Marie Thibault

FDA has published draft guidance to give industry a better idea of what the agency looks at when deciding whether to approve an Investigational Device Exemption (IDE) application. More specifically, the guidance points out what information is helpful in making a decision about the benefit-risk tradeoff of the proposed study.

IDE applications must be submitted when a company or sponsor wants to conduct a study on a life-sustaining or life-impacting investigational medical device. The draft guidance, “Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions (IDEs),” lays out the elements that influence an approval or disapproval decision. FDA makes it clear in the opening paragraphs of the draft guidance that it will usually turn down an IDE application if the benefits or the knowledge from the study do not outweigh the risks to the study subjects. Much of the guidance document is dedicated to explaining how FDA makes this distinction.

IDE applications do not follow a set template, so sponsors may be uncertain of what to include and how to format the application. In a memo on the draft guidance, Morgan Lewis partners M. Elizabeth Bierman and Phoebe Mounts highlighted key items the guidance suggests including in an application. They point out that “The FDA’s draft guidance recommends that IDE sponsors include a section in their applications that summarizes the primary or most important considerations in the IDE benefit-risk assessment. The Agency states that the summary should not be a comprehensive review, but rather, should focus on those factors likely to significantly affect the FDA’s decision.”

These factors include patient preferences, a description of the device, an assessment of potential device risks, likelihood and types of risks and benefits, risk management, disease description, available alternatives, and level of uncertainty in light of the development stage, among other items.

Jennifer Newberger, an associate at Hyman, Phelps & McNamara, wrote about the draft guidance for the firm’s FDA Law Blog. She wrote, “It is encouraging that FDA continues to focus on the balance between benefits and risks, recognizes that pre-clinical data can reduce clinical requirements, and recognizes that there may be different considerations for different patient populations.”

In an appendix of the draft guidance, FDA outlines hypothetical situations. The first is a pivotal study to treat a life-threatening disease with alternative therapies that work only temporarily and come with serious adverse events. In this case, the agency is likely to allow the pivotal study to proceed because the high risk and uncertainty is outweighed by non-clinical and feasibility data, the lack of good alternatives, and the disease progression without therapy.

The second hypothetical scenario involves a device feasibility study for “a life-limiting condition with reasonable alternative treatments.” In this case, FDA would decide not to approve the application until biocompatibility data is provided to allay biocompatibility concerns. In the third situation, the hypothetical IDE application is for an early feasibility device study for a life-threatening disease with no alternatives. In this case, FDA would decide to approve the application because of the lack of alternative therapy, clinical use of a similar device showed no likelihood of “catastrophic failures,” patients are likely to benefit, and any other nonclinical study would not give more useful information.

Comments on the draft guidance are requested by September 16.

Marie Thibault is the associate editor at MD+DI. Reach her at [email protected] and on Twitter @medtechmarie. 

[Image courtesy of STUART MILES/FREEDIGITALPHOTOS.NET]