S4-41.2 – Naim Alkhouri on clinical trial populations and the future decisions of FDA

S4-41.2 - Naim Alkhouri on clinical trial populations and the future decisions of FDA
In this conversation, the Surfers are joined by Naim Alkhouri to discuss the recent FDA workshop for NIT development. The group discusses loss aversion, screen fail rates, the future of FDA decision making and more.

In Season 4, Episode 41, the surfers (Jörn Schattenberg, Louise Campbell and Roger Green) review highlights from the FDA’s NIT workshop in three seperate interview sessions with guests Naim Alkhouri, Laurent Castera and Veronica Miller. Each guest participated in some form at the meeting and shares slightly differing but incredibly insightful perspectives.

Roger starts this conversation by discussing loss aversion – the idea that an individual’s decision does not reflect an overall fear of risk, but a specific fear of risking something they do not want to lose. Researchers and advocates define loss in terms of time to bring a drug to market or screen fail rates, but for government regulators, “loss” can refer to prestige or credibility when they make what are publicized as “wrong decisions.” Naim registers the issue, but notes that in his clinic, no more than 5% of people test in a way that might qualify them for clinical trials, and probably 1-2% ever participate altogether. He contrasts the high percentage of population with identifiable MASH (which might be 15%) with the 1 in 1,000 “unicorns” in the population that can qualify for trials, suggesting this demonstrates how strongly we need to reconsider testing standards and protocols. Jörn mentions that the FDA commentator in the final session suggested we avoid surrogates altogether and instead look into a million person trial. Naim agrees that this is not feasible. From here, the group moves to other issues, including cause of death among patients with F0 to F3 fibrosis. As the conversation continues, Louise comments on the idea of doing broader, multi-organ or multi-outcome trials with drugs, as have been done with vitamins. She ends her comment by stating, “I would rather go into the future making the wrong decisions for the right reasons than the right decisions for the wrong reasons.” This statement represents a broader look at how we approve MASH drugs. Naim and Jörn both agree. In the last part of this conversation, Roger asks what Naim anticipates FDA might do next. Naim talks about studies generating more data with more NITs that might lead to what he describes as his “dream” – continuing the biopsy pathway but providing one alternative pathway based on a combination of NITS.

Plenty more ideas are explored as this is both a fascinating and pivotal workshop which covers a range of topics on NITs with presentations by the some of the field’s most innovative and knowledgable contributors. If you have questions or comments around the workshop, NITs, drug development or any other themes addressed in this episode, we kindly ask that you submit reviews wherever you download the discourse. Alternatively, you can write to us directly at questions@SurfingNASH.com.

Stay Safe and Surf On!