There is a heightened sense of urgency by all stakeholders to accelerate development of therapies for rare disease. The FDA shares this urgency, but it also must protect the public safety and follow the well-defined laws and regulations governing the process.
The challenge (or opportunity) is to develop creative approaches, which accelerate access and stay within the regulatory boundaries – or find ways to shape the boundaries. Intermediate Clinical Endpoints, or ICE, could represent one such approach.
You can download the slides for your referience, and leave a comment below if you have questions. I hope you enjoy.