Family’s Story Highlights Need for Right to Try Expansion

Imagine hearing devastating news about your child’s health and knowing that a potentially lifesaving treatment exists, but that bureaucratic red tape is keeping it just beyond your reach. This was the harsh reality for Kendra Riley, an Arizona mother of three who faced a heart-wrenching race against time to save her infant daughter Keira.

Now, there’s a reform that can help families like the Rileys, Kendra and Goldwater Senior Fellow Naomi Lopez said in a recent interview on the Top of Mind podcast, emphasizing the urgent need for Goldwater’s Right to Try expansion.

Two of Kendra’s children were diagnosed with metachromatic leukodystrophy (MLD), a rare and progressive disease that impacts the white matter in the brain and then the nervous system. First, Kendra’s middle daughter, Olivia, lost the ability to walk and talk due to the condition. Then came the devastating news that Keira had the same gene mutation for the disease. But a ray of hope emerged from Italy, where an individualized gene therapy treatment that could help was available. Unfortunately, the treatment wasn’t available in the U.S., at least not in time to save Keira’s life—onerous U.S. Food and Drug Administration (FDA) restrictions force patients to wait months and years that they don’t have to access experimental, highly personalized treatments that could help.

Determined to save their daughter as quickly as possible, Kendra and her family embarked on a campaign to make it to Italy. “I saw cases of kids who are older and doing wonderful who had the treatment, so that was our hope,” Kendra said. The family had to fundraise half a million dollars through their own hard work and relocate to Italy to secure the treatment. Thankfully, Keira is now doing amazing—she’s swimming and attending school. Tragically, it was too late to save Olivia—she’s now in hospice care, her condition a stark contrast to Keira’s improved health.

Families like the Rileys shouldn’t have to travel across the globe to receive the care they need. That’s why the Goldwater Institute crafted a new law to ensure patients can access innovative, personalized treatment when they need it: the Right to Try for Individualized Treatments. Generally, the FDA requires all treatments to pass a first phase of basic tests, followed by lengthy clinical trials. But for cases like Keira’s, there may not be enough patients to put together a clinical trial or move into commercialization. This innovative law recognizes that individualized treatments should not be evaluated the same as those intended for large populations and allows patients to access them without begging the federal government for permission.

Right to Try reflect the idea that the federal government should not be the ultimate decision-maker when it comes to treatments your physician deems appropriate. The Goldwater Institute passed its original Right to Try law in 41 states and then at the federal level in 2018, protecting terminally ill patients’ access to experimental treatments that have not yet received full FDA approval. As Lopez succinctly put it on the podcast: “We want to make sure it’s not illegal to save your life.” The Right to Try for Individualized Treatments—which Goldwater already passed in Nevada and Arizona, and which we’re now taking nationwide— is the natural next step: it ensures that no one is cut off from potentially lifesaving personalized medical treatments due to outdated FDA protocols.

While the FDA plays a role in ensuring safety, there is bipartisan agreement that reform is urgently needed. After all, when a child’s life hangs in the balance, the last thing anyone wants is for bureaucracy to prevent lifesaving care. The Goldwater Institute remains steadfast in its mission to champion healthcare freedom, because the power to decide on treatments should rest in the hands of patients and their physicians, not the federal government.

Cameron Teel is a Ronald Reagan Fellow at the Goldwater Institute.