As the monkeypox outbreak grows in the United States, many patients are struggling to access TPOXX, the only known treatment—and some experts argue federal health officials should loosen restrictions on the treatment to make it more widely available, Apoorva Mandavilli writes for the New York Times.

Why the US response to monkeypox is drawing comparisons to Covid-19

Providers and patients struggle to access TPOXX

TPOXX, also known as tecovirimat, was first approved by FDA in 2018 to treat smallpox. And, similarly to how smallpox vaccines are being used to prevent monkeypox, many health experts believe TPOXX could also be used to treat monkeypox. In fact, TPOXX was approved based on efficacy data in primates who were infected with monkeypox.

However, TPOXX is currently classified as an "investigational drug" against monkeypox and cannot be released from the strategic national stockpile without an extensive bureaucratic process. According to Lynda Dee, the executive director of AIDS Action Baltimore, the process to access TPOXX is "labor intensive and virtually impossible" for most physicians.

To prescribe the treatment, physicians must first sign up to be investigators in a clinical trial, which includes sending in resumes and informed consent forms from monkeypox patients, Mandavilli writes.

Because of this burdensome process, relatively few monkeypox patients have received the treatment—even as many patients and physicians have attested to its effectiveness in treating skin lesions that develop from the virus.

"This doesn't need to be held back the way it's being held back," said Stacy Lane, the founder and medical director of a network of clinics that serve the LGBTQ population in Pennsylvania and Ohio. "It would be ideal if we can get this Tpoxx treatment at the local pharmacies just like we can get anything else, especially as this thing continues to explode."

The debate over TPOXX's investigational status

According to Mandavilli, federal health officials last week published an article in the New England Journal defending their decision to classify TPOXX as an investigational drug.

Although the officials acknowledged that efficacy data in animals was promising and that the drug seemed safe in human patients, they said without large clinical trials, "we will not know whether tecovirimat [TPOXX] would benefit, harm or have no effect on people with monkeypox disease."

"At this time, it's unclear if or how well this drug works for monkeypox patients," said Kristen Nordlund, a CDC spokesperson.

According to Nordlund, keeping TPOXX an investigational drug "ensures we have data from patients who use this drug," which will "ultimately help us understand who will benefit most, what the true benefits are and potential risks there might be."

However, several health experts argue that since TPOXX has been shown to be safe in humans and is already approved by FDA, physicians should be able to prescribe it more broadly.

"If a drug is already F.D.A.-approved, any physician can prescribe it as off-label because F.D.A. is not permitted to regulate the practice of medicine," said Jay Varma, the director of the Cornell Center for Pandemic Prevention and Response. "It is a C.D.C. policy choice to avoid releasing or recommending a drug off-label."

"I understand that there needs to be a lot more data-gathering and post-marketing surveillance and everything else," said 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."

In addition, some experts argue FDA could quickly alter its restrictions to allow for emergency use authorization of TPOXX, much as the agency did for Covid-19 vaccines and treatments during the pandemic.

"The bureaucracy of gaining access to Tpoxx is excessive given the crisis the U.S. is facing with monkeypox," said Larry Gostin, an expert on public health law and the 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 medication that can help them." (Mandavilli, New York Times, 8/6)