Clinical trials FAQ

What is a clinical trial? Why are they performed?

Clinical trials are research studies conducted in humans. Often, the purpose is to determine whether a new investigational drug (or diagnostic or treatment approach) is sufficiently safe and effective to allow its use in the general patient population.

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What are the phases of clinical trials?

Clinical trials are conducted in four phases:

• Phase I studies are the first studies conducted in humans—they examine the safety and tolerability of a new drug in a small number of healthy male volunteers or male and female patients.
• Phase II studies examine the safety and efficacy of an investigational drug in larger number of patients and establish the optimal dose.
• Phase III studies further examine safety and efficacy in a yet larger number of patients. The new drug may be compared to placebo or the current standard of care. Phase III studies are sometimes called pivotal studies, and if successful, may lead to approval of the new drug by the competent regulatory authorities if all further regulatory and legal requirements for the approval of the new drug are fulfilled.
• Phase IV studies are conducted on approved drugs to provide broader experience with the drug. They may examine long-term effects, adverse events, and optimal use of the drug.

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What do randomization and blind/double-blind mean?

Randomization refers to the process of randomly assigning patients (usually by computer) to the different treatments being tested in a clinical trial. Randomization ensures that each treatment group contains a similar patient population. Most Phase III studies are randomized.

Blind means that the patients and/or researchers don’t know which patients are receiving which treatment.  In a single-blind study, either the patient or investigator does not know which group a patient is in. In a double-blind study, neither the patients nor the investigators know which group a patient is in. The purpose of blinding is to minimize bias.

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What is a protocol?

A protocol outlines in detail all aspects of the clinical study—including who can participate, what treatments are to be given, and how tests should be performed. Every study center follows the same protocol for every participant.

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How is the wellbeing of participants protected?

The clinical trial protocol must be approved by regulatory authorities and independent committees charged with safeguarding the rights and interests of participants. Participants remain under close and continuous observation during the trial.

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What is informed consent?

Informed consent refers to the process by which potential study participants are informed about the clinical trial through discussions with the nurses and doctors in the study as well as an informed consent document that explains the study. Participation in a clinical trial is entirely voluntary, and thus the informed consent process is designed to help patients make the decision of whether or not to participate.

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Who can participate in a clinical trial?

The requirements for eligibility are specific to each clinical trial. Criteria are based on factors such as the type and stage of disease, age, gender, and medical history. Inclusion criteria are those characteristics that are required for a person to participate in the study, while exclusion criteria are those characteristics that prevent a person from participating in the study. These criteria serve to ensure that participants are recruited appropriately for the study.

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Who sponsors clinical trials?

Clinical trials are sponsored by a wide variety of medical institutions including pharmaceutical and biopharmaceutical companies, universities, private organizations, research centers, governmental institutions, and other health care institutions.

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Where are clinical trials conducted?

Clinical trials take place in a variety of locations, including hospitals, clinics, and doctors’ offices. Each trial has its designated locations.

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What are the benefits and risks?

In every clinical trial, there are benefits and risks. Potential benefits may include access to a new, as yet unavailable treatment (which may be especially appropriate to patients whose current or previous therapy has not been particularly successful) and continuous medical care. Risks include the potential for unexpected, also serious, side effects, the chance that therapy will be ineffective, and additional out-of-pocket expenses.

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How can I find out about the results of a trial?

If the results have been published, you can find them at www.clinicaltrials.gov on the trial’s web page. Detailed instructions on how to search for the results are posted on the website.

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How do I find a clinical trial that is right for me?

Patients typically talk to their doctors first to find out whether a clinical trial might be available for their medical condition. Other ways to find clinical trials is by searching the internet (including www.clinicaltrials.gov) or contacting a patient advocacy association. A discussion with family and friends may be helpful. If you find a clinical trial you are interested in, talk to your doctor to find out more.

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How do I get started?

After you or your doctor has identified a study that you may be eligible for, your doctor can refer you to a study center location. The study center will contact you to schedule a visit to determine your eligibility.

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Can I leave a clinical trial after I’ve started the treatment?

Yes. Participation is entirely voluntary. Patients may leave at any time—before the trial starts, during the trial, or even during the follow-up period after the trial.

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