FDA Chief Wants to Break Down Rare Disease Drug Monopolies

Patients battling rare diseases could have more treatment options under an FDA plan to make clinical trials more efficient, which the agency head believes ultimately could lower drug prices.

Testing an experimental therapy for a rare disorder takes more time compared to medicines for more common diseases because it’s harder to recruit enough volunteers to demonstrate the drug meets the Food and Drug Administration’s approval standards. This means companies that are first to market a drug to target unmet medical needs “are enjoying monopolies for longer periods of time,” FDA Commissioner Scott Gottlieb said.

Rare or “orphan” drugs—therapies for ...