Americans with Rare Diseases Need a ‘Right to Try’ Too

This is the final piece in a four-part series about Right to Try at the federal and state levels and the response from the private sector. The first piece defines Right to Try and explains its importance to patients. The second piece discusses the passage of federal Right to Try legislation. The third piece talks about the effects of Right to Try on the private sector.

Right to Try has been a literal lifesaver for many Americans with deadly diseases such as cancer and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). A federal law that protects terminally ill patients’ right to access treatments that could save their lives but aren’t yet approved by the Food and Drug Administration (FDA), Right to Try gives new hope to the 97% of patients who aren’t fortunate enough to get into a clinical trial. It empowers them to try to save their own lives without having to beg the federal government for permission.

But these traditional options are often off-limits for the 1 in 10 Americans who have a rare disease. Fortunately, new technology allows medical experts to tailor-make the latest medicines for some of those individuals, based on their genetics. Yet because these treatments are personalized, they cannot go through traditional clinical trial review, and thus they are not covered by the current federal Right to Try law. Innovation is outpacing regulation, and these treatments can’t go through the FDA’s decades-old approval processes in a timely manner—processes devised long before technological breakthroughs enabled these new personalized options. So these patients are out of luck, and they need their own Right to Try.

A Chance at Life, but an Ocean Away

In March 2020, the Riley family in Arizona received devastating news. Their one-year-old daughter, Olivia, was diagnosed with a rare and fatal brain disease called metachromatic leukodystrophy. Three months later, Olivia’s younger sister, Keira, received the same diagnosis. Because Olivia was already showing symptoms, there was little the family could do. Her disease progressed quickly, and within three months, Olivia lost the ability to walk and talk. She is unlikely to survive her childhood.

However, a personalized treatment was available that could help the Rileys’ younger daughter, Keira—based on a cutting-edge technology that uses genetics to customize a treatment just for her, modifying her DNA so her body can function as it was meant to. Time was of the essence, as the Rileys had learned from Olivia’s rapid decline. But this treatment hadn’t been approved by the FDA, so the Rileys couldn’t access the treatment in the U.S.

In 2018, Congress passed Right to Try. While this reform has empowered countless patients to access potentially lifesaving care, it wasn’t an option for the Rileys because Keira’s treatment wasn’t a traditional drug; it was a new technique, personalized just for her. Right to Try applies only to medicines undergoing traditional clinical trial review. So, the Rileys had no choice but to raise hundreds of thousands of dollars to move to Italy, where the treatment is available—hard even in the best of times, let alone in the middle of a global pandemic.

In the end, the Rileys were able to afford that option. And, thanks to the cutting-edge, personalized treatment she received, two-year-old Keira is developing normally today: running, climbing and having full conversations. But there are countless other Americans with rare diseases for whom traditional treatments won’t work, who aren’t able to travel across the world to get the care they need. Those patients need a Right to Try, too.

Technology and Red Tape

As the next generation of genomics-based medicine becomes more available and accessible, so will our ability to craft these individualized medical treatments. Today, the obstacle isn’t the science or the technology; it’s the federal rules and red tape. The FDA’s outmoded drug approval process requires testing each medication on a large number of people. However, the latest kinds of treatments—especially those for rare diseases—are individualized based on a single patient’s specific genetics. By definition, they are designed for and can only work for one person—which means they can’t go through the old-fashioned FDA processes in a timely manner.

For example, a cancer treatment can be designed based on a patient’s own diseased cells or unique genetic makeup, which stimulates that patient’s immune response to target cancerous tumors. It’s truly remarkable science, enabling the body to turn the tables and fight off the disease that’s attacking the body. But that unique treatment only works for the individual patient. The FDA’s existing clinical trial system can’t work in cases like this because its safety and testing protocols don’t accommodate this kind of singular treatment.

In other words, requiring patients who need individualized treatments to undergo clinical trials is a regulatory mismatch—a square peg in a round hole. After all, the goal of a clinical trial is to learn something about the treatment that will be generally applicable to a group of patients and move the treatment closer to FDA approval for sale. Any benefit to patients participating in a clinical trial is secondary. But in clinical care, the primary goal is treating the individual patient. The current regulatory system subjects patients seeking individual care to a system designed for general study. That means it forces patients to undergo lengthy processes and beg the federal government for permission before they can receive potentially lifesaving treatment. Thus, Right to Try must be taken to the next level, expanded so that the growing number of patients seeking individualized treatments are not caught in needless red tape.

Americans don’t have to wait for FDA reform to put these patients first. Just a few years ago, all terminally ill patients were forced to endure the FDA’s slow-moving approval process to access potentially lifesaving treatments. Today, many of these patients have new hope and more options under the first Right to Try law. That’s why it’s time to take things to the next level with Right to Try 2.0.

Similar to the original Right to Try law, Right to Try 2.0 (or “Right to Try for Individual Treatments”) allows patients to access treatments that are not yet FDA-approved by working directly with their doctor and pharmaceutical manufacturer. And like its predecessor, Right to Try 2.0 has safeguards to protect patients who are pursuing treatments. Under the current Right to Try law, treatments must have passed basic FDA clinical-trial safety testing. Because Right to Try 2.0 treatments don’t undergo traditional clinical trials, the reform measures safety in an analogous way: Individualized medicines must comply with the same regulations required by federal law for the protection of human subjects in research. In fact, most facilities that would offer individualized Right to Try treatments are already following these federal safety rules. Right to Try 2.0 simply allows these revolutionary treatments to proceed without forcing patients to get government permission first.

This year, Arizona became the first state to expand Right to Try to individualized treatments. Keira’s mom, Kendra, testified before the Arizona legislature about why Right to Try 2.0 is so desperately needed. After all, if Right to Try 2.0 had been enacted just a few years ago, her family could have gotten lifesaving care right at home, rather than having to travel overseas. “It brings tears to my eyes thinking of all the other special-needs families out there who have always held onto hope for a chance like this. A chance at healing. A chance at a normal life—something every single one of us deserves,” Kendra said. She’s hoping that other states will follow suit and that Right to Try 2.0 will soon be the law of the land.

A Brighter Future

Bespoke treatments are no longer distant dreams—thanks to technological advancements, they’re becoming reality. And patients and their advocates don’t have to wait decades for federal regulations to catch up. They’ve learned that they can bring about nationwide change by working from the ground up, starting in state capitols.

The success of Right to Try shows that despite the increasing centralization of U.S. healthcare, the nation’s founding promise—the right of every person to the pursuit of happiness; in other words, the right to strive—has not dimmed. Right to Try 2.0 could be the dawn of a new era, a return to patient-centric care governed not by bureaucrats, but by the doctor-patient relationship, allowing Americans to live freer, happier and more hopeful lives.