Clinical Trials Explained

The clinical trial process is essential in bringing a new drug from the research lab to physicians and their patients. Before a drug enters into the clinical trial phases, it goes through extensive research and testing to define the drug's possible benefits and its limitations before it is tested in humans. The research and the clinical trials are carefully supervised, monitored and documented and the entire process is heavily governed by regulatory agencies across the globe and by the medical profession. After drugs go through successful completion of the final phases of trials, the FDA or foreign regulatory agencies spend time reviewing and analyzing the results to decide upon approval of the drug for market sales. This entire process takes many years depending on the complexity of the studies and the target patient population with the disease or disorder.

The Difference Between Phase I, II and III Clinical Trials

New treatments being studied have undergone preclinical testing in the laboratory with animals. If the treatment appears safe, it is then given to human patients in Phase I clinical trials. Phase I clinical trials are conducted with a small number of subjects to determine the early safety profile, maximum tolerated dose and pharmacokinetics of the product in human volunteers. Phase II clinical trials are conducted with groups of patients for whom the drug under development is intended in order to determine safety, preliminary efficacy, optimal dosages, and dose regimens. Phase III trials are large-scale, multicenter comparative trials conducted with patients afflicted with a target disease in order to provide substantial evidence for efficacy and safety of the compound as are required by the FDA and other regulatory agencies for approval.

Becoming Involved in Clinical Trials

People interested in clinical trial participation should discuss this option with their physician. It is the best way to learn whether or not an appropriate trial is available, and where it is being conducted. Patients are screened to determine if they are right for a given trial. Sometimes screening involves nothing more than a series of questions and answers. Other times it may require laboratory tests. In any case, these tests are performed only with the patient's consent. Not every disease will have a clinical trial currently underway. But this situation is constantly changing. Consult your physician or patient support group to learn if research is being planned which may be appropriate for you.

For other clinical trial information, visit www.clinicaltrials.gov and www.centerwatch.com.

Safety of Clinical Trials

Clinical trials are conducted in a healthcare setting (a hospital or clinic, doctor's office or clinical research center) and are monitored by trained healthcare professionals. An Institutional Review Board or IRB approves all clinical trials before they can begin. This is a committee made up of doctors, ethicists, and members of the general public and administrators. This group is held responsible for agreeing that a trial does not represent an unreasonable risk to patients who are participating. Trial protocols are also reviewed by the FDA. Your doctor and the clinical study physician are responsible for your well being and are the best people to decide whether or not you are eligible to participate in a trial. Whenever you agree to enter a trial you are given the name and telephone number of a contact in your local physician's office who will answer your questions as well as a member of the IRB whom you can contact if you have any doubts.

Commonly Asked Questions

What is a Placebo?

A placebo is oftentimes called a "sugar pill" and is a harmless product that is used to differentiate the drug's or treatment's actions from a "normal" response. This method of observation in the clinical trial can be conducted in a more unbiased manner since the patient and the clinician usually do not know which individual has or has not received the placebo until the study is completed.

What is a protocol?

A protocol is a written document describing, in detail, the procedures and steps to conduct a study (usually a preclinical study or clinical trial) to ensure the collection of valid data.

What is a Clinical Investigator?

A medical researcher in charge of carrying out a clinical trial's protocol. Researchers are usually doctors, nurses, pharmacists, or other health care professionals.

What is the Food and Drug Administration (FDA)?

A government agency that enforces laws on the manufacture, testing and use of drugs and medical devices. All drugs and medical devices must be approved by the FDA before they can be used by the general public.

Who is the "Sponsor" of a trial?

The pharmaceutical company, research institution, or other health organization that funds a clinical trial and designs its protocol.

What is an informed consent?

Anyone entering a clinical trial in the United States is required to sign a form indicating that they have been given information in regard to the study and have had all their questions answered about what will happen to them during the study. If the patient is a child (i.e., less than 18 years of age), a parent or guardian must sign the form and the child may be asked to sign the form giving their assent. This form will tell you what treatment will be given, what kind of problems might occur, and what other treatments might work for your condition. The amount of benefit expected from the study will be given. In phase I studies, it is usually not known whether any benefit will occur.

What is the Institutional Review Board (IRB)?

A board consisting of health care professionals from the institution where the clinical trial takes place, as well as members of the local community. The board is responsible for reviewing and approving the protocol for a clinical trial. It also scrutinizes all trial activities including recruitment, advertising, and potential risks. The IRB also makes sure that FDA regulations are being followed in a particular trial.