Patient Input Said Key for Rare Disease Drug Development

Rare disease drug development would benefit from increased patient engagement and input, speakers said Sept. 15 during a workshop sponsored by the EveryLife Foundation for Rare Diseases.

Emil Kakkis, president of the foundation and the biopharmaceutical company Ultragenyx, said that during drug development, it is important to understand what benefit would be useful to patients and what risks they are willing to take. The EveryLife Foundation for Rare Diseases is a nonprofit organization dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.

“The perception of risk and risk tolerance will vary among patients, clinicians, regulators and ...