The Right to Try Law of 2018 and the Families Who Pushed It Through

Imagine a bureaucracy so cautious that it protects a dying man from a medicine by making sure he dies without it. Now imagine that bureaucracy defending the policy as "patient safety." You do not have to imagine it. For decades that was the working description of what a terminally ill American could expect when the only drug with a chance had not yet finished its paperwork.

Let me tell you how ordinary families, without a lobbyist among them, got a federal law passed that said otherwise. It is one of the better stories in recent American self-government, and it started where most good ideas start: far from Washington, with people who had run out of time and were not willing to be polite about it.

What a "right to try" is, and what it is not

Terms first, because the whole fight turns on them.

An investigational drug is a medicine that has not been approved by the Food and Drug Administration for sale. Many of these drugs are in clinical trials, and a trial enrolls a limited number of patients who meet strict criteria. A patient who does not fit the criteria, lives too far from a trial site, or is simply too sick to wait is left outside the door.

Expanded access, sometimes called compassionate use, is the FDA's own program for letting a patient outside a trial receive an investigational drug. It runs through the agency. A doctor submits a request, the manufacturer has to agree to supply the drug, and the FDA and a review board have to sign off.

A right to try law is different in one respect that matters. It says that if a patient has a life-threatening illness, has exhausted approved options, cannot get into a trial, and has a willing doctor and a willing manufacturer, then the government's agreement is not a precondition. The patient, the doctor and the company can proceed under a written informed consent.

Notice what it is not. It is not a right to demand a drug from a company that does not want to give it. It is not a mandate on anybody. It is a removal of one gatekeeper from a transaction between consenting parties, which is a very old conservative idea applied to a very new kind of medicine.

2001: a young woman and a father who would not stop

The movement has a beginning you can date. Abigail Burroughs was a college student who was diagnosed with a head and neck cancer. She was told that two drugs in development showed promise, and she was unable to get either one. She died in 2001. Her father, Frank Burroughs, started the Abigail Alliance for Better Access to Developmental Drugs, and he did the thing that most grieving parents cannot summon the energy to do. He turned his loss into a lawsuit.

The Alliance's argument was bold and, in plain terms, simple. A competent adult facing death has a liberty interest, protected by the Fifth Amendment's Due Process Clause, in trying a drug that has passed basic safety testing in humans but has not completed the long march to approval. If the Constitution protects a person's liberty to refuse life-saving treatment, the argument went, it ought to protect the liberty to pursue it.

That is not a frivolous legal theory. A panel of the D.C. Circuit agreed with it in 2006. For a moment, it looked as if a federal court was going to say that the government could not stand between a dying person and a drug with early evidence of safety.

2007: the court that said no

Then the full court heard it. In Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach, the D.C. Circuit, sitting en banc in August 2007, reversed the panel and held that there is no fundamental constitutional right of access to experimental drugs for the terminally ill. The Supreme Court declined to take the case in early 2008.

Let me state the court's best case fairly, because it is a serious one. The en banc court reasoned that the Due Process Clause protects rights that are deeply rooted in the nation's history and tradition. It looked at the long record of federal and state regulation of drugs and found no tradition of unrestricted access to unproven medicines. It added that the government has a legitimate interest in protecting patients from unsafe drugs and in preserving the integrity of the approval process, and that Congress, not the judiciary, is the place to rebalance those interests.

I think the legal reasoning on the tradition question is arguable, but notice the last sentence. The court did not say the policy was wise. It said the remedy belonged to the legislature. That is a roadmap, and the families read it as one. If the courts will not recognize the right, then the people will legislate it, starting with the legislatures closest to them.

That is how a free republic is supposed to work. A lost lawsuit is not the end of a cause; it is a notice about which door to knock on next.

2014: the states open the door

The first state to enact a Right to Try law was Colorado, in 2014. The model language was developed with help from the Goldwater Institute, a think tank in Arizona, which turned the principle into a bill that a part-time legislator could read in an afternoon. Other states followed in a rush. By the time Congress acted, a large majority of the states had passed their own versions, and the bills moved through legislatures of both parties with unusually lopsided votes.

