FAQ

In healthcare, a clinical trial or research study is done to determine the safety and effectiveness of an investigational drug or other therapy, often compared to what is already on the market. Sometimes we study a medication approved for one condition to see if it helps a different condition.

There are many reasons why people choose to participate in a study. Volunteers gain access to investigational drugs that are otherwise unavailable to the public. They get the opportunity to help researchers learn more about health and disease, and possibly improve the health of others. Volunteers will also learn more about their own health condition from medical professionals dedicated to clinical research.

In addition to the benefits of receiving medication, participants are typically compensated for their time and travel with an amount of money approved by the IRB. The amount is based on the specifics of the  trial, and in some cases no compensation is given.

Participation is based on meeting specific conditions, called “Eligibility Criteria”. Every study has detailed criteria specifying the age, sex, and physical conditions that must be met for qualification. Your participation in a research study will include a careful screening evaluation to make sure you meet these requirements.

Every medication, approved or investigational, has potential side effects; however, the side effects of investigational drugs may be largely or completely unknown. Some people may react to a medication more than others, such as an allergic reaction. An important aspect of participation in a clinical trial is the frequency of visits to the study offices to meet with the study doctor and to check for possible side effects. All medications used in clinical trails have been previously tested in animals before human trials can begin, but some side effects may not be known until they are tested in humans.

Each clinical trial has a specific study plan. All clinical studies begin with an informed consent process in which the potential participant reads a document (the Informed Consent) that explains all study procedures, risks and potential benefits. The potential participant and the study doctor will discuss the study, procedures, and what is currently known about the study medications. No study procedures will be completed until the participant fully understands the trial, has had all questions answered, and agrees to participate. The study procedures usually involve multiple visits to the study offices and general medical procedures such as a brief physical examination, an ECG and/or blood draw. The study Informed Consent contains detailed information about the procedures required for a specific study.

The length of a clinical study will vary depending on what is being studied. Participants are told how long the study will last before enrolling. Some trials can last less than a year, while others can last several years.

Every clinical study is led by a principal investigator, who is always a medical doctor. You can read about our principal investigator here, along with the rest of our team. Clinical studies have research teams that may include doctors, nurses, social workers, and other health care professionals.

Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations, in addition to Federal agencies such as the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs. Physicians, health care providers, and other individuals can also sponsor clinical research.

When participating in a clinical trial, you need to sign an Informed Consent form. On this form is a detailed explanation of the trial’s purpose and the types of treatment being studied. It also addresses the probability of you receiving a treatment, the known risks and benefits of the treatment, and any costs related to the study that you may be expected to pay. The form also lets you know who to contact for more information, and explains your rights in the trial, including the right to confidentiality, the right to medical treatment for trial-related injury, and the right to withdraw from the study without penalty or loss of other medical care.

Clinical trials are conducted in a series of phases, or steps. Each phase is designed to answer a separate and specific question.

Phase I — Phase I studies are primarily concerned with assessing the drug’s safety in healthy volunteers. This initial phase of testing in humans is done in a small number of healthy volunteers (20 to 100), who are usually paid for participating in the study. The study is designed to determine what happens to the drug in the human body–how it is absorbed, metabolized, and excreted. A phase I study will investigate side effects that occur as dosage levels are increased. This initial phase of testing typically takes several months. About 70 percent of experimental drugs pass this initial phase of testing.

Phase II — Phase II study is conducted once a drug is proven to be safe. It must then be tested for efficacy. This second phase of testing may last from several months to two years, and involve up to several hundred patients. Most phase II studies are randomized trials. One group of patients will receive the experimental drug, while a second “control” group will receive a standard treatment or placebo. Often these studies are “blind”–neither the patients nor the researchers know who is getting the experimental drug. In this manner, the study can provide the pharmaceutical company and the FDA comparative information about the relative safety of the new drug, and its effectiveness. Only about one third of experimental drugs successfully complete both phase I and phase II studies.

Phase III — Phase III study is conducted and tested in several hundred to several thousand patients. This large scale testing provides the pharmaceutical company and the FDA with a more thorough understanding of the drug’s effectiveness, benefits, and the range of possible adverse reactions. Most phase III studies are randomized and blinded trials. Phase III studies typically last several years. 70 to 90 percent of drugs that enter phase III studies successfully complete this phase of testing. Once a phase III study is successfully completed, a pharmaceutical company can request FDA approval for marketing the drug.

Phase IV — Phase IV studies are performed after obtaining a license to market the drug. Contrary to Phases II and III, problems associated with long term use of the drug or rare adverse events can be detected. In late phase III/phase IV studies, pharmaceutical companies have several objectives: (1) studies often compare a drug with other drugs already in the market; (2) studies are often designed to monitor a drug’s long-term effectiveness and impact on a patient’s quality of life; and (3) many studies are designed to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies.

Placebo-controlled studies are a way of testing a medication in which, in addition to a group of subjects that receive the treatment being evaluated, a separate group receives a “placebo” treatment which is specifically designed to have no real effect. The purpose of the placebo group is to account for the placebo effect, that is, effects from treatment that do not depend on the treatment itself. Such factors include knowing one is receiving a treatment, attention from health care professionals, and the expectations of a treatment’s effectiveness by those running the research study.

A randomized study is when two or more alternative treatments are selected by chance, not by choice. The treatment chosen is given with care and expertise, then the results of each treatment are compared. Analyses are done at intervals throughout the trial. As soon as one treatment is found to be superior, the trial is stopped. This way, the fewest number of patients receive the less beneficial treatment.

A randomized study is when two or more alternative treatments are selected by chance, not by choice. The treatment chosen is given with care and expertise, then the results of each treatment are compared. Analyses are done at intervals throughout the trial. As soon as one treatment is found to be superior, the trial is stopped. This way, the fewest number of patients receive the less beneficial treatment.

In single- or double-blind studies, the participants don’t know which medicine is being used. This allows them to describe what happens without bias. Blind studies are designed to prevent anyone from influencing the results. This creates more accurate conclusions. In single-blind studies, only the patient is not told what is being given. In a double-blind study, only the pharmacist knows; the doctors, nurses, patients, and other health care staff are also uninformed. If medically necessary, it is always possible to find out what the patient is taking.

Clinical trial results are often published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups. Once a new approach has been proven safe and effective in a clinical trial, it may be applied as standard practice.

The researchers look carefully at the data collected from the trial before making decisions about the meaning of the findings and further testing.

Clinical trial results are often published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups. Once a new approach has been proven safe and effective in a clinical trial, it may be applied as standard practice.

– See more at: http://www.fccc.edu/cancer/clinicalTrials/faq.html#end-trials

The researchers look carefully at the data collected from the trial before making decisions about the meaning of the findings and further testing.

Clinical trial results are often published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups. Once a new approach has been proven safe and effective in a clinical trial, it may be applied as standard practice.

– See more at: http://www.fccc.edu/cancer/clinicalTrials/faq.html#end-trials

The researchers look carefully at the data collected from the trial before making decisions about the meaning of the findings and further testing.

Clinical trial results are often published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups. Once a new approach has been proven safe and effective in a clinical trial, it may be applied as standard practice.

– See more at: http://www.fccc.edu/cancer/clinicalTrials/faq.html#end-trials

Learn about AAMRC  Current Studies now Enrolling and if you’d like to participate in one of our research program please Request an Appointment. One of our highly dedicated staff members will contact you and explain the study, go over required procedures, and determine if you may be eligible. If you qualify for one of our research programs, we will schedule an appointment for you to meet with the study coordinator and study physician.