COMMENTARY

Why the Right-to-Try Law Is a Complete Failure

Arthur L. Caplan, PhD

Disclosures

April 16, 2019

This transcript has been edited for clarity.

Hi. I'm Art Caplan, head of the division of medical ethics at the New York University School of Medicine.

Some of you may remember this year's State of the Union Address, when President Trump discussed many ideas he had, including the possibility of eliminating HIV and driving down drug prices. One of the things he said he was especially proud of was the Federal Right-to-Try [Act] that he, Vice President Pence, and others in Congress helped to enact.

That law has been on the books for roughly 1 year. The president made a point of noting that Matt Bellina, who was at the signing ceremony at the White House when the law was passed, had been able to receive a drug for his ALS (amyotrophic lateral sclerosis) because of the law. Trump said the Right-to-Try law was a major step forward, something he was very proud of, and one of the big health achievements of his administration.

Well, I'm sorry to say that I don't agree that the Right-to-Try law is something to be proud of. I don't believe it is working, and in many ways, it shows more of an ideological commitment on the part of the president than it does a practical commitment to getting people access to drugs not yet approved by the US Food and Drug Administration (FDA).

In a nutshell, the Right-to-Try law says that if you want to try a drug that hasn't yet passed muster with the FDA—is still in early phases of testing—then you should be able to do that without FDA approval, and without the approval of an institutional review board or research ethics committee. You should just be able to get whatever drug you need.

That sounds great, but the law didn't create any duty or obligation to give anyone drugs, devices, or experimental vaccines. So you can ask—or more likely, beg—a sponsor, usually a drug or device company, to give you a drug that hasn't yet been approved for sale or prescription by the FDA. But most companies won't do it.

Indeed, in the case of Matt Bellina, the gentleman who received an ALS drug from a company called BrainStorm, the company has said that it can't afford to give anyone any drug anymore.[1] Just this one individual has been helped. Over the past year there have been two cases, one of which arguably isn't really a right-to-try case, where someone has gotten access. The president promised that tens of thousands, hundreds of thousands of people would be getting access to unapproved drugs. So far, only one person has.

This is a law that has failed. It needs to be fixed. It needs to be changed and it needs to take into account the realities of getting unapproved drugs. The reality is that if you don't budget any money for a law such as this, then small companies like BrainStorm can't afford to give anybody anything. They're not going to be able to give away their drugs for very long. They are just too small. Other companies will say they are not going to do something without the FDA—"We don't trust that we can get our drug approved if we don't talk to the FDA at all points in time."

Moreover, there's no reason to get the FDA out of the way because the FDA's expanded-access program, which has been on the books for many, many years, has given thousands and thousands of patients access to unapproved drugs. Often, the FDA has proprietary information about dosing and the frequency of administration of drugs that a drug company may not be aware of. So the FDA can actually be a help, not a hindrance.

Right-to-Try sounds good; it beats up on agencies like the FDA and on regulators, and says they're in the way. Get them out of the way and people will have access to drugs much more quickly. The reality, after a year of the federal law and a couple of years of state laws, is that no one is getting access. Until you find ways to reassure companies and pay them for giving away their products, they are not likely to want to act, even when asked.

I'm Art Caplan at the Division of Medical Ethics at NYU. Thanks for watching.

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