Right-to-Try Laws Could Increase Gene Therapy Access

The United States leads the world in approved gene therapies. Europe and China aren’t far behind. That sounds like good news, and it is, until you see how far all of them remain from what the technology can do.

What’s the holdup? Unsurprisingly, regulation.

Before a gene therapy reaches a patient, it runs a gauntlet. It answers not only to the FDA but also to the Center for Biologics Evaluation and Research, and historically to bodies such as the Office of Biotechnology Activities and the Recombinant DNA Advisory Committee. A typical therapy takes 12 to 15 years to win approval, partly because the agency struggles to write testing rules for treatments tailored to a single patient. Thousands sit backlogged in line.

Basic economics tells us what over-regulation buys: higher prices and less access. Zolgensma, the one-dose treatment for spinal muscular atrophy, arrived at $2.1 million, the most expensive drug in the world. By one estimate, moving a single gene therapy through approval costs nearly $5 billion and roughly $1 million per trial participant.

Those price tags ripple outward. Insurers hesitate to cover a seven-figure treatment, so physicians hesitate to recommend it, and patients go without. Not because the medicine failed, but because the system priced it out of reach.

And these aren’t cosmetic treatments. Spinal muscular atrophy, the disease Zolgensma treats, is the most common genetic cause of death in infants. The patients who need gene therapy are among the sickest in the country, and there aren’t many of them.

The FDA says reform is coming. It usually does. But in nearly ten years of writing about the agency, I can count the deregulatory reforms that actually stuck on one hand.

So where’s the fix? Not in Washington. It’s in the states, and it goes by a familiar name: right-to-try. Right-to-try won’t lower the sticker price on an approved drug. What it does is get experimental treatments to patients before the FDA signs off, and its newest form aims squarely at individualized gene therapies. The idea is grassroots, and it took hold long before the FDA even noticed.

That push has a face. Abigail Burroughs was twenty-one when she died of head and neck cancer in 2001, after the system kept her from an experimental drug her oncologist thought could help. Her father founded the Abigail Alliance and sued for the right of dying patients to try treatments the FDA hadn’t approved. He lost in court. The movement didn’t die with the case.

It became right-to-try, and it delivered legitimate hope. Ted Harada, who told an experimental procedure wouldn’t help his ALS, tried it anyway under Georgia’s law. His symptoms reversed. A man who couldn’t open a Ziploc bag was soon hiking with his children. Others reached treatments for brain cancer. Not every story ended that way, but real people got chances they would otherwise have been denied.

In 2018, right-to-try became the law of the land. And promptly underperformed. Hemmed in by fine print, it required patients to be terminally ill and to have exhausted every approved option first. Who decided a patient was qualified? The FDA, of course.

So the action moved back to the states, this time through a revamped generation of right-to-try laws. Arizona went first. In 2022, it passed a “Right to Try for Individualized Treatments” law, allowing access to personalized therapies, including gene therapies, that haven’t completed the FDA’s first phase of testing. Montana went further, dropping the terminal-illness limit in 2023 and, in 2025, licensing the country’s first experimental treatment centers. Roughly seventeen states have now passed individualized right-to-try laws, and more are moving.

None of this is a cure-all. The laws are young, and critics are fair in noting that no statute can force a company to hand over its drug or an insurer to pay for it.

But strip away the fights over cost and access, and something simple remains. Gene therapy is a reminder that medicine is personal, as personal as a treatment tailored to one child’s genome. Federal policy is the opposite. It treats everyone the same, which is exactly why it so often fails the people who don’t fit the mold. State policy, imperfect as it is, gets us closer to how medicine should be practiced: one patient, one decision at a time.