The draft guidance has been in development since 2011 and is the result of collaboration between the FDA and the European Medicines Agency (EMA). Together, the agencies gathered feedback from patient, scientific, and drug development communities to develop “Gaucher disease: A strategic collaborative approach from EMA and FDA.”
The document was released today as an FDA guidance, using Gaucher disease as a model. However, the described approach could be extended to other rare pediatric diseases, according to the FDA.
“The proposed approach, if adopted by sponsors, has the potential to ultimately help speed products to market by minimizing the time it takes to conduct drug studies,” FDA trade press officer Jeremy Kahn told Outsourcing-Pharma.com.
“Having an approach agreed upon by FDA and European regulators can facilitate collaboration among study sponsors and regulators and get studies started in a time efficient manner,” he added.
As Kahn explained, the proposed strategy includes two elements: “the use of statistical models and simulations that may eliminate the need for certain clinical studies and the development of multi-arm, multi-sponsor clinical trials that reduce the total number of patients needed for placebo control groups.”
The FDA previously encouraged the use of modeling and simulation as it unveiled its plans to implement aspects of the 21st Century Cures Act.
The FDA is seeking comments on the draft guidance for 60 days.