The FDA over the weekend issued its first emergency authorization for COVID-19 therapies. But absent any solid data on the drugs, medical and legal experts questioned the move.
The “emergency use authorization” (EUA) from the FDA applies to two drugs, hydroxychloroquine sulfate and chloroquine phosphate. President Donald Trump and a smattering of doctors around the world have boosted the drugs’ potential for weeks despite limited clinical data supporting them.
Versions of the drugs have been used for years to treat malaria, lupus and rheumatoid arthritis. In fact, Americans with those condition have experienced shortages of the tried-and-true treatments for their conditions as doctors rush to fill prescriptions for the drugs — and sometimes hoard them for themselves.
In a press release Sunday, the HHS office for Preparedness and Response, which manages the U.S. Strategic National Stockpile, emphasized that the emergency order would “ease supply pressures for the drug” for patients who’ve long used it for existing treatments.
Still, the drugs aren’t backed by proven results from the large-scale, controlled studies on COVID-19 patients that would normally be required before FDA-approved use.
Armed with little more than anecdotes of successful treatment and very limited trials, doctors across the country have legally prescribed chloroquine and hydroxychloroquine in an “off-label” capacity — meaning for something other then their approved use — and some hospitals have begun using the unproven drugs in cases severe enough to justify taking a risk.
New York state recently began a large-scale trial of the drugs, and the World Health Organization included chloroquine and hydroxychloroquine in a global study it recently announced of several potential therapies.
Sunday’s EUA allowed the government’s Biomedical Advanced Research and Development Authority, or BARDA, to distribute millions of doses of the drugs — which were recently donated to the government by Bayer and Novartis’ generics division, Sandoz — to public health officials for this off-label use.
“The EUA is specific to how BARDA distributes the drug from the strategic national stockpile,” Holly Fernandez Lynch, a professor of bioethics and law at the University of Pennsylvania, told TPM. Without the EUA, BARDA would be violating federal law if it distributed the drugs to treat COVID patients.
And while the FDA said the emergency-authorized treatments should be limited to patients for whom “a clinical trial is not available, or participation is not feasible,” the language was “wishy washy” and difficult to enforce, Fernandez Lynch said. It could also have an unintended consequence: Decreasing the amount of potential data available on the drugs’ effectiveness.
Doctors for patients who are otherwise eligible to participate in trials, Fernandez Lynch said, “could just say ‘Oh yeah, there’s a trial, but I don’t want you to participate in that, I’m just going to give you the drug.'”
“That’s really, really the wrong thing to do, because it will mean that the physician will be in no better position treating their 150th COVID patient than they were treating their first COVID patient, because they will just be relying on anecdotes, not evidence.”
Steve Joffe, a pediatric oncologist and bioethicist, wrote on Twitter Monday that the EUA “and widespread use of an unproven drug is going to make it devilishly hard to figure out whether that drug, as well as every other promising drug, actually works.”
As things stand, there’s little proof of the drugs’ effectiveness against COVID-19.
The FDA’s chief scientist, in her letter of authorization to BARDA, simply said that based on the “totality of scientific evidence available to the FDA,” it was reasonable to believe the drugs may be effective, and that their known and potential benefits outweigh their known and potential risks.
Later, the letter noted that guidelines published by several other countries recommended using the drugs against COVID-19 based on “limited in-vitro and anecdotal clinical data.” But it did not note studies to the contrary.
“It is within FDA’s authority to decide whether the standard is met,” Fernandez Lynch said. “But they have not made a very convincing case.”
As the agency’s former acting chief scientist Luciana Borio wrote Sunday, there is a “total lack of scientific evidence that chloroquine/hydroxychloroquine are beneficial in the treatment of COVID-19.”
“EUA is supposed to be issued when the evidence indicates that benefits outweigh the risks,” Borio added.
Zachary Brennan, editor of the online journal of the Regulatory Affairs Professionals Society, told TPM in an email that the drugs joined a tiny handful of therapeutics ever approved for emergency use by the FDA — alongside Doxycycline, as an anthrax treatment, and freeze-dried plasma, used to control hemorrhage from battlefield trauma.
“Will be curious to track if any hospitals receive donations from the national stockpile but their doctors decline to use them [because] of the scant evidence,” Brennan wrote.
One man hasn’t spent much time dwelling on the scant evidence. Trump has boosted the drugs relentlessly in recent weeks, speculating recently that a combination of hydroxychloroquine and azithromycin could turn out to be “one of the biggest game changers in the history of medicine.”
Last week, an Arizona man died after consuming chloroquine phosphate in the form of a fish tank additive. His wife subsequently told NBC News that the couple had seen the President and others promote the product as “pretty much a cure.”