Bringing the Patient Voice to CNS-Targeting Drug Development in Myotonic Dystrophy
Hyatt Regency San Francisco
5 Embarcadero Center, San Francisco, CA 94111
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In response to requests from drug development professionals to help identify CNS endpoints appropriate for the regulatory review process, and to accelerate the development of CNS-targeting DM therapies, IDMC-11 and the Myotonic Dystrophy Foundation will jointly host a multi-panel discussion to bring patient perspectives on disease burden and clinically meaningful endpoints to efforts to develop endpoints for CNS-targeting DM therapies. The meeting format will mirror the very successful Patient-Focused Drug Development (PFDD) meeting hosted by MDF with the FDA in September 2016. Panels will be seated with people affected by DM, and DM caregivers. The session will be moderated by James Valentine, J.D., who also moderated the September 2016 PFDD meeting.
The session will begin with a presentation on outcome measure development to help the lay audience understand the importance and role of endpoints in drug development and approval, and to frame the goals for the session. Panel presentations will be followed by moderated audience discussion and a presentation from an industry representative regarding possible future DM CNS endpoints, and CNS endpoints used in other neurological diseases that might inform upcoming drug development efforts in DM.
A publication summarizing the workshop is planned.
The annual Drug Development Roundtable brings together leaders from academia, biotechnology and pharmaceutical industries to discuss opportunities, barriers and pre-competitive space collaborations to facilitate the pathway to drugs and biologics for DM.