Use of Investigational Drugs in Human Clinical Studies

Use of Investigational Drugs in Human Clinical Studies

What is a clinical study of an investigational new drug?

In a clinical study, human volunteers receive investigational drug therapy. The effects of the treatment are compared with a control group of human volunteers who receive either the standard treatment or a placebo (a sugar pill that has no therapeutic benefit). Researchers are able to conclude whether the experimental drug had a better outcome than the standard treatment. The experimental drug might produce a better outcome, but it might not work. It could prove dangerous or even deadly.

Who conducts clinical trials of investigational new drugs?


The federal government, through the National Institutes of Health (NIH), conducts clinical trials of investigational or experimental new drugs. Research universities also conduct studies on new drugs using federal funds, private money, or drug company funding. Drug companies also fund private studies on investigational new drugs.

Are clinical trials required before a new drug can be approved?


The Food and Drug Administration (FDA) has responsibility for regulating drug development. Clinical studies are a required part of the FDA’s approval process for a new drug.

How do patients gain access to clinical trials?


Patients learn of clinical trials through their doctors. There are also Web sites that list current clinical trials. The clinical study protocol establishes eligibility criteria for patient participation. Occasionally, a compassionate exemption is made which allows a patient who does not meet the explicit criteria to participate in the study.

Who has access to investigation drugs outside of clinical studies?


The FDA can permit people with serious or life-threatening illness to take investigational drugs, usually cancer or AIDS drugs, while the products are still being tested in a clinical trial. This sometimes occurs when the initial study results of the drug have shown it to be promising and safe.

Can approval of cancer therapies be expedited?


The Food and Drug Administration Modernization Act, which was passed in 1997, authorized the FDA to speed up drug development and expand cancer patients’ access to unapproved therapies. Experimental drugs that show preliminary promising results can be made available to cancer patients early in the drug evaluation process. Also, cancer drugs can be fast tracked and given priority review by the FDA.

Is there access to cancer therapies approved in other countries?


The FDA has identified and approved certain cancer drugs that have already been approved for use in other countries.

How many cancer patients participate in clinical trials?


NIH-sponsored clinical studies enroll more than 100,00 patients each year.

Copyright 2012 LexisNexis, a division of Reed Elsevier Inc.