Does anyone have insight into or experience with reporting concerns to the FDA regarding their approval of drugs based on inappropriate statistical methods and endpoints, such as but not limited to comparing median percent change from baseline (PCHG) in seizures between groups, and comparing the responder rate derived from PCHG between groups?
A paper discussing the lack of a breakthrough in seizure control despite an exponential growth of approved anti-epileptic drugs provides evidence that the use of the above poor methods and endpoints has persisted for over 30 years and remains the golden standard for epilepsy drug trials to this day. Here are a few recent studies of the sort (L-DEE-2024, E-FS-2023, Z-FS-2021, O-MS-2020). Iām afraid this trend will persist for as long as the regulatory agencies deem these acceptable. I hope to see real breakthroughs in this field, which would require applying the appropriate methodology and endpoint for these studies, and we need this message to come directly from the regulatory agencies.