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
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
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
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
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.