Frequently Asked Questions
- Why participate in a clinical trial?
- Who sponsors clinical trials?
- What is a protocol?
- What is a control or control group?
- What is a blinded study?
- What are the phases of clinical trials?
- Who can participate in a clinical trial?
- What happens during a clinical trial?
- What is informed consent?
- What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Does a participant continue to work with a primary healthcare provider while in a trial?
- What are the benefits and risks of participating in a clinical trial?
- How is the safety of the participant protected?
- Can a participant stop participating in a clinical trial after it has begun?
Why participate in a clinical trial? Back
People who participate in clinical trials have the opportunity to play a more active role in their own health care and to help others by contributing to medical research. Participants can gain access to new treatments before they become widely available, or receive existing treatments that are being evaluated for additional information on risks, benefits and optimal use.
Who sponsors clinical trials? Back
Clinical trials are sponsored or funded by a variety of organizations such as pharmaceutical companies, medical institutions, foundations and voluntary groups, in addition to federal agencies such as the National Institutes of Health (NIH).
What is a protocol? Back
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 the types of people who 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 monitored by the research staff to determine the safety and effectiveness of treatment.
What is a control or control group? Back
A control is the standard by which experimental therapies are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the other group, the control group, is given either a standard treatment or a placebo. A placebo is an inactive agent that has no treatment value.
What is a blinded study? Back
In a blinded study neither the participants nor the researchers know who is getting the experimental treatment and who is receiving a placebo or control treatment. At enrollment, researchers inform all patients that they may receive a placebo and that certain risks are associated with delaying treatment.
What are the phases of clinical trials? Back
Clinical trials are conducted in phases. Each phase has a different purpose and helps scientists answer different questions:
In Phase I trials, researchers test a new treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and well-tolerated dosage range, and identify side effects.
In Phase II trials, the experimental drug or treatment is given to a larger group of people (100-300) to identify an effective dosage and information about effectiveness.
In Phase III trials, the experimental treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness when compared to other treatments or a placebo and to monitor for side effects and collect safety information.
Who can participate in a clinical trial? Back
All clinical trials have guidelines about who qualifies to participate, called "inclusion and exclusion criteria." An important principle of medical research, these criteria help produce reliable results. Inclusion criteria dictate whether a person can participate in a clinical trial; conversely, exclusion criteria prevent a person from being able to join. These criteria are based on age, gender, the type and stage of a disease that the applicant has, previous treatment history, and other medical conditions. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. Inclusion and exclusion criteria are used to identify appropriate participants and keep them safe during the course of the study.
What happens during a clinical trial? Back
The exact process depends on the kind of trial being conducted. The clinical trial team may 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 it is completed.
Some clinical trials involve more tests and doctor visits than the participant would otherwise have for an illness or condition. Regardless of the trial type, the participant works closely 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.
What is informed consent? Back
Informed consent is the process of learning the key facts about the benefits and risks of a clinical trial before deciding whether to participate. It continues throughout the study as a way to provide information to participants. To help someone decide whether to participate, doctors and nurses first explain the details of the study. If the participant's native language is not English, translation assistance can be provided. As a second step, the research team gives the participant an informed consent form with details about the study, including its purpose, duration, procedures and key contacts, as well as risks and potential benefits. The participant then decides whether to sign the document. Once signed, informed consent is not a contract, and the participant may withdraw from the trial at any time.
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor? Back
The participant should plan ahead and write down possible questions. When it's time to speak to the research coordinator or doctor, the participant should ask a friend or relative to come along for support.
Does a participant continue to work with a primary healthcare provider while in a trial? Back
Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care.
What are the benefits and risks of participating in a clinical trial? Back
Benefits: Well-designed, well-executed clinical trials may be the best way for certain qualified individuals to play a more active role in their health care, gain access to new experimental treatments before they become widely available, obtain expert medical care at leading healthcare facilities during the trial, and help others by contributing to medical research.
Risks: Treatment may cause unpleasant, serious or even life-threatening side effects.
The treatment may not be effective. The protocol may require more of the participant's time and attention than a non-protocol treatment, including repeat trips to the study site, additional treatments, longer hospital stays, and complex dosage requirements. Surgical procedures related to a trial may pose potential risks.
How is the safety of the participant protected? Back
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, which is a plan that details what researchers will do in the study. As the clinical trial progresses, researchers report the results at scientific meetings, to medical journals and to various government agencies. Participants' identities are kept confidential.
Every clinical trial must be approved and monitored by an Institutional Review Board (IRB). 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 must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Can a participant stop participating in a clinical trial after it has begun? Back
Yes. A participant can leave a clinical trial at any time. When withdrawing from the trial, the participant should alert the research team and describe the reasons for leaving the study.