FREQUENTLY ASKED QUESTIONS
- What is a clinical trial?
- What is a protocol?
- Where are clinical trials carried out?
- What happens during a clinical trial?
- What happens when a clinical trial is over?
- What are the potential risks and benefits of clinical trials?
- How safe are clinical trials?
- What is informed consent?
- Who pays for the patient costs on a clinical trial?
- Who is eligible to take part in a clinical trial?
- What are your rights if you take part in a clinical trial?
What is a clinical trial?
Clinical trials, also known as clinical studies, are research studies in which scientists and doctors test new drugs and treatments to see if they will improve health. Many of today's treatments for cancer are based on the results of past clinical trials. Because of progress made through clinical trials, many people treated for cancer are now living longer.
Clinical trials are divided into four phases.
Phase 1 trials: These trials are the first time a new drug or treatment is given to humans. They are normally carried out in a small number of volunteers (typically 6-20 people) who may include healthy volunteers or patients with the disease for which the product is intended as a treatment in order to find out how safe the treatment is. They also look at how a new drug should be given (by mouth, injected into muscle or the bloodstream, etc.), how often and at what dose. Phase 1 trials can also involve patients for whom standard therapies have failed and for whom no other therapies are available.
Phase 2 trials: These trials involve larger numbers of people (typically 12 - 50). Phase 2 trials continue to look at safety of the therapy but also test how well the new drug or treatment works in patients with different disease types.
Phase 3 trials: These are large studies (100+ people) that look at how well a new drug or treatment works in comparison to current therapies to see which treatment is better. Those taking part are usually divided into two treatment groups: standard treatment versus new treatment.
Phase 4 trials: These trials are usually carried out after the drug or treatment has been approved by the FDA and is readily available for use in the general patient population. The purpose of Phase 4 trials is to continue to study the effects of the drug or treatment on different populations and to look for side effects associated with long-term use.
What is a protocol?
Clinical trials use written guidelines called protocols. The protocol explains what the trial hopes to accomplish, how the trial will be carried out, and why each part of the trial is necessary. For example, the protocol includes:
- The reason for doing the trial
- How many people will be in the trial
- Who is eligible to take part in the trial
- What study drugs participants will need to take
- What medical tests participants will have and how often
- What information will be gathered
Every doctor or research center that takes part in the trial uses the same protocol. This makes sure that patients are treated identically no matter where they are receiving treatment, and that information from all the centers taking part can be combined and compared.
Protocols are written by the trial sponsors (those conducting the trial) and must first be reviewed by an Institutional Review Board (IRB) at each participating hospital or medical center before they can begin. IRBs are committees that oversee the safety of people who take part in clinical trials.
Where are clinical trials carried out?
Depending on the type of trial, patients may be treated at one or hundreds of clinical sites at the same time. Clinical sites can include large specialty treatment centers, university hospitals, local medical centers or a doctor's office. Each patient takes part in the trial under the guidance of a team including his/her own doctor and other health care professionals, who report the patient's experience back to the center responsible for the trials' overall coordination. Experts then use the information from all participants to see if the treatment being tested is safe and effective.
What happens during a clinical trial?
If you decide to participate in a clinical trial, you will work with a research team. Team members may include doctors, nurses, social workers, and other health care professionals. They will provide your care, monitor your health carefully, and give you specific instructions about the study.
Taking part in a trial may mean that you will have more tests and doctor visits than you would if you weren't in the study. Team members also may continue to stay in contact with you after the trial ends. To make the trial results as reliable as possible, it is important for participants to follow the research team's instructions. That means having all doctor visits and tests, taking medicines on time, and completing logs or answering questionnaires.
What happens when a clinical trial is over?
After a Phase 1 or 2 trial is completed, the researchers look carefully at the data collected during the trial and decide whether to:
- Move on to the next phase trial with the treatment, or
- Stop testing the treatment because it is not sufficiently safe or effective
When a Phase 3 trial is completed, the results maybe submitted to the FDA to consider whether to approve the new treatment for commercial sale.
What are the potential risks and benefits of clinical trials?
Potential benefits include:
- Access to investigational new drugs and therapies before they are available on the market
- A more active role in your own health care
- Close monitoring of your health care and any side effects
- An opportunity to make a valuable contribution to medical research
Potential risks include:
- Investigational new drugs and therapies may have unknown side effects
- Side effects may be worse than with standard therapies
- Even if the approach has benefits, it may not work for you
How safe are clinical trials?
The U.S. government has a system designed to protect people who take part in clinical trials. Before a clinical trial can begin, the trial plan (also called a protocol) must be approved. During the trial, review committees such as Institutional Review Boards (IRBs), make sure that the plan is being followed and participants are being protected.
Researchers performing studies are required by law to inform patients about a study's treatment and their possible benefits and risks before a patient decides whether to take part in the study. This process is called informed consent and continues as long as you are in the study. You will also be told of any new information discovered during the study that might affect your willingness to stay in the study.
What is informed consent?
Informed consent is a process in which a patient learns all the key facts about the clinical trial before deciding whether to take part. A doctor or research nurse may explain all that is involved. In addition, these facts will be given to you in a written consent form that you may take home to read and discuss. The consent form will included details about:
- The study approach
- The possible risks and benefits
- The tests and procedures you will have if you take part
Patients interested in taking part are encouraged to ask questions until they have all the information needed. Informed consent continues as long as you are in the study. You may change your mind at any time and leave the study whenever you want. Should you wish to stop participating in the study, standard therapy will remain available to you.
Who pays for the patient costs on a clinical trial?
Even if you have health insurance, your coverage may not include some or all of the costs associated with a clinical trial. This is because some health care plans view clinical trials as 'experimental' or 'investigational' procedures. The National Cancer Institute (NCI) is working with major health care providers to find a solution to this coverage problem.
For Vical studies, Vical will be responsible for providing the study drug and for all evaluations, scans, and treatments required by your participation in the study. Either you or your insurance company will be billed for any routine medical care for problems or complications of your disease unrelated to the administration of the study drug. This includes routine blood tests, x-rays, scans, medications and physician charges.
You will not be paid for participation in any study. Your doctor will make available or arrange for appropriate care and treatment for any physical injury resulting directly from participating in the study.
Who is eligible to take part in a clinical trial?
Each study has guidelines for who can take part. These are called eligibility criteria. Generally those who take part in the study are alike in certain aspects such as type and stage of disease, age, gender or previous treatments. The eligibility criteria are included in the study plan (also called a protocol).
What are your rights if you take part in a clinical trial?
Taking part in a study is voluntary. You may choose not to take part or may leave the study at any time. Leaving the study will not result in any penalty or loss of benefits to which you are entitled.
The study sponsor (those running the trial) will tell you about new information that may affect your health, welfare, or willingness to stay in the study.