President Trump announced on Sunday that the U.S. Food and Drug Administration has allowed for the emergency use of an unproven treatment known as convalescent plasma therapy for COVID-19 patients, stating it can reduce the chances of dying in some by 35 percent. But scientists were quick to cast doubt on the claim, with one calling it "outrageous."
Convalescent plasma therapy involves extracting blood from people who have been infected with the coronavirus, and removing components including red and white blood cells and platelets to leave behind a straw-coloured liquid which contains antibodies. It is hoped that injecting this liquid into COVID-19 patients could help them recover.
Trump said during a press conference regarding the FDA's emergency use authorization (EUA) that a study on the treatment launched by the Mayo Clinic and the federal government had "proven to reduce mortality by 35 percent."
Health Secretary Alex Azar told the briefing: "We saw about a 35 percent better survival in the patients who benefited most from the treatment, which were patients under 80 who were not on artificial respiration.
"I just want to emphasize this point, because I don't want you to gloss over this number. We dream in drug development of something like a 35 percent mortality reduction. This is a major advance in the treatment of patients. This is a major advance."
The EUA did not indicate where the 35 percent figure originated from.
The USCovidPlasma.org website for the program states: "COVID-19 convalescent plasma has not yet been demonstrated to provide clinical benefit in patients affected by this disease. It's not known if this treatment will or will not help those with COVID-19 or if it will have any harmful effects, but this is one of the only treatments that we have at present."
In May, the FDA deemed convalescent plasma an "investigational product" so it could be used in clinical trials and administered to seriously ill patients.
In June, the Mayo Clinic released a safety report involving 20,000 people that suggested the treatment was safe. This was followed by a study released as a pre-print on the server medRxiv earlier this month which indicated that giving convalescent plasma to patients with severe COVID-19 within three days of them being diagnosed could lower their risk of death.
Submitting data as a pre-print means it hasn't been through the rigorous peer review process required to publish in scientific journals. Releasing data this way enables scientists to prompt debate on a topic, and it is useful in a fast-moving situation like a pandemic.
The study did not have a placebo group and was not a randomized clinical trial, the gold standard for testing treatments are safe and effective, leading experts to express skepticism about the data and the rationale behind issuing an EUA.
Last week, top federal health officials called for the FDA to put its EUA for the treatment on hold, arguing the data was too weak, The New York Times first reported.
Eric Topol, founder and director of the Scripps Research Translational Institute, tweeted after the EUA was issued that to claim based on a pre-print, observational (not randomized control trial) study that the therapy improved mortality rates by 35 percent was "outrageous."
"There's no evidence to support any survival benefit," he wrote.
On Sunday, Dr. Thomas M. File president of the Infectious Diseases Society of America said in a statement that while the data shows "some positive signals" that it could be helpful in treating COVID-19 patients, particularly those in the early stages of the disease, "we lack the randomized controlled trial data we need to better understand its utility in COVID-19 treatment."
"For this reason, IDSA supports the continued collection of data in randomized clinical trials to better understand the benefits of convalescent plasma treatment before authorizing its wider use in patients with COVID-19," he said.
Duncan Young, a professor of intensive care medicine at the University of Oxford, U.K., told Newsweek: "The biggest problem with an EUA for convalescent plasma therapy is that we will not know if it works, as the EUA essentially causes clinicians to give the treatment rather than entering patients in clinical trials. There may also be a rationing problem as it may be in short supply."
Ashish Jha, director of the Harvard Global Health Institute, tweeted that "sadlyy" the FDA "took [a] big hit to credibility in the manner in which they announced EUA for plasma."
"To be clear, FDA has the authority & data might even meet the criteria. But manner of role out: hyperbole, mischaracterization of evidence by @SteveFDA [FDA Commissioner Stephen Hahn] very disappointing."
He said: "Bottom line for EUA is this: if you end up hospitalized, your doctor won't know if plasma is helpful or not. That's why we do science. And EUA sets us back. Makes it harder to do science right."
Echoing their concerns, the Journal of the American Medical Association tweeted an explainer article on the treatment, alongside the message: "People who've had #COVID19 develop anti-#SARSCoV2 antibodies in plasma, the yellow liquid portion of blood. This convalescent plasma can be transfused into COVID19 patients, but the effectiveness of the treatment hasn't yet been shown in RCTs [randomized control trials]."
A spokesperson for NHS Blood and Transplant, which manages blood donation services in England and transplant services across the U.K., said in a statement that the "observational studies coming from America are promising" but "they are not conclusive.
"Randomised control trials are the gold standard for determining the effectiveness of a new treatment."
A White House spokesperson told Newsweek the FDA commissioner addressed the concerns raised in this article on Sunday. Newsweek has contacted the FDA, HHS, and Mayo Clinic for comment.
This article has been updated with comment from Duncan Young and a White House spokesperson.