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May 28, 2018 at 06:03 PM
The U.S. House of Representatives passed a bill this week that will give terminally ill patients access to unapproved experimental drugs. President Donald Trump will likely sign it into law soon.
This national “right-to-try” legislation seems like a sensible idea at first blush. Dying patients could get treatments that otherwise wouldn’t be available to them until it’s too late. But it’s far more likely that the measure will foster false hope in desperate families, while potentially saddling them with costs for drugs that aren’t likely to do much good. This explains why patient groups, past FDA commissioners, and a variety of other organizations oppose it.
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The bill does set some restrictions aimed at protecting patients. Only drugs that have completed Phase 1 trials and are in active development will be available. In order to get an unapproved medicine, patients will have to have a “life-threatening illness” and they can’t be eligible for clinical trials. They’ll also need to have exhausted all available treatment options.
But these protections and limits don’t go all that far. For one, as far as testing goes, Phase 1 trials are the lowest of hurdles. They’re tiny and not especially rigorous tests of safety and dosage, and offer no guarantee that a medicine is effective. According to a recent study by Andrew Lo, an economist at the Massachusetts Institute of Technology, 13.8% of drugs that enter a Phase 1 study get approved. The success rate for cancer drugs, which will likely be a key focus of the right-to-try program, is 3.6%. That leaves the door open for a lot of drugs that are highly unlikely to help.
As for regulatory supervision, the Food and Drug Administration won’t directly oversee the right-to-try process. Drug makers will only be required to report safety problems once a year as they give out medicine under the program, which could limit the FDA’s ability to intervene and increase the likelihood that patients will take dangerous or ineffective medicines.
A particularly grating thing about this legislation is that it just isn’t needed. The FDA already has a program that gives patients access to experimental treatments. In congressional testimony last year, FDA commissioner Scott Gottlieb said that the agency approves 99% of such requests and that they are rapidly processed. The FDA also makes a concerted effort to get drugs that can help patients without good options to market, to the point where it has approved some medicines that might not actually work.
