San Diego-based Mapp Biopharmaceutical's experimental Ebola drug, a combination of monoclonal antibodies whose development has been completely financed by the U.S. government, hasn't even reached the stage where it can be tested in humans. Such trials first require successful safety testing in animals before the FDA will approve safety testing in healthy people.
Only after it passes that test can human clinical trials begin under the usual FDA procedures. But what would be the shape of an Ebola clinical trial? Double-blind, randomized trials—the gold standard for regulators—are out of the question. Can you imagine anyone dying from Ebola volunteering for a trial where one group received a placebo?
The current outbreak has created a situation where compassionate-use policy isn't relevant. There is no drug and there are no trials. Companies with biotech capabilities are gearing up to manufacture the experimental compound, a very difficult process as Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases at the National Institutes of Health, told CNN earlier this week. But once it arrives, how can it be distributed without undermining future research designed to learn if it actually works?
Here's where the FDA should take a second page from its history. In the 1960s, more than half of children stricken with various forms of cancer died from the disease as oncologists floundered through the existing armamentarium looking for useful drug combinations.
But rather than celebrating each survival miracle, while having no sense of what happened to the larger group, every child given a set of drugs or procedures was enrolled in a clinical trial. The protocols and outcomes data were rigorously collected and recorded. Over time, protocols were adjusted to reflect the best outcomes. Today, because of those adjustments and the arrival of new medicines and procedures that were fed into the system, over 90% of children stricken with cancer survive until adulthood.
The experimental Ebola therapy could be treated the same way. Its developers already have two data points for its first-stage safety trial—the two western aid workers who received the drugs.
As more of the drug gets manufactured, the FDA and the World Health Organization could sanction its use in West Africa, as long as health authorities and patients there give informed consent that they are aware of the risks and understand that it might not work. While there's a history of western drug companies violating informed consent rules while testing medicines in Africa, in today's environment, with nearly 1,000 dead and millions more living in fear, it's unlikely that such consent will be hard to obtain.
To ease the way, and more importantly, to further science, health authorities should tell every patient that he or she will be enrolled in a massive, real-time clinical trial where medical authorities will track the results of what is likely to be an unprecedented human experiment.
Follow Merrill Goozner on Twitter: @MHgoozner
