Clinical Trials

Frequently Asked Questions (FAQs)

What are clinical trials?
Clinical trials are research studies that test new drugs and treatments for both safety and effectiveness in human beings. Clinical trials are carefully controlled and conducted by physicians, nurses, and other healthcare professionals. The results of clinical trials help determine if a particular treatment should be approved and made available for broader use to improve the overall standard of care.

What are the potential benefits of participating in a clinical trial?

• Clinical trials allow access to new treatments before they become available to the public and allow you to expand your treatment options.
• Clinical trials offer the possibility of quality medical care at leading institutions.
• Clinical trials provide an opportunity to contribute to science and medical research for future disease prevention and treatment.

What are the potential drawbacks of participating in a clinical trial?

• You may find that the drug or treatment is not effective for you.
• You may not receive the treatment being studied (see "placebos and controls").
• You may experience side effects or adverse reactions to medications or treatments.
• You may be required to visit a physician and undergo evaluations more often than you would otherwise.

Who conducts a clinical trial?
Clinical trials are typically sponsored by government agencies (such as the National Institutes of Health), and by biotechnology, pharmaceutical, and medical device companies that develop new drugs and treatments. Clinical trials take place at a variety of investigative sites such as hospitals, medical centers, and doctors' offices. As a participant, your contact is the healthcare provider (investigator) at the investigative site.

What does clinical trial "phase" mean?
Before any drug, device or treatment is tested in humans, it is tested in animals. This is done to ensure the basic safety of the treatment. No treatment is administered to animals or humans unless the researchers have a scientific basis for believing that the treatment will have a positive rather than a negative effect. Once the treatment has been determined to be safe for testing in humans, clinical trials are conducted in up to four standard phases:

• Phase I trials involve a small number of people (fewer than 100) and are used to evaluate the safety of the treatment, determine the appropriate dose, and identify side effects.
• Phase II trials are larger (100-300 people) and involve further evaluation of the safety and efficacy of the treatment.
• Phase III is the largest phase (with trials involving 1000-3000 people). Again, researchers evaluate the efficacy of the treatment, compare it to other existing treatments, and monitor the rate of side effects.
• Phase IV trials are conducted after a treatment has been approved by the FDA (Food and Drug Administration) and is available to the public. This phase continues to test the treatment and collect valuable information on its effects in different people, including any side effects associated with long-term use.

What are placebos and controls?
Not all participants in a clinical trial will receive the same treatment. In order to evaluate how the treatment being studied compares to existing treatments, investigators usually gather data from participants receiving the target treatment and from participants receiving other care. The allocation of treatments is determined by a randomization process that ensures statistical validity. Participants receiving other types of care are in "control" groups. Some control groups may be administered a "placebo," which is an inactive treatment, such as a sugar pill. Placebos are used to ensure that any effects of receiving the target treatment are not due solely to the experience of receiving care (the positive benefits of medical attention and the psychological advantage of believing a treatment will be effective). Not all clinical trials include placebos; if you are concerned about this possibility, ask the investigator for more information.

What is a "blind" or "masked" clinical trial?
You may hear that a treatment is being tested through a "blind" or "masked" clinical trial. This means that participants don't know whether the treatment they are receiving is the target treatment or a control treatment. If a trial is "double-blind" or "double-masked," that means that neither the trial participants nor the investigators know who is receiving the target versus control treatments. This is done so that results will not be interpreted based on participants' or researchers' expectations or hopes that one treatment will be proven more effective than others. In blind/double-blind or masked/double-masked clinical trials, treatments typically are not revealed except in extreme circumstances.

What happens to the clinical trial data?
Researchers review the clinical trial data after each phase to determine if the next phase should begin. After Phase III has been completed and the treatment judged to be safe, effective, and an improvement upon existing treatments, the treatment is submitted for review by the FDA. The FDA may then approve the treatment for use in the general public or mandate that additional research be conducted. Results of clinical trials are sometimes published in medical journals.

How are trial participants selected?
Investigators develop a set of eligibility criteria for selecting clinical trial participants. These criteria are used to ensure that the treatment is appropriate and safe for participants. For example, researchers may choose to investigate the effect of a drug on female stroke patients without a history of other illnesses, rather than on stroke patients with other complications. In some cases, researchers need to restrict participation in a clinical trial to protect patients from known risks. For example, some drugs can only be given to patients with healthy kidneys; patients with kidney disease would therefore not meet the eligibility criteria for clinical trials involving those drugs.

What are inclusion and exclusion criteria?
Inclusion criteria are characteristics that a patient must have to participate in the trial. For example, a patient must have a confirmed diagnosis of breast cancer in order to be a candidate for a trial testing a drug's effectiveness in treating breast cancer.

