Don't Do Different Things - Do Things Differently! Drug Development in Rare Diseases: The Patient's Perspective.

The pharmaceutical industry and regulatory authorities have traditionally made risk-benefit decisions without consulting patients. Today there is a movement to engage the patient in these decisions. This is particularly pertinent for the rare-disease patient population, who have limited access to effective diagnostics and treatments. We argue that their involvement at the earliest point of drug development will lead to more appropriate decision making, and potentially reduce the attrition rate. [ABSTRACT FROM AUTHOR]