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 designated tecovirimat, also called Tpoxx, an “experimental drug,” which they say means it cannot be released from the national strategic 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 detailed patient information.
It doesn’t have to be, experts say: there is no law preventing federal officials from change these rules and make the drug more widely available.
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 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 given to patients with monkeypox. As a treatment for smallpox, its use against monkeypox is considered experimental. But vaccines developed for smallpox were assumed to be effective against both diseases. Why not the treatment?
Experts say the FDA restrictions are a policy choice that can be quickly changed.
“The bureaucracy to access Tpoxx is excessive given the crisis the United States faces with monkeypox,” said Larry O. Gostin, 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 considerable flexibility to use scientific assessments to ensure those in need get the drugs 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 failed to take an extra step that would have allowed the FDA to grant emergency use authorizations for vaccines and treatments, as the agency did during the coronavirus pandemic. coronavirus.
In an article published Wednesday in the New England Journal of Medicine, federal health officials defended his decision treat tecovirimat as an investigational drug.
While acknowledging that the animal data was promising and 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. on people with monkeypox”.
What 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 has mainly been found in parts of West and Central Africa, but in recent weeks it has spread to dozens of countries and infected tens of thousands of people, overwhelmingly men with sex with men. July 23, the World Health Organization has declared monkeypox a global health emergency.
“At this time, it is not known if or how well this drug works for patients with monkeypox,” said Kristen Nordlund, 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. “It will ultimately help us understand who will benefit the most, what the real benefits are and what potential risks there might be.”
Restrictions on tecovirimat were even more complicated early in the outbreak, and — after numerous complaints from doctors — the CDC relax some rules. But the system remains cumbersome.
Doctors who want to prescribe the drug must first sign up to become investigators in a clinical trial, sending in resumes and informed consent forms signed by patients with monkeypox – a process that is “intensive labor-intensive and virtually impossible” for most physicians, said Lynda Dee, executive director of AIDS Action Baltimore.
“If it wasn’t such a serious emergency, it would make for a really good bureaucratic joke,” she added. They said “Unfortunately the joke is once again on the gay community.”
The rules are so complex that some patients have had to educate their doctors on 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 suffered damage to his face and rectum.
The nurse practitioner he saw had no idea how to treat him, Mr Thompson said. Based on a friend’s experiences, I 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 of paperwork to get it.
When Mr. Thompson said he wanted her to note in her chart that she did not want to prescribe him the drug, she told him the doctor would have to decide. The doctor finally called him on Sunday, July 31, nearly two weeks after he first reached out.
“She was like, ‘I contacted the CDC, I contacted the health department. I contacted multiple doctors in different states, I contacted multiple pharmacists in different states,'” he said .
At that time, it didn’t matter. His wounds were healing, the pain had eased and he was on the mend.
Dr. Stacy Lane, founder and medical director of a network of seven clinics in Pennsylvania and Ohio serving the LGBTQ population, said many of her patients with monkeypox were turned away by three or four doctors who couldn’t do in the face of convoluted federal rules regarding tecovirimat.
In early July, Dr. Lane saw a patient with eye lesions characteristic of monkeypox infection, which can lead to blindness. She tried to acquire tecovirimat, but the Pennsylvania Department of Health insisted that the patient first have an eye exam.
It was a Friday afternoon, and Dr. Lane didn’t want the patient waiting in a crowded emergency room, so she rushed to find an eye doctor instead of waiting for the exam to be performed. “in a very controlled manner in an outpatient office” the following Monday.
Pennsylvania has since dropped the requirement for a diagnosis, but Ohio has not.
“It doesn’t need to be restrained the way it is restrained,” Dr. Lane said of tecovirimat. “It would be ideal if we could get this Tpoxx treatment from local pharmacies like we can get anything else, especially as this thing keeps exploding.”
Dr. Lane and others said they understand there are still questions about the safety and effectiveness of the drug in humans, but noted that it has already been shown to be safe in humans and had been approved by the FDA.
“If a drug is already FDA-approved, any doctor can prescribe it as off-label because the FDA is not allowed to regulate the practice of medicine,” said Cornell Director Dr. Jay Varma. Center for Pandemic Prevention and Response.
“It is a policy choice of the CDC to avoid publishing or recommending an off-label drug,” he added.
Many patients and physicians, including Dr. Lane, have reported that tecovirimat appears to melt away skin lesions within 24 hours. No serious adverse events were 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 there needs to be a lot more data collection and post-marketing surveillance and everything else,” said Dr. James Lawler, director of the University of Nebraska’s Global Center for Health Security. “But you can do it in a way that doesn’t create barriers.”