The FDA already has an expanded access program for compassionate use for certain seriously ill patients to apply to gain access to experimental treatments, this legislation bypasses the application approval, although there is still a process and certain criteria must still be met. Critics say this undermines the authority of the FDA to regulate drugs and it may leave patients vulnerable to medicines that may not work or may be harmful, while others say it really isn’t changing anything except saving precious time, as the FDA already approves the almost all requests for access, and it gives hope to patients who are terminally ill who have exhausted all other options.
This law allows certain patients who met criteria with life threatening conditions who have exhausted all other options and are unable to participate in clinical trials to try medicines that have cleared the Phase 1 safety testing FDA approval process that are still undergoing testing and have not been approved by the FDA yet. While the drug makers are not obligated to provide the patients the experimental medicines they have requested this law still provides hope.
In a statement Scott Gottlieb, FDA Commissioner says the agency grants about 99% of the requests from patients applying for the expanded access program for compassionate use, and the FDA is ready to implement the right to try legislation.
“The FDA is dedicated to achieving the goals that Congress set forth in this legislation, so that patients facing terminal conditions have an additional avenue to access promising investigational medicines,” says Gottlieb.
It can take 12+ years for an experimental drug candidate to travel from lab to market in the rigorous FDA approval process, if they even make it. President Trump has said he doesn’t understand why passing this bill was so difficult since it can take years for drugs to undergo clinical trials.
While signing the bill President Trump said, “Right to try. That’s such a great name. Some bills, they don’t have a good name. Really. But this is such a great name, from the first day I heard it. Right to try. And a lot of the trying is going to be successful. I really believe that. I really believe it.”