Exclusion criteria are characteristics that a patient cannot have in order to participate in the trial. If a treatment requires that a patient have no history of hypertension, for example, then a participant with high blood pressure will not be eligible.

How are clinical trial participants protected?

• IRB Review: Before a treatment is administered to patients in a clinical trial, all aspects of the treatment are thoroughly reviewed by a qualified panel of experts, called an Institutional Review Board, or IRB.
• Informed Consent: Before participating in a trial, you are asked to provide your "informed consent." This means that you agree to participate in the clinical trial, and that you have been provided with extensive information about the trial. It is also important to know that agreeing to participate in a clinical trial puts you under no obligation to begin or complete the trial process. You have the right to withdraw from the trial at any time without penalty. You must be given the following information:
  • Goals of the trial.
  • Why it is being conducted.
  • Information about the treatment you will be administered.
  • Exactly what participation will involve.
  • The known risks and benefits of participation.
  • Alternative treatments that are currently available.
  • Contact information for the trial investigators.

What is a clinical trial "protocol?"
A clinical trial protocol is a description of the treatment each participant will undergo, including what medications they will receive or procedures they will experience, the frequency and manner of these interventions, the schedule of tests to determine results, and the length of the clinical trial. The protocol also includes information about participation requirements.

What types of treatment will I receive?
The actual treatment you receive varies considerably from trial to trial. In general, however, you can expect:

• A team of healthcare professionals, such as physicians and nurses, will administer the trial.
• You will receive an evaluation at the beginning of the trial to determine your health status at the time.
• You will be given specific, easy-to-follow instructions on how to proceed through the trial.
• You can expect to be monitored carefully during the trial, and you may be contacted for follow-up after the trial has ended.
• Participation in a clinical trial may involve visiting a healthcare professional more frequently than you otherwise would, and/or receiving more medical interventions (medications or procedures).
• The trial is likely to continue for several months.

Are there any costs involved in participation?
Research costs, such as the cost of hiring personnel and managing data, are covered by the organization sponsoring the clinical trial. Some clinical trials will pay you for joining the trial, although this is not a universal nor mandated practice. You may also receive reimbursement for expenses you incur as a result of participation, such as transportation costs. Patient care costs may be covered by medical insurance, although this varies by policy and by trial. You should seek more information about this arrangement from the trial investigator and from your insurance provider.

Do I need to coordinate with or notify my physician?
If the healthcare provider who has been treating your condition is not the one conducting the clinical trial, you are strongly encouraged to consult him/her about trial participation.

How does the drug development process work?

• The First Step: Laboratory Work
  Before a drug can be prescribed to patients, it must undergo a lengthy development process. The first step is a discovery phase, where scientists undertake laboratory work to identify compounds that have some promise of assisting in human healthcare. Molecular biologists, organic chemists, toxicologists, and other scientists do this work.
• The Second Step: Animal Studies
  Only a small percentage of the chemical compounds studied by scientists show promise for eventual use as treatment for human disease. These drugs are then studied further through administration to animals. Researchers take this step very seriously, administering the treatment to as few animals as possible and taking care to treat each animal humanely. This stage allows researchers not only to identify side effects, but also to learn how the drug interacts in a living being - how it is absorbed in the blood, for example.
• The Third Step: Request to the FDA to Conduct Human Studies
  The Food and Drug Administration, which will provide ultimate approval for wide use of a drug, becomes involved in the development process at this stage. Drug developers and researchers must present the results of their laboratory and animal research to the FDA, which can then approve a plan for testing the drug in humans.
• The Fourth Step: Clinical Trials
  Clinical trials occur relatively late in the process of drug development, at a point when the treatment has already undergone intense scrutiny. As discussed above, clinical trials must occur in three or four phases, each of which has a different objective. Subsequent phases cannot begin until the results of earlier phases have been obtained and studied to assess the impact of the treatment on humans.
• The Fifth Step: Application for FDA approval
  Before a drug can gain approval from the FDA, its developers must demonstrate both its safety and its effectiveness. Officials review the relative risks and benefits to patients, and will approve a drug or treatment only if its proven benefits outweigh its risks. Although the FDA considers its role to be of major importance, it also recognizes that delaying drugs with years of testing may be detrimental to patients. Efforts are often made to complete the evaluation process as quickly as possible.

Where can I find more information about clinical trials?
It is important to understand the clinical trial process, as well as the features of the particular trial you are considering, before you agree to become a participant. For further information, the website clinicaltrials.gov includes a list of questions to ask about trial participation, and links to additional resources. Also, remember that you should discuss trial participation with your healthcare provider, as well as with your loved ones.