Presentations: ADEPT 6, Pediatric Clinical Trial Endpoints for Rare Diseases with a Focus on Pediatric Patient Perspectives
November 12, 2019
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- Understanding the Patient Perspective on Age-Appropriate Measures of Growth in Rare Disease Drug Development
Ali Mohamadi, BioMarin Pharmaceutical, Inc. - Age-Appropriate Endpoints to Assess Neurodevelopmental Outcomes
Heather Adams, University of Rochester Medical Center - Measuring Pain and Symptoms in Children with Chronic Conditions
Tonya M. Palermo, Ph.D., Seattle Children's Research Institute - Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease and Pediatric Trials
Lili Garrard, FDA - Individualized Endpoints in Pediatric Rare Disease Trials: a clinical perspective
Patroula Smpokou, FDA - Using Pediatric PROMIS (Patient Reported Outcomes Measurement Information System) to Evaluate Symptom Burden Experienced by Children with Brain Tumors
Jin Shei Lai, Northwestern University - Pediatric Clinical Outcome Assessments: The PROMIS® Approach
Courtney K. Blackwell, Ph.D., Northwestern Feinberg School of Medicine - Quality of Life in Congenital Heart Disease Survivors: Creating New Models of Intervention to Promote Resilience and Minimize Disability
Bradley Marino, Ann & Robert H. Lurie Children’s Hospital of Chicago - Pediatric Migraine Registry
Christoph Hornik, Duke Clinical Research Institute - NESTcc & Real-World Evidence
Robbert Zusterzeel, M.D., Ph.D., MPH, Medical Device Innovation Consortium - Electronic Tools for Self-Management
Lori E. Crosby, PsyD, Cincinnati Children's