The development of a pharmaceutical drug is a slow, deliberate process, and it can take years of back-and-forth between scientists and health officials before medicine makes it onto the market.
But the worst Ebola outbreak in history has changed all that. The Food and Drug Administration is in crisis mode, and its process of reviewing applications for new drugs has been pushed into overdrive. The arrival of the deadly virus to American soil has created an unprecedented situation for the regulatory agency—especially when it comes to experimental drugs.
Now, FDA officials are reviewing new drug applications to fight Ebola in a matter of days instead of months or years. In August, two American health care workers who contracted Ebola in Libera received an experimental drug called ZMapp in the U.S., and made a full recovery. The FDA also approved an anitviral drug called TKM-Ebola for use in "emergency cases" to fight Ebola. Last week, Thomas Eric Duncan received brincidofovir, an antiviral drug originally developed to treat smallpox, after the FDA approved it for emergencies only. He died two days later.
Until this year, none of these drugs were approved for use, let alone deemed safe, by humans by the FDA.
Ebola—fatal 60 percent of the time when it's caught early, and 90 percent when it's found too late—has changed the game. "It's like a race against time," Dr. Luciana Borio, assistant commissioner for counterterrorism policy and acting deputy chief scientist at FDA, told National Journal. In Liberia, Sierra Leone, and Guinea, the disease is well ahead of medical response efforts, killing at least 4,447 people so far, according to the latest numbers from the World Health Organization.
In the U.S., when physicians ask the FDA for permission to use experimental drugs on their patients, as they have recently in Dallas, the agency responds in a matter of hours—and often, less than one hour. In that time, the treating physician and an FDA doctor talk about the patient's condition and weigh the benefits of receiving an experimental drug with the risks. "We want to make sure that we are not hurting individuals," Borio said.
But there's no way to know that. "We don't know right now if these products work. We don't know if they hurt, we don't know if they do nothing, and we need to find that out," she said. But in the meantime, Borio said, if a patient is dying of Ebola in the U.S., the FDA has to change its rules.
FDA regulations prohibit the agency from disclosing information about experimental drugs, as well how many doctors have asked for them. The agency has oversight over any experimental medicine administered to Ebola patients in the U.S.
The FDA has had to bend the rules in its review of applications for new drugs, too. Most of the drugs that the FDA has approved for emergency use against Ebola have not even reached clinical trials yet.
"We are working with the developers and government colleagues in much more real time to, before the application is in, to discuss what are the basic data elements that will be required for us to make the decision?" Borio said. "What data elements, for example, might be deferred until a later time? Do you really need to have A, B, and C before you go on to D, or can you do A, B and then get C later?"
The use of drugs in their early stages of development means that doctors can't be sure what about them works—and what doesn't. Some patients who have received such medicine have recovered entirely; others, like Duncan, have died. "I can't stress enough that we will not really learn about the use of these products in these circumstances where they're used in individual patients," Borio said. The effect of one drug on a single patient is not a good measure for an entire population. But, "we think it's the right thing to do when a clinician asks for treatment in these particular situations."
Borio, along with Tom Frieden, director of the Centers for Disease Control and Prevention, will testify on the U.S. response to the Ebola outbreak before the House Energy and Commerce subcommittee on Thursday afternoon. Lawmakers are unlikely to focus on the science of the situation, and bear down on the speed of it instead. With another new case of Ebola reported in the U.S. this week, are government agencies like the FDA and CDC moving fast enough to contain its spread here and abroad?
"We're working as fast as we can," Borio said. "I'm here late, but I walk the corridors and there are many people who are here late too, and that includes weekends. People are 24/7, all-hands-on-deck."
She added: "Ultimately we want to offer more than hope. We want to offer products that are safe and effective, not only for this epidemic, for a future one."