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Lynne Eldridge MD

What is an Investigational Drug?

By December 30, 2012

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"I heard about a new investigational drug that may help with lung cancer. Is there any way that I can be treated with this new drug?"


Variations of this question are something I hear often. And with the heart-wrenching statistics surrounding lung cancer treatment, these are questions worth asking. In fact, according to the National Cancer Institute, people with lung cancer should consider enrolling in clinical trials that test these drugs.

But what exactly is an investigational or experimental drug? The terms alone conjure up images of star trek reruns portraying medical treatments of the future - treatments that lend a sense of intrigue if not fear, to those who are already confused by the foreign language of cancer.

So perhaps I can translate a bit.

An investigational (experimental drug) is one that has not yet been approved by the FDA, and for that reason, can't be legally marketed or sold in the United States.

This doesn't mean that the drug hasn't been studied.

Many of these drugs have been studied on thousands of people. How many depends on what phase of a clinical trial an investigational drug is being studied in. If it's a phase 1 trial, the goal is to see if the drug is safe and what the best dose may be. If it's a phase 3 trial, researchers may be trying to answer the question, "is this treatment really better than others we have?" Even before the phase 1 trials, there is a great deal of research that has gone into an investigational drug.

You don't necessarily need to in a clinical trial to use an investigational drug.

Yes, most of the time these drugs are used as part of a clinical trial. But there is also something known as "compassionate use" - in which one of these medications may be used outside of a clinical study - after meeting strict criteria - for someone who has failed other treatments that may help with their cancer.

Learn more about the definition of investigational drugs and the criteria for using them here:

Photo: National Cancer Institute, Bill Branson (photographer)

January 5, 2013 at 8:44 pm
(1) Annie says:

There is no medical cure for our lung cancer. But I see it different. My cure is in my heart,mind,feelings and eyes. These parts of me have healed or is healing. My eyes see things they’ve never seen before. Oh it’s so beautiful to see with my eyes wide open. People are so colorful,smiles are so much bigger,everthing I see is so much brighter. Life may be a little shorter,but its much more richer. I feel its like a gift. I guess we could grive for ourselves or see the richer side of life. This is something most people can’t and will not see. They are not as special as I(we). Sorry if I offended anyone. I just choose to live my life different. Bless you all.

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