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Clinical Trials

FDA on Clinical Trials: What Patients Need to Know

FDA on Basics About Clinical Trials

Debunked: Clinical Trial Myths

Myth: Clinical trials are only for people who have no other standard treatment options.

A wide range of people patriciate in clinical trials, for many different reasons. Although some participate after they’ve exhausted all approved treatment options, others join to test new combinations of drugs, new surgical procedures, or so researchers can look at other areas of care, like improving quality of life. Many healthy volunteers also participate in clinical trials!

Myth: Children can’t participate in clinical trials.

New drugs and treatments need to be studied in children in the same way they need to be studied in adults. Clinical trials in children help to gather important information on safety, efficacy, and dosing. Children are enrolled in clinical trials with the consent of their parent or guardian. 

Myth: Clinical trials are not safe.

There is a risk of experiencing serious adverse side effects when participating in a clinical trial, but several safeguards are in place to make every clinical trial as safe as possible. An Institutional Review Board made up of doctors, scientists, and laypeople must approve all clinical trials in the United States and are dedicated to ensuring participants are not exposed to unnecessary risks. Before joining a clinical trial, potential participants must undergo the informed consent process where they are made aware of the risks, benefits, and alternatives to participating. 

Myth: If I participate in a clinical trial, I might get a placebo instead of a real treatment.

Depending on the type of study, participants may be randomly assigned to a test group receiving the experimental treatment or a control group receiving a placebo/standard care. This allows researchers to compare the results from the two groups and determine if they happened by chance or because of the intervention. Patients who are randomly assigned to a placebo (often referred to as the control group), cannot be more likely to die or experience substantial harms or severe discomfort, than those in the treatment group. Furthermore, patients who receive a placebo at the beginning of a trial may have the opportunity to receive the study drug at a later time.

Myth: I can’t leave a clinical trial after I started if I change my mind.

Even after providing Informed Consent and starting treatment, a participant can withdraw at any time, for any reason.

Myth: Clinical trials are too expensive, and my insurance won’t cover the cost.

When participating in a clinical trial, most patients won’t have an increase in their typical cost of care. Generally, a participant’s health insurance will cover charges related to tests and treatments for standard of care, and participants are often not billed for things done solely for research purposes. A section of the informed consent will breakdown what costs patients can expect.

Myth: My doctor will tell me about any potential clinical trials that may help me.

Doctors are not always aware of all clinical trials, so they may not know if a patient is potentially eligible for one. Patients can learn about current clinical trials at www.clinicaltrials.gov.

Phases of Clinical Trials

After undergoing pre-clinical research to determine if a new drug or treatment has the potential to improve the care of patients, it enters into clinical trials. Clinical trials assess the safety and efficacy of a drug or treatment in people.

Phase 1

Phase 1 trials are primarily done to test a treatment of drug’s safety. A small group of 15 to 30 patients are given a new drug or treatment to help researchers identify side effects and determine a safe dosing range. If the treatment or drug is deemed safe, it can be tested in a Phase 2 trial.

Phase 2

In Phase 2 trials a larger group of patients are given the treatment. Researchers use this phase to further evaluate safety and determine if the treatment is effective. The new drug or treatment is not usually compared to the standard-of-care treatment at this point in the process. If the treatment is found to be effective, it can be tested in a Phase 3 trial.

Phase 3

Phase 3 trials enroll a large number of patients—usually 100 or more. Researchers continue to monitor treatment safety and efficacy and compare the new treatment or drug to the standard-of-care treatment to assess which works better. If one treatment is found to have much better results, the study will be stopped early.

Phase 4

Phase 4 trials take place after a treatment or drug is approved by the U.S. Food and Drug Administration. By testing the treatment in larger groups of patients, researchers can gather additional information on safety and side effects as well as how well it works and how it can best be used.

Phases of clinical trials. National Comprehensive Cancer Network.
What Are the Different Types of Clinical Research? U.S. Food and Drug Administration.


Chemotherapies and Surgery


Before enrolling its first patient, the study was stopped.


These clinical trials have finished normally and treatment and/or follow-up visits have ended.


These clinical trials have ended early and will not start again. When a clinical trial is terminated, it means participants stop being treated or followed. 


These clinical trials have been stopped early but they may start again.

Active, not recruiting

These clinical trials are already underway and new participants are not being enrolled.

Enrolling by Invitation

These studies are not open to the general public even if a person meets all eligibility criteria. Instead, a select group of patients determined by the researchers are invited to join the trial.


Patients who meet eligibility criteria are currently being enrolled in these clinical trials

Not yet recruiting

The researchers have not yet started recruiting patients for these clinical trials

Study Design

Cross-over assignment

Groups of participants are treated with multiple interventions in a specific order. For instance, participants in one group will receive drug A first and drug B second, while participants in another group with receive drug B first and drug A second.

No intervention arm

Participants do not receive any intervention or treatment.

Sham comparator arm

Participants receive a device or procedure that is made to look the same as the device or procedure that is being studied in the clinical trial, but does not have any active components

Placebo comparator arm

Participants receive a placebo, a treatment that has no therapeutic effect.

Active comparator arm

Participants receive a type of treatment that is already known to be effective

Experimental arm

Participants receive the treatment that is the subject of the clinical trial.


A group or subgroup of participants in a clinical trial (usually an interventional study) that receives the same intervention or no intervention, consistent with the study protocol. 


A group of participants who share the same characteristics. Cohorts are often used in observational studies. 

Observational study

A type of clinical study design where researchers observe participants but do not control the treatment they receive. These types of studies are usually done when it is difficult or unethical to randomly assign participants to experimental groups.

Interventional study

 A type of clinical study design that allows researchers to test a potential treatment (or intervention) in participants.

Informed Consent

The process used to make participants in clinical trials aware of potential risks and benefits of participation. 


An adolescent’s agreement to participate in a clinical trial. Before giving assent, the clinical trial must be explained in a way the child can understand. Additionally, both parents need to permit participation.

Inclusion Criteria

Systemic therapy

Cancer treatment that targets the entire body. Chemotherapy is the most common form of Systemic cancer therapy. 

Local control

Cancer that has not grown or spread past the primary site.


Cancer that has spread from where it started (call the primary site) to other parts of the body.


Cancer is not responding to treatment.


Cancer has returned after treatment and after a period where no cancer was detected.


Cancer or tumor that can be removed completely with surgery


Cancer or tumor that cannot be removed completely with surgery


All-cause mortality

Used to measure deaths from all causes that occurred during a clinical trial.

Serious adverse event

Any unwanted medical occurrence that is considered life-threatening, leads to hospitalization or increases existing hospitalization, disrupts the ability to conduct normal life functions, leads to congenital anomalies or birth defects, or results in death. 

Adverse event

Any unwanted medical occurrence that takes place during a clinical trial or within the follow-up period. These events may or may not be caused by participation in the clinical trial.