A clinical trial is a research study designed to evaluate potential new medical treatments. These studies are the result of a long and deliberate process that often takes many years to complete. Clinical trials test the safety and effectiveness of new or modified drugs, new drug doses, and varied combinations of treatments. Clinical trials are an integral component for improving the treatment of medical conditions because they lead to higher standards of care with state of the art medication.
Clinical trials essentially fall into two general categories:
- The first general category of clinical trials are designed to evaluate new drugs, compounds, or biologic agents that have not yet been approved. All substances must go through several phases of clinical trials to document their safety and effectiveness before approval is granted. Prior to approval, these substances are only available through clinical trials; however, after approval, they may be commercially available.
- Clinical trials may also evaluate drugs, compounds, or biologic agents already approved for use. These substances have already been determined to be safe by previous trials, and are now being evaluated for different doses or for new indications of use.
The informed consent that you will receive from the doctor should explain the major elements of the trial and answer questions you might have about the motivations for the study, what is required of you in order to participate, potential risks and benefits, alternatives for treatment and potential costs. Questions you may wish to ask include:
- Why is the study being done?
- Has the drug or treatment in this study been tested before?
- Who is sponsoring the study?
- What will happen during the study?
- What am I required to do to participate in the study?
- Will I need to be hospitalized to participate? If not, how often will I need to come to the study center?
- What are the possible risks or discomforts?
- What are the possible benefits of taking part in the study?
- What other treatment alternatives do I have?
- Will I receive the study drug or treatment during the clinical trial, or is it possible that I may be part of a control group receiving a placebo instead?
- Are there any costs involved in participating in the study or receiving the care as part of the study?
- Will I receive any compensation for participating in the study?
- What happens if I am injured as a result of my participation?
- What do I do if I need emergency care during the study?
- What are my rights as a participant in the study?
- What will be done with the information gathered from the study?
- Will I be able to get information about the results of the study?
- Who can I call with questions or problems?
Once you have had your questions answered to your satisfaction, you can consult with your health care providers, family and friends to decide whether participating in a clinical trial might beneficial for you.
The Institutional Review Board can stop a study if it appears to be causing unexpected harm to participants, or if there is evidence that the risks outweigh the benefits. An IRB can also stop a study if there is clear evidence that the new treatment is effective, in order to make it widely available.
Patients participating in a clinical trial may leave the study at any time, since their inclusion is voluntary.
Clinical trials are rigorously regulated. As with any new medication or surgical procedure, treatments are first studied thoroughly in the laboratory. If basic research establishes that a new method or chemical seems likely to succeed safely and effectively in patients, it can be submitted for approval for a clinical trial.
Not only must the submission meet regulatory guidelines established by organizations such as the Medicines Control Council and the Food and Drug Administration (FDA), but also it must first be submitted to the Institutional Review Board (IRB), a panel of scientists, researchers and representatives in the community who additionally approve the proposal for safety and ethical issues.
Aside from reviewing the scientific basis for the trial, as well as its safety and efficacy (the ability of the treatment or drug to produce its intended effect), the IRB gives serious consideration to the proposed trial's "informed consent" - the document that doctors give to patients explaining the trial and its potential risks.
All clinical trials must have a protocol, or action plan, which describes in detail what will be done in the study, how it will be conducted and why the various portions are necessary.
Risks are involved in clinical research, as in routine medical care and activities of daily living. In thinking about the risks of research, it is helpful to focus on two things: the degree of harm that could result from taking part in the study, and the chance of any harm occurring. Most clinical studies pose risks of minor discomfort, lasting only a short time. Some volunteer subjects, however, experience complications that require medical attention. The specific risks associated with any research protocol are described in detail in the consent document, which you are asked to sign before taking part in research. In addition, the major risks of participating in a study will be explained to you by a member of the research team, who will answer your questions about the study. Before deciding to participate, you should carefully weigh these risks. Although you may not receive any direct benefit as a result of participating in research, the knowledge developed may help others.
