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Goldwater Advances New Pathways to Treatment Ahead of Rare Disease Day

February 5, 2024

February 29 is Rare Disease Day, and patient advocates across the country are spending this month raising awareness about the challenges facing Americans suffering from rare diseases, including limited access to cutting-edge medical treatments. The Goldwater Institute is working to address this critical accessibility issue with a state-level reform known as the Right to Try for Individualized Treatments Act. This innovative reform, which the Institute has already enacted in Arizona and Nevada, empowers patients to work with their physicians to access promising investigational treatments that are designed just for them.

Over 30 million Americans suffer from rare diseases. Of those, nearly half are children. In the United States, rare diseases are considered to be those which impact fewer than 200,000 Americans. In some cases, there may be only a handful of people in the country, if not the world, who suffer from a given condition. Orphan diseases are those for which the chance of treatment is, sadly, nearly as rare as the disease itself.

With nearly 80% of rare diseases estimated to be genetic in nature, it is no surprise that access to innovative, individualized gene therapies is top of mind for many patients. Unfortunately, an outdated regulatory framework at the U.S. Food and Drug Administration (FDA) means many promising treatments are not reaching patients in time. According to the National Human Genome Research Institute, 95% of the nearly 8,000 known rare diseases do not have a single treatment approved for patient use by the FDA. This distressing reality is the result of an antiquated FDA drug approval process that requires large patient populations and huge amounts of investment from research companies: from start to finish, the costs of bringing a new treatment to market are estimated to be as high as $1 billion, with a time investment of nearly 10 years. For patients with rare or ultra-rare diseases, the hope of an FDA-approved treatment is almost certainly out of reach. The FDA has just made it too expensive and too burdensome for researchers to spend time and resources to develop trials for these smaller patient populations.

Fortunately, new technologies and recent advances in modern medicine have made it possible for researchers to tailor treatments to an individual patient using his or her own genetics. Due to their highly personalized nature, these kinds of treatments do not fit into the traditional FDA approval pipeline. The Right to Try for Individualized Treatments Act protects the right of patients to pursue these customized treatments without having to first beg the federal government for permission.

As with the original Right to Try, landmark Goldwater legislation enacted in 41 states and at the federal level in 2018, patient protections are an essential part of this new reform. Treatments must be investigational in nature, individualized to a patient based on his or her own genetics, and developed in facilities which comply with the same safety and ethics protections as those that participate in clinical trials. Patients must have been diagnosed with a life-threatening or severely debilitating illness and have considered approved treatment options, and a physician must agree to recommend the investigational treatment. Physicians are not compelled to recommend treatment nor are manufacturers compelled to provide treatment, nor to make a profit on the treatment. These considerations ensure that patients are informed and protected throughout the treatment process.

Now law in Arizona and Nevada, the Right to Try for Individualized Treatments Act is under consideration in other states this legislative session. Recognizing that patients should not be punished for an outdated FDA approval process failing to keep pace with medical innovation, lawmakers from California to Iowa to Maryland have introduced versions of the reform. As Maryland’s Delegate Matt Morgan noted in a recent article explaining his support for the reform, “everyone deserves a fighting chance” to save their own lives.

The Goldwater Institute thanks the many patients, advocacy organizations, and supporters who have worked tirelessly both this month and year-round to protect the right of patients everywhere to seek innovative treatments to save their own lives.

Heather Curry is the Director of Strategic Engagement at the Goldwater Institute.



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