Thanks to private-sector innovation and public investment, medical innovation in America and across the globe is advancing more quickly than ever before. This progress, and the development of new treatments for deadly diseases, represents a tremendous opportunity to save lives.
But it does take significant time for new treatments to be developed. Patients who face long odds and terminal illnesses do not always have access to the latest drugs in clinical trials.
They don’t want to give up, but they don’t have years to wait for new drugs to receive FDA approval.
That is why President Trump is so committed to making sure these patients have the chance to try an experimental medicine if it shows promise for their condition.
At the Department of Health and Human Services, we are working to empower patients and their families to make informed decisions and, if they wish, to try a potential cure.
As of this month, we now have more opportunities to deliver on these goals. President Trump successfully fought for and signed a bill from Congress that will provide more access to promising new drugs.
Named after four activists and their families who fought for its passage, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 establishes a new pathway for access to unapproved, investigational treatments. These four Americans fought for the Right to Try because they didn’t want to give up.
Like many facing terminal illnesses, they wanted to be able to have as many choices and options available to them as possible — they were willing to risk the unknown if there was potential it could lead to a cure.
The Food and Drug Administration works to protect the interests of all patients and provide them with reliable information about the potential effects of treatments. But government rules should not stand in the way of potentially lifesaving therapies for those who do not have much time or any other options.
This administration believes in putting American patients first. That means giving them the power to make decisions over their own medical treatment, with the support and assistance of their doctors and families and the best medical information possible.
The new Right to Try law is a significant step in putting those principles into practice. It builds on extensive efforts already underway at the FDA to enable more patients with serious medical conditions to have access to promising treatment options.
For many years, the FDA has facilitated seriously ill patients’ access to investigational drugs, devices and biological products through a program known as “expanded access” or “compassionate use.” The FDA has authorized more than 99 percent of expanded access requests, sometimes in a matter of days or hours.
Recently, under Commissioner Scott Gottlieb, the FDA has taken new steps to further streamline this program.
Now, the Right to Try law will open up still more opportunities for patients who are diagnosed with a broad range of life-threatening diseases or conditions, who have exhausted all other approved treatment options and who cannot participate in clinical trials.
The medicines that will be available under Right to Try have already undergone a Phase I safety trial, but the benefits offered by these novel, and still unapproved, treatments are typically unclear.
This new law follows HHS’s practice of promoting access while protecting patients, and we look forward to implementing it.
Americans are blessed with a can-do spirit — we don’t give up, even in the face of a daunting challenge like a life-threatening illness.
Every patient in such circumstances deserves the Right to Try, and thanks to President Trump’s voice on this issue, more of them will have it.
Alex Azar is US secretary of health and human services.