Comment from Bower, Kaylin

This comment regards FDA docket FDA-2026-N-3947: "Impacts of Patient-Focused Drug Development Meetings; Established of a Public Docket; Request for Information and Comments": Attachment: “Railroading at the FDA” Editorial published 08 November 2016 in Nature Medicine (22, 1193). *Key excerpts: -“During the meeting, committee members found themselves ‘under intense and near-incessant pressure from a large public audience’ packed with patients and advocates, all but one of whom gave testimony in favor of approval.” -”...this decision sends a signal that evidence from poorly designed clinical trials-if presented in the context of vocal patient advocacy-can be sufficient to obtain accelerated approval of a rare-disease drug” -”Crucially, because commercial drug development is focusing increasingly on rare diseases, the factors that complicated the Exondys decision-very small clinical trials, desperate patients lacking therapeutic options and reliance on surrogate endpoints-will come up again and again. With each rare-disease drug application, these challenges will be confronted anew, and they will risk more internal FDA disputes and staff resignations” -”Exondys’s approval is a stark illustration of the challenges of balancing science with patient input in regulatory decisions.” *Commentary: This is NOT what should be considered “patient-focused drug development” and should NOT be the outcome of PFDD! This egregious Exondys FDA approval should serve as the epitome of what PFDD should NOT be and should never be allowed to be! PFDD should NEVER be allowed to serve as an excuse, justification, or cover for the bullying, abusing, and harassing of FDA staff! PFDD should also NEVER be allowed to serve as an excuse, justification, or cover for the FDA to approve drugs that do not meet the statutory requirements for approval! The FDA MUST take a strong stance against this type of harmful, egregious, inappropriate, and unacceptable behavior from the “patient advocacy community” and must NEVER accept it under the auspices of “PFDD”! Sadly, the FDA’s handling of this egregious Exondys approval has set the precedent that such inappropriate, abusive, unethical, and egregious “patient advocacy community” behavior leads to the desired outcome for these “communities.” The FDA must now do everything in its power and ability to send the strong message and stance that decisions like eteplirsen will no longer happen, no matter how much bullying, pressure, abuse, intimidation, and “advocacy” efforts are directed at them (the FDA) by the “patient advocacy community.”