(Bloomberg View) -- There is a worrisome sign that President Donald Trump is willing to abandon one of the Food and Drug Administration’s core missions: ensuring that the medicines Americans take are both safe and effective.
A national “right to try” law, supported by Vice President Mike Pence and scores of Republicans in the the House and Senate, is meant to circumvent the FDA’s regulatory authority by giving patients who are terminally ill the right to use drugs that the agency hasn’t yet approved. The idea sounds reasonable; in the past few years, bipartisan majorities in two-thirds of state legislatures have passed essentially the same law.
In reality, however, these laws give patients no new rights at all. They do nothing to compel drug makers to provide experimental medicines to the dying, or insurers to pay for them. They merely eliminate a patient’s right to sue for any injuries that might arise -- that is, if any patient ever gets an untested drug in this way. (So far, thankfully, no one has.)
In contrast, the FDA’s existing expanded-access program has granted thousands of requests -- 99 percent of those it has received -- for “compassionate use” of experimental drugs. Some patients say they have been frustrated by the amount of time this process takes, but the FDA has shortened its application process and, in any case, the longest wait comes in persuading drug makers to provide medicines still in the pipeline.
These delays are understandable: The pharmaceutical companies are being asked to provide drugs that they may possess only in limited supply for clinical trials, and without the confidence in their safety that the full FDA approval process provides. No one wants even a terminally ill patient to be harmed unnecessarily, to die sooner or in pain. Right-to-try laws envision dying patients using drugs after Phase I trials, which are preliminary tests of a drug’s safe dosage level and side effects.
Support for right-to-try laws is based in part on the popular myth that if the FDA focused on safety alone, then people would have faster and cheaper access to lifesaving medicines. This assumes, of course, that the FDA forces drug makers to perform a lot of unneeded studies. In fact, the FDA approves drugs significantly faster than its counterparts in Europe and Canada. It moves especially quickly when a new medicine is designated a “breakthrough therapy.”
And it’s essential that regulators require tests of a drug’s effectiveness. Few medicines, after all, are perfectly safe. What doctors and patients need to know is whether they’re effective enough to justify the possible side effects. Medicare and private insurers need this information, too, to tell whether a drug is worth paying for.
Unfortunately, the candidates Trump is said to be considering for FDA commissioner also favor right-to-try laws. The person he nominates should appreciate the need for competent federal oversight of prescription drugs -- and respect the FDA’s approval process enough to keep it running well.
--Editors: Mary Duenwald, Michael Newman.
To contact the senior editor responsible for Bloomberg View’s editorials: David Shipley at davidshipley@bloomberg.net.
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