The only drug available to treat monkeypox is so difficult to access that only a fraction of the nearly 7,000 patients in the United States have received it.
Health officials have called tecovirimat, also known as Tpoxx, an “investigative drug,” which they say means it cannot be taken from the strategic national stockpile without a series of complicated bureaucratic steps. But most doctors don’t have the time or resources to complete the required 27-page application or provide the detailed patient information.
It doesn’t have to be, experts say: There’s no law that prohibits federal officials from changing those rules and making the drug more widely available.
The Food and Drug Administration approved tecovirimat as a smallpox treatment in 2018, based on safety data in humans and efficacy data in primates — who were actually infected with monkeypox before the trial. The so-called animal rule allows the agency to approve drugs when testing them in humans would be unethical.
Until the current outbreak, tecovirimat was rarely given to monkeypox patients. As a treatment for smallpox, its use against monkeypox is considered experimental. But vaccines developed for smallpox are believed to be effective against both diseases. Why not the treatment?
Experts say the FDA’s restrictions are a policy choice that can be changed quickly.
“The bureaucracy to access Tpoxx is excessive given the monkey pox crisis facing the US,” said Larry O. Gostin, a public health law expert and director of the O’Neill Institute for National and Global Health Law. at Georgetown University.
“The law gives the agency significant flexibility to use scientific assessments to ensure people in need are getting the medication that can help them,” he added.
The Department of Health and Human Services declared monkeypox a national public health emergency on Thursday. But Secretary Xavier Becerra took no additional step that would have allowed the FDA to authorize emergency use of vaccines and treatments, as the agency did during the coronavirus pandemic.
In an article published Wednesday in the New England Journal of Medicine, federal health officials defended their decision to treat tecovirimat as an investigational drug.
While acknowledging that animal data showed promise and that the drug appeared safe in healthy patients, they wrote that, without large clinical trials, “we won’t know whether tecovirimat benefits, harms, or has no effect on people with monkeypox disease.”
What you need to know about the Monkeypox virus?
What is monkey pox? 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 mainly found in parts of Central and West 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.
“At this point, it’s unclear if and how well this drug works for monkeypox patients,” said Kristen Nordlund, a spokeswoman for the Centers for Disease Control and Prevention.
By only offering Tpoxx as an investigational drug, “we make sure we have records of patients taking this drug,” she said. “This will ultimately help us understand who will benefit the most, what the real benefits are and possible risks.”
The restrictions on tecovirimat were even more complicated at the start of the outbreak, and — after much complaining from doctors — the CDC relaxed some rules. But the system remains cumbersome.
Physicians wishing to prescribe the drug must first apply to become investigators in a clinical trial, by submitting resumes and informed consent forms signed by monkeypox patients — a process that is “labor-intensive and virtually impossible” for most physicians. said Lynda Dee, the executive director of AIDS Action Baltimore.
“If this wasn’t such a dire emergency, it would be a really good bureaucratic joke,” Ms Dee said. “Unfortunately, the joke is again about the gay community.”
The rules are so complex that some patients had to inform their doctors about the process.
Adam Thompson, a 38-year-old cook in 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, said Mr. Thompson. Based on a friend’s experiences, he convinced her to prescribe hydrocortisone suppositories and gabapentin, a drug used to treat nerve pain.
They didn’t help. The nurse practitioner had heard of tecovirimat, but she told him it would take hours of paperwork to obtain it.
When Mr. Thompson said he wanted her to write in his file that she was unwilling to prescribe the drug, she told him that the doctor had to make a decision. The doctor finally called him on Sunday, July 31, nearly two weeks after he first contacted him.
“She said, ‘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,’ he said.
By then it didn’t matter. His lesions were healing, the pain was gone, and he was on the mend.
dr. Stacy Lane, the founder and medical director of a network of seven clinics in Pennsylvania and Ohio for the LGBTQ population, said many of her monkeypox patients had been turned away by three or four doctors who couldn’t handle the tortuous federal rules regarding tecovirimat. .
In early July, Dr. Lane, a patient with eye lesions characteristic of monkeypox infection, which can lead to blindness. She tried to get tecovirimat, but the Pennsylvania health department insisted that the patient get an eye exam first.
It was a Friday afternoon and Dr. Lane didn’t want the patient to have to wait in a crowded emergency room, so she rushed to find an ophthalmologist instead of waiting for the exam to be done “in a very controlled manner in an outpatient clinic.” the following Monday.
Pennsylvania has since dropped the requirement for a diagnosis, but Ohio has not.
“This doesn’t have to be held back the way it’s held back,” said Dr. Lane about tecovirimat. “It would be ideal if we could get this Tpoxx treatment at the local pharmacies just like we can get anything else, especially as this thing keeps on exploding.”
dr. Lane and others said they understood there were still questions about the drug’s safety and effectiveness in humans, but noted that it had already been shown to be safe in humans and approved by the FDA.
“If a drug is already FDA-approved, any doctor can prescribe it as off-label because the FDA isn’t allowed to regulate medical practice,” says Dr. Jay Varma, the director of the Cornell Center for Pandemic Prevention and Response.
“It is a CDC policy choice to prevent a drug from being released or recommended off-label,” he added.
Many patients and doctors, including Dr. Lane, have reported that tecovirimat appears to melt away skin lesions within 24 hours. No serious side effects have been reported.
Given the well-known and emerging evidence of the drug’s effectiveness in patients and the urgency of the outbreak, experts say there’s little reason to restrict access.
“I understand there’s a lot more data to collect and post-marketing surveillance and everything else,” said Dr. James Lawler, a director of the University of Nebraska’s Global Center for Health Security. “But you can do that in a way that doesn’t create barriers.”