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 known as Tpoxx, as a “research drug,” which they say means it can’t be released from the strategic national stockpile without a series of convoluted bureaucratic steps. However, most physicians do not have the time or resources to complete the required 27-page application or provide the detailed patient information.
It doesn’t have to be this way, experts say: There’s no law stopping federal officials from changing those rules and making the drug more widely available.
The Food and Drug Administration approved tecovirimate as a smallpox treatment in 2018 based on safety data in humans and efficacy data in primates — who were actually infected with monkeypox for the purposes of the study. The so-called animal rule allows the agency to approve drugs when testing on humans would be unethical.
Until the current outbreak, tecovirimate was rarely given to monkeypox patients. As a smallpox treatment, its use against monkeypox is considered experimental. However, vaccines developed for smallpox were believed to be effective against both diseases. Why not the treatment?
Experts say the FDA’s restrictions are a political decision that can be changed quickly.
“The bureaucracy to access Tpoxx is excessive given the monkeypox crisis the U.S. is facing,” 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 reviews to ensure those in need get the medicines that can help them,” he added.
The Ministry of Health declared monkeypox a national health emergency on Thursday. But Minister Xavier Becerra did not take an additional step that would have allowed the FDA to issue emergency use authorizations for 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 tecovirimate as an investigational drug.
While acknowledging that animal data is promising and that the drug appears to be safe in healthy patients, they wrote that “without large clinical trials, we will not know whether tecovirimate would benefit, harm, or have no effect in people with monkeypox.” .
What you should know about 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 been found mostly 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 time, it’s unclear whether or how well this drug works in monkeypox patients,” said Kristen Nordlund, a spokeswoman for the Centers for Disease Control and Prevention.
Making Tpoxx available only as an investigational drug “ensures we have data from patients using this drug,” she said. “This will ultimately help us understand who benefits most, what the real benefits are, and what the potential risks might be.”
Restrictions on tecovirimate were even more complicated early in the outbreak, and — after many complaints from doctors — the CDC relaxed some of the rules. But the system remains cumbersome.
Physicians wishing to prescribe the drug must first register to become investigators in a clinical trial, submit resumes, and submit informed consent forms signed by monkeypox patients — a process that is “labor-intensive and virtually impossible” for most physicians, Lynda Dee said. the executive director of AIDS Action Baltimore.
“If this weren’t such a bad emergency, it would be a very good bureaucratic joke,” said Ms Dee. “Unfortunately, once again, the joke lies with the gay community.”
The rules are so complex that some patients have had to educate their doctors about the process.
Adam Thompson, a 38-year-old Atlanta chef, first developed a headache and body aches on July 17 and two days later developed lesions on his face and rectum.
The nurse he saw had no idea how to treat him, Mr Thompson said. Based on a 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 had heard about Tecovirimat but told him it would be hours before they got any paperwork.
When Mr. Thompson said he wanted her to put a note on his chart that she didn’t want to prescribe the drug, she told him the doctor had to decide. The doctor finally called him on Sunday, July 31, nearly two weeks after he first called.
“She said, ‘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.
Until then it didn’t matter. His lesions healed, the pain had subsided and he was on the mend.
dr Stacy Lane, the founder and medical director of a network of seven clinics in Pennsylvania and Ohio that serves the LGBTQ population, said many of her monkeypox patients have been turned away by three or four doctors who couldn’t handle the tortuous federal regulations on Tecovirimat.
In early July, Dr. Lane a patient with eye lesions suggestive of monkeypox infection that can lead to blindness. She attempted to purchase 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 to keep the patient waiting in a crowded emergency room, so instead of waiting for the exam to be performed “in a very controlled manner in an ambulance,” she scrambled to find an eye doctor “The following Monday.”
Pennsylvania has since dropped the requirement for a diagnosis, but Ohio has not.
“This doesn’t need to be held back the way it is held back,” said Dr. Lane about tecovirimat. “It would be ideal if we could get this Tpoxx treatment at local pharmacies just like we can get anything else, especially as this thing keeps 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 had been approved by the FDA
“If a drug is already approved by the FDA, any doctor can prescribe it off-label because the FDA is not allowed to regulate the practice of the medicine,” said Dr. Jay Varma, the director of the Cornell Center for Pandemic Prevention and Response.
“It is a CDC decision to avoid approving or recommending an off-label drug,” he added.
Many patients and doctors, including Dr. Lane, have reported that tecovirimate 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, there is little reason to restrict access, experts say.
“I understand there needs to be a lot more data collection and post-marketing monitoring and everything else,” said Dr. James Lawler, director of the University of Nebraska’s Global Center for Health Security. “But it’s done in a way that doesn’t create barriers.”