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Experimental Treatments? Unapproved But Not Always Unavailable

Getting Access

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 Research.

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.

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