The only drug available to treat monkeypox is so difficult to obtain that only a small fraction of the nearly 7,000 patients in the United States receive it.
Health officials have designated tecovirimat, also referred to as Tpoxx, as a “research drug,” which they say means it cannot be released from strategic national stockpiles without a series of convoluted 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 shouldn’t be like this, experts say: there is no law prohibiting federal officials changing these rules and make medicine more accessible.
The Food and Drug Administration approved tecovirimat as a treatment for smallpox in 2018 based on safety data in humans and efficacy data in primates, which, for the purposes of the study, were indeed infected with monkeypox. The so-called animal rule allows the agency to approve drugs when testing them on humans would be unethical.
Prior to 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 treatment?
Experts say FDA restrictions are a political choice that can be changed quickly.
“The bureaucracy involved in gaining access to Tpoxx is overwhelming given the crisis the US is facing with monkeypox,” said Larry O. Gostin, public health law expert and director of the O’National and Global Health Institute. Neal at Georgetown University. .
“The law gives the agency considerable flexibility in using scientific assessments to ensure that those in need receive the drugs that can help them,” he added.
Department of Health and Human Services on Thursday monkeypox declared health emergency. But Secretary of State Xavier Becerra has not taken additional steps to allow the FDA to grant emergency use approvals 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 their decision consider tecovirimat as an experimental drug.
While acknowledging that the animal data were promising and that the drug seemed safe in healthy patients, they wrote that without large clinical trials, “we will not know whether tecovimat will benefit, harm, or have no effect in humans with monkey disease.” .
What you need 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 has mostly been 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. July 23 The World Health Organization has declared monkeypox a global health emergency..
“It’s unclear at this time if this drug works in monkeypox patients, and how well,” 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 on patients who use this drug,” she said. “This will ultimately help us understand who will benefit the most, what are the real benefits and the possible risks.”
Restrictions on tecovirimat were even more difficult at the start of the outbreak, and – after numerous complaints from doctors – the CDC loosened some rules. But the system remains cumbersome.
Doctors who want to prescribe the drug must first register to become investigators in clinical trials by submitting resumes and informed consent forms signed by monkeypox patients, a process that is “time-consuming and next to impossible” for most doctors, Linda Dee said. executive director of AIDS Action Baltimore.
“If it wasn’t such a terrible emergency, it would be a very good bureaucratic joke,” she said. Dee said. “Unfortunately, the joke touches the gay community again.”
The rules are so complicated that some patients have had to teach their doctors the process.
Adam Thompson, a 38-year-old chef from Atlanta, had his first headache and body aches on July 17, and two days later developed rashes on his face and rectum.
The nurse practitioner he saw had no idea how to treat him. Thompson said. Based on his friend’s experience, 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 told him it would take hours to get the paperwork.
When Mr. Thompson said he wanted her to mark on his chart that she was unwilling to prescribe him medication, she told him that the doctor would make the decision. The doctor finally called him on Sunday, July 31, almost two weeks after he first contacted him.
“She said, ‘I contacted the CDC, I contacted the health department. I contacted several doctors in different states, I contacted several pharmacists in different states,” he said.
By then it didn’t matter. His wounds have healed, the pain subsided, and he is on the mend.
Dr. Stacey Lane, founder and medical director of a network of seven clinics in Pennsylvania and Ohio serving the LGBT community, said many of her monkeypox patients were turned away by three or four doctors who couldn’t handle twisty federal regulations regarding tecovirimat.
At the beginning of July, Dr. Lane saw a patient with eye lesions consistent with monkeypox infection, which can lead to blindness. She tried to get tecovirimat, but the Pennsylvania Department of Health insisted that the patient first have an eye examination.
It was Friday afternoon and dr. Lane didn’t want a patient waiting in a crowded emergency room, so she struggled to find an ophthalmologist instead of waiting for the exam to be done “in a very controlled manner in an outpatient’s office” the following Monday.
Pennsylvania has since dropped the diagnosis, but Ohio has not.
“It doesn’t need to be contained the way it is contained,” says the doctor. Lane talked about tecovirimate. “It would be ideal if we could get this smallpox remedy from local pharmacies like everything else, especially since this thing keeps exploding.”
Dr. Lane and others said they understand there are still questions about the drug’s safety and efficacy in humans, but noted that it has already been proven safe in humans and has been approved by the FDA.
“If a drug is already approved by the FDA, any doctor can prescribe it off-label because the FDA has no right to regulate medical practice,” the doctor said. Jay Varma, director of the Cornell Center for Pandemic Prevention and Response.
“This is a political choice by the CDC to avoid releasing or recommending the drug off-label,” he added.
Many patients and physicians, including D. Lane, have reported that tecovirimat appears to dissolve skin lesions within 24 hours. No serious adverse events were reported.
Given the known and emerging evidence of the drug’s efficacy in patients and the urgency of the outbreak, experts say there is little reason to restrict access.
“I understand that a lot more data collection, post-marketing surveillance and everything else is needed,” the doctor said. 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.”