Frequently Asked Questions
You have questions. We have answers.
What is a clinical trial?
A clinical trial is a research study involving volunteers which is carefully conducted by pharmaceutical and biotechnological companies to answer specific health questions. Qualified investigators and other research professionals supervise clinical trials that are approved and thoroughly regulated by the U.S. Government's Food and Drug Administration (FDA). Clinical testing of a new drug is a step-by-step process that ensures patients to receive careful medical attention. Clinical trials can tell researchers which treatments are more effective than others, and ultimately help in the development of new drugs and treatment strategies.
Why participate in clinical trials?
Participants in clinical trials play an active role in their own health care as well as gain access to potential new treatments at no cost before they are available. Participants also help others by contributing to medical progress.
What are the phases of clinical trials?
As part of the lengthy process of drug development, clinical trials are conducted in phases (I to IV) - with different purpose and questions to be answered.
- Phase I: Scientists test drugs on small groups of healthy volunteers and patients to evaluate its safety, determine a preliminary dose range, and identify potential side effects.
- Phase II: The study drug is given to a larger group of patients to test its effectiveness in a certain disease and to further evaluate its safety.
- Phase III: The investigational drug is given to larger groups of patients suffering from the targeted disease. The purpose is to demonstrate substantial evidence of effectiveness and safety of the drug. These studies usually serve as basis for the new drug approval process, performed by the FDA.
- Phase IV: Post marketing studies for additional information on the optimal use, efficacy, and tolerability.
What are the procedures of clinical trials?
This process includes several steps:
- After signing an informed consent form, a volunteer patient is screened for study eligibility. That is to find out if he/she meets the inclusion/exclusion criteria laid out in a study protocol, approved by the FDA. The interested person will receive a physical examination, a review of the medical history, and a detailed description of the specific clinical trial.
- If eligible, the volunteer will be enrolled in the study and randomized to either of the pre-defined treatment groups.
- The volunteer is asked to come in to the trial site for regular visits, where various tests and procedures are performed and the study drug is distributed.
- The final visit signalizes the end of the study for the participant.
- Some studies provide a follow-up period to gain further information on the long-term efficacy, safety, and tolerability of the study drug.
Why are clinical trials conducted?
- To see if a new drug or device is safe and effective for people to use.
- To compare existing treatments to determine which is better.
- To study different ways to use standard (approved) treatments, so they will be more effective, easier to use, and/or decrease the side effects.
- To learn how to best use the treatment in a different population, such as youth, in whom the treatment was not previously tested.
What are the risks of my participation?
- There may be unpleasant, serious, or even life-threatening side effects resulting from the treatment.
- The treatment may not be effective.
- Participation in the trial may be demanding and time consuming.
How are volunteers protected?
- To protect the rights and welfare of clinical research participants, U.S. Federal Agencies including the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) oversee much of the medical research in the United States.
- Institutional Review Boards (IRBs) oversee the centers where clinical research studies are conducted. IRBs review and approve study protocols to ensure that a clinical trial is ethical and that volunteers' rights are protected.
- A participant in a clinical trial has access to the IRB that is overseeing the research and access to a volunteer advocate, the physician and staff conducting the trial.
- Federal agencies inspect individuals and institutions conducting research. They also inspect the IRBs.
- Some IRBs are accredited much like hospitals can be accredited and some research investigators and staff are certified as research professionals.
What questions should I ask before participating in a clinical trial?
- "What is the main purpose of this study?"
- "Does the study involve a placebo or a treatment that is already on the market?"
- "How will the treatment be given to me?"
- "How long is the study going to last and what will I be asked to do as a participant?"
- "What has been learned about the study treatment and are any study results published?"
- "Do I have to pay for part of my study? Will my insurance cover these costs?"
- "Is there any reimbursement for travel costs or childcare?"
- "Will I be able to see my own doctor?"
- "If the treatment works for me, can I keep using it after the study?"
- "Can anyone find out whether I'm participating in the clinical trial?"
- "Will I receive any follow-up care after the study has ended?"
- "What will happen to my medical care if I stop participating in the study?"
- "Does the physician/investigator have any financial or special interest in the clinical study?"
- "What are the credentials and research experience of the physician and study staff?"