Experimental Treatments? Unapproved But Not Always Unavailable
The Intervention of AIDS continued...
With the activism around AIDS and the demands of people with
other serious illnesses for access to unproven treatments, the medical
community, including FDA, began to appreciate that the traditional risk/benefit
models may have been inappropriate for people with serious and life-threatening
diseases. Dying patients were willing to take bigger risks for even the
slenderest hope of benefit.
"The hope part of it is that it might work and keep them
alive a little longer," says Theresa Toigo, Associate Commissioner for the
Office of Special Health Issues. "Even if it is only two months, by then
there might be a cure. It is a wonderful survival instinct."
For patients in search of a cutting edge treatment, the
possibilities have improved dramatically. First of all, there are more clinical
studies under way than ever before. FDA has on file more than 13,000 active
drug and biologic studies. These range from a few dozen patients to as many as
50,000 participating in a single investigational new drug trial. More than
100,000 patients are enrolled each year in National Institutes of
Health-sponsored studies conducted all over the United States.
Studies with investigational new drugs can be conducted by
the federal government, primarily through the National Institutes of Health; by
research universities, usually with federal funding, though also through
private foundations or drug companies; and by private, for-profit companies on
behalf of pharmaceutical manufacturers.
Clinical trials are essential to the development and
approval of new drugs. In these studies, a group of human volunteers receiving
the investigational therapy are compared with another group that receives
either the standard treatment or a placebo. Placebos, sometimes called sugar
pills, are any fake treatment that has no therapeutic benefit. This allows the
researchers to compare the effect of the treatment to no treatment in otherwise
similar patients. When the control group is given the standard treatment,
researchers are able to determine whether the experimental treatment provides a
better outcome than what is already available.
The clinical trial setting helps ensure that risks are
minimized because the research protocol, the set of rules by which the clinical
trial is conducted, have been scrutinized by FDA and a local ethics committee
called an institutional review board.
"We want to encourage people to participate in the
clinical trial process because that is where information is best developed
about the drug product," says David Lepay, M.D., Director of the Division
of Scientific Investigation in FDA's Center for Drug Evaluation and
The downside of being in a clinical trial, indeed the
downside of using any unproved medication, is that the new drug may not work.
It may even be dangerous and, sometimes, deadly.
Not everyone who wants to participate in a clinical trial
can do so. Limits on the number of participants and specific eligibility
criteria keep some people out. In addition, it is often inconvenient for the
patient to travel to the research center.
When individuals are unable to participate in a clinical
study, FDA provides alternative mechanisms for patients and their doctors to
get their hands on a promising new drug.