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Last week, Jeff McIntyre of the Global Liver Institute introduced a draft report from the Institute for Clinical and Economic Review (ICER) on resmetirom and obeticholic acid for NASH. Jeff, an expert reviewer of the report, returns with the Surfers to expand on the contents, its shortcomings and potential implications. Among the panelists are Veronica Miller of the Liver Forum and new guest, Hannah Mamuszka, who is founder and CEO of Alva10.

10:05 Introducing the Report
Jörn Schattenberg briefly revisits the relatively large datasets on these two drugs. Veronica is “quite astounded” that this report would be generated before the emergence of a formal peer-reviewed publication. While Jeff is pleased with ICER’s inclusion of patient participation in this report, he underscores critical concern around a statement claiming that NASH and NAFLD are not progressive diseases.

21:31 Assessment of Disease Prevalence
Hannah asserts that the report “understates the problem of the size of population" and fails to consider the strength of data linking NAFLD and NASH to cardiovascular outcomes, liver cancers and beyond. She suggests that commercial payers are looking to address the following questions in their assessment of the drug candidates: 

• What is the size of the patient population and what is going to be the effect of coverage in terms of how many patients are we going to prevent from progressing to liver transplant? 
• How many patients are we going to keep out of the hospital with chronic events? 

Ultimately for Hannah, the report's analysis misses many of the potential cost savings of an effective drug.

29:06 Keeping Pace with Field Advancements
Louise notes the intensity and speed in which the Fatty Liver field has developed over the previous decade. Provided this explosion in knowledge, she wonders whether we are able to effectively collect and implement the changes reflected by all this emerging data. Jeff and Roger add that socioeconomic shifts and the COVID-19 pandemic amplify already swelling rates of incidence. Jörn poses a cogent line of questioning to consider in establishing cost-effective interventions:

• Who was enrolled in the clinical trials? 
• Where's the evidence for a subgroup of patients benefiting from therapy?
• How can we identify them moving forward and outside of clinical trials?

38:55 Biopsy and Biomarkers
Veronica describes the dichotomy of biopsy. On one hand, AI-assisted histological assessment of fibrosis lends fascinating insights into the nuances of the liver as an organ. On the other, biopsy is still a very blunt tool inadequate in assessing the effectiveness of a drug. She states that it “hurts to see that this is what happens when we are not applying the right diagnostic markers and tools” and that “in a couple of years we'll be in a totally different data zone” in terms of having much more quantitative data to employ when assessing the safety and efficacy of these drugs.

48:36 Implications and Final Thoughts
As the session winds down, the group considers various other aspects for which this document can potentially have an impact. Comments range from notes on pricing to what FDA factors for approval.

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