"Compassionate Use" and the Law on Unapproved COVID-19 Drugs
President Trump has touted experimental drugs approved for other uses, like malaria and lupus, as a potential treatment for COVID-19. What is the law when it comes to accessing unapproved medicines in a time of crisis? The Food and Drug Administration (FDA) in the U.S. regulates the process of new drug approvals and allows “compassionate use” in certain circumstances under a program called Expanded Access. Professor Glenn Cohen explains the FDA’s process and how it applies to experimental COVID-19 treatments.
Additional Resources
FDA's Expanded Access Program
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Expanded access may be appropriate when all the following apply:
- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
(Quatations in various part from FDA website "Expanded Access")
Federal Right to Try Law
The Right to Try Act, or the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, was signed into law May 30, 2018. This law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.
An eligible patient is a patient who has:
- Been diagnosed with a life-threatening disease or condition
- Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
- And has provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician
An eligible investigational drug is an investigational drug:
- For which a Phase 1 clinical trial has been completed
- That has not been approved or licensed by the FDA for any use
- For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA
- Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA
(Quatations in various part from FDA website "Right to Try")
The Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law was founded to promote interdisciplinary analysis and legal scholarship in these fields.
"Compassionate Use" and the Law on Unapproved COVID-19 Drugs Brief Transcript
You may have heard a lot in the news about the president and others talking about drugs they think might be helpful or could help or might work for people who are in crisis due to the COVID pandemic. "Well, there was this great drug I'm hearing about, that there's not a lot of evidence for, there's no clinical trials, but I think it'll work wonders. I have a feeling or whatever." I want to talk a little about the law that governs the use of those druge. My name is Glenn Cohen, I'm a professor at Harvard Law School and I'm an expert at the intersection of law and medicine.
Now first off, I want to remind you there's a lot of risks involved in taking a drug that has not been cleared by FDA or by others for a particular use. There's even greater risk that people might hear these messages and really become confused. There' s a tragic story of somebody who consumed a form of a drug used for fish in a koi pond and then ended up dead, essentially because of this. So we want to just be very careful about our health in these instances. But you might legitimately wonder what's the law on this. So FDA, the Food and Drug Administration, has the primary authority in approving drugs in this country. It reviews three phases of clinical trials aimed at showing that the drug is safe and efficacious and has a reasonable side effect as to a particular indication and a particular use. The long process is expensive and many drugs don't make it out of this process, to be honest, but it is a process that is time tested and honored in this country.
Nonetheless, FDA realizes that there are instances where people need something quicker, their are lives are at risk, they are terminally ill, for example, and it has a process called "expanded access," where for an individual, for a small group of people, or even widespread use, it can approve a use of a drug that has not go through these three phases of approval already. There is also, in many states now in the federal government, an even more capacious way of getting access to these drugs called "right to try laws." They shift the onus and regulatory burden from FDA largely to doctors and the states instead.
Okay, well beyond unapproved drugs, there are also drugs that have been approved for a particular use that someone wants to use them for a different use. Does FDA control that? Short answer: no. FDA has said, "That's the practice of medicine, we don't do the practice of medicine.” So you as a doctor, if you want to use a drug that has been approved for usage for X and you want to use it for Y, you may do so, and FDA can't say you are violating the Food, Drug, and Cosmetic Act. Instead, you may face some tort liability if you do, but you're allowed to do that as a physician as part of the practice of medicine.
What happens if a drug company tries to promote a drug for an unapproved use, including in the COVID pandemic situation? Well, here it gets a little bit tricky. Historically, FDA has taking the position that it does have authority over the promotion of off-label uses, but that authority has been challenged and in a number of cases, first in the District Court, then in the Second Circuit, FDA has lost and it's been told they would violate the First Amendment for it to claim a violation of its statute for the promotion of off-label uses. They never pushed the issue to the U.S. Supreme Court, so it's a bit dicey whether you could promote off-label or not and get away with it under the Constitution. Look for this as a future issue that a future Supreme Court might tackle.
Well, I hope I've taught you a little bit about the use of drugs off-label and unapproved uses of drugs for the COVID pandemic. I'm Glenn Cohen, and I hope you enjoyed watching this.