Editorial: 'Right to try' laws could impede medical progress

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.

It's true that patients nearing death from cancer and other fatal diseases are often excluded from clinical trials. Manufacturers want to run experiments on a patient population with the best chance of benefiting. But if they give the drug to people outside the trial, the manufacturers must still report adverse events. Drug developers rightfully fear those reports could jeopardize the ultimate approval, or result in label warnings that could eventually dampen sales.

Given the drug industry's recent pricing policies, its economic reasons for resisting right-to-try generate less sympathy. But they help explain why there's been virtually no use of the state laws that have already passed.

The laws require drug companies to sell their experimental drugs at the cost of manufacturing. Those relatively low costs are not something pharmaceutical companies are too keen on publicizing.

Moreover, there's also nothing in “right-to-try” laws that compels insurers to pay for the drugs or physician charges. That reserves this new “right” to those who can pay or raise enough money to pay for the treatments.

But the most serious concern in broad use of “right-to-try” is ethical. The law will wind up doing far more medical harm than good. Most drugs for life-threatening diseases have very serious side effects. That includes the targeted immunotherapies that are now the great hope for progress in the war on cancer.

Most drugs in clinical trials for life-threatening diseases turn out to be ineffective or extend life only briefly. The patients taking them in those cases will be suffering all the harms with little or no hope of benefit. That's something no doctor true to his oath would or should willfully condone.

The “right-to-try” bill sponsored by Sen. Ron Johnson (R-Wis.) already has 42 co-sponsors. Similar legislation in the House is all but guaranteed to pass. The U.S. appears poised to take a major step toward ending the FDA's status as the gold standard for drug regulatory agencies around the world. Coupled with the loosened regulatory standards in the 21st Century Cures Act, what we're witnessing is an unchecked slide toward regulatory mediocrity, which will eventually undermine the public's faith in the entire medical enterprise.