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Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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Page 101
Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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Page 102
Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×
Page 103
Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×
Page 104
Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×
Page 105
Suggested Citation:"Appendix C: Registered Attendees." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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Page 106

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C Registered Attendees Susan Amara Nathalie Breysse National Institute of Mental Lundbeck Research USA Health Katja Brose Aisar Atrakchi Neuron U.S. Food and Drug Administration Wilson Bryan U.S. Food and Drug Alex Bailey Administration U.S. Food and Drug Administration Neil Buckholtz National Institute on Aging Bruce Baron Sanofi Elzbieta Chalecka-Franaszek U.S. Food and Drug Bruce Bebo Administration National Multiple Sclerosis Society Jiu-Chiuan (J. C.) Chen University of Southern Francesca Bosetti California Keck School of National Institute of Medicine Neurological Disorders and Stroke Charles Cywin National Institutes of Health Linda Brady National Institute of Mental Daryn David Health National Institutes of Health 101

102 APPENDIX C Hirsch Davis David Hawver National Institute on Drug U.S. Food and Drug Abuse Administration Jamie Driscoll Thomas Heinbockel National Institute of Mental Howard University College of Health Medicine Emmeline Edwards John Hsiao National Center for National Institute on Aging Complementary and Alternative Medicine Carol Hubner National Institute on Drug Stephanie Fertig Abuse National Institute of Neurological Disorders and Alisa M. Hughley Stroke enBloom Linda Fossom Thomas Insel U.S. Food and Drug National Institute of Mental Administration Health Lois Freed Bram Jacobs U.S. Food and Drug University of Groningen Administration David Jett Brandy Fureman National Institute of National Institute of Neurological Disorders and Neurological Disorders and Stroke Stroke Petra Kaufmann John Gardenier National Institute of Independent Neurological Disorders and Stroke Turkan Gardenier Independent John Kehne National Institute of Jessica Goodman Neurological Disorders and Prize4Life Stroke

APPENDIX C 103 Roxanne Khamsi Elizabeth McNeil Nature Medicine National Institute of Neurological Disorders and Violetta Klimek Stroke U.S. Food and Drug Administration David Michelson Merck Research Laboratories Elizabeth Kwasnik Parkinson’s Action Network Stephen Mikita SMA Foundation Woody Lin National Institutes of Health Marilyn Miller National Institutes of Health Yuan Luo Center for Scientific Review, Eric Nelson National Institutes of Health National Institute of Neurological Disorders and Ernie Lyons Stroke National Institute of Neurological Disorders and Birgit Neuhuber Stroke National Institutes of Health Vince Macri Margaret Ochocinska PreMotor Exercise Games National Institute of Neurological Disorders and Laura Mamounas Stroke National Institute of Neurological Disorders and Suzana Petanceska Stroke National Institute on Aging Ron McKay Creighton Phelps Lieber Institute for Brain National Institute on Aging Development John Porter Matthew McMahon National Institute of National Eye Institute Neurological Disorders and Stroke

104 APPENDIX C Erica Rosemond Emily Underwood National Institute of Mental Science Health Joukja van der Naalt Kate Saylor University of Groningen National Institute of Neurological Disorders and Molly Wagster Stroke National Institute on Aging Mercedes Serabian Kevin Walton U.S. Food and Drug National Institute of Administration Neurological Disorders and Stroke Ming Shih National Institute of Philip Wang Neurological Disorders and National Institute of Mental Stroke Health Paul Sieving Susan Weiss National Eye Institute National Institute of Neurological Disorders and Judy Siuciak Stroke Foundation for the National Institutes of Health David White Biomarkers Consortium National Institute on Drug Abuse Phil Skolnick National Institutes of Health Vicky Whittemore National Institute of Roland Staal Neurological Disorders and Lundbeck Research USA Stroke Amir Tamiz Alan Willard National Institutes of Health National Institute of Neurological Disorders and Craig Thomas Stroke Eli Lilly and Company Dwain Tolbert Lundbeck Research USA

APPENDIX C 105 Lois Winsky Robert Zalutsky National Institute of Mental National Institute of Health Neurological Disorders and Stroke Brad Wise National Institute on Aging Jie Zhang Lieber Institute for Brain Celia Witten Development U.S. Food and Drug Administration Danna Zimmer University of Maryland School Lei Xu of Medicine U.S. Food and Drug Administration Haichen Yang Eisai Inc.

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Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline.

There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

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