The federal government's actions surrounding the use of an untested, unapproved drug to treat the Ebola virus
highlight the lack of protocols for use of such drugs in other countries, some experts say.
The Food and Drug Administration
has declined to say whether it gave approval to the manufacturer of the experimental Ebola drug ZMapp to provide three doses of the drug to treat two American healthcare workers and a Spanish priest in West Africa. Some observers say there is no way the manufacturer would have provided the drug
without receiving the FDA's nod.
“Ebola highlights the fact that we never had rules for trying experimental drugs at the international level,” said Arthur Caplan, founding director of the Division of Medical Ethics in NYU Langone Medical Center.
The FDA has the sole authority to decide whether an experimental drug produced by a U.S. drugmaker can be used when alternative treatments are not available. Under the agency's emergency use authorization, unapproved drugs can be allowed for use to prevent serious disease or conditions. The FDA also can allow access to drugs and devices that have yet to go through human clinical trials through the agency's emergency investigational new drug application program.
But such programs are designed for U.S. domestic use of experimental drugs.
So far, only a few infected patients have received the experimental treatment ZMapp, produced by San Diego-based Mapp Biopharmaceutical. The company said it had exhausted its supply of the drug after sending three courses to Liberia, whose government requested the treatment for two Liberian doctors after they got infected while treating Ebola patients.
Mapp had come under scrutiny by some who felt the initial use of the drug in two American healthcare workers and a Spanish priest unfairly gave priority to patients from Western countries, depriving Africans most affected by the Ebola outbreak of potentially lifesaving treatment.
It is unclear whether Mapp received emergency investigational new drug application approval before distributing ZMapp. An FDA spokeswoman said the agency does not discuss specific products but it does have a process for approving experimental drugs upon request.
“The FDA has one of the most flexible regulatory frameworks in the world,” said Dr. Luciana Borio, FDA assistant commissioner for counterterrorism policy and acting deputy chief scientist. “(It's) a framework that allows us to be very responsive to outbreaks of this sort. We have to be really flexible and nimble to meet the public health need with the information we have at hand for the products we have at hand.”
The issue of using experimental drugs for Ebola that have not been tested for safety or effectiveness in humans gained wide attention when the World Health Organization convened a meeting of ethicists on Aug. 11 and decided it was ethical to use experimental drugs to treat Ebola in light of the deadly threat the outbreak posed.
“In the particular circumstances of this outbreak, and provided certain conditions are met, the panel reached consensus that it is ethical to offer unproven interventions with as yet unknown efficacy and adverse effects, as potential treatment or prevention,” WHO stated.
So far, there have been close to 2,000 Ebola cases in three West African nations and Nigeria, and more than 1,000 people have died since the outbreak began in March. Health systems in Guinea, Sierra Leone and Liberia have been overwhelmed by the number of patients. Fear of infection has led to health workers in those countries refusing to treat Ebola patients, forcing the closure of a number of hospitals and clinics.
“Ebola probably drove forward in a very interesting way the idea that it is acceptable in some circumstances to try experimental drugs or vaccines in poorer countries,” Caplan said. “The major concern prior to that was that it would be seen as exploitation. The difference in this case is they're trying something experimental to help local people.
But Dr. Jonathan Moreno, a professor of bioethics at the University of Pennsylvania Perelman School of Medicine, said any use of an experimental drug or vaccine must be done with strong consideration of the possibility that the treatment won't work or harm patients. “There's really good reason to catch your breath,” Moreno said.Follow Steven Ross Johnson on Twitter: @MHSjohnson