S2-E54 - Go Inside a Pivotal Event: the NASH Patient-Focused Drug Development Meeting

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Global Liver Institute Founder, President and CEO Donna Cryer and her fellow patient and patient advocate Terri Milton join Stephen Harrison, Louise Campbell and Roger Green to review the ground-breaking NASH Patient-Focused Drug Development meeting held on November 4, 2021.

This patient-focused drug development meeting could be viewed as the culmination of seven years of effort by Global Liver Institute to encourage regulators and other stakeholders to think differently about NASH. In this episode, one post-transplant patient (Donna) and another patient living with disease while awaiting a transplant (Terri) share the comments and perspectives they brought to the November 4 patient-focused drug development meeting, while Stephen, Louise and Roger share their own points of view, which vary somewhat from the patients and each other. This is less scientific than the typical SurfingNASH session, but far more important in reminding all of us who we are here to help and what their goals for therapy are.

Highlights include:
2:51 – Roger Green introduces patient and patient advocate Terri Milton
3:11 – Terri tells her 20-year story of living with NAFLD, NASH, cirrhosis, HCC and other related health challenges
6:23 – Roger comments on “Dr. Google” and Stephen Harrison discusses Terri’s treatment history
8:19 – Terri finishes her story by discussing her own accountability for her health going forward and shares “one fact you wouldn’t know”
12:29 – Icebreakers from the panelists start with Louise Campbell discussing a high-profile UK athlete needing a liver transplant at a young age due to PSC
20:38 – Donna Cryer kicks off by discussing the history of Patient-Focused Drug Development 
22:10 - The inception of Global Liver Institute and its work on policy in regulatory science for liver disease
24: 50 – Co-development of Patient-Focused Drug Development meeting under the aegis of GLI in collaboration with FDA
27:19 – Terri describes the messages she hoped to deliver to FDA,  those she has delivered to researchers and her favorite moment of the  NASH Patient-Focused Drug Development event 
29:17 – Donna describes the pivotal points FDA officials experienced in the NASH Patient-Focused Drug Development meeting
30:08 – Roger’s surprise that FDA has not learned that patients value stopping disease progression as an end goal
30:48 – Louise: Does it matter that NASH is “a catalog of…system and health failures” when patients  progress to cirrhosis?
32:07 – Donna on how she has lived with her IBD and how this applies in NASH
33:30 – Terri raises her Primary Care MD’s awareness of the diabetes -NAFLD link
34:36 – Donna discusses challenges GLI wants to present to FDA vs. those it does not want to present
35:29 – Roger: more important to see progress made on non-invasive diagnostics or getting a first drug to market, even with a not-great efficacy:safety ratio?
36:02 – Stephen discusses his reasons for selecting non-invasive testing and the steps he is taking to drive this discussion forward
37:37 – Stephen’s thoughts on FDA and its perspective on these issues
39:02 – Louise’s perspective on Roger’s question, those with "severe disease" will take more risks
40:07 – Donna: “It’s not just people with severe disease,” but anyone who understands the disease path, complications and challenges
41:05 – Donna: the patient’s goal is to have a full life, not merely avoid negative medical events.
42:16 – Roger is struck by the “paternalism” of institutional medicine
43:42 – Louise: this reminds me of the debate around false positives in testing
45:24 – Roger’s final question: what’s the one thing each of you wants to see come out of this?
45:34 – Terri,  Louise, Roger answer
48:07 – Donna answers: “It’s not the answer that p