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
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–
–
cognitive
emotional
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to
motor
sensory
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• 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
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• 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
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to edit Master title
style
Emotion
Motor
Sensation
Cognition
Working Memory
Executive Function
Inhibitory Control
Working Memory
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Cognitive Flexibility
Processing Speed
Episodic Memory
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Visual
Auditory
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title
Language
Vocabulary
style
Comprehension
Reading Decoding
Attention
9
Emotion
Emotion
Positive
Affect
Negative
Affect
Stress and
Coping
Social
Relationships
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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
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Master
title style
Balance
(Non-vestibular)
Lower Extremity
11
Sensation
Sensation
Taste
Olfaction
Odor Identification
Olfactory Sensitivity
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Audition
Auditory
Processing
Hearing Loss
Hearing Threshold
Middle Ear
Function
Vestibular Balance
Preference Intensity
Vestibular-ocular Reflex
Bitter Perception
Vestibulospinal Function
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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
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edit
Masterversions
title style
• English
and
Spanish
• Supplemental set of additional
instruments– Tool Shed
Online Resources
www.nihtoolbox.org
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•
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NIH Toolbox Training Manual
NIH Toolbox Administration Manual
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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
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Program Director: Claudia S. Moy, PhD
Office of Clinical Research
National Institute of Neurological Disorders and
Stroke