The only drug available to treat monkeypox is so hard to come by that only a fraction of the nearly 7,000 patients in the United States have received it.
Health officials have designated tecovirimat, also called Tpoxx, as an “investigational drug,” which they say means it can’t be released from the strategic national stockpile without a series of intricate bureaucratic steps. But most doctors don’t have the time or resources to fill out the required 27-page application or provide detailed patient information.
It doesn’t have to be this way, experts say: There is no law that prevents federal officials changing those rules and make the drug more available.
The Food and Drug Administration approved tecovirimat as a smallpox treatment in 2018, based on safety data in people and efficacy data in primates, which, for the purposes of the trial, were actually infected with monkeypox. The so-called animal rule allows the agency to approve drugs when testing them on people would be unethical.
Until the current outbreak, tecovirimat was rarely administered to patients with monkeypox. As a smallpox treatment, its use against monkeypox is considered experimental. But vaccines developed for smallpox are supposed to be effective against both diseases. Why not the treatment?
Experts say the FDA restrictions are a policy option that can be changed quickly.
“The bureaucracy to gain access to Tpoxx is excessive given the crisis the US is facing with monkeypox,” said Larry O. Gostin, public health law expert and director of the O’Neill Institute for Health Law. National and Global at Georgetown University. .
“The law gives the agency considerable flexibility to use scientific evaluations to ensure that those who need it get the medicine that can help them,” he added.
The Department of Health and Human Services on Thursday declared monkeypox a national health emergency. But Secretary Xavier Becerra did not take an additional step that would have allowed the FDA to grant emergency use authorizations for vaccines and treatments, as the agency did during the coronavirus pandemic.
In an article published in the New England Journal of Medicine on Wednesday, federal health officials defended his decision to treat tecovirimat as an investigational drug.
While acknowledging that the animal data was promising and that the drug appeared safe in healthy patients, they wrote that without large clinical trials, “we will not know whether tecovirimat would benefit, harm, or have no effect in people with the disease.” monkey pox”.
“At this time, it’s not clear if this drug works or how well it works for monkeypox patients,” said Kristen Nordlund, a spokeswoman for the Centers for Disease Control and Prevention.
Providing Tpoxx only as an investigational drug “ensures that we have data from patients using this drug,” he said. “Ultimately, this will help us understand who will benefit most, what the true benefits are, and what potential risks there might be.”
What to know about the monkeypox virus
What is monkeypox? Monkeypox is a virus similar to smallpox, but the symptoms are less severe. It was discovered in 1958, after outbreaks occurred in monkeys kept for research. The virus was found mainly in parts of central and western Africa, but in recent weeks it has spread to dozens of countries and infected tens of thousands of people, mostly men who have sex with men. On July 23, The World Health Organization declared monkeypox a global health emergency..
Tecovirimat restrictions were even more complicated at the beginning of the outbreak and, after many complaints from doctors, the CDC loosened some of the rules. But the system is still cumbersome.
Physicians who want to prescribe the drug must first sign up to become investigators in a clinical trial by submitting resumes and signed informed consent forms from monkeypox patients, a process that is “laborious and virtually impossible” for most doctors. said Lynda Dee, the executive director of AIDS Action Baltimore.
“If this wasn’t such a dire emergency, it would be a very good bureaucratic joke,” Ms. Dee said. “Unfortunately, the joke is once again on the gay community.”
The rules are so complex that some patients have needed to educate their doctors about the process.
Adam Thompson, a 38-year-old cook from Atlanta, first developed a headache and body aches on July 17, and two days later he had lesions on his face and rectum.
The nurse practitioner he saw had no idea how to treat him, Thompson said. Based on the experiences of a friend of hers, she convinced her to prescribe hydrocortisone suppositories and gabapentin, a medication used to treat nerve pain.
They didn’t help. The nurse practitioner had heard of tecovirimat, but she told him that it would take her hours of red tape to get it.
When Mr. Thompson said he wanted her to put on his chart that she was unwilling to prescribe the medication, she told him the doctor would have to decide. The doctor finally called him on Sunday, July 31, nearly two weeks after she first contacted him.
“He told me, ‘I’ve contacted the CDC, I’ve contacted the health department. I’ve contacted multiple doctors in different states, I’ve contacted multiple pharmacists in different states,’” she said.
By then, it no longer mattered. Her injuries were healing, the pain had subsided, and she was recovering.
Dr. Stacy Lane, founder and medical director of a network of seven clinics in Pennsylvania and Ohio serving the LGBTQ population, said three or four doctors turned away many of her monkeypox patients because they couldn’t deal with the torturous federal rules regarding tecovirimat.
In early July, Dr. Lane saw a patient with eye lesions distinctive of monkeypox infection, which can lead to blindness. She tried to get tecovirimat, but the Pennsylvania Department of Health insisted that the patient get an eye exam first.
It was a Friday afternoon, and Dr. Lane didn’t want the patient to wait in a crowded ER, so she rushed to find an ophthalmologist rather than wait for the exam “in a very controlled manner in a outpatient clinic”. ” the following Monday.
Pennsylvania has since dropped the requirement for a diagnosis, but Ohio has not.
“This doesn’t need to be retained the way it’s being retained,” Dr. Lane said of tecovirimat. “It would be great if we could get this Tpoxx treatment from local pharmacies just like we can get anything else, especially as this continues to explode.”
Dr. Lane and others said they understood there were still questions about the drug’s safety and effectiveness in people, but noted that it had already been shown to be safe in people and had been approved by the FDA.
“If a drug is already approved by the FDA, any doctor can prescribe it as off-label because the FDA cannot regulate the practice of medicine,” said Dr. Jay Varma, director of the Center for Pandemic Prevention and Response. from Cornell.
“It is a CDC policy choice to prevent publication or recommendation of a drug off-label,” he added.
Many patients and doctors, including Dr. Lane, have reported that tecovirimat appears to melt skin lesions within 24 hours. No serious adverse events have been reported.
Given the known and emerging evidence of the drug’s effectiveness in patients and the urgency of the outbreak, experts say there is little reason to restrict access.
“I understand that there needs to be a lot more data collection and post-market surveillance and everything else,” said Dr. James Lawler, director of the Global Center for Health Security at the University of Nebraska. “But you can do it in a way that doesn’t create barriers.”