ASENT: 2011 Annual Meeting

2011 Annual Meeting

This activity has been planned and implemented in accordance with the essential areas and policies of the Accreditation Council for Continuing Medical Edication through the joint sponsorship of the University of Vermont and the American Society for Experimental NeuroTherapeutics. The University of Vermont is accredited by the ACCME to provide continuing medical education for physicians

At the completion of the meeting participants will be able to

Describe new FDA guidance on clinical trial outcome measures
Outline the role of neuroinflammation in brain disease
Discuss the issues relating to the proper conduct of clinical trials with internation aspects, including pharmacoepidemiology and regulatory consideration

To view a session, click PPT Icon to view a PowerPoint slide presentation, Audio Icon to hear the audio, and Video Icon to view a video.

Thursday, February 24, 2011

Whose Outcome Matters Most?: Update on Clinician and Patient Reported Outcome Measures

Whose Outcome Matters Most?: Update on Clinician and Patient Reported Outcome Measures Co-Chairs: Karen Johnston, MD, MSc, University of Virginia Cynthia Joyce, Spinal Muscular Atrophy Foundation Nancy Santilli, BSN, MSN, Endo Pharmaceuticals, Inc PROMIS, Neuro-QOL and The NIH Toolbox: Three new tools to advance and standardize patient-centered measures of health Over the past five years, several new tools have been developed to measure outcomes that matter in patents’ lives. These tools have all been sponsored by The National Institutes of Health, and should be useful in clinical research and quality improvement in clinical practice. They also have the potential to streamline discussions with regulatory authorities regarding patient-reported outcomes that are fit for purpose. They are available in English and Spanish, and can be used in children as well as adults. The first of these tools is the Patient Reported Outcomes Measurement Information System (PROMIS), which provides efficient (i.e., reliable-yet-brief) measures of generic health concepts (common symptoms and physical, mental and social health status). It allows one to choose from a large item bank of validated questions to construct an internet-based website that scales a patient's sense of fatigue, physical function, pain, emotional distress, and social participation. PROMIS is already in use today across a wide range of research and clinical settings (www.nihpromis.org). The second, Neuro-QOL (www.neuroqol.org) is also available for use now and can best be considered a neurology-specific version of the more general PROMIS. Neuro-QOL has the advantage of being directly linked to PROMIS across most of its item banks, yet it provides more specificity with regard to common neurologic conditions. PROMIS and Neuro-QOL may be particularly useful in multicenter neurological treatment studies because they are customizable, quick and easy to use. They can be utilized for remote patient assessment, and have potential for consideration in discussion with regulatory authorities as a standardized basis for approving experimental therapeutics. The third new tool is the NIH Toolbox (www.nihtoolbox.org). Scheduled to be available for public use in 2012, it extends standardized patient-centered assessment beyond self-report (PROMIS and Neuro-QOL are exclusively self-report systems). Toolbox measures of health range from motor function, to sensory function (vision, audition, taste, smell, somatosensation, vestibular balance), to cognitive function and emotional health. The NIH Toolbox provides this wide array of brief, standardized tools for measuring neurologic function and will include normative values for each assessment. Neurologists and neurological investigators should consider how these tools may improve assessment of clinically important patient-reported and patient-centered outcomes. Investigators should consider how to use these standardized measures to enhance the quality and scope of data collected in their studies.

Title	Speaker(s)	Duration
Got Outcome Measures?: What Clinicians and Investigators Want	Lisa Shulman, MD, University of Maryland	22 min. 58 sec.
What is the New FDA Guidance? FDA Study Endpoint and Label Development Division	Elektra Papadopoulos, MD, FDA	22 min. 21 sec.
Is There Promise in the NIH PROMIS Initiative?	David Cella, PhD, North Western University	25 min. 59 sec.
NeuroQoL	Claudia Moy, PhD, NIH/NINDS	15 min. 11 sec.
The Practical Art of Endpoint Selection: Industry Perspectives	Glen Phillips, PhD, Sunovion Pharmaceuticals, Inc	21 min. 07 sec.
From the Reviewing Division	Russell Katz, MD, FDA	18 min. 05 sec.
What is Clinically Meaningful?	Anne Rutowski, MD, Cure CMD	22 min. 15 sec.

