A clinical trial is a research study conducted to answer specific questions about new treatments, or to investigate new ways of using known treatments. In most cases, the therapies and drugs investigated in clinical trials are not approved by Regulatory Agencies (such as the Food and Drug Administration (FDA) in the United States), and the benefits and risks of taking the treatment are not known. Clinical trials are used to determine whether new treatments are safe and effective. The clinical testing of experimental treatments or drugs is generally done in three phases, each successive phase involving a larger number of people. The knowledge gained from years of clinical trials regarding the safety and efficacy of new treatments or drugs ultimately may allow pharmaceutical companies to offer new and better options to patients.

 

A Phase I clinical trial is primarily concerned with evaluating the drug's safety. This initial phase of testing in humans is done in a small number of patients (20 to 100). The drug’s side effects are carefully monitored and recorded. This phase is also designed to determine how the drug is absorbed, metabolized, and excreted by the patient.

A Phase II clinical trial rigorously tests the drug for efficacy, may last from several months to a few years, and may involve several hundred patients. Some Phase II studies are randomized trials. In a randomized trial, one group of patients receives the experimental drug, and a second "control" group receives a treatment commonly used for the disease or a placebo. These studies may also be "blinded." This means that neither the patient nor the study physician knows which patient is receiving the experimental drug. Performing a blinded study can provide the pharmaceutical company and the Regulatory Agencies with comparative information about the safety and effectiveness of the new drug. Approximately one-third of experimental drugs successfully complete both Phase I and Phase II clinical trials.

A Phase III clinical trial assesses the drug on a much larger scale—in several hundred to several thousand patients. This type of study provides the pharmaceutical company and the Regulatory Agencies with a more thorough understanding of the safety and effectiveness of the experimental drug, and offers better insight into possible drug-related side effects. Most phase III clinical trials are randomized, and some Phase III studies may also be blinded. Upon successful completion of a Phase III study, a pharmaceutical company may request Regulatory Agencies’ approval to market the experimental drug.

By participating in a clinical trial, a patient may experience unpleasant, serious, or even life-threatening side effects of the drug or treatment being studied. The drug or treatment may not be effective for patients. Every clinical trial has a protocol which describes exactly what the physician and patient must do during the trial. The patient may be required to visit the clinical trial investigation site often and undergo numerous tests to evaluate the drug or therapy.

To minimize the risks and protect patients’ rights, all clinical trials must be approved and monitored by an Institutional Review Board or Ethics Committee. These review boards/committees also examine the study's protocol to validate that the patient’s rights are protected, and that the study does not present an undue or unnecessary risk to the patient.

In addition, anyone participating in a clinical trial in the United States is required to provide informed consent, this is typically documented when the patient signs and dates an "informed consent form”. This form explains the conduct of the clinical trial in detail, the risks involved, the potential benefit of the experimental treatment or drug, what a patient can expect during the clinical trial, and assures the patient that he/she has the right to leave the study at any time with no consequence to their health.

 

Patients participating in a clinical trial may benefit by having access to the latest research and treatments not available to the general public. These patients often are able to get advanced medical care at a leading health care center for little or no cost to them. Participation in a clinical trial also allows patients to have an active role in their own health care, as well as help others by contributing to medical research.

Patients considering participation in a clinical trial should discuss it first with their doctors. Patients should also be sure that they understand all risks and potential benefits by asking questions and ensuring the questions are answered to their satisfaction. Below is a list of suggested questions to help guide a patient's conversation with a study doctor or a delegate to the study doctor (nurse coordinator):

At any given time, there are thousands of doctors throughout the world who are in the process of conducting clinical trials. If you are interested in participating in a clinical trial sponsored by Biogen Idec and if you would like to know if there is a doctor enrolling patients with your condition near you, please click here.