Pause on that for a moment. This was a federalist success story. The Constitution's design invites states to serve as laboratories, and here was a case where fifty laboratories ran the same experiment under different local conditions and reported back. The sky did not fall. No wave of poisoned patients appeared. What appeared, instead, was a body of state law and a lot of publicity about families that wanted the freedom and had been refused.

I should be candid about the limitation. A state law could not bind the FDA, and a state law could not override federal rules on the shipment of an unapproved drug across state lines. State laws were a statement of principle, a model for Congress, and a way to build a record. They were not, by themselves, a pipeline. If you want a clear illustration of why federalism is a strength and a limit at the same time, this is it.

You can read more on the logic of states as laboratories in Federalism Lets Fifty States Experiment, and the Constitution Planned It.

2017 and 2018: Washington finally notices

Congress moved in 2017. The Senate passed a federal Right to Try bill by unanimous consent that year, and the House took it up in March 2018. President Trump had spoken in favor of it in his State of the Union address of January 30, 2018. The bill reached his desk, and he signed it on May 30, 2018.

Look at the name, which is a mouthful and a tribute: the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act. Those are the names of patients and families who became public advocates for it. Congress rarely names a statute after the people it was written for, and the long title is a reminder that somebody had to stand in front of a committee and tell the truth about what waiting costs.

Here is what coverage of the signing mostly left out. The bill was not a partisan product. It passed Congress with support from members of both parties, and it was the result of years of work by families who did not much care which side of the aisle a legislator sat on. The framing in some outlets was that it was a favor to the drug industry. The industry, as it happens, has long been wary of unapproved-use programs, because a bad outcome in a desperate patient can complicate the approval of a drug it has spent a decade developing. A favor to a lobby does not usually look like a law that gives the lobby new things to worry about.

What the Act actually does

Read the statute the way you would read a contract, because that is what it is: a short list of conditions.

1. The patient must have a life-threatening disease or condition, as diagnosed by a physician, and must have exhausted approved treatment options.
2. The patient must be unable to participate in a clinical trial of the drug.
3. The drug must have completed a Phase 1 clinical trial, which is the first stage of human testing, mainly about safety. It must be under active development and in an ongoing application or trial track.
4. The patient must give written informed consent, and a physician must be in the loop.
5. The manufacturer must be willing. Nobody can be forced to supply the drug.
6. Liability is limited. Manufacturers and sponsors are shielded from liability for providing a drug in compliance with the Act, and prescribers and dispensers are protected too, except in cases of reckless or willful misconduct, gross negligence, or an intentional tort under state law.
7. Reporting follows. Manufacturers must report the use of the drug to the FDA annually.

The statute also says the FDA may not use clinical outcomes from a Right to Try use to delay or adversely affect review of the drug, unless the agency determines that using the outcome is critical to protecting public safety. That clause matters more than it looks. One of the old fears was that a company would not release a drug to a dying patient for fear that a bad result would sink its application. The law took that fear away, or at least turned it down.

How that differs from expanded access

Here is the comparison in plain words.

• Expanded access is a request to the agency. The FDA has long said that it authorizes the great majority of requests it receives, and the program remains available. But it is a permission system. The patient asks, the doctor files, a review board weighs in, and the agency has the last word.
• Right to Try is a pathway that does not run through the agency for permission. The statute names the conditions, and if they are met, the parties can proceed.

Supporters of the new law argued that the existence of a permission system, even a mostly permissive one, is the problem. The paperwork takes time. Time is the one thing a person with a fast-moving disease does not have. And a right that must be requested from the government is a privilege. I think they are correct on the principle, though I will say that the practical effect of the federal Act has been modest compared with the symbolism, since the drug still has to be supplied by a willing manufacturer and many companies have preferred to use the FDA's program.

Five objections, answered

Let me do what any honest argument has to do. Here are the critics' best points, and here is my answer to each.

