Is a Clinical Trial the Right Choice for Me?
All medications and medical devices that we use have gone through a clinical trial. This is the way that new products are shown to work and to be safe before they are available to be prescribed or purchased over the counter. A clinical trial is a scientific study that outlines processes to discover or determine how a new medicine or medical device works in people.
A new drug or device, called an Investigational Product, goes through several stages:
1) First, it is tested in the laboratory.
2) Following careful scientific analysis of the results, it may be tested in animals.
3) Finally it may be tested in humans. This last stage, where the drug or device is tested in humans, is known as a clinical trial.
The FDA approves new medications and devices or new uses for existing medications and devices based on the results of clinical trials.
Clinical trials are done to find a better standard of care. They are important because there is no way to make a precise duplication of the human body or its responses to products being investigated. Not every clinical trial leads to a cure, but each can provide another piece of knowledge toward solving the puzzle.
Participation in a Clinical Trial is voluntary. A health care provider may talk to you about participating in a clinical trial because you have symptoms, a condition, or a disease that is currently being investigated. When participating in a clinical trial, you help others by contributing to medical research.
You will receive a consistent level of health care by a carefully selected team. You can take a positive action to help yourself by playing an active role in your own health care. You may have the ability to participate in alternative treatment options before they are widely available.
The treatment might not work; there could be serious, harmful, or even life-threatening side effects. You can only take treatments approved by the study; some of your current treatments may not be allowed. After you have completed your participation in the study, you may not be able to continue receiving the investigational product, even if it worked for you.
Patients’ rights are protected on many levels.
Locally, any research that is being conducted must be approved by an Institutional Review Board (IRB)
. This is a group comprised of physicians, scientists, nonscientists, clergy, and community activists. They carefully review each study to make sure
1) that it does not put the participants at undue risk
2) the research is necessary
3) the research is being conducted in an ethical and responsible way.
After the IRB says that a study can be conducted, it then periodically reviews the study.
On the national level, the sponsor periodically reviews the data to find if there is any indication that the participants are experiencing any new risks. If this happens, the study may be changed or stopped to prevent additional problems with study participants.
Also, the Food and Drug Administration (FDA) closely monitors all trials. The FDA has to approve the study before it can begin and monitors the study on a regular basis. It can stop a study, either at one location or all locations, if it finds problems with the study or risks to the people participating.
The Language of Clinical Trials
Phase I: Phase I studies are done to establish a drug’s safety. The drug is given to small groups of healthy people (20 to 100) for the first time. These studies are designed to determine what happens to the drug in the human body. Researchers also examine the side effects that occur as dosage levels increase. This process generally takes several months. About 70% of experimental drugs pass through this phase of testing.
Phase II: Phase II studies are done to establish a drug's efficacy. The drug is given to several hundred people who have the disease or condition. These studies are generally randomized trials to provide the FDA with comparative information about the safety and effectiveness of the new drug. Only about one-third of experimental drugs successfully complete both Phase I and Phase II studies.
Phase III: The drug is now given to several hundred to thousands of people to determine how well it works and expand what is known regarding the drug’s safety with selected doses when given to a larger number of people.
Phase IV: This is any research study when the drug has been approved by the FDA. This is a very large group of subjects, larger than Phases I-III, to continue to monitor safety in larger populations.
The majority of clinical trials done at Ochsner Health Systems are Phase III or IV trials.
The people who are working on the trial are known as the study staff. This group is made up of doctors, pharmacists, nurses, coordinators, and others.
The sponsor is the individual or company who is conducting the research.
The protocol is a manual that explains the scientific basis for the research, what the study is trying to prove, and how the study acquires the data. The manual explains every procedure, test, and other evaluation that the participants of the study will experience. At each site, the study staff agrees to perform the study according to the protocol. This guarantees that the study is being conducted in the same way at each location.
Informed consent is a process where the study; its purpose, risks, and benefits; and detailed information of what being involved with the study will mean is explained to people who are considering participating. Possible participants are encouraged to ask any questions or talk about their concerns. A member of the study staff will talk about each until the participant feels comfortable with the information.
Once a person decides to participate, a document called the Consent Form, with every aspect of the study written out, is signed. The document will tell who is conducting the study, both at the site and nationally/internationally (if the study is a multicenter study). Participants receive a copy of their signed Consent Form, so they have a copy of the information that has been presented to them. The Informed Consent process does not end once the document is signed. Participants always have the right to ask questions about their participation in the study and the study-related procedures.
The goal of clinical trials is to provide treatments to similar patients, free of bias. This is accomplished by randomization; it is similar to flipping a coin. Patients are placed in different treatment groups regardless of what treatment group the doctor or the patient might prefer.
A placebo is an inactive substance that can be made to look like the active investigational product. A placebo is not used as part of a clinical trial if it might jeopardize the patients’ health.
Clinical trials are performed to see if a new treatment is better than the preexisting ones. One way to prove this is to compare the new treatment against doing nothing. Almost any medical treatment seems to have at least a temporary effect, even using a placebo. Evidence showing how well the placebo works can be compared to how well the investigational product worked.
The control group contains participants who are not receiving the investigational drug/device. This group may receive a placebo or they may receive “standard of care,” which is the treatment already available. By comparing the results between the control group and the investigational group, researchers can determine if the investigational product is working, safe, and effective.
In a blind trial, the participants do not know if they are in the group receiving the investigational treatment or in the control group. A trial is double-blind if neither the participant nor the physician are aware. At the end of the trial, the participants and the study staff are informed which treatment was received.