Q: What is a Clinical Trial?

A: When a promising, new, potentially therapeutic compound has been identified, the first step in turning it into a drug is preclinical testing which usually takes from one to six years during which researchers determine what the drug is good for, the appropriate dose, and its potential side effects. Depending on the data gleaned from this testing, research is either abandon or the potentially therapeutic compound moves on to clinical trials. A clinical trial is a carefully designed study that involves people like you who volunteer to receive investigational treatments under close supervision by a physician and other research professionals. Pharmaceutical and biotechnology companies have developed these treatments. These companies have then selected physicians, also called investigators, who are qualified to conduct clinical trials. It is through these trials that investigational drugs may show their benefits.


Q:  What can I expect?

A:  A clinical trial is a carefully designed study that involves people like you who volunteer to receive investigational treatments under close supervision by a physician and other research professionals. Pharmaceutical and biotechnology companies have developed these treatments. These companies have then selected physicians, also called investigators, who are qualified to conduct clinical trials. It is through these trials that investigational drugs may show their benefits. The clinical testing of an investigational drug is a step-by-step process that ensures you receive careful medical attention. All clinical trials are reviewed by the U.S. government's Food and Drug Administration (FDA) and by Institutional Review Boards (IRBs). The clinical testing of an investigational drug is a step-by-step process that ensures you receive careful medical attention. All clinical trials are reviewed by the U.S. government's Food and Drug Administration (FDA) and by Institutional Review Boards (IRBs).  IRBs are independent committees whose job is to make sure your rights are fully protected and that you are not exposed to any unnecessary risks. IRBs also are responsible for ensuring you sign a consent form before agreeing to participate in a trial. Clinical trials usually are conducted in three phases (I, II, III). A small number of people participate in Phase I trials. Each latter phase involves a larger number of people.  In a clinical trial, a volunteer is usually assigned a specific study group. Sometimes volunteers in one study group will receive an investigational treatment or study drug. Sometimes volunteers will receive a placebo or a treatment already available.  A placebo is an inactive product used as a basis of comparison for the results of the study drug. You, your physician and your research staff may not know who is receiving a placebo and who is not. In this way, volunteers in a clinical trial can be observed by the physician and research staff more fairly. Whether you receive the placebo or the investigational drug, the level of medical attention and care that you receive is the same.   A placebo is an inactive product used as a basis of comparison for the results of the study drug. You, your physician and your research staff may not know who is receiving a placebo and who is not. In this way, volunteers in a clinical trial can be observed by the physician and research staff more fairly. Whether you receive the placebo or the investigational drug, the level of medical attention and care that you receive is the same. I


Q:  What happens after a trial

A:  Phase I trials are small-scale tests of the safety of a drug. About 20 to 100 healthy volunteers take different dosages so researchers can learn how the human body absorbs and eliminates the drug, and identify any potential side effects. If the potential benefits of the new drug are deemed to outweigh the side effects that turn up in Phase I trials, the FDA allows it to proceed to Phase II. Phase II clinical trials are longer, larger-scale versions of Phase I trials. Researchers recruit patients with the condition they are using the new drug to treat. They give them varying doses of the drug to identify the dosage/effectiveness ratio. Phase II trials typically run for about two years. While they primarily focus on effectiveness and dosage, the side effects of the drug are still being closely observed and documented. If the new drug appears to be effective at a practical dose without side effects or with minimally toxic side effects, the FDA will then approve its use for Phase III trials.  By the time a drug reaches Phase III trials, researchers have reasonable expectations regarding the safety, appropriate dosage and effectiveness of the new drug on a given condition. The Phase III trials are typically conducted for two to four years usually involving the participation of several thousand individuals in trials at various medical centers around the country. After completion of Phase III trials, the pharmaceutical company that has sponsored these clinical trials, submits its findings to the FDA. The FDA then approves or denies the drug for use, or indicates a requirement for further testing and resubmission prior to approval.

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