Georgia now has a 'right to try' law so terminal patients can take experimental medications

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Elijah Stacy

Credit: Goldwater Institute

Elijah Stacy is going to die. Soon.

The 23-year-old suffers from a rare genetic muscle wasting disease known as Duchenne muscular dystrophy (DMD), which has a global prevalence of 1 in 3,500 to 5,000 male births.

The average life expectancy for someone with DMD is about 30 years, according to a 2021 study published in Neurology.

Stacy was diagnosed at age 6, after his parents noticed him walking on his toes. Now, he uses a powered wheelchair.

"I lost my ability to walk when I was 11 years old," he said. "And then I started to decrease upper limb function as well."

He can no longer raise his arm. 

His diaphragm and heart muscles will deteriorate and Stacy will lose the ability to breathe on his own.

"That's why the disease is fatal," Stacy said. "It's really just a whole life of suffering because even when you could walk, you fall a lot on the floor; you can't keep up with your friends."

If there was an experimental treatment that might help, Stacy would try it.

The Hope for Georgia Patients Act, which passed with bipartisan support, allows terminally ill patients the “right to try” individualized experimental treatments with permission from their doctors. 

State laws modeled after Goldwater Institute's Right To Try legislation include patients with a "life-threatening" disease or condition, and not just "serious," as with the U.S. Food and Drug Administration’s current expanded access program. 

Georgia will be the ninth state to enact these reforms when signed into law, Goldwater Institute health policy expert Brian Norman said.

Arizona was the first state to pass this reform in 2022, followed by Nevada in 2023 and Maryland, North Carolina, Louisiana and Mississippi in 2024. 

Tennessee, Kansas, Arkansas, South Dakota and Colorado passed right to try laws this year.

Most treatments are designed for a large patient population, and the clinical trial process is designed to evaluate a mass market drug and its impact on larger populations, Norman said.

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The issue is individualized treatments vary from person to person.

"If you understand the science of my disease in particular, the disease is very heterogeneous, meaning that no one's disease is really the same," Stacy said. "So everyone's mutation is unique. It makes a lot of sense that you would need individualized treatment."

Experimental medications are available through right to try laws only after completing Phase 1 clinical trials, and treatments accessed through the expanded access program can be administered during a Phase 1 study. 

"Essentially, what happens is a manufacturer evaluates and analyzes your genetic information and they tailor a treatment specific to you that is meant to target the particular disease that you have," Norman said.

This innovation holds particular promise for individuals with rare diseases, 80% of which are genetic, and 95% of those rare diseases do not have an FDA-approved treatment option, Norman said.

There's no guarantee that an experimental treatment is going to save someone's life, but the pathway is paved, Stacy said.

"There's definitely more options now if you live in Georgia than there is if you're living in a state without this law," Stacy said.