DM Voice of the Patient Report Submitted to the FDA

"We will not forget your faces or your stories. Please know that all of your efforts will make a contribution to making [the regulatory review] process go smoother when a new treatment comes to the FDA to be reviewed."  -Dr. Larry Bauer, regulatory scientist, U.S. Food and Drug Administration (FDA)

DM PFDD Meeting Report Submitted to FDA

As many of our community members know, MDF held the first myotonic dystrophy (DM) Patient-Focused Drug Development (PFDD) meeting with key senior leaders from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference in September 2016. The DM PFDD meeting, which was the first Externally-Led PFDD Meeting sanctioned by the FDA as well as the first PFDD meeting focused on DM, presented community member perspectives on the most burdensome and most prevalent DM symptoms and what affected individuals would consider meaningful benefit from future therapies.

Well over 200 community members, industry professionals, academic researchers and FDA representatives attended the DM PFDD meeting live and via the live stream MDF provided. FDA leadership including Dr. Janet Woodcock, who oversees all drug evaluation and research at FDA as the Director of CDER; Dr. William Dunn, who is the Director of the Office of Drug Evaluation 1 – Division of Neurology Products, the division that will review all DM therapies; Dr. Jonathan Goldsmith, Associate Director of the Rare Diseases Program, Office of New Drugs, CDER/FDA; Dr. Larry Bauer, a regulatory scientist in the Rare Diseases Program; and a number of additional representatives from the Office of Health and Constituent Affairs, the Office of Translational Sciences, the Study Endpoints team, and others also attended.

The final task in the DM Externally-Led PFDD initiative was the creation and submission of a Voice of the Patient Report (VOP) to the FDA with comprehensive results of the meeting, including a comprehensive summary of the meeting and discussions, detailed polling results, audience demographics, a listing of FDA participants and panelists, as well as a report on the Benefits/Risks Study MDF conducted with the Silicon Valley Research Group in 2015. MDF submitted the VOP to FDA in May 2017 for distribution to internal FDA leadership, dissemination to the Neurology Products Review Division for inclusion in the benefit/risk framework used to assess potential DM therapies, and to ensure that FDA decisionmakers understand DM disease impacts as described by those living with the disease, and what our community would find clinically meaningful from future therapies.

MDF thanks our PFDD panelists, FDA leadership who helped bring the meeting to life, and Ionis Pharmaceuticals for providing attorney and former FDA professional James Valentine as a consultant on the project.


Contact Molly White at MDF via email or phone  415-800-7777.

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