Systematic Review of Patient Focused Drug Development Meeting Reports for Conditions Affecting Neurodevelopment

Area of Science:

• Neuroscience
• Rare Diseases
• Clinical Outcome Assessment Development

Background:

• Researchers face challenges in identifying concepts and outcomes for rare neurodevelopmental diseases.
• The collective commonness of rare diseases is underutilized for creating large-scale clinical outcome assessments.
• Identifying shared concepts across conditions can accelerate outcome assessment development.

Purpose of the Study:

• To systematically review patient-focused drug development (PFDD) meeting reports.
• To identify concepts prioritized by patients and caregivers across rare neurodevelopmental conditions.

Main Methods:

• Systematic review of 16 PFDD meeting reports for rare neurodevelopmental conditions.
• Coded responses on troublesome symptoms and ideal treatment targets into general concepts.
• Aggregated endorsement rates of concepts across conditions.

Main Results:

• No single symptom or treatment target achieved full consensus across all conditions.
• For 11 of 16 conditions, over 30% of respondents prioritized Communication or Cognitive/Developmental concepts.
• These prioritized concepts were relevant as both troublesome symptoms and ideal treatment targets.

Conclusions:

• Empirical evidence supports shared prioritization of developmental concepts across diverse rare diseases.
• This shared prioritization is crucial for unifying clinical outcome assessment development.
• Unification efforts can enhance clinical trial readiness for rare disease populations.