Our week of “Greatest Hits” episodes from the vault continues with this conversation from the episode reviewing the NASH Patient-Focused Drug Development meeting sponsored by the Global Liver Institute and held at the FDA on November 4, 2021. This conversation focuses on the perspectives that patient advocates brought to this session.
Late 2021 and early 2022 traced a series of events that sensitized FDA to the need to look “beyond the biopsy” (or at least semi-quantitative reads) to an approach that would more accurately reflect the benefits of NASH drugs in development and the extreme need patients and their advocates felt for new medications. One critical event on this path was the patient-focused drug development (PFDD) meeting the Global Liver Institute organized and held at FDA on November 4, 2021. At this meeting, FDA reportedly acknowledged that if they had understood patients’ perspectives better a couple of years earlier, the path of NASH drug development might have been different.
As I wrote at the time: In this conversation, Global Liver Institute Founder, President and CEO Donna Cryer describes the history of the basic Patient-Focussed Drug Development concept and this particular NASH event. Patient and patient advocate Terri Milton discusses the message she hoped regulators would take away from this session and why she believes the group may have succeeded. Finally, the group discusses why regulators and advocates vary so greatly in terms of how they view the efficacy:safety tradeoff for first-in-class drugs.