Situation
Opting for a rare clinical event endpoint meant that a medical device company’s proposed clinical trial would require thousands of subjects and a years-long enrollment period. Options were limited as all prior device studies in this therapeutic space had used this same rare endpoint, too.
Despite being clinically relevant, the rare clinical endpoint was constraining study designs, lengthening timelines, and increasing financial burdens for companies – in effect, preventing novel therapies from being evaluated and commercialized in the U.S.
Patients suffer when innovation lags. The medical device company reached out to Bright for a solution.
Solution
Bright proposed a novel technical endpoint to the client – one that assessed device mechanical effectiveness versus a clinical endpoint. In doing so, the study sample size could be reduced, and the study timeline shortened, therefore making the study viable for this start-up company.
The client’s leadership was willing to go to the FDA to discuss how the novel technical endpoint could drive innovation in this therapeutic space while still providing scientifically sound data. Bright helped the client navigate the FDA’s pre-submission process – a route for medical device companies to get early, interactive feedback from the FDA about their regulatory and clinical strategies. Together with the client, Bright crafted a compelling science-backed argument and hosted a congenial discussion with FDA experts.
However, during the pre-sub meeting, the FDA said “no” to the proposed novel strategy, claiming the rare clinical event endpoint was still the standard. Bright and the client headed back to the drawing board. To everyone’s surprise, the next day, the client received a follow-up message from the FDA. The FDA’s discussion had continued offline, and they agreed to accept the proposed novel technical endpoint– a huge win for patients and for medical device innovation in this therapeutic space.
Significance
The client’s clinical trial is ongoing, and pursuing the novel technical endpoint is expected to result in several benefits:
- The sample size for the clinical trial was considerably reduced, which will shorten the enrollment period, expedite follow-up timepoints, and overall shrink the study timeline.
- With a shorter timeline, the client anticipates significant cost savings for their clinical trial and a faster pathway to commercialization.
- By leveraging the pre-submission process, the client received valuable, early feedback from the FDA and built an amicable relationship with the review team which continues today.
Getting the FDA on board with a novel technical endpoint was one huge step toward innovation in this therapeutic space, with the long-term goal being to benefit patients with better technology. Be brave with Bright!
