“Pediatric rare disease is complex. Phenotypic variability both within and across clinical conditions poses significant challenges for researchers and the broader community. Through this collaboration, DiMe has advanced identification and consensus of key outcome domains for pediatric rare disease natural history studies and future clinical trials. I am particularly excited about the tool to customize core outcome measure sets that are aligned with patient-, caregiver-, and scientific priorities and tailored to the developmental characteristics most relevant to an individual study.”

– Aaron J. Kaat, PhD | Associate Professor and Director of Educational, Department of Medical Social Science, Northwestern University Initiatives Care