The nation’s top health officials say they’re rushing to make sure Americans can get widespread access to the experimental drug remdesivir after a study showed that it shortens the recovery time for some coronavirus patients.
The push to give the drug the regulatory approval it needs for broader distribution comes a day after the government’s top infectious disease expert, Anthony Fauci, called the drug “an important proof of concept” and compared it to the discovery of the earliest HIV treatments in the 1980s. Investors sent U.S. stocks surging after the news was announced.
But there are still major questions about how well remdesivir works, when the government might make it available to the public, and whether drugmaker Gilead can keep pace with rising global demand for the drug. Here’s what to watch as more data is uncovered.
This isn’t a cure
The results that made headlines Wednesday came from a trial sponsored by the National Institute of Allergy and Infectious Diseases, which Fauci runs.
The study of 1,063 patients revealed that those who received remdesivir had a 31 percent faster time to recovery than those who received a placebo — 11 days versus 15 days, on average. The findings also suggested that patients given remdesivir were slightly less likely to die. But that effect was so small that you would need to treat 28 patients with remdesivir to save one life, said Vincent Rajkumar, a physician at the Mayo Clinic.
A leak this month of early data from the NIAID trial had suggested more significant benefits for those taking remdesivir. But the findings released Wednesday tempered expectations.
Public health experts have also warned for months that finding a slam-dunk coronavirus drug will not be what ends the pandemic. Rather, widespread testing and the development of an effective vaccine — which could still be more than a year away — will rein in the virus and curb hospitalizations.
“This is not a magic cure,” said Jeffrey Gold, a doctor and chancellor of the University of Nebraska Medical Center, the lead researcher in the NIAID remdesivir study. But “if you marry that to more testing, contact tracing and the ability to identify earlier patients … all that adds into a ray of light and seeing our way through this.”
We still haven’t seen the actual data
One of the biggest head-scratchers is the way in which the results of the NIAID trial were announced.
Gilead, which makes the drug, put out a press release Wednesday morning saying the NIAID trial met its primary endpoint (even though the company was not involved in running the trial). Several hours later, Fauci disclosed some of the data to reporters during an Oval Office appearance. Later in the day, NIAID issued a press release echoing what Fauci said and adding a few extra details.
But no one outside of NIAID, Gilead and the hospitals where the trial was conducted have seen the actual data. Some scientists and health experts are wondering why there wasn’t a simultaneous data release alongside the announcements.
“I understand the pressure, but if there is enough certainty to announce it to the nation, then they should be able to post a pre-print that details the methods and results,” said Harlan Krumholz, professor of medicine at Yale School of Medicine. “Now we have heard results without any ability to evaluate them. I am hopeful that more complete information and actual data will be available soon.”
Administering it is complicated
Remdesivir isn’t a pill. Instead, it’s an intravenous infusion that patients have to take anywhere from five to 10 days. Gilead says data it released Wednesday from its own trial, which did not include a control arm, suggests five days could be just as effective as longer dosing. But dispensing the medicine for any length of time still requires skilled doctors and nurses, IV supplies and capacity in hospitals and clinics to treat patients.
Those logistical hurdles make it likely that remdesivir will go to severely ill and hospitalized patients first, even though the company has said it may be more effective when given early in the course of infection. “The IV delivery is likely to relegate the treatment to severe hospitalized patients who paradoxically may benefit least from a drug that reduces” the amount of virus in the blood, RBC Capital analyst Brian Abrahams wrote in March.
The bottom line, Rajkumar said, is that the drug’s impact will be limited by the difficulty of dispensing it.
It’s not yet available for wide use
NIAID may have run the latest remdesivir trial, but it is the Food and Drug Administration that will decide when the drug will reach the market.
There are a few options if regulators want to get the drug to people quickly. FDA can issue an emergency use authorization, as it has done for several Covid-19 tests and the still-unproven drug hydroxychloroquine. That is a bar below approval and can come with limitations about when drugs can be used. “An EUA can be crafted as broadly or narrowly as the situation warrants,” said Steven Grossman, president of the policy consultancy HPS Group.
The agency could also issue an accelerated approval, which is rare but not unprecedented for a drug backed by a small amount of clinical data. An accelerated approval would signal FDA’s confidence in the results it has seen, but the process could take longer than granting an EUA.
FDA is working at “lightning speed” to assess the data, Commissioner Stephen Hahn told Bloomberg on Thursday. “There will be a lot of factors that go into all the regulatory decisions."
Manufacturing it is tricky
Because remdesivir is an infusion and not a pill, the process by which Gilead and its supply chain partners make the treatment is more complicated and difficult to set up.
Gilead CEO Daniel O’Day has said the company began ramping up manufacturing in January. He’s promised to donate the company’s existing supply — 1.5 million doses, or enough to treat about 140,000 people by the end of May — for use in clinical trials or compassionate use programs. But those doses may end up going further than previously thought, because the results of Gilead’s trial released Wednesday show that a 5-day treatment course was just as effective as a 10-day treatment course.
O’Day told STAT News in an interview that Gilead ordered enough raw materials in January to scale up manufacturing of remdesivir through the end of the year.
Abrahams estimates that Gilead could have sufficient remdesivir supplies for 1.8 million patients by the end of 2020.