The WHO decision came amid mounting anger in Guinea, Liberia, Nigeria and Sierra Leone that the few doses of the experimental drug ZMapp had been given to two U.S. healthcare workers, who survived and are now in Atlanta. A Spanish priest died from the disease despite receiving some of the drug, which is being developed by Mapp Biopharmaceutical in San Diego.
Since it may be months before more of the drug is available, there's time to educate the public here and abroad about the best way to handle unproven therapies in the midst of an epidemic. The U.S. response to the early days of HIV/AIDS, when dying patients—mostly young, outspoken gay men—demanded access to experimental drugs, provides the historical precedent.
The movie “Dallas Buyers Club,” released last year, portrayed the desperation of those years. After years of stormy protests, the FDA created a compassionate-use program that enabled companies to give patients experimental drugs. All had been successfully tested in animals and humans for safety, so the drugmakers could be reasonably assured that they wouldn't cause additional harm to those patients, even if the drugs didn't work.
But that's where the analogy breaks down. Normal procedure at the FDA and other global regulatory bodies requires that drugs in line for approval be tested in randomized trials to determine if they are effective. For a potential Ebola drug, a randomized trial—the gold standard for regulators—is 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. As companies gear up to manufacture their experimental drugs and vaccines, the FDA, the National Institutes of Health and the WHO should rip a second page from the history books.
In the 1960s, more than half of children stricken with cancer died as oncologists floundered through the existing armamentarium looking for useful drug combinations. Some survived. But rather than celebrating each survival miracle and winding up with no sense of what happened to the larger group, every child was enrolled in a clinical trial.
The regimens and outcomes of their treatments were rigorously collected and recorded. Over time, regimens 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 universal testing system, more than 90% of children stricken with cancer survive until adulthood.
The experimental therapies from Mapp and Tekmira Pharmaceuticals could be treated the same way. Its developers already have three data points for its first-stage safety trial—the two U.S. health workers and the Spanish priest who received the drug. Their results—two survivors and one death—also provide data for an efficacy trial.
While waiting for more of the drug to be produced, authorities should take steps to educate the African public about the experimental nature of the therapy and lay the groundwork for meaningful informed consent. Every potential patient should be assured that when the drug is available, they will be enrolled in a clinical trial so that medical authorities can track the results.
Follow Merrill Goozner on Twitter: @MHgoozner
