“Patients with terminal conditions should have access to experimental treatments that could potentially save their lives,” declared President Donald Trump in his first State of the Union. “It is time for the Congress to give these wonderful Americans the ‘right to try.’”
But what does “right to try” really mean? What is Congress’ role? And how do patients benefit?
About Right to Try
“Right to try” refers to allowing terminally ill patients to receive experimental medicines. That is, drugs that the Food and Drug Administration hasn’t yet approved. It’s also known as “compassionate use” or “expanded access.”
Before being considered for use of an unapproved drug, patients must have already tried all other available treatments. In short, this unapproved drug is their last opportunity to get better.
While 37 states have right-to-try laws in place, the federal government does not. Related legislation unanimously passed the Senate last August, but has remained stalled in the House since. Compromise language emerged this week.
There’s just one catch: The right to try already exists.
Who Decides when It’s Right to Try?
The FDA’s current expanded access process requires physicians to submit an application to the FDA on behalf of their terminally ill patients. By most accounts, it is timely and effective. The Food and Drug Administration granted 99.3 percent of compassionate use requests between January 2005 and December 2014, according to a recently published study.
Under the current system, patients can request access to drugs that the FDA has reviewed for safety and efficacy but not yet approved. Under the Right to Try Act, however, patients could access drugs that have gotten through only the first of three phases of clinical trials.
That raises concerns about patients’ safety – concerns that may be justified. Less than 10 percent of drugs in phase one trials ultimately wind up being approved. It also brings into question whether the right-to-try system envisioned by the legislation would undermine the FDA’s role of ensuring drugs’ safety and efficacy.
Most all Americans sympathize with terminally ill people’s desire to try experimental treatments. But in debating current legislation, policymakers must ask some complex questions. How much risk is justified in treating terminally ill patients? Who decides which drugs are safe enough to take? And, ultimately, is the Right to Try Act right for patients?