By Brian Norman
Imagine hearing that your 6-year-old child has a rare, debilitating disease that will likely take her life. You learn about an innovative new treatment that could help your child by tailoring a gene therapy to her DNA, but there’s a problem: the U.S. government won’t allow it.
For thousands of Iowans suffering from rare diseases, outdated federal regulations are standing in the way of cutting-edge treatments that could mean the difference between life and death. But now, Iowa lawmakers have an opportunity to place life-and-death decisions back where they belong—in the hands of patients and their doctors—by enacting a new law that protects a patient’s right to try to save their own life.
This problem is tragically real. Take the case of Elijah Stacy. At just 6 years old, doctors gave him a diagnosis no child or parent ever wants to hear. He had Duchenne Muscular Dystrophy, a rare, incurable, and fatal genetic disease that slowly erodes muscle strength and robs young people of their independence. By their teenage years, they begin losing strength in their arms, and eventually, the disease attacks the heart and the muscles that control breathing. Most succumb to the illness by their mid-twenties.
For someone with this disease, time is a luxury they just don’t have. 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. Elijah is fighting for his life—and for the lives of thousands of others who share his desperate need for hope—by advocating for the Right to Try for Individualized Treatments.
The reform, which is now law in six states and currently under consideration in the Iowa Legislature with SF 233 and HF 320, challenges the slow, bureaucratic machinery of federal drug approval. It recognizes that those facing debilitating, rare diseases don’t have time to wait on the government. And it allows patients to access cutting-edge therapies tailored to their personal genetic profiles that have not yet been approved by the Food and Drug Administration.
“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 Iowa, live with rare diseases. While new technology is ushering in a golden age of personalized medicine—where treatments can be customized to a patient’s unique DNA—regulations have not been able to keep up with the pace of innovation. Too often, those suffering from degenerative or rare diseases have to wait years for a therapy that could help them today while their treatment goes through stringent reviews by government agencies.
By enacting the Right to Try for Individualized Treatments, Iowa lawmakers can restore agency to those who have exhausted every other avenue and shift the balance of power from distant regulators to patients and their doctors.
“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.”
Brian Norman is the Director of State Affairs at the Goldwater Institute, where he assists in the development and implementation of Goldwater’s national legislative affairs strategy.
Author: Brian Norman
Brian Norman is the Director of State Affairs at the Goldwater Institute, where he assists in the development and implementation of Goldwater’s national legislative affairs strategy.
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