Federal Agencies Progress
From securing the first Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting for Angelman syndrome to representing our community at FDA roundtables — see how advocacy is opening doors with regulators.
EL-PFDD Meeting
In April 2025, ASF and FAST hosted the first-ever Externally-Led Patient-Focused Drug Development meeting for Angelman syndrome — giving families a direct line to FDA regulators to share their lived experience and treatment priorities.
In 2012, following intensive advocacy by patient advocates with their members of Congress, President Obama signed into Law The Food and Drug Administration Safety and Innovation Act (FDASIA). The law mandated the FDA take a structured and transparent approach to incorporating the views of patients and families into the drug development process and review. In response, FDA launched a new initiative called Patient Focused Drug Development (PFDD) and created mechanisms for regulators to hear directly from families about the conditions they and their loved ones live with every day. Since then, a series of Patient Focused Drug Development meetings have taken place, the end result of a report that lives on FDA’s website providing regulators with critical information to make informed decisions about pending new drugs.
Each year, FDA agrees to attend a small number of these meetings. In February 2023, ASF and FAST submitted a letter of intent to FDA on holding an Externally Led PFDD meeting for our community. We were thrilled that the FDA agreed to the need for the meeting, which occurred on April 7, 2025.
FDA Cell and Gene Therapy Roundtable
See FAST’s Chief Science Officer, Dr. Allyson Berent, speak at the U.S. Food and Drug Administration Cell and Gene Therapy Roundtable on FDA’s You Tube Channel! This meeting was an invited panel of cell and gene therapy experts who are providing important insights to new FDA Commissioner Marty Makary as FDA listens to these key stakeholders on the challenges that communities face in developing novel therapies.