Design and rationale for a real-world observational cohort of patients with nonalcoholic fatty liver disease: the TARGET-NASH study.

Challenge

Drug development for NAFLD/NASH was hindered by the absence of longitudinal, real-world cohort data spanning the full disease spectrum—from simple steatosis through cirrhosis—with the depth of clinical, laboratory, and patient-reported information required to support regulatory-grade natural history studies.

Solution

The TARGET-NASH study was designed as a multi-site, longitudinal observational cohort enrolling pediatric and adult NAFLD patients at academic and community sites across the US, with centrally abstracted electronic health record data, biospecimen collection, and annual patient-reported outcomes.

Impact

Establishing the TARGET-NASH registry provided the foundational infrastructure for all subsequent natural history, endpoint validation, treatment pattern, and patient characterization studies in the hepatology portfolio, directly enabling the evidence generation capabilities that now support multiple pharma partnerships.