The Modernization of Orphan Drug Development

Orphan drugs affect 350,000 people worldwide. Model-informed drug development (MIDD) approaches, such as PBPK and PopPK, embraced by sponsors and regulators, play a key role in modernizing and accelerating orphan drug development. Future advances in technology and science will continue to accelerate orphan and rare disease development and help to expedite the delivery of safe and effective medications to treat patients with unmet medical needs.