An Introduction to Clinical Trials
Choosing to participate in a clinical trial is an important personal decision. The following frequently asked questions provide detailed information about clinical trials. In addition, it is often helpful to talk to a physician, family members, or friends about deciding to join a trial. After identifying some trial options, the next step is to contact the study research staff and ask questions about specific trials.
FAQs Regarding Clinical Research:
- What is a clinical trial?
- Why participate in a clinical trial?
- Who can participate in a clinical trial?
- What happens during a clinical trial?
- What are the benefits and risks of participating in a clinical trial?
- What are “blind” or “masked” studies?
- What are side effects and adverse reactions?
- How is the safety of the participant protected?
- What should people consider before participating in a trial?
- What is an institutional review board (IRB)?
- Can a participant leave a clinical trial after it has begun?
- Where do the ideas for trials come from?
- Who sponsors clinical trials?
- What is a protocol?
- What are the different types of clinical trials?
- What are the phases of clinical trials?
- What happens after the trial?
- Can I expect to be paid for my participation in research?
A clinical trial or clinical research study is a study in human volunteers that seeks to answer a specific scientific or health question. Clinical studies may involve the testing of an unproven drug, cosmetic product, an investigational medical device, a diagnostic tool, a food or drink, or an existing therapy used in a new but untested way to see if the product is helpful in preventing, diagnosing, or treating a disease or condition. Clinical research studies could be either interventional or observational. Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured. Observational studies are those in which individuals are observed and their outcomes are measured by the investigators.
People who volunteer to participate in a study may be called human subjects, research participants, or another term that identifies their role as volunteers in a clinical research study.
The doctor or healthcare professional who conducts the clinical research study may be called:
- Clinical Investigator,
- Principal Investigator,
- Study Doctor.
It is important to remember that the role of a clinical investigator is very different from the role of the health care provider. While your health care provider is responsible for making health care decisions for you based upon your medical condition, a clinical investigator is conducting a research study and is responsible for carrying out the study as described in the study protocol or study plan.
This means that while the clinical investigator is responsible for carrying out the study in a way that minimizes research participant risks and maximizes the study benefits, the clinical investigator is limited by the requirements of the study with regard to the treatment options that can be provided to you, the research participant.
If you volunteer to participate in a research study, it is important that you maintain contact with your primary health care provider (your regular doctor) and other specialists before, during, and after your participation in the study to make sure that you have the best information about your health care decisions.
The people who assist the clinical investigator are part of the research team or study staff and generally include other doctors, nurses, study coordinators, and other healthcare professionals.
Finally, studies involving a product are generally paid for and supported by the company that developed the product. This company is called a sponsor. Some government agencies, such as the National Institutes of Health, also act as sponsors in funding clinical research.
Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.
All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called “inclusion criteria” and those that disallow someone from participating are called “exclusion criteria”. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion 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.
The clinical trial process depends on the kind of trial being conducted (See below ”What are the different types of clinical trials”?) The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.
Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. The research team will provide an informed consent document to read; this form will includes details about the study, such as its purpose, duration, required procedures, and key contacts. To help someone decide whether or not to participate, the doctors and nurses involved in the trial will explain the details of the study. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time; however to participate in clinical trial, the consent form must be signed.
Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the trial.
- Help others by contributing to medical research.
There are risks to clinical trials.
- There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
- The experimental treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
In many clinical trials, one group of patients will be given an experimental drug or treatment, while a control group is given either a standard treatment for the illness, or a placebo (a harmless “fake” drug), or no treatment at all.
In a “blinded” or “masked” study, participants do not know whether they are getting the drug being tested, or whether they are in the control group. The goal is to prevent the so-called “placebo effect” from influencing the results of the experiment. The placebo effect is the phenomenon of patients feeling better simply because they think they are receiving a helpful drug or treatment.
Sometimes, clinical trials are “double-blind” or “double-masked.” That means that neither the participants, nor the study staff members, know who is receiving the experimental drug and who is in the control group. Studies are performed in this way so that neither the patients’ nor the doctors’ expectations about the experimental drug can influence the observations and results.
Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants’ names will remain secret and will not be mentioned in these reports.
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
- What kinds of tests and experimental treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the experimental treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the experimental treatment is working? Will results of the trials be provided to me?
- Who will be in charge of my care?
Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.
Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.
Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, cosmeceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran’s Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics.
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages, study products; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.
Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
Screening trials test the best way to detect certain diseases or health conditions.
Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:
In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.
In Phase IV trials, post marketing studies delineate additional information including the drug’s risks, benefits, and optimal use.
Our staff will make sure that all questions have been answered to the participants’ satisfaction, at the conclusion of a clinical trial. Common questions center on the future availability of the study product or medications and, additional available treatments. The MIAAR staff and the study physician are committed to insure that volunteers leave each trial with a plan for continuing treatment and, when warrant, plans to be followed-up by your family physician. The staff at McDaniel Institute of Anti-Aging Research considers each trial volunteer as a participating partner. This partnership between volunteer and study doctor is based on the achievement of a common goal, which is the dedication to improving quality of life through the meticulous study of skin care products, medicines and devices for better treatment.
Can I expect to be paid for my participation in research?
Compensation for participating is a study varies from one study to next; this is often a decision made by the study sponsors. You may receive some type of reimbursement, whether in the form of actual payments, or specific compensation that may range from a small gift card or reimbursement of travel costs and meals.
The above information is provided by www.clinicaltrials.gov, a service of the National Institute of Health. It was last updated: 20 Sept 2007.