More than 1 million Americans die each year from a terminal illness. For those patients who are still fighting, recent passage of the federal Right to Try Act will expand access to potentially life-saving treatment before it’s too late. But this will only happen if the Food and Drug Administration faithfully applies the law and doesn’t stand in the way of patients seeking new hope.
The law bears the name of Matthew Bellina, one of the many Americans who stand to benefit.
As a Navy pilot, Bellina found himself plagued by balance problems, muscle aches and hand cramping.
He was a 31-year-old father of two young sons when he was definitively diagnosed with ALS, also known as Lou Gehrig’s disease. ALS is a progressive neurodegenerative disease that gradually robs its victims of the ability to walk, control their hands and arms, chew food, and even breathe.
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There are federally approved drugs that can slow the disease’s progression, but at the time of his diagnosis in 2014, Bellina did not qualify for them. His illness was too advanced for some to be effective and not advanced enough for others.
With few options remaining, Bellina became an advocate for laws giving terminally ill patients access to medication and treatments that have yet to receive final FDA approval. As his wife Caitlin put it, “Knowing that Matt can’t try experimental drugs when he has a terminal illness that’s going to kill him anyway is the dumbest thing ever.”
Bellina’s advocacy paid off. On May 30, he joined President Trump in the White House to witness the signing of the Right to Try Act. After exhausting all other options, patients facing the most dire circumstances will no longer need to wait on slow-moving Washington regulators to receive potentially life-saving treatments and medication that have passed Phase I of FDA drug trials, which tests for safety and side effects.
For terminally ill patients and their families, Right to Try offers renewed hope in their fight for more time. Bellina says two reputable drug companies have indicated they would attempt to treat him under the new rules.
Forty states had already passed their own version of Right to Try, but without federal approval these measures were often ineffectual. Doctors and providers worried about prescribing treatments that had not been approved by the FDA, even if the state government approved.
The new federal policy should provide much-needed certainty for patients, but troubling new statements from FDA Commissioner Scott Gottlieb suggest his agency doesn’t think the new law is as clear cut as it seems and that the FDA may continue to interject itself in the process by issuing new regulations that could limit access to experimental treatments.
Sen. Ron Johnson, R-Wis., the author of the law and long a champion of Right to Try, wrote to Gottlieb, “this legislation is fundamentally about empowering patients to make decisions in cooperation with their doctors and the developers of potentially life-saving therapies. This law intends to diminish the FDA’s power over people’s lives, not increase it.”
To be clear, the passage of this law does not curtail previous FDA authority; it simply creates a new pathway for access to experimental drugs consistent with the existing FDA approval process. All Americans want to know that the drugs they take are safe and the FDA will still play that role.
But the agency must not stand in the way of terminally ill patients like Matt Bellina who want nothing more than the chance to save or extend their lives. Doing so would violate the spirit of this important new law.
David Barnes is policy manager for Americans for Prosperity.
