Frequently Asked Questions

1. What are clinical trials?

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to find treatments that work in people, and new ways to improve health.
There are different kinds of clinical trials, including those to study:

• prevention options
• new treatments or new ways to use existing treatments
• new screening and diagnostic techniques
• options for improving the quality of life for people who have serious medical conditions

Clinical trials are conducted according to a plan called a protocol. The protocol describes what types of patients may enter the study, schedules of tests and procedures, drugs, dosages, and length of study, as well as the outcomes that will be measured. Each person participating in the study must agree to the rules set out by the protocol.

2. Why are clinical trials done?

Many clinical trials are done to see if a new drug or device is safe and effective for people to use. Clinical trials are also done for other reasons. Some compare existing treatments to determine which is better. The current, approved treatments are called the "standard treatments." Sometimes clinical trials are used to study different ways to use the standard treatments so they will be more effective, easier to use, and/or decrease side effects. Sometimes, studies are done to learn how to best use the treatment in a different population, such as children, in whom the treatment was not previously tested.

For most trials, researchers, doctors, and other health professionals administer the clinical trials according to strict rules set by the Food and Drug Administration (FDA). FDA sets the rules to make sure that people who agree to be in studies are treated as safely as possible.

3. Where can people find out about clinical trials?

People can find information about clinical trials now being conducted now by searching ClinicalTrials.gov. ClinicalTrials.gov is an interactive online database, managed by the National Library of Medicine. It provides information about both federally and privately supported clinical research in human volunteers. ClinicalTrials.gov is updated regularly and offers information on each trial's purpose, who is eligible to participate, locations, and phone numbers to call for more information.

Since ClinicalTrials.gov is only available online, individuals without Internet access can use the database at the public library or other publicly available internet portal.

4. What types of clinical trials are available?

There are many different kinds of clinical trials, including:

• Trials that test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
• Prevention trials, that look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
• Diagnostic trials, that are conducted to find better tests or procedures for diagnosing a particular disease or condition.
• Screening trials to test the best way to detect certain diseases or health conditions.
• Quality of Life trials (or Supportive Care trials), that explore and measure ways to improve comfort and the quality of life for individuals with a chronic illness.

Usually, clinical trials compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product, or an existing, approved therapy. In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks like the test product).

Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products. However, placebos are not used where a patient would be put at risk, particularly in the study of treatments for serious illnesses, by not having effective therapy. Most studies of this kind compare new products to an approved therapy. Potential participants are told before they enter a trial whether placebos are going to be used in the study.

5. Who should consider clinical trials and why?

It is important to test drugs and medical products in the people they are meant to help. It is also important to conduct research in a variety of people because different people may respond differently to treatments. FDA seeks to ensure that people of different ages, races, ethnic groups, and genders are included in clinical trials.

Some people participate in clinical trials because they have exhausted standard (approved) treatment options - which either did not work for them, or they were unable to tolerate certain side effects. Clinical trials may provide another option when standard therapy has failed.
Other people participate in trials because the want to contribute to the advancement of medical knowledge.

For each clinical trial, researchers develop eligibility criteria, such as the age, sex, type and stage of disease, previous treatment history, and other medical conditions. These criteria help to reduce the amount of variation in the study, without threatening the scientific integrity of the trial, by removing medical variations that might complicate analyzing the results.

Not everyone who applies for a clinical trial will be accepted. Volunteers may be excluded based on the eligibility criteria and/or the number of participants needed by the researchers to collect enough information to determine the safety and effectiveness of a therapeutic agent.

6. Where are clinical trials conducted?

Clinical trials can be sponsored by an organization such as a pharmaceutical company, a federal agency - for example, the National Institutes of Health or Veterans Administration -or an individual, such as a physician or health care provider. The sponsor determines the location(s) of the trials which are usually conducted at universities, medical centers, clinics, doctor's offices, and/or at hospitals, federally- and industry-funded research sites.

Source: U.S. Food and Drug Administration. Basic Questions and Answers about Clinical Trials. Available at: http://www.fda.gov/oashi/clinicaltrials/clintrialdoc.html Accessed June 6, 2008.
Choosing to participate in a clinical trial is an important personal decision. The following frequently asked questions provide detailed information about clinical trials. In addition, it is often helpful to talk to a physician, family members, or friends about deciding to join a trial. After identifying some trial options, the next step is to contact the study research staff and ask questions about specific trials.

7. Why participate in a clinical trial?

Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.

8. Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. Using ClinicalTrials.gov inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" and those that disallow someone from participating are called "exclusion criteria".

These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

9. What happens during a clinical trial?

The clinical trial process depends on the kind of trial being conducted.  The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the ClinicalTrials.gov protocol is carefully followed and there is frequent contact with the research staff.

9. What is informed consent?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided.

Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

10. What are the benefits and risks of participating in a clinical trial?

Benefits: Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

• Play an active role in their own health care.
• Gain access to new research treatments before they are widely available.
• Obtain expert medical care at leading health care facilities during the trial.
• Help others by contributing to medical research.

Risks: There are risks to clinical trials.

• There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
• The experimental treatment may not be effective for the participant.
• The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

11. What are side effects and adverse reactions?

Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

12. How is the safety of the participant protected?

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.

13. What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

• What is the purpose of the study?
• Who is going to be in the study?
• Why do researchers believe the experimental treatment being tested may be effective?
• Has it been tested before?
• What kinds of tests and experimental treatments are involved?
• How do the possible risks, side effects, and benefits in the study compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the experimental treatment?
• Will I be reimbursed for other expenses?
• What type of long-term follow up care is part of this study?
• How will I know that the experimental treatment is working?
• Will results of the trials be provided to me?
• Who will be in charge of my care?

PROTOCOL: A study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Source: National Library of Medicine, "Understanding Clinical Trials". Available at: http://www.ClinicalTrials.gov/ct2/info/understand. Accessed June 24, 2009