Key result
The LITMUS consortium highlights that successful biomarker qualification for NAFLD and NASH requires rigorous analytical validation, clear context of use, and early regulatory engagement.
Why the study?
Biomarkers can accelerate drug development, yet few have been approved through regulatory biomarker qualification pathways.
Sharing knowledge of interactions with regulatory agencies regarding biomarker qualification can help accelerate drug development across disease indications.
Regulatory lessons from LITMUS may expedite biomarker qualification; leaves open accelerated development across indications.
Biomarkers have the potential to accelerate drug development, as early indicators of improved clinical response, to improve patient safety, and for personalised medicine. However, few have been approved through the biomarker qualification pathways of the regulatory agencies. This paper outlines how biomarkers can accelerate drug development, and reviews the lessons learned by the EU IMI2-funded LITMUS consortium, which has had several interactions with regulatory agencies in both the US and EU regarding biomarker qualification in patients with non-alcoholic fatty liver disease and non-alcoholic steatohepatitis. Sharing knowledge of such interactions with the scientific community is of paramount importance to increase the chances of qualification of relevant biomarkers that may accelerate drug development, and thereby help patients, across disease indications. A qualified biomarker enables a decision to be made that all understand and support in a common framework.
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Rasmussen et al. (2022) conducted a review in Non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Biomarker qualification was evaluated. The LITMUS consortium highlights that successful biomarker qualification for NAFLD and NASH requires rigorous analytical validation, clear context of use, and early regulatory engagement.
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