Family behind new Ala. ‘Right to Try’ looks beyond the bill

Orignally posted in Washington Times by AP

MONTGOMERY, Ala. (AP) – Gabe Griffin lobbied his first bill at the age of 9. It became law when he turned 10. He’ll be in a wheelchair when he turns 12. Without the right drugs, his parents say he’ll be dead in a decade.

But Gabe doesn’t know he’s dying. In fact, he doesn’t even know what a law does. He knows it could help him, but when you ask him how, he pulls his black baseball cap all the way over his red hair, over his freckled face and asks his mom and dad when they’ll stop peppering him with questions so he can go eat cheese dip.

Gabe has Duchenne Muscular Dystrophy, a rare muscular degenerative disease that gradually and then rapidly leads to incapacitation before ultimately to an early death. Right now, he can whack a baseball with a bat like any boy, but tires before first base. He dreams of being an “armian”__his way of pronouncing Army man__but has to be carried upstairs.

The 10-year-old became a familiar face in the Alabama Legislature during the 2015 legislative session, as his family visited time after time from their Birmingham-area home to ask lawmakers to pass a bill that might someday get him drugs that don’t yet exist.

The Gabe Griffin Right to Try Act, which Gov. Robert Bentley signed into law on Wednesday, seeks to allow access to drugs that have completed a Phase 1 clinical trial but that haven’t been approved for general use by the U.S. Food and Drug Administration.

Gabe’s mother and father, Traci and Scott Griffin, have been searching for treatment since he was diagnosed seven years ago. The Griffins, and others facing a number of terminal diseases, think the FDA approval process is too tedious, too selective and too time-consuming to provide any hope.

“It’s one of those things that you can see and you can smell but you can’t touch,” Scott Griffin said.

Two drugs in clinical trial for Duchenne patients__one by Sarepta Therapeutics Inc. and the other by BioMarin Pharmaceutical Inc.__have promise. Neither is yet suitable for Gabe’s case, but when they are, the Griffins want to take the risk.

Alabama is one of several states that passed a “Right to Try” law during their 2015 session, a legislative trend that advocates say provides patients with new options and that others say creates new risks.

“We talk about side effects often in safety, and I get it, I get it,” Scott Griffin said. “But my son is going to have tubes coming in and out all part of his body, with a steel rod shoved in his back, his Achilles heel chords cut, not being able to roll over, not being able to lift his head up. I don’t think it gets much worse than that.”

According to the Goldwater Institute, one of the groups behind other states’ Right to Try laws, 19 states have such laws; in another three states, legislation is awaiting the governor’s signature.

The FDA, tasked with the duty of ensuring drugs are safe and effective, has not taken a position on any state’s “Right to Try” legislation.

“It is critical for the public to understand that FDA is not a barrier to accessing investigational drugs or medical devices,” FDA spokesman Jeff Ventura said. “The FDA is an important part of the process and helps to ensure patients are adequately protected from receiving a potentially harmful or ineffective treatment.”

The agency is trying to work with terminally ill patients who fear they’re out of options. Through its Expanded Access__or “compassionate use”__program, the FDA from 2010 to 2014 allowed 99 percent of the applications it received to proceed. Earlier this year, the agency announced plans to simplify and accelerate the application process for patients who want to try unapproved drugs.

The pace of drug discovery for orphan diseases__those affecting a relatively small population__has accelerated. According to Pharmaceuticals and Research Manufacturers of America, the FDA has approved around 500 drugs for orphan diseases since the passage of the Orphan Drug Act of 1983, which aimed to hasten the approval of drugs for orphan diseases. Over the past decade it approved 200. Last year, 17 of the 41 drugs approved by the FDA were for orphan diseases.

PhRMA has not taken an official stance on Right to Try legislation or on related bills at the federal level, but Sascha Haverfield, PhRMA’s vice president of scientific and regulatory affairs, said in a statement that the organization has “serious concerns” with alternative approaches to making investigational medicines available.

But the Griffins don’t know how long they’ll have Gabe. Some Duchenne patients live into their twenties. Others die of heart failure as teenagers.

Meanwhile, the Griffins have started a nonprofit, Hope For Gabe, to raise awareness in the state and across the country. Last summer, Birmingham lobbyist Michael Staley and Indiana University student Wes Bates rode their bicycles across the U.S. to advocate for Duchenne. Staley, the former chief of staff of former U.S. Rep. Spencer Bachus, R-Ala., has been helping the Griffins push for changes at the state and federal level since he first met Gabe during the Griffins’ visit to Washington, D.C.

“As a dad, and as mom, you’re ready to jump front of a train, a moving train, to save your child,” Scott Griffin said. “Duchenne is that moving train, and we are going to do everything humanly possible to try to make Gabe, and this generation of Duchenne boys, the first generation to survive this disease. I don’t know how it’s going to turn out, but man, we’re close. We’re close.”