Rising to the rare disease challenge: Key considerations in large-molecule orphan drug development


Rare diseases present a unique set of challenges within drug development due to small-scale manufacturing, limited time/data, small sample sizes, and costs. Learn how to overcome these challenges with:

  • Process design decisions for clinical batches across the three stages of validation
  • Key formulation considerations for sterile dosage forms based on the physical, chemical, and biologic characteristics of the drug substances
  • Optimal API quantities for preclinical development and phase I studies