S3-E11.4 – NAIL-NIT: Retrospective Analysis & Prospective Studies

S3-E11.4 - NAIL-NIT: Retrospective Analysis & Prospective Studies
Work flows for NAIL-NIT: Retrospective analysis planning to produce results in 1-2 years, then prospective studies planning to produce results in 4-6 years.

This wrap-up conversation of our NAIL-NIT episode provides greater detail on NAIL-NIT’s research plan. The plan itself includes two elements: a retrospective analysis of data collected to date by drug developers in their own clinical development programs and a six-year prospective study currently estimated to include 1,000 – 1,300 patients, with major adverse liver outcomes (MALOs) anticipated to report in four years and final results in six.

In response to a question, Mazen Noureddin suggested that we might see some significant guidance to drug developers and manufacturers within a year on the topic of screen failure rates. Over time, the retrospective data will also afford the opportunity to test some of the newer composite test measures like FAST and MAST against larger samples of patients. Sen Sundaram expressed a different target for one-year results: guidance from one or more regulatory agencies about how many patients they will want to see in key clinical trial patient cohorts and sub-populations.

In terms of the prospective studies, Louise Campbell asked whether certain segments of patients who had screened out of previous studies would be appropriate for the prospective sample. Stephen Harrison noted that one particular clinical trial, North Sea Therapeutics, included over 100 patients with F3 and NAS of 3 but no balloon hepatocytes. He also noted that by using the Summit Clinical Trial network to recruit a significant share of patients, they would be recruiting a significant share of patients through community trial sites, which can produce patients faster than academic sites. In response to the closing question, NAIL-NIT panelists made a plea for more companies to join this effort.

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