FDA’s N-of-One call to action for academics

CDER Director Janet Woodcock invites academic researchers to create therapies for individual patients using FDA’s new N-of-One pathway, but she emphasizes the program will not allow companies to skirt traditional drug development.

The advent of gene therapy and antisense oligonucleotides has opened the door to tailoring therapies to specific mutations found in single patients. The lack of commercial incentive, however, means these are almost never pursued...