CMHC Pulse Blog

From a clinical trials perspective, studies from phase 2b and on need to have a liver biopsy per the FDA guidance.12 Prior to phase 2b, other less invasive assessments can be performed (e.g., imaging), although the different clinical accuracies of the respective processes can be expected to lead to differences in the characteristics of the populations studied pre and post phase 2b. The risk of performing a liver biopsy is a hurdle for enrollment in many studies. Increasing awareness about the implications and complications of NASH is important, but at this time, NASH is still an underappreciated disease for which many people do not want to get tested. This is compounded by the current absence of approved therapies and thus the enticements to encourage patients to undergo a liver biopsy are currently comparatively limited. It is expected that this will be partially alleviated by providing a better understanding of the exciting new therapies for NASH that are currently in development

Another challenge that impacts site engagement and, consequently, enrollment rates is the analysis of the liver biopsy and the differences in assessments performed by the local pathologist and the central specialist pathologist. A diagnosis of NASH by a local pathologist may lead to the conclusion that the patient is suitable to be enrolled in the trial. However, it is not uncommon that the central specialist pathologist deem the patient to be unsuitable for the study when the NAFLD Activity Score (NAS)13 criteria are used to assess suitability of the subject against the enrollment criteria of the clinical study protocol. The NAS scoring system is designed to allow for assessment of changes with therapy during the course of a clinical trial and is not commonly utilized at local level. Such situations of discordance can demotivate sites if they are unable to enroll subjects who, they feel, are appropriate for the study. Improving communication between the local pathologist, investigator and the central pathologists is crucial to ensure understanding of the role of the central pathologist, i.e., to ensure uniformity of the study population and the consistency of assessment throughout the study.

Subscribe

Sign up to receive updates on educational opportunities, complimentary content, exclusive discounts, and more.