Keira Riley turned 3 years old in January. She loves mac and cheese, would buy a Barbie Lego if she won $1 million, and wants to be Elsa from Disney’s Frozen when she grows up. That she is growing up in the first place is what makes her story both bittersweet and extraordinary.

Three years ago, Keira’s older sister Olivia was diagnosed with a rare and highly progressive disease called metachromatic leukodystrophy, or MLD. Olivia was 2 years old at the time. Keira, then a baby, received a similar diagnosis shortly thereafter.

The National Institutes of Health explains that MLD “causes progressive destruction of white matter in the brain,” resulting in “progressive deterioration of intellectual functions and motor skills,” and that affected people eventually “lose awareness of their surroundings and become unresponsive.” What this really means is that, instead of running, playing, and singing, these young children fade away physically and mentally until death, which usually occurs by age 5.

Keira had not shown any symptoms yet, and there was a very short window in which a promising gene therapy that was not yet available in the United States might dramatically halt or slow the progression of this unimaginably cruel disease. Treatment was too late for Olivia, but there was still hope to help Keira.

The Riley family quickly raised hundreds of thousands of dollars, relocated to Italy for several months during the height of the COVID pandemic, and obtained the treatment for Keira in October 2020. It wasn’t until almost one year later that the same treatment would be provided to the first patient in the U.S., which would have been too late for Keira.

Olivia and Keira’s story illustrates why removing the obstacles and barriers that limit the most innovative treatment options is of the utmost importance. The current clinical trial evaluation system in the U.S., created more than half a century ago, was designed for treatments intended for hundreds or thousands of patients. It does not take into consideration the extraordinary circumstances in which patients with rare and ultrarare diseases find themselves. That’s why the Right to Try for Individualized Treatments should be available to these patients so Americans can access innovative, potentially lifesaving treatments that are tailor-made for them without waiting for the federal government’s go-ahead.

Obtaining a personalized treatment for a rare disease should be the norm, not the exception. Rapid medical innovations are increasingly delivering the right treatment and care to the right patient at the right time.

This extends well beyond therapeutic treatments. Take, for example, Best Buy’s recent announcement that the company is partnering with Atrium Health to expand its hospital at-home model that it piloted during the pandemic. Patients who are transitioning from the hospital back to their homes or who just need monitoring and support can now receive these services remotely from the comfort of their home, thanks to modern technology.

Moreover, during the pandemic, the federal government and states across the country relaxed needless rules and red tape that had been limiting the availability of telehealth services, which allow patients to access high-quality virtual care from home using a computer, smartphone, or landline. Since then, telehealth has become more readily available, unleashing the potential to give the public faster and easier access to many forms of needed healthcare services and in new, innovative ways.

Patient empowerment, wherein patients gain greater knowledge of and control over important healthcare decisions, is only possible where those options and choices exist. And when the scientific knowledge and technology do exist, patients who are under their doctor’s care and following medical recommendations should never be forced to beg the government for permission to seek treatment or be blocked from it entirely.

That is why it’s imperative that policymakers make it a top priority to reimagine healthcare regulation. Government rules, regulations, and red tape are too often completely out of step with the promise of 21st-century medicine. Lawmakers must embrace innovation.

Today, Keira is a thriving 3-year-old. Indeed, one would never know that she had been diagnosed with a rare genetic disease and had traveled overseas before her first birthday to receive cutting-edge gene therapy in the hope of saving her life. Sadly, her sister Olivia, who never had the option to access this potentially lifesaving genetic treatment, is now enrolled in hospice care.

Lawmakers and policymakers need to consider an important question: Will they keep up with the promise of 21st-century medicine, or will they leave even more patients behind in an outdated regulatory system that’s tangled in red tape ?

Lives are hanging in the balance.

Naomi Lopez is the vice president of Healthcare Policy at the Goldwater Institute.