Platform Technologies

Mitchell Brin, MD, Allergan, Inc. Martha Morrell, MD, NeuroPace, Inc.

Title	Speaker(s)	Duration
What is a Platform Technology?	Mitchell Brin, MD, Allergan, Inc
Platform Technologies for Drug Discovery	Daniel Gil, PhD, Allergan, Inc	18 min 58 sec.
Platform Technologies for Medical Devices	Peter Konrad, MD, PhD, Vanderbilt Univeristy Medical Center	24 min. 32 sec.
Platform Technologies for Gene Therapy	Matthew During, MD, PhD, Ohio State University College of Medicine	28 min. 02 sec.
Regulatory Pathway for Platform Technologies	Quynh Hoang, MS, FDA	24 min. 13 sec.

Dinner Session - How to Engage the Public in Neuroscience

CO: Mitchel Brin, MD, Allergan,; Kathleen Clarence-Smith, MD, PhD, KM Pharmaceuticals Consulting, LLC

Title	Speaker(s)	Duration
How to Engage the Public in Neuroscience Introduction	Mirchell Brin, MD and Kathleen Clarence-Smith, MD, PhD	2 min 35 sec
How to Engage the Public in Neuroscience - Ostrow	Peter Ostrow, MD, PhD, Suny Buffalo School of Medicine and Biomedical Sciences	33 min 57 sec
How to Engage the Public in Neuroscience - Fleysher	Ellen Fleysher, University of Miami School of Communication	26 min. 33sec.
How to Engage the Public in Neuroscience - Rutz	Dan Rutz, MPH, Center for Disease Control and Prevention	51 min 44 sec

Friday, February 25, 2011

Neuroinflammation

Co-chairs: Jacqueline French, MD, NYU Comprehensive Epilepsy Center; William Theodore MD, NIH

Title	Speaker(s)	Duration
Welcome and Introduction - NeuroInflammation	Jacqueline French, MD	6 min. 05 sec.
The Promise of Anti-Inflammatory Therapy for CNS Disease	Serge Rivest, PhD, Universite Laval	26 min. 07 sec.
Imaging Neuroinflammation	William Theodore, MD, NIH	16 min. 27 sec.
Inflammatory Mechanisms in Epilepsy	Annamaria Vezzani, PhD, Mario Negri Institute	25 min. 11 sec.
Multiple Sclerosis: A Paradigmatic Neuroinflammatory Disease?	Bibiana Bielekova, MD, NIH	32 min. 12 sec.
Harnessing the Body's Immune Defenses as a Neuroprotective Strategy for Parkinson's Disease	Howard Gendelman, MD, University of Nebraska Medical Center	29 min. 36 sec.

Saturday, February 26, 2011

International Symposium

Co-Chair: Thomas Chase, MD, Chase Parmaceuticals Jill Rasmussen, MBCHB, Psi-Napse Ira Shoulson, MD, University of Rochester

Title	Speaker(s)	Duration
Pharmacogenetics: Inter-Ethnic Pharmacokinetic/Pharmacodynamic Differences In Relation to Clinical Trials and Acceptability of Foreign Data	Issam Zineh, PharmD, FDA	30 min. 50 sec.
Placebo and IRB Issues in EU Clinical Trials	Karl Broich, MD	24 min. 16 sec.
Mega Clinical Studies in Japan	Norio Tanahashi, Saitama Medical Univeristy International Medical Center	21 min. 12 sec.
Acceptability of Foreign Data from the EMA Perspective	Sir Michael Rawlins, NICE	23 min. 08 sec.
Global Clinical Development and Acceptance of Foreign Data in Japan	Yoko Fujimoto, MD, PhD, Pfizer Japan, Inc	16 min
Acceptability of Foreign Data by the US	Russell Katz, MD, FDA