President Trump announced Sunday that he had helped break through a regulatory “logjam” to grant emergency authorization of convalescent plasma to treat covid-19, a “powerful therapy” that he claimed “had an incredible rate of success,” despite the fact that his own scientists are calling for more studies to definitively show it works.
The announcement, at a news conference where Trump was flanked by Food and Drug Administration commissioner Stephen Hahn and Health and Human Services Secretary Alex Azar, drew criticism from physicians and scientists, who said their statements misled the public by overstating the evidence behind a therapy that shows promise but still needs to be rigorously tested.
“I watched this in horror,” said Eric Topol, an influential physician and scientist and director of the Scripps Research Translational Institute. “These are basically just exploratory analyses that don’t prove anything. It’s just extraordinary to declare this as a breakthrough … All this does is jeopardize ever getting the truth.”
The Infectious Diseases Society of America released a statement noting that while there are “some positive signals that convalescent plasma can be helpful in treating individuals with covid-19,” the society believed its benefits needed to be demonstrated in clinical trials that randomly assign patients to receive either plasma or a placebo before it is authorized for wider use.
The Sunday briefing came on the eve of the Republican National Convention where Trump hopes to resurrect his flagging popularity, which has nosedived over his handling of the pandemic. He has put extraordinary pressure on federal agencies to test and approve treatments and a vaccine against the novel coronavirus, which has killed more than 170,000 Americans.
It also landed a day after the president without evidence accused the FDA of impeding enrollment in clinical trials for coronavirus vaccines and treatments for political reasons. “The deep state, or whoever, over at the FDA is making it very difficult for drug companies to get people in order to test the vaccines and therapeutics,” Trump said on Twitter on Saturday. “Obviously, they are hoping to delay the answer until after November 3rd.” He tagged Hahn in the tweet.
An administration official who spoke on the condition of anonymity because they weren’t authorized to discuss the issue said that the announcement follows two weeks of “insane fights.” The official said that Trump held more conversations on the issue Saturday. Some scientists at the National Institutes of Health have argued that the efficacy data wasn’t strong enough — but NIH does not control FDA decisions, the FDA pointed out last week.
At least one individual who has been following the issue closely said he had expected the announcement to come from the FDA this week in any case, but closer to the middle of the week.
At the briefing, Hahn struck a more measured tone than Trump, stating that convalescent therapy, in which the yellowish liquid portion of blood is taken from recovered patients and transfused into ill people, had met the standard for emergency authorization — a lower standard than full approval — and showed “promising efficacy.”
But Azar said this was a “major advance” and the kind of result pharmaceutical company executives dream about, even though the authorization states that more studies are needed, including medical trials that test the treatment against a placebo. The authorization specifically says plasma “should not be considered a new standard of care for the treatment of patients.”
A notice to patients who receive the treatment says that it "might be effective in improving the likelihood of you recovering from the disease.”
Hahn stressed his agency’s independence on a call with reporters on Sunday, saying “the expert scientists at CBER [the FDA’s Center for Biologics Evaluation and Research] made this decision solely on the data and nothing else,”
Still, he was criticized on Twitter and elsewhere for not defending the FDA in Trump’s presence. “Hard to watch @SteveFDA stand by as the president attacked the integrity” of FDA employees, said Josh Sharfstein, principal deputy FDA commissioner during the Obama administration. He described the news conference as a “campaign style press announcement” that didn’t provide adequate details. “Worse may be in store.”
As Trump, Azar and Hahn left the White House briefing room, reporters yelled asking Hahn if there was pressure on him to authorize convalescent plasma. The three kept on walking.
While the treatment may have met the FDA’s standard for authorization — that it may be effective and appears safe — outside scientists said that administration officials who touted a 35-percent reduction in death risks to non-elderly patients who were breathing on their own were overstating the conclusions that could be drawn from suggestive studies without comparison groups.
They also worried that the strong statements that the treatment is remarkably effective will make patients less likely to enroll in gold standard medical trials, where they are randomly assigned to receive the experimental intervention or a placebo, which are needed to draw more definitive conclusions.
“The urgency of the crisis has elided with a false sense we should skip over rigorous studies of interventions because we don’t have enough time,” said Peter Bach, director of Memorial Sloan Kettering’s Center for Health Policy and Outcomes.
Bach and others have been critical that the United States has given the treatment to more than 70,000 patients through an expanded access program facilitated by the FDA and the Mayo Clinic, instead of testing it rigorously in a clinical trial. Many have pointed out that if even a small fraction of those patients had gone into such a trial, physicians and scientists would have a much better idea of how well it worked.
Convalescent plasma is a century-old treatment that was deployed against the novel coronavirus on the theory that virus-fighting antibodies in the plasma could be helpful against a new pathogen. Because of the sound medical rationale for using it and the lack of treatments for patients fighting covid-19, researchers came together to make it available and have so far treated at least 70,000 patients.
Former FDA commissioner Scott Gottlieb said the blood product — derived from patients who have survived covid-19 — clearly meets the criteria for an emergency use authorization.
He said it was “probably of incremental benefit” for covid-19 patients, but “we have to be careful not to overstate the results.”
Carlos del Rio, executive associate dean of the Emory School of Medicine, said that it was an exaggeration to call plasma a “breakthrough.” He called plasma an “interesting strategy” and said the data so far was a “nice hint” that it could be helpful, but stressed that it was “not going to win the game.”
“The problem is, the President, in my mind, has lost total credibility because of what he’s done with hydroxychloroquine. He’s touted so many things that don’t work,” del Rio said. “The reality is what we have today to treat covid is extremely limited.”
But Arturo Casadevall, chair of molecular microbiology and immunology at the Johns Hopkins Bloomberg School of Public Health, expressed support for an emergency use authorization earlier this week, saying it could make it somewhat easier to access the treatment. The lack of an authorization “makes it harder, particularly in hospitals that don’t have the resources to do all the paperwork that is needed to be part of the expanded access program” facilitated by the Mayo Clinic, he said. “Simply because they’re so busy, the hospitals are stretched.”
Kate Fry, chief executive of America’s Blood Centers, which represents blood banks, said her organization was supportive of the emergency authorization. The expanded access program was never designed to provide plasma supply long term. “It really has gotten so large that it has sort of gone past its intended purpose,” she said, adding that the FDA authorization would ease the burden for clinicians and physicians.
Until recently, Trump had praised the FDA for moving quickly on coronavirus treatments and vaccines.
Besides undermining his political standing, the coronavirus has repeatedly upended Trump’s desire for a lavish political coronation this week, as a dispute with North Carolina’s Democratic governor over virus protocols prompted Republicans to shift their convention festivities to Jacksonville, Fla., only to have those plans called off because of the worsening pandemic conditions there. Though some convention activities will still take place in Charlotte — the initial site of the RNC — the president will deliver his formal acceptance speech from the Rose Garden on Thursday night.
Trump is scheduled to travel to North Carolina on Monday. One speaker after another at last week’s Democratic National Convention, topped by Joe Biden himself, hammered Trump and his administration for its management of the pandemic and stressed that the former vice president was prepared to handle the health and economic crisis.
Correction: A previous version of the story incorrectly described the patient information on plasma. The patient information says it “might be effective in improving the likelihood of you recovering from the disease.”
