2011 Annual Meeting

ASENT's 13th Annual Meeting

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

Platform Technologies

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

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

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

Friday, February 25, 2011

Neuroinflammation

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

Title Speaker(s) Duration Presentation Audio Video
Welcome and Introduction - NeuroInflammation Jacqueline French, MD 6 min. 05 sec. Presentation Audio  
The Promise of Anti-Inflammatory Therapy for CNS Disease Serge Rivest, PhD, Universite Laval 26 min. 07 sec. Presentation Audio  
Imaging Neuroinflammation William Theodore, MD, NIH 16 min. 27 sec.   Audio  
Inflammatory Mechanisms in Epilepsy Annamaria Vezzani, PhD, Mario Negri Institute 25 min. 11 sec. Presentation Audio  
Multiple Sclerosis: A Paradigmatic Neuroinflammatory Disease? Bibiana Bielekova, MD, NIH 32 min. 12 sec. Presentation Audio  
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. Presentation Audio  

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