NAFLD and NASH biomarker qualification in the LITMUS consortium – Lessons learned

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...

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Published inJournal of hepatology Vol. 78; no. 4; pp. 852 - 865
Main Authors Rasmussen, Daniel Guldager Kring, Anstee, Quentin M., Torstenson, Richard, Golding, Bruno, Patterson, Scott D., Brass, Clifford, Thakker, Paresh, Harrison, Stephen, Billin, Andrew N., Schuppan, Detlef, Dufour, Jean-François, Andersson, Anneli, Wigley, Ioan, Shumbayawonda, Elizabeth, Dennis, Andrea, Schoelch, Corinna, Ratziu, Vlad, Yunis, Carla, Bossuyt, Patrick, Karsdal, Morten Asser
Format Journal Article
LanguageEnglish
Published Netherlands Elsevier B.V 01.04.2023
Subjects
accelerated approval
Biomarkers - metabolism
context of use
Drug Development
drug development tool
EMA
FDA
health authorities
health authority
Humans
Non-alcoholic Fatty Liver Disease - diagnosis
regulatory interactions
context of use
health authorities
FDA
health authority
drug development tool
EMA
accelerated approval
regulatory interactions
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Summary: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.
ISSN:0168-8278
1600-0641
DOI:10.1016/j.jhep.2022.11.028