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Current Pathways For Rare Disease Drugs Are Not Optimal, US FDA’s Califf Says

26 Mar 2024

Anticipating a ‘tsunami of therapies’ for rare diseases, commissioner says the agency will have to think of creative approaches and employ regulatory flexibility for them. FDA considers copying the oncology center’s Project Facilitate for expanded access to other diseases.

Creative pathway — Creative approach is needed for approval of rare disease treatments • Source: Shutterstock

The US Food and Drug Administration is aware of the difficulties of getting approval of rare disease treatments, Commissioner Robert Califf said, and the agency needs stakeholders help to develop creative approaches to overcome them.

Califf gave a closing keynote address at a recent conference sponsored by the New York University Grossman School of Medicine...