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Experimental Treatments? Unapproved But Not Always Unavailable

Single-Patient INDs continued...

Companies sometimes say that they cannot make the drug available to a patient because FDA won't allow it, but that is rarely true. FDA only denies access when there is evidence that the risk of using the experimental drug clearly outweighs any potential benefit to the patient.

If a drug is frequently used in single-patient INDs, FDA streamlines the process for obtaining permission. One example is thalidomide, a drug initially associated with birth defects in the 1950s but now being used experimentally to treat cancer. (FDA approved thalidomide in 1998 to treat leprosy.)

FDA has similar rules that give patients access to investigational new medical devices.

A Difficult Decision

All things being equal, is it worth it for a patient to get access to an experimental medication?

For society the additional safety information about the new drug may prove useful. And sometimes it does make a difference for individual patients. For example, people with AIDS who participated in the clinical trials for a category of drugs called protease inhibitors probably benefited because this class of drugs proved so dramatically effective. But for many other INDs, the success rates are much less impressive, such as tacrine (Cognex) for the treatment of Alzheimer's disease.

Even if access does not change long-term survival, it may provide for the patient and the family a sense that they are doing something and are not simply victims of some serious disease. Biomedical research advances rapidly and breakthroughs come from unexpected places, all feeding the hope that the next experimental drug will be the one that cures our ills.

Finding Information about Investigational New Drugs

While finding and getting into an appropriate clinical trial for your individual disease is something like a scavenger hunt, the Internet has made it much easier to track down these studies. The following is a listing of major Internet sites where you can search for a clinical trial that may benefit you.

Information Program on Clinical Trials, (www.lhncbc.nlm.nih.gov/clin) mandated by the FDA Modernization Act of 1997, is a joint FDA/National Institutes of Health resource. While initially containing only NIH studies, it will eventually include all federally and privately financed clinical studies.

CancerNet (http://cancernet.nci.nih.gov) is run by NIH's National Cancer Institute (NCI). It provides information on clinical trials. Information is also available through NCI's Cancer Information Service at 1-800-4-CANCER.

ACTIS (www.actis.org), the AIDS Clinical Trials Information Service, provides a wide range of information on current AIDS research, including drug trials, vaccine trials, and other educational material. Sponsored by the U.S. Public Health Service, including FDA, NIAID, Centers for Disease Control and Prevention, and the National Library of Medicine, ACTIS also can be reached at 1-800-TRIALS-A.

WebMD Public Information from the FDA

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