President Donald Trump, battling COVID-19 over the weekend, extolled the drugs he has received, including one experimental drug not available to the general public, as “miracles coming down from God.”
The president’s praise of Regeneron’s monoclonal antibody treatment and his apparently improved health will likely drive demand for a drug that remains out of reach for most Americans.
“It is almost certain that the use of the monoclonal antibodies for POTUS' treatment will increase interest in others in receiving the drug, and potentially other promising [monoclonal antibodies],” said Jesse Goodman, a former Food and Drug Administration chief scientist and deputy commissioner, in an email.
“I’m quite sure Regeneron is swamped,” said Alison Bateman-House, a bioethicist and professor at the New York University Grossman School of Medicine.
The unprecedented nature of a president taking an experimental drug poses many ethical and legal questions. The drug has limited data behind it, and has not been approved or even authorized on an emergency basis by the FDA.
Drug companies usually test treatments on patients before they have been approved through a clinical trial. But Trump received the drug through an expanded access program, otherwise known as “compassionate use.”
Bateman-House said drug companies should take one of three approaches to compassionate use: granting access to the drug to everyone who asks; to nobody; or to a select population of people under clearly stated circumstances.
After news broke that Trump had received the treatment, Regeneron CEO Leonard Schleifer told CNBC last week that fewer than 10 people had received the drug for free this way.
A spokesperson citing patient privacy concerns declined to share details on how or why this handful of people received it, their home states or how they got the company’s attention.
The company would only say the drug is given in “rare and exceptional” cases.
“Given limited supply of the drug and need for continued clinical trials, this is not intended to be the main mechanism of access,” Regeneron spokeswoman Alexandra Bowie said in an email. “Clinical trials are actively enrolling (not only for our drug but for many others in this space), and many more people are able to access investigational treatments through that route.”
But experts anticipate that the president’s enthusiasm will drive many people to seek monoclonal antibody treatments through the same pathway he did. Participating in a clinical trial risks being a part of the control group that doesn't get the drug.
“I have no doubt people will look at this and ask, ‘Why can’t I get it?’” said Kenneth Kaitin, director of the Tufts Center for the Study of Drug Development. “Why would I enroll in a clinical trial if I may get a placebo?”
Under a 2016 law, companies must publicly list the criteria by which they make drugs available through compassionate use. Trump does not appear to meet those criteria, and why the company decided to make the drug available to him and others remains murky.
Regeneron’s general guidelines state that access to its unapproved drugs are intended for the most dire cases. The program "is intended for patients with serious or life-threatening conditions who do not have any viable or available treatment options, and are unable to participate in ongoing clinical trials.”
Schleifer said in the CNBC interview Monday that treating the president like a participant in a double-blind clinical trial would be risky because he could be given a placebo.
“Giving it to [Trump] or to others who might not be able or don’t qualify for clinical trials is the right use of compassionate use. That’s for small numbers of people, for these exceptions,” Schleifer said.
Schleifer and Trump are personally acquainted; Schleifer is a member of Trump’s private country club in Westchester County, N.Y.
While it may be understandable that the president would get special consideration, experts worry about a system with vastly different rules for the rich and powerful.
“It gives the impression that we do clinical trials just to satisfy the regulators. We save drugs for the important people while average people do clinical trials,” said Vinay Prasad, a University of California San Francisco professor of medicine.
Bateman-House said researchers recruit clinical trial participants by promising that a placebo is no worse than a drug until it’s proven to be safe and effective. She described this episode as “massively unsettling” to that pact.
“They didn’t even consider Trump for a clinical trial. That shows inequity, and it’s not in keeping with the regulations,” she said. “It delegitimizes clinical trials in the eyes of the public, and that impact could last longer than the life of any one person.”
The company also proactively offered the drug to Vice President Joe Biden, who is not currently diagnosed with COVID-19 and also does not meet the company’s publicly stated requirements for its compassionate use program.
Questions about illegal marketing
Some experts worry that in discussing Trump’s treatment on television and offering the drug to Biden, the company transgressed the law that prevents drug companies from marketing unproven treatments.
When asked on CNBC whether Trump's use of the unproven drug benefits the company, Schleifer said Regeneron was simply trying to "help an individual win this battle."
“We think giving that antibody makes a lot of sense because it does what the immune system is supposed to do but hasn’t quite done yet," Schleifer said. "And that’s what we hope happened with the president.”
Schleifer added a note of caution: “You’re asking me did we help him? I’d like to think so. It’s impossible to know with one patient.”
Asked whether Schleifer's comments could amount to marketing an unproven drug, Bateman-House said that while she’s not a lawyer, “if it walks like a duck and quacks like a duck, it’s probably a duck.”
On the other hand, Michael Carome, an FDA expert at the consumer group Public Citizen, said the executive’s statements are not out of the ordinary in a pandemic that has given rise to rampant speculation about unapproved treatments. These treatments are usually touted in press releases before the public sees any evidence.
“It’s absolutely consistent with a pattern of drug companies seeking to promote the products they’re developing by putting a positive spin on the early data and benefiting from that in terms of their stock prices,” Carome said.
Regeneron's Bowie said the company's executives "have gone on news shows when invited and explained what antibodies are, how our investigational antibody cocktail operates against the virus and what the recently announced clinical data were. As physician-scientists who founded and run a biotechnology company, they see scientific education as an important part of their role."
Some worry Trump’s access to the drug could jeopardize its availability for most people in the long term. By generating demand before FDA can make a decision about the drug, the president has made it more difficult for researchers to recruit clinical trial participants.
This could be especially true if the FDA issues an emergency use authorization for it, which experts speculate is likely given the low bar for authorization and Trump’s enthusiasm.
“The bigger issue is definitely enrollment in clinical trials,” Kaitin said. “We have a population justifiably afraid and desperate for something that works.”
Even after any FDA authorization, the drug is likely to be pricey and face shortages. Experts warn the manufacturing capacity for monoclonal antibody treatments is limited.
Another of Trump’s treatments, the antiviral drug remdesivir, faced shortages for months after an EUA was issued.
A five-day course of remdesivir typically costs $2,340, according to drugmaker Gilead Sciences. Trump also received dexamethasone, an old and widely available steroid, which costs $10 to $20 for 30 tablets, according to the GoodRx website.
Carome worries Trump could create false hope.
“It’s the type of conduct we’ve seen since the early days of the pandemic, when Trump was touting hydroxychloroquine despite a lack of evidence. He gloms onto things and runs with them,” he said. “He’s downplaying the seriousness of COVID and suggesting that if you get it, don’t worry, these drugs will work for everyone. That’s simply not true.”