The United States Food and Drug Administration (FDA) released a new draft guidance as part of its stated commitment to assist sponsors and expedite drug development for rare pediatric diseases, Outsourcing-Pharma.com reports. FDA collaborated with the European Medicines Agency (EMA) to develop the draft guidance. The draft guidance was based on the document “Gaucher disease: A strategic collaborative approach from EMA and FDA,” which FDA and EMA began developing in 2011 by gathering feedback from patient, scientific, and drug development communities.
FDA released the new draft guidance, “Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry,” on December 6, 2017
FDA trade press officer Jeremy Kahn told Outsourcing-Pharma.com that “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 studies.” Kahn went on to explain that “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.”1
The proposed strategy contains two key elements. First, using statistical models and simulations might eliminate the need for particular clinical studies. Second, the development of multi-arm and multi-sponsor clinical trials could reduce the total number of patients necessary for placebo control groups. Previously, FDA encouraged the use of modeling and simulation when the agency revealed its plans to implement aspects of the 21st Century Cures Act.2
Biopharmaceutical companies discover and develop innovative products to solve today’s biggest healthcare issues. Policies continue to change as new pathways emerge for rare pediatric diseases, but regulators’ expectations are not lowering. Learn more here about how we help small startup to large multinational biopharmaceutical companies traverse product development pathways for rare pediatric diseases.