Alternate monkeypox treatment caught in regulatory delays

• Calls for easier access are intensifying with the monkeypox outbreak now covering all but two states and accounting for 6,326 confirmed cases.
• While the CDC waived some requirements, doctors and clinics still must submit more than a dozen pages of forms, conduct follow-up visits and collect patient data.
• "Some clinics don't have the administrative capacity to fill out paperwork so they're referring patients to other providers ... it's a highly variable situation across the United States, and this should not be the case," David Harvey, director of the National Coalition of STD Directors, told Axios.

What we're hearing: The San Francisco AIDS Foundation, which has a 10,000-person waitlist for monkeypox vaccines, just began administering TPOXX treatment after weeks of patient requests.

• The administrative burden of paperwork, follow-up visits and getting patient consent had previously been too much for to take on with resources already stretched, Tyler TerMeer, the foundation's CEO, told reporters this week.
• The access problems are coming on top of missed diagnoses, inappropriate treatments and other problems for patients who are in pain and trying to navigate an unprepared and short-staffed medical system, Kaiser Health News reported.

Context: TPOXX is among the countermeasures the government keeps on hand to protect against potential biological attacks.

• It falls under the CDC's regulations for investigational new products. To obtain such a drug for serious or life-threatening conditions, providers have to take steps like scheduling follow-up visits, taking photographs and making patients keep a diary.
• Even after the CDC eliminated some of these steps, the time needed to care for patients becomes more onerous as more people seek treatment, Marshall Glesby, associate chief of the infectious diseases division at Weill Cornell Medicine, told Axios.

Driving the news: Some advocacy organizations and providers are asking the CDC to release TPOXX from the national stockpile, freeing it from the regulatory process.

• "We're saying completely remove the hurdle," Garrett Wilkinson, policy officer at Partners in Health, told Axios.
• The treatment could be more easily accessed if the Biden administration declared a public health emergency or even significant potential for one, and the FDA grants emergency use authorization, like it did for COVID-19 vaccines.
• But even if the administration did so, "FDA would need to consider the circumstances and appropriateness of an EUA for a particular medical countermeasure and make a determination that the criteria for issuance of an EUA have been met," an FDA spokesperson wrote in an email.

Where things stand: TPOXX is the lead product of SIGA Technologies, which has contracted with the federal government for the last 20 years and tested the drug on animals infected with other orthopoxviruses, including monkeypox.

• In 2018, the FDA granted TPOXX approval for use against smallpox.
• Since the monkeypox outbreak began early this summer, SIGA has also struck agreements with Canada and countries in Europe and Asia.
• The company is restocking expiring doses in the U.S. stockpile with 363,000 new ones and has the capability to replenish that supply further.
• "We don't know where the outbreak (will go), but right now we think we're able to respond" to increased demand, Chief Executive Phillip Gomez told Axios.

Yes, but: Some clinicians say TPOXX still should be put through clinical trials before it's widely used for monkeypox, especially since there is no data on the drug's use in humans to date.

• SIGA has partnered on some human-based trials in Africa, but those results have not been published.
• Currently, some patients treated with TPOXX are seeing improvement, but Glesby said it is too soon to draw sweeping conclusions because the natural course of infection is still murky.
• A placebo-controlled clinical trial would answer that question, Glesby said, adding that it's important that people with serious cases have immediate access to treatment, as well.
• The only TPOXX clinical trial in the United States evaluating it for treatment of orthopox viruses is limited to military personnel.

The bottom line: The way monkeypox is proliferating is making some rethink risks and benefits, especially for populations at highest risk.

The FDA’s apparent reasoning, Wilkinson said, is that "the risk of giving gay and trans people a drug that has already been deemed entirely safe by the FDA outweighs the risk of delaying by days, if not weeks, access to this antiviral."

Editor's note: The story has been updated to clarify that Wilkinson's remark about risks and benefits was referring to the FDA's apparent reasoning.