Letter to the Editor: “FDA’s Compassionate-Use Program Offers Early Access Without Compromising Drug Trials”

The April 22 front-page article “FDA relents in battle against a cruel disease,” about parents urging the Food and Drug Administration to approve a promising drug for Duchenne muscular dystrophy, pulled on the heartstrings. However, there is a better way to get an experimental drug to patients than lowering approval standards.

If the pharmaceutical company is willing, parents who are understandably desperate to help their children can utilize the FDA’s compassionate-use program, which allows a patient with a life-threatening disease to receive an unapproved drug from the company. This provides patients with early access to drugs. If the patients do well, that early access can help products obtain FDA approval.

Paul Brown, Washington

The writer is government relations manager at the National Center for Health Research.