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months to obtain a gene therapy treatment that doesn’t fall within the U.S. Food and Drug Administration’s (FDA) outdated clinical trial system.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. The Golden State has already been a leader in putting patients’ rights first and cutting through medical red tape. In 2016, the state enacted the original Right to Try Act with nearly unanimous bipartisan support. Under this first Right to Try law, patients have the right to seek treatments that are safe enough to be used in clinical trials but remain under clinical evaluation for final FDA approval. The federal Right to Try law was later signed into law in 2018 and is now the law of the land.
We know that Right to Try works, and we’ve seen a great example in Orange County. An aggressive form of brain cancer, glioblastoma, has a five-year survival rate of only about 5 percent. Too often, patients are left with no promising treatment options. Thanks to the liability reforms and reduced red tape that is part of the Right to Try law, some patients who were ineligible for the clinical trial can now access this immunotherapy treatment, which is in a clinical trial being led by a physician at UC-Irvine. The trouble is, this law needs to be upgraded and modernized to account for rapid advancements in medicine, such as gene therapy, which aren’t covered under the original law. That’s where Right to Try for Individualized Treatments—or “Right to Try 2.0”—comes in.
Many of the medical innovations being pioneered in California have made it possible to take an individual’s genetic information and create a treatment for that individual person. But the current clinical trial evaluation system—created more than a half-century ago—is based on treatments for large populations, not an individual patient. The end result is that an individualized treatment is still subject to the same clinical trial process as a single treatment that is intended for hundreds or thousands of patients. But that doesn’t recognize how these new individualized treatments work. Right to Try for Individualized Treatments accounts for new innovations—and it helps get those innovations to the patients who need them.
California has the opportunity to continue to lead on the important goal of getting the right treatment, to the right patient, at the right time. While there are unrealized economic opportunities, there are also benefits to California’s patients. A 2019 report from the USC Schaeffer Center for Health Policy & Economics documents that, in addition to the economic benefits that come with medical innovation (such as job creation), the benefits of reducing disease burden, such as promoting a higher quality of life, higher productivity, and independent living to Californians also provide enormous value.
California is already home to some of the world’s leading medical innovators and facilities. Removing the government red tape that stands in the way of a doctor’s treatment options does not require additional taxpayer investment and can be achieved in a manner that ensures patient safety and informed consent. California lawmakers have the authority, as well as the legislative vehicle, to unleash the potential of today’s medical innovation to further benefit patients. It is time for California to embrace this opportunity, continuing to lead the nation on this important issue.
Naomi Lopez is the director of healthcare policy at the Goldwater Institute.
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