"Hopes of family, firm collide on unproven drug" by Tracy Jan | Globe Staff, January 31, 2014
WASHINGTON — Jamie and Jason Fowler arrived early at a Chicago airport hotel on a recent Saturday morning, eager to meet the pharmaceutical executive from Massachusetts they hoped would help save the life of their 6-year-old son, Jack.
So began an encounter that provides a window into one of the most heart-wrenching dilemmas in modern medicine: should an experimental drug be given to a dying patient if it is unproven and might unravel a carefully designed clinical trial?
It is a dilemma that the federal government has given its own special name, “compassionate use.” From the point of view of the Fowlers, it would be compassionate indeed if Shire, an Irish-based drug company that has a rare diseases division in Lexington, Mass., agreed to provide the drug that potentially could save their child.
“Jason and I made a promise the day Jack was diagnosed that we would do everything and anything possible to ensure he is happy, healthy, and living,” Jamie Fowler said. “It’s a living nightmare to ride this roller coaster of hope and fear, hope and fear, and then trying to find the strength within all that.”
But from the point of view of the company....
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