SANDEFUR: One Year On, Right To Try Is Empowering Terminally Ill Patients


Christina Sandefur Goldwater Institute
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One year ago, what seemed impossible became reality when the president signed the Right to Try Act — a federal law that protects patients’ right to seek medicines that have received basic safety approval by the Food and Drug Administration (FDA), but have not yet been fully approved for sale.

The Right to Try movement was genuine grassroots reform, passed by a majority of states before arriving in Congress. It received strong bipartisan support all the way.

It’s pretty unusual for state and federal legislators of both parties to come together on anything nowadays, especially a major piece of healthcare reform legislation. But patients have been trying for decades to break the bureaucratic inertia that has slowed their access to innovative treatments that could improve or even save their lives. They knew their time had finally come.

The Right to Try movement began because every year, thousands of Americans suffer and die while potentially lifesaving treatments are mired in a bureaucratic process that takes, on average, 15 years before they can obtain approval for sale. Right to Try makes a statement that in our free country, people, in consultation with their doctors, have the right to decide for themselves whether to try medicines that could save their lives. Their lives, after all, belong to them.

Right to Try has already helped patients across the country. When Marc Hayutin was diagnosed with neuroendocrine cancer, he was told he had only months to live. Today, six years later, he is still alive, after being able to access a treatment that at the time was not yet FDA-approved. In fact, he was in the midst of treatment when federal bureaucrats halted the study he was participating in because they decided they didn’t need any more data. Marc was desperate and out of options. But thanks to Right to Try, he was allowed to complete his treatment — and he credits Right to Try with saving his life.

Matt Bellina has been a vocal advocate for Right to Try ever since he was diagnosed with ALS in 2014. His illness had progressed too far for one FDA-approved treatment and not far enough for the other, so before Right to Try, he was out of options — and out of hope. He could barely stand or speak. But after Right to Try became federal law, Matt was given access to an investigational drug, and after the first round of treatment, he could speak and swallow, could pull himself up a standing position, and no longer needed to sleep with a breathing machine.

Of course, nobody can know what the future has in store, but these dramatic improvements mean he’s been able to better enjoy time with his wife and three sons. Most importantly to Matt, he was able to make that treatment decision himself, rather than being at the mercy of a federal bureaucrat.

In addition to changing lives, Right to Try has changed the culture in Washington, D.C. It’s one of the few times in recent history that Congress has reined in FDA overreach. And it began not in the boardroom of bureaucratic staff members, but among citizens themselves in their own state capitals. Right to Try marks an important step on the path toward more patient-centric healthcare in America — and it serves notice that government must strive to maximize patient choice, not government paternalism.

At its inception, the FDA focused on ensuring that products sold to the public were safe and accurately labeled, to help patients make their own choices safely. But over time, the Agency’s focus has drifted from empowering patients to making patients’ choices for them. That change has been well-intended, but the question of what risks a patient is willing to take is not a scientific question. It’s a decision that should rest ultimately with the patient.

There’s plenty left to do. Lawmakers across the country can adopt additional reforms to protect patients’ freedom of choice and increase access to treatments, such as “conditional approval” for promising treatments, granting U.S. approval for medicines already approved by designated countries, and permitting manufacturers to communicate directly with doctors regarding “off-label” benefits of drugs — something that’s currently not allowed.

Right to Try shows that our founders’ promise of life, liberty, and the pursuit of happiness is still alive. Legislators should take the next steps now, to improve access to safe and effective treatments, by empowering patients and doctors to decide for themselves how to improve — and even save — their lives.

Christina Sandefur (@CmSandefur) is executive vice president at the nonprofit Goldwater Institute, an Arizona-based nonprofit dedicated to limited government.

 The views and opinions expressed in this commentary are those of the author and do not reflect the official position of The Daily Caller.