Transcript 1 - ASENT
February 20, 2014 ASENT Annual Meeting, Bethesda, MD Petra Kaufmann MD MSc Director, Office of Clinical Research National Institute of Neurological Disorders and Stroke Background • For many neurological diseases there is no suitable outcome measure for therapeutics development – Properties of measures inadequate for purpose – Methodological robustness varies – Developed for academic research rather than regulatory pathway – Insufficient data available using the measure Challenges • Multiple measures for each domain • De novo outcomes development – Redundancy – Lack of comparability Opportunity • Build on existing components – Robust methodology – Increased comparability – Less development time and cost NIH Assessment Tools • NIH Toolbox www.nihtoolbox.org/default.aspx – Assessment of Neurological and Behavioral Function • Neuro-QOL www.neuroqol.org/default.aspx – Quality of Life in Neurological Disorders • The EXAMINER http://examiner.ucsf.edu – Neurobehavioral Evaluation and Research • Phen-X www.phenx.org – Consensus Measures for Phenotypes and Exposures • PROMIS® www.nihpromis.org – Patient-Reported Outcomes Measurement Information System Joint Adult & Pediatric Measures (self/proctor) Cognition 8 Motor 6 (peg board) Audition 2 Vision 1 Olfaction 1 Balance 1 Somatosensation 2 Taste 1 Subjective objective Patient Observer Clinician Naturalistic Artificial X PROMIS, NeuroQoL NIH Toolbox for Assessment of Neurological and Behavioral Function • Brief, unified set of measures (assessment tool) • Multiple indicators of neural and behavioral health functioning – – – – cognitive emotional Click to motor sensory edit Master title style • For use in large epidemiological studies and clinical trials NIH Toolbox for Assessment of Neurological and Behavioral Function • Measures the same constructs over lifespan • Where possible, objective measures over selfreport DOWNSTREAM Click to edit Master title style • Could be used as a form of “common currency” across diverse study designs and populations • Would maximize yield from large, expensive studies with minimal increment in subject burden and cost Toolbox Domains Cognition Click to edit Master title style Emotion Motor Sensation Cognition Working Memory Executive Function Inhibitory Control Working Memory Click to Cognitive Flexibility Processing Speed Episodic Memory edit Visual Auditory Master title Language Vocabulary style Comprehension Reading Decoding Attention 9 Emotion Emotion Positive Affect Negative Affect Stress and Coping Social Relationships Click to edit Master title style Sadness Happiness Fear Life Satisfaction Well-Being Anger General Distress Apathy Perceived Stress Coping Strategy Coping Self-efficacy Social Support Social Network Integration Loneliness 10 Motor Endurance Locomotion Strength Dexterity Extremity Click to editUpper Master title style Balance (Non-vestibular) Lower Extremity 11 Sensation Sensation Taste Olfaction Odor Identification Olfactory Sensitivity Click to Audition Auditory Processing Hearing Loss Hearing Threshold Middle Ear Function Vestibular Balance Preference Intensity Vestibular-ocular Reflex Bitter Perception Vestibulospinal Function edit Master title style PROP Sensitivity Central Involvement Somatosensation Pain Proprioception Temperature Texture Sensory Feedback Vision Visual Acuity Function Visual Function HRQL Peripheral Vision 12 Toolbox • Four domain-level batteries • Domain batteries to take no more than 30 min to administer; entire Toolbox administration max of 2 hours Click to edit Masterversions title style • English and Spanish • Supplemental set of additional instruments– Tool Shed Online Resources www.nihtoolbox.org • • • • NIH Toolbox Training Manual NIH Toolbox Administration Manual Click to editTechnical Master title style NIH Toolbox Manual NIH Toolbox Scoring and Interpretation Guide • NIH Toolbox Accessibility and Accommodations - Quick Reference Principal Investigator: David Cella, PhD Chair, Department of Medical Social Sciences Director, Center for Patient-Centered Outcomes - Institute for Public Health and Medicine Northwestern University Click to edit Master title style Program Director: Claudia S. Moy, PhD Office of Clinical Research National Institute of Neurological Disorders and Stroke