Guest Author
Four words. That’s all it takes to unravel a parent’s world: “Your child is sick.”
The diagnosis is rare, the prognosis grim. He’s only six, but there’s hope: a new kind of treatment that can be customized to his DNA — a marvel of modern medicine promised for decades that is now a reality.
The potential cure has a gatekeeper, though. While the clock continues to tick, the government won’t allow your child to try this last chance at saving his life.
More than 2 million Floridians suffer from rare diseases, and for thousands of them, outdated federal regulations and the entanglement of bureaucracy continue to block cutting-edge treatments that could mean the difference between life and death. But now, lawmakers in Florida have an opportunity to place life-and-death decisions back in the hands of patients and their doctors, where they belong.
Florida leaders can enact a new law, the Right to Try for Individualized Treatments, that allows Floridians with rare diseases to access innovative, highly personalized treatments without first begging the federal government for permission.
The problem isn’t hypothetical. It’s tragically real.
Take Elijah Stacy, for example. At just 6 years old, doctors gave Elijah a crushing diagnosis. He had Duchenne Muscular Dystrophy, a rare, incurable, and fatal genetic disease that slowly erodes muscle strength and robs young people of their independence. As they reach adolescence, DMD patients start losing strength in their arms. Eventually, their heart and diaphragm weaken, too. Death typically comes by the mid-twenties.
Waiting on the government to go through reviews and testing, granting approvals, and making recommendations isn’t a luxury someone like Elijah can afford.
When Elijah was only 11, the disease had already taken away his ability to walk. Now, at 23, he’s struggling to use his arms. But that hasn’t stopped him and he’s not saying goodbye. Elijah has led the fight for his life — and the lives of thousands of others — by advocating for the Right to Try for Individualized Treatments.
The reform, which is now law in nine states and currently under consideration in the Florida Legislature with HB 1333/SB 680, defies a federal system that prizes red tape over relief, rules over recovery, and leaves dying patients trapped as they wait for the end to come. It recognizes that those facing debilitating, rare diseases need action now and allows patients to access cutting-edge therapies tailored to their genetic profiles, even if the Food and Drug Administration has yet to approve.
“Right now, I can be willing to try a new treatment, the manufacturer of the treatment can be willing to allow me to try it, and a doctor can be willing to administer it — yet Uncle Sam is not willing and stops me from receiving a lifesaving treatment,” Elijah said. “This is completely backwards. After all, doctors know their patients better than a far-off bureaucrat who doesn’t even know the patient exists. Why should I have to beg the federal government for permission to try to save my own life?”
Elijah’s story is far from unique. Tens of millions of Americans, including thousands in Florida, live with rare diseases and want to try anything they can. Although new technology has ushered in an age of personalized care customized to a patient’s unique DNA, regulations have not kept pace, leaving many stranded. Too often, those suffering from degenerative or rare diseases have to wait years for a therapy that could help them today.
By enacting the Right to Try for Individualized Treatments, Florida lawmakers can advocate for some of the state’s most vulnerable, empowering them to fight to save their own lives despite distant regulators standing between them, their doctors, and a treatment.
“The reality for me is that I’m going to die, likely before I reach middle-age,” Elijah explains. “The average lifespan for people with Duchenne is 25. But it doesn’t have to be that way.”
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Brian Norman is the director of State Affairs at the Goldwater Institute, where he assists in developing and implementing Goldwater’s national legislative affairs strategy.
