Description

Informed by conversations in 2023 around challenges and opportunities in research and therapeutic development for rare neurological diseases, the Rare Neurological Diseases Action Collaborative was formed as an ad hoc activity under the auspices of the Forum on Neuroscience and Nervous System Disorders. Representatives from federal, clinical, academic, and pharmaceutical organizations will contribute to the action collaborative, which aims to foster collaboration and information-sharing among various partners and highlight paths forward.

The action collaborative may plan activities to amplify patient and families’ voices, coordinate global and federally based efforts, enhance clinical trial design and readiness, improve usage of patient-centered outcome measures and enhance data harmonization, among other priorities.

Collaborative participants can pursue a range of outputs, which may include:

• Laying the groundwork for action on the part of other stakeholders
• Publishing perspectives in peer-reviewed journal
• Spurring formation of public-private partnership
• Envisioning a charge for a workshop or consensus study
• Developing resources

The collaborative is an ad hoc activity convened under the auspices of the Forum on Neuroscience and Nervous System Disorders at the National Academies of Sciences, Engineering, and Medicine (the National Academies). The work it produces does not necessarily represent the views of any one organization, the Forum, or the National Academies, and is not subjected to the review procedures of, nor is it a report or product of, the National Academies.

Participants:

Scott Baraban, University of California, San Francisco

Terry Jo Bichell, COMBINEDBrain

Michelle Campbell, Food and Drug Administration

Anne Claiborne, Chan Zuckerberg Initiative

Eva Feldman, University of Michigan

Veronica Moore, Amgen

Jill Morris, National Institute of Neurological Disorders and Stroke

Terry Periovalakis, CureSPG50

Maria Picone, TREND Community

Scott Pomeroy, Boston Children’s Hospital

Marsie Ross, Harmony Biosciences

Tiina Urv, National Center for Advancing Translational Sciences

Vanessa Vogel-Farley, Independent consultant