On Monday, 37-year-old Leila Saedian learned from her local Walgreens that her refill for Plaquenil — the brand name for the drug hydroxychloroquine, which she uses to treat her lupus — would be delayed indefinitely. After calling 10 pharmacies in her Los Angeles neighborhood, she finally obtained 10 days’ worth of Plaquenil; normally, her refills would include enough for three months. Now, she has no choice but to keep calling local pharmacies, until she finds one that can replenish her supply. She worries about experiencing a flare-up — which can cause so much pain and swelling, she can barely get out of bed — without any Plaquenil to alleviate it. “I’ve been on this drug for 18 years,” she tells Mic. “Never have I seen this problem.”
President Trump lauded chloroquine and hydroxychloroquine as potential treatments for COVID-19 last week. According to the Centers for Disease Control and Prevention, chloroquine is used to treat and prevent malaria, while hydroxychloroquine is used to treat lupus, rheumatoid arthritis, and porphyria cutanea tarda, a rare condition that can cause the skin to become thin or fragile. Since Trump heaped praise on these drugs, there have been reports of increased demand for them, leading to shortages for those, like Saedian, who rely on them. People have begun taking them, and products containing the same active ingredients, on their own, without a doctor’s supervision; an Arizona man died earlier this week after taking after taking a form of chloroquine phosphate used to treat parasites in fish to stave off COVID-19, NBC News reports. This is exactly why drugs need to be thoroughly tested before we use them to treat a specific disease.
In a press briefing last Thursday, President Trump said that chloroquine and hydroxychloroquine have shown “very, very encouraging early results” for treating COVID-19, and claimed that they’d already been FDA-approved for the disease, according to the White House website.
Where'd his assertion come from, exactly? Chloroquine and hydroxychloroquine prevented the novel coronavirus from infecting lab-grown cells in one study, and a very modest-sized preliminary clinical trial of hydroxychloroquine in France, which Trump referred to in a tweet, has yielded encouraging results, STAT reports. But as CNN points out, the FDA hasn’t approved them, or any drug for that matter, to treat COVID-19. Indeed, the agency maintains that “there are no FDA-approved therapeutics or drugs to treat, cure or prevent COVID-19.” FDA Commissioner Stephen Hahn said, at the Thursday briefing, that chloroquine would go through testing in "a large, pragmatic clinical trial," per CNN.
All of us would love nothing more than a treatment or vaccine to be made available immediately, but there’s a highly rigorous method to the madness to ensure any new intervention is used such that it not only works, but doesn't seriously harm people.
Physicians can legally prescribe chloroquine or hydroxychloroquine for what’s called “off-label” use to treat COVID-19, per CNN — but that doesn’t mean the FDA has deemed it safe or effective for this use, the agency explains on its website.
Indeed, although the the FDA has approved chloroquine and hydroxychloroquine to treat the conditions described above, these drugs still have to undergo the FDA’s entire vetting process for the agency to approve it as a COVID-19 treatment. For approval of this new use, the FDA requires what’s called a New Drug Application, or NDA, according to the ASCO Post. An NDA includes data from clinical trials of the drug that test its safety and efficacy in patients, along with a bunch of other documentation, per the FDA website.
Designing a clinical trial is an exhaustive process that involves reviewing existing data on a drug to formulate research questions and objectives, as well as develop a study protocol, the FDA explains. Researchers then decide who can enroll, ways to limit bias, how the data will be collected, and much more. The “gold standard” of clinical trials is a randomized controlled trial, in which one group of patients gets the treatment and the other doesn’t, as decided by chance.
Scientists typically carry out clinical trials in four phases. In a phase I trial, they test a treatment in a small amount of patients to assess its safety and figure out a safe dosage range, per the FDA. They also note any side effects. In a phase II trial, they test the treatment in a larger group of patients to further assess its safety and whether it’s actually effective.
Next, in a phase III trial, they test it in even more patients to confirm its effectiveness, compare it to widely used treatments, and gather data that’ll be used to administer it safely. After the FDA approves the treatment, a phase IV trial sheds additional light on the best context in which to use it, as well as its benefits and risks. The process, as you can imagine, can't be rushed.
It’s very difficult to get a drug approved by the FDA for a new use, even if it’s already been approved to treat other medical conditions.
It’s not clear how long it would take for chloroquine or hydroxychloroquine to undergo this process. The French study that Trump tweeted about was promising, but it was tiny: It included only 36 patients and wasn’t stringently designed, STAT points out. The outlet also cites data from the Biotechnology Industry Organization about how infectious disease drugs that make it to phase 2 trials end up on pharmacy shelves only 27.5% of the time. Even when it's fast-tracked, a single drug trial can last weeks or months, according to the Associated Press.
In other words, it’s very difficult to get a drug approved by the FDA for a new use, even if it’s already been approved to treat other medical conditions. All of us would love nothing more than a treatment or vaccine to be made available immediately, but there’s a highly rigorous method to the madness to ensure any new intervention is used such that it not only works, but doesn't seriously harm people.
And spewing misinformation about how chloroquine and hydroxychloroquine are hugely promising, and already FDA-approved for treating COVID-19 is downright reckless. Saedian believes Trump’s enthusiasm about hydroxychloroquine was not only premature, but misleading. She also points out that he hasn’t mentioned its side effects, which can include retinal damage in rare cases. And now she’s scrambling to find enough of the drug to keep her debilitating flare-ups at bay. “His praise of it… completely disregarded what the medication is actually used for.”