Clinical Trial FAQs

Clinical research involves testing an investigational drug, device, or treatment method in humans. Each clinical trial must use a protocol that has been written according to government regulations and that is also approved by the pharmaceutical company sponsoring the study. An investigational drug may be:

• A drug that has not yet been approved for marketing (i.e., a drug that is not allowed to be sold);

• A drug that is currently on the market but that is being studied for a non-approved indication or a new route of administration, strength, or formulation;

• A drug that is being sold on the market by one company but that is being considered for in-license or acquisition by another company;

• A drug that contains known and established ingredients but has not yet been marketed by the company;
• A drug that is currently on the market but is being studied in a population for which the drug has not yet been approved, such as in children or in elderly patients.

Before an investigational drug, device, or treatment method can be studied in humans, its safety and effectiveness is carefully studied in the laboratory. However, this early research cannot predict how safe and effective the drug will be in humans. With any new treatment, there may be benefits as well as known and unknown risks. Clinical research trials are conducted to determine the degree to which promising new treatments will be safe and effective for patients.

Phase 1 studies involve testing the investigational drug in a small number of healthy adult volunteers. The only focus of these studies is safety. These trials are designed to determine how the drug is metabolized, or processed by the body and what pharmacologic actions the drug has. One goal of these studies is to determine the best way to administer the drugs (i.e., the preferred route).

Phase 2 studies help determine how safe and effective a drug is, common short-term side effects of the drug, pharmacokinetic effects of the drug (i.e., the amount of drug that gets into the bloodstream, how quickly it gets there, and how long it stays there) and pharmacodynamic effects of the drug (i.e., the effects of the drug on the body). These studies involve a larger group of patients, typically 100 to 200, and are usually conducted in patients who have the medical condition for which the drug is being studied. These studies are used to identify the dose range of the drug that will be tested in subsequent larger studies (i.e., Phase 3 studies).

Phase 3 studies provide additional information about safety and efficacy when the drug is used in the way it is intended to be marketed. These studies are initiated only after earlier studies have demonstrated that there is an acceptable risk to benefit profile (i.e., the risk of taking the drug is outweighed by the potential benefit of the drug to the patient). These large studies are usually conducted at many different study centers; up to 10 to 30 study centers and between 500 and 5,000 patients may be included. These studies are used to confirm the indication and best dosage for the drug.

Phase 4 studies are conducted after the drug is available for sale (i.e., post-marketing studies), and they evaluate how safe and effective the drug is when exposed to the “real” population. These studies evaluate parameters such as different formulations, dosages, treatment durations, and drug interactions. The Food and Drug Administration (FDA) may require a company to conduct certain Phase 4 studies as a provision for product approval. These studies may include new age groups, various races, and other types of patients. These studies are useful for detecting and defining previously unknown or inadequately quantified adverse events and related risk factors. The size of these studies is broad and can range from less than 100 to over 20,000 patients.

Clinical trials are sponsored by government agencies such as the National Institutes of Health (NIH), pharmaceutical companies, individual physicians or investigators, healthcare institutions such as health maintenance organizations (HMOs), and organizations that develop medical devices or equipment. Clinical trials can take place in a variety of locations, such as hospitals, surgical centers, universities, or doctors’/dentists’ offices.

A Principal Investigator (PI) is a person qualified by education, training, and experience to assume responsibility for the proper conduct of a trial. They are usually physicians. The PI bears ethical responsibility for the study and is the leader of the study team. Final responsibility for the quality control of study procedures and the integrity and scientific merit of study findings rests with the PI.

Study Coordinators, who are often nurses, are the “experts” of the protocol aside from the Principal Investigator. They have a high level of expertise and devote a great deal of time to the planning and conduct of a research project. They will assist you throughout the study during the consenting process, taking the study medication, performing evaluations, and answering any questions you may have. An experienced Study Coordinator is a valuable source of knowledge and guidance for patients.

People choose to take part in clinical trials for many reasons. Usually, they hope for benefits for themselves such as hope for a cure of disease, a longer time to live, an increased quality of life, or a way to decrease or eliminate pain. Some people choose to participate because of the financial compensation they receive for time and travel. Others may want to contribute to a research effort for more altruistic goals, knowing that medical research is essential to advancing healthcare and improving lives through new and better medicines.

People who participate in clinical trials are among the first to receive new research treatments before they become widely available. Sometimes these people have the first chance to benefit from a cutting-edge treatment. However, there is no way to know whether an investigational treatment will be effective for a patient. Even standard treatments, although effective in many patients, do not guarantee benefits for everyone. Some people in a study may not receive the active treatment and instead will receive a placebo (i.e., a sugar pill). Although all patients in clinical trials are carefully monitored both during and after conclusion of the trial, there are always known and unknown potential risks associated with new treatments. Patients should decide whether to participate in a study only after they understand both the potential risks and benefits.

