Family behind new Alabama “Right to Try” law looks beyond the bill
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. Gabe doesn’t know he’s dying, though. 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 Wednesday, seeks to allow access to drugs that have completed a first 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 parts 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. 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 about 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. During 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 prepared 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 attorney 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 Alabama U.S. Rep. Spencer Bachus, a Republican, 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 in 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.” Republished with permission of The Associated Press.
Lawmakers grant final approval to Right to Try bill
Alabama lawmakers granted final passage Thursday to a bill that would allow physicians to prescribe terminally ill patients promising, but unapproved medical treatments. House Bill 463, known as the Right to Try Act, passed the House of Representatives by a vote of 97-0. The bill says that doctors caring for people with terminal illnesses can prescribe medications that the Food and Drug Administration has deemed promising, but not yet ready for mass consumption. Thursday’s vote makes Alabama one of at least 12 states that have passed right to try legislation this year. House Bill 463 and Senate Bill 357 were inspired by 9-year old Gabe Griffin of Shelby County. At age 3, Gabe was diagnosed with Duchenne, one of nine types of Muscular Dystrophy. The disease causes generalized weakness and muscle wasting that increases over time and with muscle activity. Not only is the disease incurable, according to the Muscular Dystrophy Association, boys with Duchenne typically did not survive beyond their teens. Two Alabama lawmakers, Sen. Cam Ward and House health committee chair Rep. April Weaver, agreed to sponsor legislation that would help Gabe and other terminal patients work with their doctors to access medication in the earliest stages of FDA approval. Ward’s bill has already passed the Senate. When he introduced the Senate version of the bill, Ward said in a prepared statement: “Terminal patients should have a right to try, and as a father and an elected official I believe we should do everything we can to help remove unnecessary bureaucratic red tape from the process so that doctors and terminally ill patients can determine the best course of action in each individual situation.” The initial barriers, according to Gabe’s father, Scott Griffin, came from the Food and Drug Administration when the family tried to gain accelerated access to experimental treatments for Gabe. In a recent interview with Alabama Today, Griffin recalled one of his conversations with FDA administrators. “The first argument was that the patient population of one of the studies was too small: just 12 kids. Then they said the results in the study may have been just the natural difference in the disease … They said, ‘Well we have the best drug approval process in the world’ and ‘You have to understand that we have to worry about long-term side effects.’ And I said to them, ‘Explain to me what a long-term side effect is to a child who is going to die.’” The Food and Drug Administration has acknowledged that the accelerated approval process can be daunting for patients and medical providers. In a statement on the FDA website, assistant commissioner Peter Lurie M.D. said that although approvals can happen within days – or even hours – of a finished application, the process has given them cause for concern. “It called for 26 separate types of information and seven attachments,” Lurie wrote. “In fact, it was originally designed for manufacturers seeking to begin human testing, not for physicians seeking use by single patients.” According to Lurie, those concerns are what drove the FDA to announce a new streamlined process to allow patients access to experimental drugs. The FDA website says that the new forms “will be used for requesting the medications, and is designed to greatly simplify and accelerate the process by which a physician can request that FDA permit the use of an experimental — so-called ‘investigational’ — drug or biological product while it’s still being tested to establish its safety and effectiveness.” However, the forms are still going through the administrative rulemaking process and not yet available to the public. The official website gave no indication on when the final guidelines would be available for patients or providers. For the Griffins, the FDA roadblocks helped fuel their decision to lobby Alabama lawmakers. “We believe those drugs could save him, but we can’t get them because they’re not FDA approved,” Griffin said. “And after years of petitioning the FDA, we decided to take our fight to the state level.” The Griffins aren’t alone. According to the National Conference of State Legislatures, a growing number of states are considering legislation to work around those barriers and help patients gain access to experimental drugs. Thursday’s vote makes Alabama one of 12 states that have passed right to try bills so far this year. The NCSL reports that lawmakers in at least 36 states proposed “right to try” measures in 2015.
#Hope4Gabe family excited to see “Right to Try” Act move closer to finish line
One family’s winding journey to secure medicine they hope could save their son’s life is several steps closer to the finish line. A group of Alabama lawmakers voted Wednesday in favor of House Bill 463, also known as the Right to Try Act. The bill says that doctors caring for people with terminal illnesses can prescribe medications that the Food and Drug Administration has deemed promising, but not yet ready for mass consumption. The Griffin family and their son, Gabe, are at the center of the legislation. Scott Griffin and his wife Traci live in Birmingham with their four children: Turner, age 14; Cooper, age 11; and 9-year-old twins, Gabe and Addie. In an interview with Alabama Today, Griffin said he and his wife had suspected since the twins were babies that Gabe wasn’t developing as quickly as his sister. “When you have a twin right beside him and she’s hitting all her benchmarks, and he’s not, it becomes a little evident that something’s not right,” Griffin said. “Our doctors kept saying, ‘Nothing’s wrong. He’s just a boy twin and they’re slower to develop.’ So we just kind of dismissed it. But something didn’t seem right.” The Griffin’s neighbor was the first to suggest that Gabe might need to be tested for muscular dystrophy. An occupational therapist, she sometimes babysat the kids and noticed subtle differences in Gabe’s attempts to stand and walk. She told the Griffins about Duchenne Muscular Dystrophy and recommended that 3-year-old Gabe see a doctor. Duchenne is one of nine types of Muscular Dystrophy. It causes generalized weakness and muscle wasting that increases over time and with muscle activity. The disease primarily effects boys and shows up between ages 3 and 5. According to the Muscular Dystrophy Association, boys with Duchenne typically did not survive beyond their teens. But advances in medical treatments and care have increased their life expectancy: “Survival into the early 30s is becoming more common and there are cases of men living into their 40s and 50s.” Scott Griffin says those medical advances could save Gabe’s life. “He’s 9 now. The typical progression of the disease is that by 10 or 12, they’re wheelchair bound. And then, between 17-20, they usually die.” He says that’s why the family started looking into cutting-edge therapies to slow – or even reverse – the degeneration of Gabe’s muscles. However, the drugs that the Griffins and their doctors feel would help Gabe are still going through the FDA approval process. “We believe those drugs could save him, but we can’t get them because they’re not FDA approved,” said Griffin. “And after years of petitioning the FDA, we decided to take our fight to the state level.” The Griffins started a foundation called Hope for Gabe (H4G) to raise awareness about Duchenne. They also enlisted the help of Michael Staley, former chief of staff to U.S. Congressman Spencer Bachus and current lobbyist at Birmingham law firm Waller Landsen Dortch and Davis, to educate legislators about the proposed law. Two lawmakers, Sen. Cam Ward and health committee chair Rep. April Weaver, agreed to sponsor legislation that would allow doctors to prescribe medication in the earliest stages of FDA approval to terminally ill patients. Sen. Ward’s bill has already passed the Senate. Today, Rep. Weaver’s legislation received a favorable report from the health committee and can move to the House floor for open debate. “Right to Try is the right thing to do,” Weaver said just after the committee vote. “I’m a mother and a nurse with more than 20 years in health care. I believe that anything that we can do to help doctors and terminal patients work together on their plan of care and remove barriers to treatment that might be beneficial is just the right thing to do.” Griffin said that the timing of the legislation is critical to securing medical treatment for Gabe. “Getting him these drugs now means that we can stop the disease before he needs a wheelchair or breathing tubes. They could even turn his condition around.” For Gabe, passing the Right to Try Act means being able to “ride a bike, run, and climb stairs like everybody else.” He said that he’s also looking forward to excelling in his favorite subject in school: physical education. Weaver said she hopes to see the bill head to the House floor by late next week.
