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Experimental Treatments? Unapproved But Not Always Unavailable

Finding Information about Investigational New Drugs continued...

Information regarding clinical trials for rare disease can be found at, a database compiled by NIH's Office of Rare Diseases.

CenterWatch Clinical Trials Listing Service ( is published on the Internet by CenterWatch Inc., a multimedia publishing company in Boston, MA. It provides information on more than 5,000 active clinical trials as well as other information.

When a clinical trial is not an option, FDA facilitates access to an investigational new drug or an investigational medical device through other programs. For information on programs for, or access to, an unapproved investigational new drug, call FDA's Office of Special Health Initiatives at 301-827-4460.

Is the Risk Worth It?

No matter how promising a clinical trial or investigational new drug seems, there is no way to know about all the risks before the study begins. While the hope is that the study will produce a cure, it's important to recognize that risks can prove significant. For example, in 1992, tests for a promising hepatitis B drug severely damaged the liver in 10 patients. Some died and others required liver transplants.

Because of these inherent uncertainties the health-care professionals conducting the study must ensure that the patient understands the risks as well as the benefits beforehand and is willing to proceed.

Here are some questions patients might want to ask to make sure they understand the consequences of entering a study or using an investigational new drug:

1. What are the potential benefits from the treatment being studied? What have the animal or other human studies shown about the effectiveness of the drug?

2. What are the potential dangers from using this drug? Again, what do other animal and human studies show about the side effects?

3. In what phase is this clinical trial?
Clinical trials are generally performed in three phases. A phase 1 trial is primarily designed to assess the safety profile in a small number of patients. Phase 2 tests the effectiveness of the treatment in a relatively small number of patients. Many drugs never progress beyond phase 2 because they are not effective. In phase 3, a large number of patients receive the drug to substantiate that the effectiveness seen in phase 2 is real and to work out the details of its use. Individual patients are most likely to benefit from drugs in the later phases of development.

4. Will there be a control group?
For a clinical trial to produce useful information, the group of patients receiving the new treatment needs to be compared with patients who receive something -- or nothing -- else. Often, patients in the control group receive whatever is the current standard therapy for the disease. Sometimes, the control group patients will receive a placebo -- so-called sugar pills that produce no therapeutic benefit. In a clinical study, patients are randomly assigned to either the group treated with the experimental drug or to a group receiving the standard therapy or placebo.

WebMD Public Information from the FDA

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