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Concept to Dosing: Challenges in New Orphan Drugs

Summary: As drug developers look to address unmet medical needs for orphan diseases, working with experienced partners can help in navigating key challenges such as a limited active pharmaceutical ingredient supply, efficient small-scale manufacturing for small patient populations and distribution logistics

Developing Optimised Formulations with Minimal Drug Substance

Advances in in silico and experimental techniques mean that APIs and prototype formulations can be thoroughly characterised using multiple material-sparing assays, allowing the most promising formulation candidates to move on to in vivo studies.