This issue of Informed Decisions provides a brief overview of the clinical trial process. Of course, every clinical trial is different; each study is conducted according to a detailed plan known as a protocol which specifies what is supposed to happen and when. The protocol includes a schedule for when to take the study medication and when to come in for clinic visits for physical exams, tests, and other procedures. The goal of this overview is to help you make a well-informed decision about clinical trial participation.
First Visit: Clinical Trial Participation
If you find a clinical trial that may be appropriate for you, you may want to discuss this with your doctor, the study coordinator, and other family or friends. The next step is to schedule a visit to the clinic or hospital where the study is being conducted to determine if you qualify for the study. During this first visit, often called a screening visit, you will be asked questions about your medical history to see if you match the eligibility criteria for the clinical trial.
Every clinical trial has a list of criteria specifying who can take part in the study. These guidelines are called eligibility criteria or inclusion/exclusion criteria. The criteria are listed in the study plan or protocol and include such characteristics as gender, age, health problems, and previous or current treatments.
The eligibility criteria are designed to ensure the scientific validity of the study and to protect participants in the study. These criteria are important is in producing scientifically reliable results. For example, only people with a confirmed diagnosis for the relevant disease or condition are eligible to participate to ensure the study drug is being properly evaluated. The criteria exclude those who might have difficulty with the study drug or procedures involved in the research. For example, a person who takes a medication that has been shown to have an adverse effect when taken with the study drug will not be allowed to participate.
Before enrolling in a clinical trial, the study doctors and nurses will explain the key facts, risks, and benefits about the study and answer any questions that you may have. This is a process that is known as informed consent. Informed consent begins before you agree to participate in a trial and continues as long as you are in the study.
The process of informed consent includes verbal discussions and the written informed consent document. You will be given a written consent form that you can take home to read and discuss. You will be asked to sign the written form before beginning the clinical trial.
The March 15, 2002 issue of Informed Decisions, Practical Guide to the Informed Consent provides additional information about the informed consent process and includes a sample informed consent form and explanations of why each section of the written form is necessary and important.
Participation in a clinical trial begins when you qualify for the study at the screening visit and sign the informed consent form. In studies involving more than one study drug or a study drug and a placebo, you will be randomly assigned to receive one of the study medications. You will be given a schedule of study visits and procedures with instructions describing what to do, when to take study drug and where the visits will take place. Studies could include the following types of visits: home visits with a visiting nurse, telephone visits, outpatient visits to the hospital or clinic and inpatient stays in the hospital. You will have contact numbers of study staff if you have questions at any time. It is important for you to follow all instructions, for example, going to all scheduled visits, having all tests and study procedures and taking study drug on time.
Most studies have roughly three periods or phases: baseline, treatment and follow-up.
Baseline visits usually occur before you receive the study drug or investigational procedure. This is also called a lead-in period. This may consist of one visit or more visits. In some studies, there is a wash-out period when participants are asked to stop taking the medication prescribed for the condition being studied for a brief period before starting the study medication. During the baseline period, some of the procedures listed in the study plan will be performed including measurement of vital signs, physical examination or blood and urine testing. These results will later be compared with additional tests and procedures performed after you receive study drug to see how the drug is affecting you.
After the baseline period, treatment with the assigned study medication begins. The amount of time you will take the study drug is defined in the study protocol. Treatment may consist of taking only one dose of study drug or may consist of taking the study drug for a longer period of weeks or months. You will usually take the study drug for the first time at the dosing visit. The testing procedures listed in the study plan will be performed within a specified amount of time after you take the study drug. If treatment consists of more than one dose of study drug, after completing the dosing visit, you will be given study drug, a schedule and instructions for the rest of the treatment period.
If there is a possibility of receiving a placebo (sugar pill or a substance that looks like the treatment but contains no active drug), you will be told this before you start participation in a study. In some studies, when one group receives placebo during the study, they will be given the opportunity to participate in an open-label extension study where all participants receive the active study drug for a specified amount of time after completing the clinical trial.
Each study will then have a series of one or more follow-up visits at which you will have procedures performed to monitor the effects of the drug over time. The study staff will compare the results from these tests and procedures to baseline results. The staff records any side effects or reactions to the study drug that occur.
To ensure patient safety, the protocol or study plan must be reviewed and approved by an Institutional Review Board (IRB) at each hospital or institution where the study will be conducted. This board consists of people with a variety of backgrounds including clergy, health professionals and patient advocates. In the review of the protocol, the IRB tries to ensure that participants in the study will not be exposed to unreasonable or unethical risks.
Drugs used in clinical trials have been extensively tested in laboratory experiments and animals prior to use in humans. However, some side effects do not become apparent until the drugs are given to humans. Side effects can vary from patient to patient. Periodic reviews of test results and other procedures are carried out while the trial is underway to monitor for side effects. If the study drug is found to be harmful to the study participants or if the risks appear to outweigh the benefits, the trial will be stopped. A trial may also be stopped if it is going exceptionally well and the new treatment or procedure appears to be so effective that the study drug should be made available to the general public.
If any new information becomes available during your participation that may affect your decision to participate in the study, for example, serious side effects or risks, you will be notified by the study staff. You may end your participation in the study at anytime with no penalty. If you do withdraw or the study is stopped early, you may be asked to complete follow-up procedures to ensure your safety.
After the Clinical Trial
After you finish participating in a clinical trial, your name and address will be kept on file by the study staff in case they need to contact you in the future.
Throughout the course of a study, researchers collect data in order to find out how well the treatment under investigation is working. At the end of the study all of the data is carefully evaluated to decide what the next step for the investigational treatment will be. For treatments that have completed early phase trials, researchers will decide if the treatments should move into the next phase, or if they should halt testing because the treatment is either not effective or not safe. When a final phase (phase III) trial ends, the data is evaluated in order to determine if it is effective or more effective than the standard treatment it was compared to in the trial.
Results from some studies are presented at conferences or published in scientific or medical journals. The results will also undergo a process of review by the FDA (Food and Drug Administration) or agencies from other countries, where experts will review the study results to make sure its analysis and conclusions are accurate and valid. Once a treatment has been shown to be safe and effective it is approved by the FDA and becomes generally available as another treatment option for patients and physicians to consider.
Informed Decisions article by John Yee, M.D., M.P.H. Veritas Medicine
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