Great news for our friends in the the Gaucher community!
The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur the development of new treatments for Gaucher disease.
Gaucher disease is a rare condition characterized by a buildup of certain fatty substances in certain organs, particularly the spleen and liver. This causes these organs to enlarge and can affect their function. Symptoms include abdominal pain, bone issues, blood disorders, and more.
According to the strategy document, these agencies outlined two possible approaches to better facilitate investigations of treatment for those with systemic, types I and II Gaucher disease:
- Extrapolation across age groups within a single drug development program, including through modeling and simulation techniques, to better predict how a medicine may work in children and adolescents based on studies conducted in adults or other pediatric populations
- A multi-arm, multi-company clinical trial, which addresses the feasibility of developing multiple drug products in a time-efficient manner and would mean the same control arm is used to compare more than one medicine under evaluation thereby reducing the total number of children included in trials.
Our government at work!
Furthermore, these strategies can be applied to other drug development of other rare diseases! To the nameless FDA employees who too often do good work with not much recognition:
Thank you!
