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 http://rarediseases.info.nih.gov/ord/research-ct.html,
a database compiled by NIH's Office of Rare Diseases.
CenterWatch Clinical Trials Listing Service (www.centerwatch.com) 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
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
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.