(Washington, D.C., January 29, 2021) Thank you to the Food and Drug Administration (FDA) for the informative briefing earlier today titled, “Regulatory Perspectives for Development of Drugs for Treatment of NASH.” We appreciate that the FDA has followed through on our 2020 line of dialogue and provided a response to many of our previously communicated concerns around acceptable endpoints, biomarkers, non-invasive diagnostics, and accelerated approval.

We appreciate the FDA’s acknowledgement of the seriousness of NASH, but based on the evidence so far, there is still a lack of urgency. It is critical for the FDA to not underplay the value of stopping progression. As the FDA stated in today’s presentation, there is immature evidence on the rate of progression in patients, but the assumption of length of use (chronic) used to balance safety is still not necessarily the answer. To fill this gap in knowledge, the FDA must consider the patient with NASH perspective and explicitly invite patient input on safety tolerance and benefit-risk acceptability. Our critical message to the FDA remains the same – delay has a risk. Doing nothing has a risk. Striving for perfection in the midst of complexity has a risk. Our patients are risking their disease progressing to stages with life-threatening complications like cirrhosis, liver cancer, and death.

We also understand that it is the responsibility of sponsors to prove scientific advancement and reasonable evidence of efficacy. However, it is also necessary for a complementary relationship to exist between regulators and sponsors to aid their respective efforts to support patients. The field needs a set threshold or level of acceptance provided by regulators to lead to safe and effective approved therapies. On top of this, it was disheartening to hear the Division of Hepatology and Nutrition (DHN) stance on NAFLD. While we understand that therapy approvals may fall within another division of the FDA, it is still disappointing to hear the lack of willingness to collaborate and explore NAFLD solutions.

As the voice of the liver patient community, including those with NASH, we patiently await the release of the FDA’s updated guidance later this year. We hope that the FDA will expand upon and clarify the Agency’s viewpoints even further. The lives of liver patients depend on it.

View the presentation from the January 29 briefing here: https://sbiaevents.com/past-sbia-events/


About Global Liver Institute

Global Liver Institute (GLI) is a 501(c)(3) tax-exempt not-for-profit organization, headquartered in Washington, D.C., United States, with offices in the U.S. and Europe. GLI’s vision is for liver health to take its place on the global public health agenda commensurate with its prevalence and impact. GLI’s mission is to improve the lives of individuals and families impacted by liver disease through promoting innovation, encouraging collaboration, and supporting the scaling of optimal approaches to help eradicate liver diseases. Follow us on TwitterFacebookInstagram, and LinkedIn.