When ‘right to try’ isn’t enough: Congress wants a single ALS patient to get a therapy never tested in humans
WASHINGTON — A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding.
Just last week, the FDA gave Jaci Hermstad, a 25-year old Iowan who is dying from a rare form of ALS, an early sign that she will receive the first dose of an experimental drug never before tested in humans. The FDA’s move, which was confirmed to STAT by Jaci’s family and doctor, is a breakthrough for the Hermstads.
For months, the family has circulated petitions, tweeted at President Trump, and called on some of Washington’s most controversial and powerful politicians — including the speaker of the House and a Republican under fire for racist comments — all to convince the FDA to waive even the most basic of studies before the drug is injected into Jaci’s already fragile immune system.
The Hermstads’ progress reopens an ardent debate in Washington over the FDA’s role in determining when sick or dying patients can access experimental therapies — and what role career politicians should play in the nation’s decisions about how and when to let patients access untested treatments. It also underscores a fundamental question about the role of lawmakers: Is it fair for a member of Congress to intervene on behalf of a single constituent?
“Do you really want the FDA responding to one-by-one requests by families from congresspeople?” asked Arthur Caplan, the founding head of the New York University School of Medicine’s division
