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.
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.
According to Gottlieb’s testimony, the biggest barrier to gaining access to these experimental products isn’t regulation, but drug makers themselves. Pharmaceutical companies don’t have a huge amount of extra drug product sitting around, because they don’t manufacture at scale until a product is approved. And they worry that if a drug doesn’t work or causes harm, it might lead to negative publicity that could make it harder to get patients to enroll in clinical trials.
In a right-to-try scenario, medicines would be given to very sick patients outside of a carefully managed trial setting, so bad results that scare off potential clinical trial participants or investors are far more likely. That could ultimately slow a drug’s path to market, and limit future access.
There’s nothing in this legislation that would compel drug makers to increase the availability of medicines that are most likely to be beneficial. But it could make things easier for companies that are more interested in squeezing revenue from marginal products than they are in winning FDA approval. Meanwhile, the full financial burden of these treatments is likely to fall on patients and families.
As it is, insurers are reluctant to cover approved drugs that have limited evidence of efficacy. Why would they be willing to shell out for medicines deemed even riskier than that? People may end up putting themselves or their families into financial jeopardy for treatments that are highly unlikely to help.
Some lives might be saved or improved by this legislation. But that will likely come at a heavy cost.
— For more columns from Bloomberg View, visit http://www.bloomberg.com/view.
Max Nisen is a Bloomberg Gadfly columnist covering biotech, pharma and health care. He previously wrote about management and corporate strategy for Quartz and