Clinical Trial FAQs
What is a clinical trial?
A research study is the name given to a scientific study used to find out if and how a new or existing drug/treatment works. These can also be called clinical trials, clinical studies, or clinical research studies. A drug will not be approved to treat patients without going through these comprehensive studies.
Even after a drug is approved for doctors to prescribe, clinical research studies are conducted to increase our understanding of how a particular drug can be used most effectively and to compare it to other available treatments.
The drug being investigated in a research study is called an “investigational drug”.
Who can participate in a clinical trial?
All clinical trials have guidelines about who can participate. Using 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.
Why do people participate in research studies?
People participate in research studies for various reasons including:
- Learning more about their condition
- Gaining access to new investigational drugs not yet widely available
- Helping researchers understand if and how an investigational drug works
- Helping future generations to potentially benefit from new or improved treatments.
Without research studies; new drugs cannot be developed.
What are the benefits and risks of participating in a clinical trial?
Clinical trials that are well-designed and well-executed are good ways for eligible participants to:
- Play an active role in their own healthcare
- Gain access to new research treatments before they are widely available
- Help others by contributing to medical research
There are also risks to clinical trials:
- They may be unpleasant, serious or even life-threatening side effects to treatment
- The treatment may not be effective for all participants.
All potential benefits and risks will be reviewed with you prior to you joining a research study, called informed consent. For more information about this, see the “What is informed consent” topic in this FAQ.
It is also important to note that the protocol may require more of your time and attention than a non-protocol treatment, including trips to the study site, more treatments, testing, monitoring, hospital stays or complex dosage requirements.
What happens during a clinical trial?
The clinical trial process depends on the kind of trial being conducted (See “What are the different types of clinical trials” topic in this FAQ). The clinical trial team can include doctors and nurses as well as social workers and other healthcare 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.
Many research studies compare an investigational drug to an alternative treatment/s or another investigational drug. The alternative treatment might be an existing drug which doctors know to work well in the condition being studied, or it might also be a ‘placebo’. A placebo is drug which contains no active medicine.
Before enrolling in a study, potential participants are given information about the research study. If they are comfortable with the information and choose to participate, they are asked to sign an informed consent form.
In most research studies, participants are randomly assigned to receive either the investigational drug, or the alternative drug/s. Often neither they nor the study doctor know which study drug they receive because the tablets are disguised to look identical. Studies using this approach are called ‘blinded’ studies.
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 protocol is carefully followed and there is frequent contact with the research staff.
Once a trial is completed, the data from the study is analyzed. The data is used to determine if the medicine is safe and/or effective. The data is often published in peer-reviewed medical journals and a summary of the data is typically made available on public external websites such as www.clinicaltrials.gov or www.clinicaltrialsregister.eu. In addition, sometimes the study participants are provided with a summary of the trial results.
What is informed consent?
Informed consent is the process of understanding the key facts about a research study before deciding whether or not to participate. Informed assent is the same process, however, it is called assent as the person assenting is too young (or unable) to give legal consent.
For research studies conducted in children, the study doctor will explain the study to both the parent or legal guardian and the child. .
If all parties decide to take part in a study, the parent/legal guardian will sign an ‘Information and Consent’ document and the child will sign a simplified document called an ‘Information and Assent’ document.
What are side effects and adverse reactions?
Side effects are any undesired actions or effects of drug or treatment. Common examples of negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. New medicines must be evaluated for both immediate and long-term side effects and adverse reactions, as well as their effect on the condition under investigation.
Adverse events (AEs) are considered to be any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Adverse drug reactions (ADRs), on the other hand, consist of all noxious and unintended responses to a medicinal product in the preapproval clinical
experience that are related to any dose.
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 and research is also reviewed by independent ethics committees who also need to approve the study. The trial follows a carefully controlled protocol, which is a study plan that details
what researchers will do during 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. For more information see the “Confidentiality Regarding Trial Participants”
topic in this FAQ.
Every clinical trial conducted is approved in monitored by Institutional Review Boards (IRB) or Ethics Committees (ECs) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants
are protected. All institutions that conduct or support biomedical research involving people in the United States must, by federal regulation, have an IRB that initially approves and periodically reviews the research In other countries around the world these review committees are usually called Ethics Committees or ECs.
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 healthcare team questions about it, the care expected while in a trial, and the burden of the trial. The following questions might be helpful for the participant to discuss with the healthcare team. Some of the answers to these questions are found in the informed consent
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
- What kinds of tests and 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 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 treatment is working? Will results of the trials be provided to me?
- Who will be in charge of my care?
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- If possible, bring a tape recorder to record the discussion to replay later or bring a pen and paper to take notes of the discussion.
Can a participant continue to see their primary healthcare provider while in a trial?
Yes. Most clinical trials provide short-term treatments related to a specific illness or condition, but do not provide extended or complete primary health care. In addition, by informing a primary care provider about the research, the participant can ensure that other medications or treatments will not conflict with the protocol.
Can a participant leave a clinical trial after it has begun?
Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.
Where do the ideas for trials come from?
Ideas for clinical trials usually come from researchers and current unmet needs of patients. After researchers test new therapies or procedures in the laboratory and in animal studies, the treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about a new treatment, its risks and how well it may
may not work.
Who sponsors clinical trials?
Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, universities, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place
in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.
What is a protocol?
A protocol is 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.
What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
What are the different types of clinical trials?
- Natural History Trials are sometimes conducted before a Phase I trial. A natural history study does not involve any treatment or medicine, but is simply a study designed to learn more about how the disease or condition progresses in a population without treatment. These studies are most often conducted in rare diseases when little is known about the symptoms
of the condition
- Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or other therapy.
- Prevention trials 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 are conducted to find better tests or procedures for diagnosing a particular disease or condition.
- Screening trials test the best way to detect certain diseases or health conditions.
- Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
What are the phases of clinical trials?
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions: For more information about the phases of clinical trials see: Clinical Trials: The Basics
- In Phase I trials, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
- In Phase II trials, the study drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
- In Phase III trials, the study drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
- In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.