Should US agencies keep requiring trial data for TPOXX?

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US Health Agencies Defend Stance on TPOXX Trial Data Requirements

US health agencies have defended their decision to require human trial data to confirm the safety and efficacy of SIGA Technologies' drug TPOXX for treating monkeypox. Despite pressure to ease access, agencies maintain that rigorous data is necessary for full approval, even as they allow broader, albeit still restricted, distribution. TPOXX, also known as tecovirimat, holds approvals in the UK and Europe for both smallpox and monkeypox, granted under pathways that acknowledge data limitations in certain circumstances.
Defense of Current Approval Standards

Authors from the Food and Drug Administration (FDA), Centers for Disease Control and Prevention (CDC), and National Institutes of Health (NIH) published an article in the New England Journal of Medicine to explain their position. They stated that the NIH is planning a US-based randomised clinical trial, the data from which will be crucial for determining whether TPOXX gains full US approval for monkeypox. This stance reflects the FDA's established standards for ensuring drug safety, which, while robust, have been noted by some experts as potentially hindering access in urgent situations.
International Approvals and Pathways

Globally, TPOXX has received approval for monkeypox in both the UK and Europe. The European Union, for instance, granted its approval under an "exceptional circumstances" pathway. This route permits marketing authorisation when comprehensive data cannot be obtained even after a drug is authorised. The European Medicines Agency, in making its determination, anticipated that side effects in infected individuals would mirror those observed in healthy subjects and concluded that the drug's benefits outweighed its risks.
Current Distribution and Prescribing Process

While awaiting full approval, TPOXX is being distributed in the US under a "compassionate use" programme. This process involves doctors requesting the drug from the CDC or their health department and enrolling each patient in a study. As of July 22, 223 individuals had received the drug through this channel. On July 21, the CDC and FDA modified the procedure to allow physicians to prescribe TPOXX before trial paperwork is finalized, though hospital institutional review board approval remains a requirement for each dose.
Challenges and Future Trial Plans

The current system, while improved, is still described as burdensome by infectious disease experts. Dr. Karen Krueger of Northwestern Medicine noted that while the process is "doable," it presents significant challenges compared to standard drug prescriptions, with additional paperwork required post-patient visit and for follow-up consultations. Northwestern has treated approximately 20 monkeypox patients with TPOXX and is slated to be a trial site for the upcoming NIH study. SIGA Technologies estimates that a 500-patient, placebo-controlled clinical trial in the United States would likely be necessary for full approval, a process that could be completed within months if patient numbers remain high. The company is also working to initiate trials in the UK, Canada, and other parts of Europe.

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Should US agencies keep requiring trial data for TPOXX?

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