Drug Trials FAQ

You see press releases and literature related to treatments that are in development for PFIC, but what do those press releases mean? Is treatment available now? What steps do they still need to go through? We are working to make sense of all of the information that is coming at our community. We hope that these therapies and future therapies will provide the much needed hope that the PFIC community needs for a better quality of life. This page is intended to make this information digestible to all, so that those who are affected with PFIC can understand what is happening in the complex world of treatment development.

Drug Trials and Therapies

How do Drug Trials Work & Get Approved?

What does this mean to me?

The answer to that will vary. We are hopeful that both of these drugs will be proven as safe and effective treatment options by the FDA and EMA for PFIC within the next 2 years. If that happens, accessing the medications still may take some work. Especially related to the multiple subtypes of PFIC. Furthermore, some countries have different regulatory processes in place, meaning the FDA and EMA approval does not mean access to medication in all countries.Our end goal would be treatment for all, but it may time a lot of time and work. Learning about the process for drug approval in your country and participating in advocacy opportunities is essential to making that happen. We are dedicated to assist in this process, but we can not do it alone. If you have questions on how you can advocate for drug approval in your country, please contact us!

If you still have questions about the drug trials, you can contact us using the Contact Us button below. We will try and get back with you as soon as possible and try our best to answer your question!