In single- or double-blind studies, the participants don't know which medicine is being used, and they can describe what happens without bias. Blind studies are designed to prevent anyone (doctors, nurses, or patients) from influencing the results. This allows scientifically accurate conclusions. In single-blind studies, only the patient is not told what is being given. In a double-blind study, only the pharmacist knows; the doctors, nurses, patients, and other health care staff are not informed. If medically necessary, however, it is always possible to find out what the patient is taking.
The clinical trial protocols are designed to assess continued safety of any participant in a clinical trial. The investigator is therefore able to assess you regularly to ensure that safety is established. Other safeguards that protect patients include:
- The Protocol Review Process
- Informed Consent Procedures
Protocol review. As in any medical research facility, all new protocols must be approved by an institutional review board (IRB) before they can begin. The IRB, which consists of medical specialists, statisticians, nurses, social workers, and medical ethicists, is the advocate of the volunteer subject. The IRB will only approve protocols that address medically important questions in a scientific and responsible manner. Furthermore, the Medicines Control Council reviews protocols to ensure that the trial is being conducted according to their stringent requirements.
Informed consent. Your participation in any clinical trial is voluntary. For every study in which you intend to participate, you will receive a document called an Informed Consent Form that explains the study in straightforward language. A member of the research team will discuss the protocol with you, explain its details, and answer your questions. You may discuss the study with family and friends. You will not be hurried into making a decision, and you will be asked to sign the document only after you understand the nature of the study and agree to the commitment. At any time after signing the consent form, you are free to change your mind and decide not to participate further. This means that you are free to withdraw from the study completely, or to refuse particular treatments or tests. Sometimes, however, this will make you ineligible to continue the study. If you are no longer eligible or no longer wish to continue the study, you will return to the care of the doctor who attends to your normal care.
Placebos are harmless, inactive substances made to look like the real medicine used in the clinical trial. Placebos allow the investigators to learn whether the medicine being given works better or no better than ordinary treatment. In many studies, there are successive time periods, with either the placebo or the real medicine. In order not to introduce bias, the patient, and sometimes the staff, are not told when or what the changes are. If a placebo is part of a study, you will always be informed in the consent form given to you before you agree to take part in the study. When you read the consent form, be sure that you understand what research approach is being used in the study you are entering.
Medical research is dogged by the placebo effect - the real or apparent improvement in a patient's condition due to wishful thinking by the investigator or the patient.
Each patient that takes part in a trial will be compensated for travel costs, time and inconvenience. The Medicines Control Council will stipulate a minimum amount of R150 per visit, however, higher amounts may be agreed upon should the need arise.
The health of millions has been improved because of advances in science and technology, and the willingness of thousands of individuals like you to take part in clinical research. The role of volunteer subjects as partners in clinical research is crucial in the quest for knowledge that will improve the health of future generations.
In addition to being part of development, patients who take part in clinical trials are usually exposed to a high standard of care, which include regular blood tests, ECGs, lung function tests, etc. Patients who routinely follow up at public health facilities may experience medication and examinations that are not part of the public health care system.
What are the benefits?
For patients with advanced disease, clinical trials offer some of the most aggressive options for treatment.
Early access to treatments
When an agent being tested in a clinical trial proves superior, patients benefit by having received a treatment they wouldn't have been able to receive otherwise.
Because clinical trial treatments are experimental, each trial has a protocol, or action plan, that specifies how and when patients are seen, given their medication and monitored with blood and other diagnostic tests. Patients involved with trials will interact closely with a physician or scientist and a research nurse.
Although the main motivation for participating in a clinical trial is the possible benefits realized by the patient, participants are also advancing progress in the discovery of causes and potential treatments and cures for cancer.
In Phase I clinical trials, researchers test a new drug or treatment for the first time in a small group of normal, healthy volunteers (about 20 to 80) to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase II clinical trials, the study drug or treatment is given to a larger group of people (about 100 to 300), including patients with the particular disease, to see if the drug or treatment is effective, and to further evaluate its safety.
In Phase III clinical trials, the study drug or treatment is given to large groups of people (from 1,000 to 3,000), including patients, to confirm its effectiveness, monitor side effects, compare it to other commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase IV clinical trials are done after the drug or treatment has been approved by the FDA and marketed for public use. These studies continue testing the drug or treatment to collect information about its effect in various populations and gather data on any side effects associated with long-term use.