All clinical trials have criteria that determine who is allowed to participate. You cannot participate in a study if you don’t meet all of the study criteria. These criteria often include such factors as age, type of disease, medical history, and current medical condition. Some studies seek volunteers with specific medical conditions and others seek healthy volunteers. It is important to note that these study entry criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Most investigative treatments can cause side effects and other health risks, which vary from patient to patient. The number of side effects, if any, that you may experience will depend on the type of treatment being studied and your medical condition. Because clinical trials evaluate new medical treatments, the potential risks of the treatments are not always known ahead of time. However, in every study several safeguards are implemented to protect the safety of the study volunteers. Before you agree to participate in a study, you should know what side effects may occur and, as much as possible, what unknown side effects or potential risks may occur. This information will be covered thoroughly during the informed consent process before you enroll in the study.

A protocol is the set of rules that must be followed during a clinical trial. It describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. Before a clinical trial can start, the FDA and IRB must review the protocol.

Informed Consent is a freely given agreement on the part of a competent person in full possession of all available, pertinent information to participate as a subject in a clinical research trial. Consent may only be given by the study subject, unless the subject is a minor or otherwise incapable of acting on his or her own behalf. If this is the case, consent may be given by the subject’s parent or legal guardian. Informed consent is documented by means of a written, signed, and dated Informed Consent form. Food and Drug Administration (FDA), International Conference on Harmonization (ICH), Good Clinical Practice (GCP), and Institutional Review Board (IRB) regulations require that Informed Consent be obtained before any subject may participate in a clinical trial.

A placebo is an inactive substance (pill, liquid, or powder) that has no treatment value. It may be referred to as a “blank” or “sugar pill.” In placebo-controlled clinical trials, experimental treatments are compared with placebos to assess the treatment’s effectiveness.

Some participants in these studies may receive placebo rather than active treatment. In pain studies, patients who receive placebo (as well as those who receive active treatment) will always have access to “rescue medication,” which is a standard pain medication given for the condition or procedure being studied. If the participant feels they need more pain medication, they will be given a rescue dose of the standard pain medication immediately. Asking for rescue pain medication will not in any way have a negative impact on study participation.

The term adverse event, as used in the context of clinical research, means any untoward medical occurrence in a patient, after the patient received a pharmaceutical product, and which does not necessarily have causal relationship with the treatment. Adverse events can be a symptom (e.g., a headache), a finding during a physical exam (e.g., elevated blood pressure), a syndrome or disease (e.g., “flu syndrome” or meningitis), or an abnormal laboratory value.

You should know as much as possible about the research study before you participate. You should feel very comfortable asking questions and that the staff answers them in a way you can understand. When preparing for a meeting with the study coordinator or doctor, you should plan ahead and write down the questions you want to ask. Review the study website to help you prepare questions to ask. You might ask a friend or relative to come with you for support and to hear the responses to your questions. You may want to ask the following questions when deciding whether to participate in a clinical trial:

• What questions should I ask?
• Why is this research being done?
• What is the purpose of the study?
• Who is sponsoring the study?
• Who has reviewed and approved this study?
• Why does the research team think the treatment, drug, or medical device will work?
• What kinds of therapies, procedures, or tests will I have during the trial?
• Will any of the therapies, procedures, or tests hurt? If so, for how long?
• How will the tests in the study compare to tests I would have outside the study?
• How long will the study last?
• How often will I have to go to the study site?
• Will I be able to take my regular medications during the trial?
• What medications, procedures, or treatments must I avoid while in the study?
• What are my responsibilities during the study?
• Will I have to be in the hospital during the study?
• Will the study researchers work with my doctor while I am in the study?
• Can anyone find out that I am participating in a study?
• Can I talk to other people in the study?
• Will I be able to find out the results of the trial?
• How do the possible risks and benefits of the study compare with approved treatments for me?
• What are the possible immediate and long-term side effects?
• Will I have to pay anything to participate in the study?
• What are the charges likely to be?
• Is my insurance likely to cover those expenses?
• What compensation should I expect for participating in the trial?
• Are there prorated compensation levels if I am unable to complete the trial?

Yes, you should continue your regularly scheduled visits with healthcare providers during clinical trial participation. Most clinical trials provide short-term treatments related to a designated illness or condition but do not provide extended or complete primary healthcare. In addition, by having your healthcare provider work with the research team, you can ensure that the other medications or treatments you are using will not conflict with the clinical trial protocol.

Yes, you can leave a clinical trial at any time. This is called “withdrawing your consent.” If you plan to stop participating in a study, inform the Study Coordinator or Principal Investigator that you would like to withdraw your consent. You should also inform the Study Coordinator or Principal Investigator of your reason for withdrawing.

You will typically be paid for your time and travel while participating in a clinical study. Surgery, medical exams, and medical procedures may also be performed at no cost to you depending on the type of study. If a study requires you to stay at a research facility for any length of time, you may also receive meals or snacks.