Situation
A medical device company’s pivotal study for a premarket approval (PMA) application encountered a major challenge: A primary effectiveness endpoint did not meet statistical objectives despite providing clear clinical benefit and maintaining an acceptable safety profile. Since the product’s benefit-risk profile supported market authorization, the critical question was: How to make this case with FDA?
Solution
It was Bright’s time to shine. Our approach to supporting the client’s PMA application was methodical, transparent, and collaborative, with the end goal of telling the clinical story given the available data:
- State the facts. Rather than attempting to argue with FDA on the merits of the statistical testing, Bright advised the client to clearly acknowledge the primary effectiveness endpoint did not meet its objective in a formal statistical sense.
- Isolate the cause. Because the primary effectiveness endpoint was composed of multiple outcomes (that is, it was a “composite” endpoint), Bright deconstructed the composite endpoint and reported the success rate of each individual component. Through this exercise, Bright determined that one specific component was driving the composite endpoint.
- Highlight the safety profile. Since the outcome tracked by this one specific component was relatively benign, it had minimal impact on the device’s clinical safety and effectiveness. Bright advised the client to emphasize the clinical benefit and strong safety profile of the investigational device to bolster the case for the device’s PMA approval.
- Bring it all together. Bright helped the client present a comprehensive view of the data, demonstrating that the strong safety profile, combined with the overall device effectiveness, supported a favorable benefit-risk ratio and ultimately justified PMA approval by the FDA.
To successfully execute the above strategy, Bright leveraged the expertise of—and relationships built between—Bright, the client, the study’s national principal investigator, and the FDA.
Significance
Bright’s approach contributed to the ultimate success: PMA approval. This means that the device can be commercialized in the U.S. and made available to patients with complex conditions.
Learn more about partnering with Bright for creative solutions to your clinical research needs.
