Medical Experts Look For New Ways To Test Ebola Drugs
Medical experts are meeting today and tomorrow at the World Health Organization in Geneva to figure out how to test potential Ebola drugs in Africa. In addition to determining which experimental drugs should be the highest priority, the experts are sorting through some difficult ethical issues.
In short, they're trying to figure out how to design tests that will provide the fastest and most trustworthy answers — and yet minimize the need for comparison groups who won't be offered the experimental treatments.
Practice in the United States has set an unrealistic standard. When American health care workers fell ill with Ebola in Africa, they flew home and received medical care vastly better than what Africans were getting, including experimental therapies.
"We still have no idea whether those treatments made any difference in their recovery," says Nancy Kass, a bioethicist at Johns Hopkins University. The patients "also got exquisitely high-quality supportive care, which undoubtedly made a huge difference."
The result was that U.S. medical researchers learned very little about the various experimental drugs dispensed to those few individuals.
In order to understand the value of these potential medications, scientists need a scientific study.
The standard way to do this the world over is to provide some people with the potential drug and a comparison group with a placebo — the so-called sugar pill. Martin Friede, who's in charge of WHO's work on Ebola drugs, says some in West Africa find that strategy objectionable.
There are other ways to come up with a comparison group — not as rigorous as using placebos, but still potentially useful, especially if a given experimental drug has a powerful effect.
"This can be done through historical records, it can be through comparing what's being done in other sites," Friede says. "There are various mechanisms of doing this."
Those are under discussion this week at the meeting in Geneva. But scientists know the farther they stray from the most rigorous standards, the less likely they are to get rapid, clear results.
"What we do not want are trials that are conducted where, at the end of the study, we are unable to say whether the drug is safe or not or whether the drug is effective or not," Friede says.
Numerous research groups are planning trials, using a variety of strategies. For example, scientists at Oxford University say they aren't going to use placebos in their trials.
U.S. scientists aren't drawing that bright line. They're hoping to start tests with Liberian health researchers early next year.
"This study initially would almost certainly be a randomized control trial," says Dr. H. Clifford Lane at the National Institute of Allergy and Infectious Diseases.
That means not everyone will get an experimental drug. The study will, however, offer everyone who enrolls a higher standard of overall medical care than they would ordinarily receive, so in theory everyone will benefit — unless the experimental drug proves to be harmful.
"People always think about investigational drugs as being wonder drugs, miracles — they're going to make the patient better," Lane says, "when, in point of fact, some of these agents can have pretty serious side effects."
Some people could actually be harmed by the treatment. "Without a control group, you wouldn't know that — or you wouldn't know it until a large number of patients might potentially have been harmed," Lane says.
All of the experimental drugs have had at least basic safety testing, but there's almost no experience in giving them to people who are sick with Ebola.
Scientists have been considering other ideas to minimize the number of people who are in a comparison group. One idea is to have a small comparison group and a large treatment group. Another idea is to give people varying doses, and see whether people who got more drug did better.
"I think this is a time, as is often true in deep ethical challenges, where thinking a little bit outside the box might be the answer that works best for everyone," Kass says.
After all, this isn't simply a test-tube experiment. Clinical trials involve real people, and they need to agree to participate, or the studies won't go forward.
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Transcript
STEVE INSKEEP, HOST:
We're also tracking this news - medical experts are meeting at the World Health Organization in Geneva today and tomorrow. And they're asking how to test experimental Ebola drugs in West Africa. Naturally people want to test whether the experimental treatments will work. The trouble is resolving ethical issues. NPR's Richard Harris reports.
RICHARD HARRIS, BYLINE: When American health care workers developed Ebola in Africa, they flew home and received medical care vastly better than what Africans were getting. Nancy Kass, a bioethicist at Johns Hopkins University, notes those treatments included experimental drugs.
NANCY KASS: We still have no idea whether those treatments made any difference in their recovery. They also got exquisitely high-quality supportive care, which undoubtedly made a huge difference.
HARRIS: But their treatment sent a clear message to Africans - the best care seems to include experimental drugs. But that's not the standard that will be applied in the tests in Africa.
KASS: People get suspicious whether there's a secondary level of care.
HARRIS: And the care seems inferior because not everyone in these trials will get an experimental drug. Researchers need a comparison group to see whether a drug makes a difference. The standard way to do this the world over is to provide some people with a potential drug and a comparison group with a placebo. Martin Friede at the World Health Organization says some in West Africa find that concept objectionable.
MARTIN FRIEDE: The word placebo it carries a lot of connotations to it that I think rather people are talking about controlled trials.
HARRIS: There are other ways to come up with a comparison group, not as rigorous, but still potentially useful, especially if a given experimental drug has a powerful effect.
FRIEDE: This can be done through historical records. It can be done through comparing to what's been done in other sites. There are various mechanisms of doing this.
HARRIS: Those are under discussion this week at the meeting in Geneva. Scientists know the farther they stray from the most rigorous standards, the less likely they are to get clear, rapid results.
FRIEDE: What we do not want are trials that are conducted where at the end of the study, we are unable to say whether the drug is safe or not or whether the drug is effective or not.
HARRIS: Numerous research groups are planning trials, and they're using a variety of strategies. For example, scientists at Oxford University say they aren't going to use placebos at all. U.S. scientists aren't drawing that bright line. Cliff Lane at the National Institute of Allergy and Infectious Diseases says they're hoping to start tests with Liberian health researchers early next year.
CLIFF LANE: This study initially would almost certainly be a randomized, controlled trial.
HARRIS: That means not everyone will get an experimental drug. That said, the study will offer everyone who enrolls a higher standard of overall medical care. So in theory, everyone will benefit. That is, unless the experimental drug proves to be harmful.
LANE: People always think about the investigational drugs as being, you know, wonder drugs, miracles. They're going to make the patient better, when in point-of-fact, some of these agents can have pretty serious side effects. And in fact, the patients might do worse for having gotten the agent. Without a control group, you wouldn't know that, or you wouldn't know it until a large number of patients might potentially have been harmed.
HARRIS: All the experimental drugs have at least had very basic safety testing, but there's almost no experience in giving them to people who are sick with Ebola. Scientists have been tossing around other ideas to minimize the number of people who are in a comparison group. One idea is to have a small comparison group and a large treatment group. Another idea is to give people varying doses and see whether people who get more drug did better. Again Nancy Kass from Hopkins.
KASS: I think that this is a time, as is often true in deep, ethical challenges, where thinking a little bit outside the box may be the answer that works best for everyone.
HARRIS: After all, this isn't simply a test-tube experiment. Clinical trials involve real people. And they need to agree to participate or the studies won't go forward. Richard Harris, NPR News. Transcript provided by NPR, Copyright NPR.
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