In the Oval Office on Wednesday the president kissed a small boy with muscular dystrophy. Behind them were two men who Trump described as battling ALS. He thanked them for their bravery. He took up his pen for the camera and announced that by signing the controversial legislation—known as “right to try”—he would be saving hundreds of thousands of lives.
“We will be saving—I don’t even want to say thousands, because I think it’s going to be much more. Thousands and thousands. Hundreds of thousands. We’re going to be saving tremendous numbers of lives.”
In fact it’s unclear if the law will save a single life, especially when weighed against how many lives it could shorten. There’s no way to know, and that is exactly the point. The law allows pharmaceutical companies to provide medications to patients that have not been tested for effectiveness, and with only minimal evidence of safety. On the long list of ways the United States could improve access to quality health care—including affordable, safe, effective medication—nowhere does “right to try” appear.
It is rather a step in the wrong direction, but one that is easy to misrepresent and to sell as good. Typically only drugs that have been deemed safe and effective by the Food and Drug Administration, based on three-phase clinical trials, can be sold to patients. “Right to try” allows that process to be circumvented—though in news coverage this often receives a more congratulatory slant. CNBC announced that Trump’s signature would “allow gravely ill patients to bypass [the] FDA for experimental treatment.” As The Washington Post said, it “[gives] desperately ill patients the opportunity to receive promising experimental drugs that do not yet have FDA approval.” This is much the same as Trump’s own words: “These are experimental treatments and products that have shown great promise, and we weren’t able to use them before. Now we can use them.”
What’s not to love about that?
I also, of course, believe that extremely ill people should have the right to try promising treatments. I believe that not just as a doctor, but simply as a human.
Even some of Trump’s most vocal critics support the legislation. Congressman Ted Lieu, who has been a vocal detractor of the president, wrote on Twitter on Wednesday: “Pleased [President Trump] signed the right to try bill. I voted for it because the current status quo is unacceptable. A close friend of mine was prescribed experimental medication but couldn’t get it because of the messed-up incentives for drug companies. She died of cancer.”
The self-described “opponent of President Trump” Ed Krassenstein similarly broke form: “I applaud Trump for signing the ‘Right to Try’ bill today. It’s not often that I applaud him for doing something, so this is a huge occasion.” (He added: “This doesn’t wipe out the fact that he conspired against the United States, obstructed justice, spread hate, and is ruining America.”)
So what’s the catch? Did something just happen that everyone loves, and will save hundreds of thousands of lives?
If that were the case, of course, the bill would’ve passed long ago. In fact, many ethicists and doctors and patient advocates quite emphatically oppose it, as do former FDA commissioners. The American Society of Clinical Oncology is among nearly 40 health organizations that have publicly dragged the bill, saying it “could do more harm than good to seriously ill patients.” In a February letter to Congress, the groups reminded legislators that the current regulatory system for medical products was created as a result of serious harm and exploitation that occurred early in the 20th century: “Birth defects resulting from Thalidomide are an example of what happens when drugs are given to humans without proper safety review and approval.”
The legislation is a product of the conservative advocacy organization the Goldwater Institute, and backed by the Koch Brothers’ Americans for Prosperity. The name is cynical but effective. As Trump said on Wednesday, “‘Right to Try.’ It’s such a great name. Some bills, they don’t have a good name. Okay? They really don’t. But this is such a great name, from the first day I heard it. It’s so perfect.”
The name is certainly catchier than the existing name for the program that already does almost exactly the same thing—allowing people with serious diseases to obtain experimental medicines. It is known as expanded access, or more commonly, “compassionate use.”
The difference is that the current program operates through the Food and Drug Administration, which retains the ability to deny some requests for drugs it has not yet approved for use in the general population. The FDA reports that it already authorizes more than 99 percent of requests—so the upcoming change could be minimal. But the potential to exploit this lack of oversight is a risk. In the rare cases when access to unapproved drugs is denied, it can be because of serious concerns about risks on behalf of the pharmaceutical company, or because a physician has overlooked an obviously better alternative.
Under “right to try” this safeguard will be gone, and drugs can be usable after just phase one of clinical trials. As David Gorski, a cancer surgeon and professor at Wayne State University, wrote on Twitter this week, “Claiming phase I testing is enough to show that a drug is ‘safe’ enough for #RightToTry ... is utterly insane, as anyone who’s ever had anything to do with clinical trials knows.”
Three phases are required before a drug can be deemed safe and effective. Even then, it is very common for new side effects to be discovered, or for a drug to turn out to not be effective. Phase one is just basic safety testing. It does not even prove that a drug is safe to be taken to market, but only that it is safe enough to be tested further. Only around 10 percent of drugs that pass phase I trials go on to be deemed safe and effective.
While the compassionate-use process is not perfect, a policy report from Rice University noted that in states that have passed “right to try” laws, obtaining drugs directly from a manufacturer can be even more burdensome than the current FDA method. That analysis found that “right to try” laws “do not provide patients with a new mechanism to access investigational drugs. Instead, the laws can be harmful to both patients and public good by delaying the testing process. Patient advocates should work with the FDA and pharmaceutical companies to improve the current federal system.”
The longer-term concern is that the law is part of an explicit agenda to weaken the FDA. This has been dismissed as conspiracy theory, but it’s also what the author of the bill said this week. Senator Ron Johnson wrote to the FDA, “This law intends to diminish the FDA’s power over people’s lives, not increase it.”
At the same time, the Goldwater Institute is working to allow pharmaceutical companies to market drugs to physicians based on “off-label” uses—those that are not supported by clinical trials. This is in keeping with the concern that lobbyists will continue to press for weakening the FDA to the point where drugs can go to market without any evidence that they actually, safely do what they claim.
Still most importantly, if we wanted to help people have access to affordable health care, there are many ways—hundreds of thousands of ways—to go about doing that.
The goal should not be a right to try, but a right to have safe, effective, affordable drugs that do not drive people to medical bankruptcy. When we remain so far from that, there is no celebration in offering people untested substances and crossing our fingers and then claiming that we have improved the health-care system, much less that we have saved even one life.