The Liver Forum aims to advance the regulatory sciences for the treatment of NAFLD/NASH and liver fibrosis by providing a neutral, independent venue for ongoing multi-stakeholder dialogue. Their work facilitates the best science-based decisions on how to study efficacy and safety in real-time by using collective knowledge and experience with therapies for advancing liver disease. Previously, Executive Director Veronica Miller had introduced ongoing work with the Placebo Arm Database Project, co-chaired by Manal Abdelmalek.
Jörn Schattenberg leads this conversation with the observation that in Manal’s past appearances on the podcast, she has discussed the value of stabilizing disease. Jörn asks whether the Forum is creating new insights on that front. Manal circles back to the challenges presented by an inability to predict an a priori placebo response rate and several issues in managing samples through trials. She notes that if researchers can integrate placebo arms from multiple trials, this will lead to far larger data sets. Additionally, this will strengthen the ability to create deeper insights and more robust solutions to issues surrounding the best way to manage and standardize these trial elements. From here, Jörn’s question shifts focus toward composite trial endpoints. After a brief discussion on this issue, the conversation turns to the value of AI and better controlled study analysis. Roger poses a question regarding reducing placebo rates. Manal suggests that the goal is not to lower placebo rates, but instead to standardize them in the most accurate ways possible. The conversation concludes with Jörn noting that he is constantly concerned about whether he is matching patient and trial to maximum benefit.