DIA Meeting on Personalized Medicines and Companion Diagnostics

The registration study for a personalized biopharmaceutical therapy also serves as the registration study for the accompanying companion diagnostic, and it is often conducted in a small, molecularly-defined rare disease population. The frequent lack of available alternative treatment options in rare diseases makes it important for industry and FDA to reach a mutual understanding on the standards and evidentiary expectations needed for co-registration of novel personalized therapies in the rare disease setting with their companion diagnostics, so that safe and effective treatment options can be made available to patients as quickly as possible. In this session, a panel of experts from industry and government will discuss the latest innovations in registration study design for biomarker-defined rare diseases.