RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > EMA Consults on Regulatory Requirements for Chronic Liver Disease Drugs

EMA Consults on Regulatory Requirements for Chronic Liver Disease Drugs

Posted 01 June 2017 | By Michael Mezher 

EMA Consults on Regulatory Requirements for Chronic Liver Disease Drugs

The European Medicines Agency (EMA) on Thursday launched a public consultation to gather input on a future reflection paper discussing the regulatory requirements for developing drugs to treat chronic non-infectious liver diseases.

According to the agency, there is an unmet medical need for drugs to treat chronic non-infectious liver diseases such as non-alcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC).

And while there are an increasing number of development programs for drugs to treat those diseases, EMA says that its "current regulatory experience reveals the need for further guidance" in order to avoid potential snags during drug development.

Specifically, EMA says the diseases pose a significant challenge for drugmakers to study, as all three have a long progression and symptoms that are either unspecific or non-predictive of long-term outcomes. Furthermore, EMA says the use of "hard" clinical outcomes like liver transplantation or death raise feasibility issues for studies, and the need for repeated liver biopsies deters patients from enrolling in studies and increases risk.

Because of these challenges, EMA says there is a strong need for a validated surrogate endpoint to reduce the need for biopsies.

"There is also a need for the identification of the most suitable patient population, balancing unmet medical needs, the mechanism of action of drug candidates, and the disease severity with regard to grade of inflammation and stage of fibrosis development," EMA writes.

EMA also says there are questions as to the appropriate requirements for observation, licensing, post-approval studies and addressing ethical questions and patient adherence.

As such, EMA is asking for stakeholders, including drugmakers, academia, scientific associations and other regulators for input on potential endpoints, surrogate endpoints, biomarkers and study designs that could help guide sponsors' development programs in this area.



© 2022 Regulatory Affairs Professionals Society.