The Trump administration has thrown its weight behind giving patients the “right to try” experimental drugs outside clinical trials. While such laws purport to offer hope for the dying, they will actually slow medical progress.
“Right-to-try” laws have already passed in 33 states at the urging of the Phoenix-based Goldwater Institute. They give patients with terminal illnesses the opportunity to buy unproven drugs that passed the Food and Drug Administration's initial safety test.
To gain full FDA approval, a drug must be proven both safe and effective. That usually requires three clinical trial phases, only the last of which offers definitive proof that the drug provides some benefit.
The FDA last year pushed back against the burgeoning “right-to-try” movement by highlighting its compassionate use program, in place since the AIDS crisis of the late 1980s. It gives dying patients the right to access promising drugs in clinical trials. The FDA authorized 99% of the 1,200 requests for compassionate use in 2015.
Whether a drug works or not isn't the primary concern of the libertarian Goldwater Institute. “We just fundamentally do not believe that you should have to apply to the government for permission to try to save your own life,” a spokesman told the Wall Street Journal.
There are actually plenty of reasons why you should be required to apply to the government to take experimental drugs—the most important of which are scientific.
If offered the option of taking experimental drugs, people with dire diagnoses will be less likely to enroll in the randomized clinical trials needed to show proof of efficacy. Why enroll when you run a 50-50 chance of receiving a placebo?
Since many fatal diseases have small patient populations, enrolling enough patients in those trials for statistical validity often takes years. Widespread use of right-to-try will slow the process even more.