FDA facing huge pressure as Ebola worsens

The Food and Drug Administration (FDA) told the US Congress yesterday it is working hard to clarify the framework for bringing experimental therapies and vaccines to patients affected by Ebola, although the agency is facing “incredible demands” as the epidemic takes hold.

Luciano Borio, director of the FDA’s Office of Counterterrorism and Emerging Threats, told a congressional hearing yesterday that it is trying to develop a flexible way to evaluate the safety and efficacy of new drugs and vaccines as quickly as possible.

Measures already underway include an acceleration in the review of Investigational New Drug (IND) applications, which are required by US law for FDA-regulated trials to proceed, said Borio, noting that the agency approved two Ebola vaccine INDs “in less than a week” to allow trials to get underway.

The key problem facing the FDA and other agencies is not restricted to getting experimental therapies to patients – which has been sanctioned by the World Health Organisation (WHO) after a meeting last month – but making sure that their use generates meaningful data to evaluate their effects.

“We cannot lose sight of the scientific fact that investigational vaccines and treatments for Ebola are in the earliest stages of development,” said Borio at the hearing.

“Data on safety or effectiveness in humans are limited or lacking, and accurate assessment (especially of effectiveness) may be impossible if adequately designed clinical trials are not performed,” he added.

On the safety front, there was some good news yesterday after Sarepta Therapeutics reported that it had completed human trials of its RNA interference-based candidate AVI 7537 for Ebola which “demonstrated no clinical or toxicologic safety concerns.”

One of the key issues is however deciding on new, flexible protocols for trials that do not depend on the use of a control group, which is unethical considering that Ebola has a 70 per cent fatality rate with supportive care.

“Developing the medical products to help bring this Ebola epidemic under control is highly complex and will, unfortunately, take time,” said Borio, adding: “We fully appreciate the gravity of the situation at hand and are exercising maximum flexibility in our activities [including] facilitating and expediting the development of medical products to diagnose, prevent, and treat Ebola virus disease.”

Thankfully, the list of companies developing Ebola candidates is getting longer by the day, with Chimerix announcing yesterday it had received the FDA’s blessing to start trials of its brincidofovir, which has already been used on a compassionate basis to treat Thomas Duncan, the first patient diagnosed with Ebola on US soil.

The US Department of Health and Human Services (HHS) also revealed this week it was providing $5.8m in funding to Baltimore-based Profectus BioSciences to help accelerate its efforts to develop an Ebola vaccine, which is now expected to start human trials in 2015, and a few days ago Toyama indicated it plans to start testing its favipravir antiviral drug candidate as an Ebola therapy.

Meanwhile, it emerged yesterday that China’s Sihuan Pharmaceutical has sent several thousand doses of an experimental drug called JK-05 to West Africa for use by Chinese aid workers and has indicated more can be made available if required.

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