Ideas for clinical trials usually come from researchers. Researchers first test new therapies or procedures in the laboratory and in animal studies. The potential treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about a new possible treatment, its risks, and how well it may work.
Clinical trials are sponsored (ie, funded) by organizations or individuals: physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, as well as government agencies. Trials can take place in a variety of locations, including 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 help 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 an investigational treatment, while the control group is given either a standard treatment for the illness or a placebo.
- Treatment trials test new possible 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.
Side effects are any undesired actions or effects of a 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 help protect you.
Each trial follows a carefully controlled protocol, which is a study plan that 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.
Every clinical study must be approved and monitored by an Independent Ethics Committee (IEC) to make sure the risks are as low as possible and are worth any potential benefits. An IEC is a committee of physicians, statisticians, community advocates, and others that helps ensure that a clinical trial is conducted in an ethical manner and that the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by regulation, have an IEC that initially approves and periodically reviews the research.
Informed consent is the process in which researchers communicate with potential and enrolled participants about a clinical trial. It provides the potential benefits as well as the risks to the patient. It informs the patients that participation in the trial is voluntary, and that they may discontinue participation at any time.
The goal of the informed consent process is to protect people who enroll in clinical studies. The informed consent process starts when a potential participant first asks for information about a study and continues until the study ends.
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
Yes. 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 other medications or treatments will not conflict with the protocol.
Yes. You can leave a clinical trial at any time. When withdrawing from the trial, you should let the research team know about it and the reasons for leaving the study.
If you don’t qualify for a clinical trial because of your age, health conditions, or other factors, but your doctors believe you would benefit from the trial treatment, sometimes you may be able to get the treatment through “expanded access.” Expanded access is sometimes called “compassionate use” but may go by other names. Expanded access may be used to provide access to an investigational treatment for people with a life-threatening or serious disease for which there is no good existing treatment.
Health authorities may approve expanded access for a trial treatment if:
- Researchers are actively studying the trial treatment in well-controlled clinical trials, or if all trials have been completed
- There is evidence that the trial treatment may work well to treat certain patients
- The benefits of the trial treatment outweigh the possible risks
Learn more about expanded access for specific treatments at: ClinicalTrials.gov