Imagine your child is suffering from a rare disease. You’ve consulted countless doctors and explored every available treatment in the United States, but your child’s condition continues to deteriorate. Then, one day, your child’s physician mentions a promising new treatment tailor-made for your child’s unique genetic makeup.
Unfortunately, there’s a major hurdle.
The treatment is only available in Europe because the US Food and Drug Administration’s (FDA) outdated approval process is delaying the treatment’s timely access in the United States. To save your child, you’d have to travel overseas and possibly relocate for many months to obtain the needed treatment.
This is not a fictional scenario. It is a heartbreaking reality for families whose loved ones are running out of time. But it doesn’t have to be this way.
In North Carolina, hope is on the horizon for suffering patients thanks to a proposal that passed the General Assembly just this week and now awaits Gov. Roy Cooper’s signature.
The Right to Try Individualized Treatments Act offers a potential lifeline for those families. This legislation allows patients suffering from severely debilitating or life-threatening illnesses to work with their physicians to seek pioneering investigational treatments. These treatments, designed for specific individuals based on their unique genetic makeup, offer a hyper-personalized approach that promises to revolutionize how we treat rare and genetic diseases.
Unfortunately, an outdated regulatory approach at the FDA means many of the most promising potential treatments are kept out of the hands of patients with rare diseases. It’s estimated that just 5% of rare diseases have an FDA-approved treatment.
The FDA’s clinical-trial process can take years to complete and is structured to evaluate drugs meant for large patient populations, not treatments designed for single patients. This mismatch between the latest science and the regulatory system means patients are denied access to the most innovative treatments because the FDA’s clinical-trial protocols aren’t designed with individualized treatments in mind. This occurs despite the fact that individualized treatments represent groundbreaking modern medicine.
One million North Carolinians are living with a rare disease — they and their families deserve better. The Right to Try Individualized Treatments Act can help solve this regulatory mismatch and get innovative treatments to the people who need them most.
North Carolina has a rich history of supporting patient-centered policies. Building on the state’s prominence as a leading global innovation hub, North Carolina passed its original Right to Try law in 2015, allowing terminally ill patients to access experimental drugs that have cleared Stage 1 of FDA clinical trials.
Right to Try has been a beacon of hope for terminally ill patients — even more so after this groundbreaking reform was signed into federal law in 2018. In 2019, the North Carolina legislature expanded the state’s Right to Try Act to allow terminally and chronically ill patients to receive experimental stem-cell treatments.
This most recent proposal builds on that success by establishing a new pathway for those patients whose doctors believe an individualized treatment offers the most hope.
This bill helps ensure that North Carolina’s Research Triangle, which is home to some of the world’s most innovative medical research, remains on the cutting edge of medical innovation.
North Carolina has a unique opportunity to bring these cures to patients and join a growing list of states that have unleashed potential innovation in the healthcare industry. By expanding the Right to Try, North Carolina can open a needed pathway for suffering patients to safely access individualized treatments and keep the state at the epicenter of global innovation.
The Right to Try Individualized Treatments Act promises to keep North Carolina at the forefront of medical innovation, ensuring that the state continues to be a beacon of hope for patients and families desperately in need of state-of-the-art medical solutions.
Sen. Benton Sawrey and Rep. Allen Chesser, members of the North Carolina General Assembly, are co-sponsoring the Right to Try Individualized Treatments Act.