1. "It gives false hope." Hope is not a harm the government gets to ration. A competent adult told the odds is entitled to decide what to do with the time that is left. The alternative, a ban on hope for the patient's own good, is paternalism at its purest, and it is hard to find a clearer example of a mistake in judgment about whose life it is.
2. "Patients could be hurt by unproven drugs." True, and the law does not hide it. Informed consent is a requirement, not a decoration. The drugs in question have cleared a first safety trial. And the patients are, by definition, those for whom approved treatment has run out. The comparison is not between an experimental drug and a safe alternative. It is between an experimental drug and no alternative.
3. "It undermines the FDA's approval process." It does not replace it. The drug is still unapproved and the FDA still regulates its sale. The Act carves out a narrow lane for a narrow group, and it explicitly protects the integrity of review by requiring reports and by preserving the agency's authority where safety is at stake. If a rule so narrow threatens the process, the process was already fragile.
4. "It exposes companies to liability, so they will not participate." The Act limits liability to reduce exactly that reluctance. The critics' premise cuts the other way: a company that is shielded is a company that might say yes.
5. "It does not give anyone anything, since the company can still refuse." That one is partly right, and I would rather concede it than defend a statute for what it does not do. The law removes a government barrier. It cannot create a supply. A person who wants the full effect should look to the next reform, which is to make the whole approval system faster and cheaper, a subject covered in How a New Drug Gets Through the FDA and Why the Trip Takes So Long.

Measure it against the Founding

The Declaration of Independence names "Life, Liberty and the pursuit of Happiness" as unalienable rights, and says that governments are instituted to secure them. A reasonable person can argue about how far that principle reaches in a courtroom. The D.C. Circuit said it does not reach a fundamental right to experimental drugs. But a republic is not limited to what its courts will enforce. The people also own the Constitution's meaning through the laws they pass, and they can decide that a free people's presumption runs toward liberty.

Think about how odd the old arrangement was. We let a person sign a form to refuse every treatment and die at home. We let a person decide whether to fight an illness with surgery or with nothing. Yet the same person, on the question of whether to take a drug that a company is willing to provide and a doctor is willing to supervise, was told the answer was "wait for permission." The inconsistency was not principled. It was inherited.

The Act does not settle the large questions about the FDA, which I take up elsewhere, including in Frances Kelsey, Thalidomide and the 1962 Law That Slowed Every New Drug on the history of the safety-and-efficacy regime and in Price Controls on Medicines Today Mean Fewer New Medicines Tomorrow on the economics of drug development. But it is a clean example of a principle working: when a rule's cost is borne by people who cannot wait for a better rule, those people are entitled to a hearing, and when the courts will not hear them, the legislature must.

The lesson for people who want to change things

You may have noticed that nobody in this story had power. A father in 2001, some families, a think tank with a model bill, and a few state representatives who were willing to carry it. Nobody got a hearing because they were important. They got a hearing because they kept showing up, telling the same plain story, and asking the same plain question: why does a dying person need your permission?

That is the lesson I would hand to anyone who thinks that the federal government is too big to move. It moves. It moves when enough states have moved first, when the ask is specific, and when the people asking are those with the most credible claim on the public's conscience. The method generalizes. It worked for this issue, and it can work for others.

There is a small sad irony in the coverage of the law too. Some stories treated it as a fringe curiosity or as a sop to a political faction. The families who pushed it did not belong to a faction. They belonged to the oldest constituency there is, the people who have run out of options, and the question they raised was one that every legislator will someday face about someone he loves.

The case in brief

1. A right to try is a narrow rule: a patient with a life-threatening illness, no approved options and no trial may try a drug that has cleared a first safety trial, if a doctor and the manufacturer agree, on written informed consent.
2. The courts did not supply it. Abigail Alliance v. von Eschenbach (D.C. Circuit, en banc, 2007) held that there is no fundamental constitutional right to experimental drugs, and pointed the argument toward legislatures.
3. The states did. Colorado went first in 2014, most states followed, and the federal Act was signed on May 30, 2018, under the names of patients who had advocated for it.
4. The critics' objections about false hope, safety and the approval process fail against the Act's actual text, which keeps consent, FDA authority and reporting, while removing one gatekeeper.
5. The deeper reform is a faster, cheaper approval system, so that fewer people have to ask for a workaround at all.

What you can do: read the text of your own state's Right to Try statute on your legislature's website, then ask your state representative whether the state's law has been updated to match the federal Act.