Emerging models point to a new operating system for rare disease innovation

Around the world, regulators, clinicians, patient groups and biotechs are stumbling into new models for creating and delivering treatments for rare diseases. There’s no overarching plan and little coordination. 

What is emerging is not just a set of experiments, but the outline of a new operating system for rare disease innovation, one that combines new rules and workflows for evidence generation, approval, payment and delivery...