The following questions are intended for anyone in the process of deciding to join a research study at the University of Virginia. We hope the information will help you make the right decision for yourself about being in a research study at UVa.
Clinical research is done to find out:
- What new drugs, devices and other treatments work and don’t work
- What causes disease
- What prevents disease and improves health
Ideas for clinical trials come from researchers, pharmaceutical companies and/or medical device companies. After researchers test new therapies or procedures in the laboratory and in animal studies, the treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about a new 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, 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.
- Treatment trials test new 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, vitamins, vaccines, 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.
It is important to test drugs and medical products in the people they are meant to help. It is also important to conduct research in a variety of people because different people may respond differently to treatments.
Some people participate in clinical trials because they have exhausted standard (approved) treatment options – which either did not work for them, or they were unable to tolerate certain side effects. Clinical trials may provide another option when standard therapy has failed.
Other people participate in trials because they want to contribute to the advancement of medical knowledge.
Not everyone who applies for a clinical trial will be accepted. Volunteers may be excluded based on the eligibility criteria and/or the number of participants needed by the researchers to collect enough information to determine the safety and effectiveness of a therapeutic agent.
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.
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. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. 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.
The clinical trial process depends on the kind of trial being conducted. 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.
Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:
- Play an active role in your own health care.
- If the new treatment works, you may be one of the first people to benefit.
- Help others by contributing to medical research.
- Access to expert medical care for the condition being studied, since investigators are often specialists in the disease area being studied.
- The trial sponsor may pay for some of your medical care or tests.
There are also risks to clinical trials:
- Side effects may be worse than those of the standard treatment.
- Side effects may occur that the doctor does not expect.
- New treatments do not always turn out to be better than, or even as good as, standard treatment.
- As with standard treatment, the new treatment may not work for you even if it works for other patients.
- The protocol may require more of your time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
Side effects are any undesired actions or effects of 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.
Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.
All participants in clinical trials are volunteers. You can choose to quit a clinical trial at any time, but talk to your doctor first. Your doctor can tell you how quitting the trial might affect your health and if there are other treatment options. Your relationship with your health care providers will not be changed by your decision.
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; 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.
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help researchers answer different questions:
- In Phase I trials, researchers test a new 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 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 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 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.
Most of the time, when you take part in a clinical trial, you will only be in that one phase of the study. Treatments move through the phases, but patients do not.
An Institutional Review Board (often abbreviated as “IRB”) is a committee or panel comprised of medical and non-medical members who study and properly consider all clinical protocols involving human subjects. The involvement of human subjects in research is not permitted until an appropriate IRB has reviewed and approved the research protocol and informed consent. When reviewing a proposed clinical research trial, the IRB assesses such things as:
- the risks to the subjects
- the anticipated benefits to the subjects and others
- the importance of the knowledge that may reasonably be expected to result
- the informed consent process to be employed
If your doctor offers you a clinical trial, you will first go through a process called informed consent. The goal of informed consent is to make sure you understand the clinical trial’s plan. A member of the research team will review the informed consent form in detail with you. This form explains the clinical trial’s purpose, plan, risks and benefits.
This is a great time to ask questions. Try to bring a family member or friend to help you ask questions and write down answers. You may also want to bring a tape recorder so that later you can listen to what the doctor said. If you decide to join the clinical trial, you will be asked to sign the informed consent form. The research team member will also sign the form, and you will receive a copy.
Take time to make your decision. If you like, you may take the informed consent form home with you to review before signing it.
You 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 you to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the main purpose of this study?
- Who is going to be in the study?
- Why do researchers believe the treatment being tested may be effective? Has it been tested before?
- Does the study involve a placebo or a treatment that is already on the market?
- How long will the study last?
- What will I be asked to do as a participant?
- What kinds of tests and treatments are involved?
- What has been learned about the study treatment and are any study results published?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- Do I have to pay for any part of the study? Will my insurance cover these costs?
- Is there any reimbursement for travel costs? Will I receive any compensation for being in the study?
- Will I be able to see my own doctor?
- Can anyone find out whether I’m participating in the clinical trial?
- Will I receive any follow-up care after the study has ended?
- What will happen to my medical care if I stop participating in the study?
- How will I know that the treatment is working? Will results of the trials be provided to me?
- If the treatment works for me, can I keep using it after the study?
- Who will be in charge of my care? Does the physician/investigator have any financial or special interest in the clinical study?
- What are the credentials and research experience of the physician and study staff?