April 6, 2022
By Naomi Lopez
There’s a new treatment for a rare disease. It’s custom-made for you, based on your own genetic profile. It offers you hope, but you can’t access it, even though your doctor says it could save your life. The reason? Federal regulations are ancient by today’s standards, and they’re not designed to allow these new, genetic treatments.
California has an opportunity to solve this problem—and save lives—by passing a new law called Right to Try for Individualized Treatments.
The federal barriers to lifesaving treatment are not hypothetical. Take the case of Keira Riley, a young Arizona girl who was diagnosed with a highly progressive and rare genetic brain disease. Her family was forced to raise hundreds of thousands of dollars and move to Italy for several months to obtain a gene therapy treatment that doesn’t fall within the U.S. Food and Drug Administration’s (FDA) outdated clinical trial system. Had the Right to Try for Individualized Treatments been in place when Keira needed it, she may have been able to access that treatment at home. Today, Keira is thriving, and her family is advocating for the reform so that other patients facing rare diseases in the United States can access these types of promising, innovative treatments.
California ought to take charge on this life-or-death issue.
Read the rest of the op-ed at The Orange County Register.
Naomi Lopez is the Director of Healthcare Policy at the Goldwater Institute.