The only available drug for monkey pox is so scarce that it has been administered to only a fraction of the nearly 7,000 patients in the United States.
Health officials have appointed tecovirimat, also known as Tpoxx, the ‘investigative drug’, which they say means it cannot be released from strategic national stockpiles without a series of intricate bureaucratic steps. However, most physicians do not have the time or resources to complete the required 27-page application or provide patient details.
Experts say it doesn’t have to be that way: there is no law preventing federal officials from changing these laws and making the drug more widely available.
The Food and Drug Administration approved tecovirimat as a smallpox treatment in 2018, based on human safety data and primate efficacy data – who were actually infected with monkey pox for the purposes of the study. The so-called animal rule allows an agency to approve drugs when it would be unethical to test them in humans.
Until now, tecovirimat has been rarely administered to patients with monkey pox. As a treatment for smallpox, its use against monkey pox is considered experimental. But it is assumed that vaccines designed for smallpox are effective against both diseases. Why not treatment?
Experts say the FDA’s restrictions are a policy choice that can be changed quickly.
“The bureaucracy in accessing Tpoxx is excessive given the crisis the United States is dealing with monkey pox,” 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 provide drugs in need that can help them,” he added.
The Department of Health and Human Services declared monkey pox a national health emergency on Thursday. But Secretary Xavier Becerra has not taken the extra step that would allow the FDA to authorize emergency 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.
Acknowledging that animal data are promising and the drug seems safe in healthy patients, they wrote that without large clinical trials, “we won’t know if tecovirimat will benefit, harm or have no effect on people with monkey pox.”
“It is not clear at the moment whether or how well this drug works in monkey pox patients,” said Kristen Nordlund, spokeswoman for the Center for Disease Control and Prevention.
Providing Tpoxx only as an investigational drug “ensures that we have data from patients who use the drug,” she said. “This will ultimately help us understand who will benefit most, what are the real benefits and what the potential risks may be.”
What You Should Know About Monkey Pox
What is monkey pox? Monkey pox is a virus similar to smallpox, but the symptoms are less severe. It was discovered in 1958 following an outbreak in monkeys kept for research. The virus has been found mostly in parts of Central and West Africa, but has spread to dozens of countries in recent weeks and has infected tens of thousands of people, mostly men who have sex with men. On July 23, the World Health Organization declared monkey pox a global health emergency.
The restrictions on tecovirimate were even more complicated at the start of the epidemic and – after many complaints from doctors – the CDC loosened some rules. But the system remains inconvenient.
Doctors who wish to prescribe a drug must first register to become investigators in a clinical trial by submitting resumes and informed consent forms signed by monkey pox patients – a process that is “laborious and virtually impossible” for most physicians, said Lynda Dee. executive director of AIDS Action Baltimore.
“If it weren’t for such a dire emergency, it would be a very good bureaucratic joke,” said Mrs. Dee. “Unfortunately, the joke is again about 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 cook from Atlanta, first developed headache and body aches on July 17, and two days later he had lesions on his face and rectum.
Mr. Thompson said the nurse he saw had no idea how to treat him. Based on his 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 had heard of tecovirimat but told him it would take hours to get the paperwork.
When Mr. Thompson said she wanted her to write on his record that she did not want to prescribe him, she told him that the doctor would have to make a decision. The doctor finally called him on Sunday, July 31, almost two weeks after he first contacted him.
“She said,“ I contacted the CDC, contacted the health department. I have contacted many doctors in different states, I have contacted many pharmacists in different states, ”he said.
Until then, it didn’t matter. His lesions healed, the pain subsided, and he was healing.
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 monkey pox patients were dismissed by three or four doctors who couldn’t deal with the intricate federal tecovirimat laws.
In early July, Dr. Lane met with a patient with an eye injury characteristic of monkey pox that can lead to blindness. She tried to acquire tecovirimat, but the Pennsylvania Health Department insisted that the patient undergo an eye exam first.
It was a Friday afternoon and Dr. Lane did not want the patient to wait in the crowded emergency room, so she tried to find an ophthalmologist instead of waiting for the examination “in a very controlled manner in the outpatient clinic.” ” next Monday.
Pennsylvania has since dropped the diagnostic requirement, but Ohio has not.
“It doesn’t have to be held back the way it is stopped,” said Dr. Lane on tecovirimat. “It would be perfect if we could get this Tpoxx therapy from local pharmacies as we can get anything else, especially since this thing continues to explode.”
Dr. Lane and others said they understood that there were still questions about the drug’s safety and effectiveness in humans, but noted that it has already been shown to be safe in humans and has been approved by the FDA
“If a drug is already approved by the FDA, any doctor can prescribe it as unregistered because the FDA cannot regulate medical practice,” said Dr. Jay Varma, director of the Cornell Center for Pandemic Prevention and Response.
“It is a CDC policy choice to avoid releasing or recommending a drug off-label,” he added.
Many patients and doctors, including Dr. Lane, have reported that tecovirimat appears to dissolve 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 no justification for restricting access.
“I understand that much more data collection, post-market surveillance and everything else is needed,” 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 creates no barriers.”