA has approved on average 27 original PMA devices, with 13 of those devices being implants.  While we oppose the EAP for any Class III device, it would be even more inappropriate for implanted medical devices.

Forgoing Manufacturing Inspections

The draft guidance states that, “On a case-by-case basis, FDA may, at its discretion, allow a sponsor to provide less manufacturing information in their PMA application for an EAP Device,” and it added FDA may “forgo inspection” of manufacturing sites as part of the premarket review of EAP devices.  We strongly oppose this.  Inspections should be required for all new PMA devices, whether EAP devices or not.  This is necessary to make a level playing field for all manufacturers and for the sake of consistency and quality of the medical devices and the health and safety of patients receiving these life sustaining and life-saving devices.

Additional Concerns


The draft guidance states that, “The labeling of a device approved through the Expedited Access PMA program would include a succinct description of the uncertainty about anticipated benefits and risks and the extent of data that supported approval and required post-approval study(ies).” It is unlikely that patients will ever see the label and we question whether health care professionals read the labels.  We are concerned that they will use the device without reading the label because it has been approved by the FDA.

Approving more than one EAP device

The draft guidance states that, “FDA may approve more than one EAP device for the same condition because of the possibility that the data from the post-approval study may not confirm certain safety or effectiveness aspects of the device under the conditions of use.”  This opens the door to unlimited numbers of EAP devices to treat the same condition.  Moreover, it contradicts one of criteria for addressing an unmet need that states, “No approved alternative treatment or means of diagnosis exists.”

Potential to address unmet needs

The draft guidance also states that for a device to qualify for an EAP it must “Demonstrate the potential to address unmet medical needs.”  The word potential is an enormous loophole in this criterion.  It could be argued that any device has the potential to address unmet needs.  That sentence should be reworded to state: “The device will demonstrate that it addresses unmet medical needs.”


Members of the Patient, Consumer and Public Health Coalition strongly oppose this draft guidance. The draft guidance shifts the risk-benefit paradigm so that patients face greater risks.  The draft guidance states that the Expedited Access PMA program “will help patients have more timely access to these medical devices.”  While patients want access to devices that meet unmet medical needs, few patients are willing to risk their lives to do so, especially if a few more months of research could result in devices that are proven to be safe and effective.

American Medical Women’s Association

Annie Appleseed Project

Breast Cancer Action

Center for Medical Consumers

Connecticut Center for Patient Safety

Consumers Union

Jacobs Institute of Women’s Health

National Center for Health Research

Our Bodies Ourselves

The TMJ Association



The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202)223-4000 or at

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