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UC Dermatology Clinical Research Q&A
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Clinical Research
Frequently Asked Questions |
What is a clinical trial?
A clinical trial is a carefully designed study of a technique, device, treatment or combination of treatments.
Why are clinical trials important?
Clinical trials are performed in order to identify new or improved diagnostic or treatment options for health problems. Clinical trials are extremely important in improving the delivery of health care. The treatments available today are in part due to the participation of others in clinical research trials.
Dermatology researchers are constantly trying to identify better treatments for skin diseases. Studies are also conducted to identify treatments with fewer side effects than currently available treatments. Most improvements in all areas of patient care have occurred as results of clinical trials.
Why do people participate in clinical trials?
People participate in clinical trials for a variety of reasons. Clinical trials may offer access to promising medications long before these drugs are approved for the marketplace. Clinical trial participants typically get excellent care with close follow-up from the trial physicians during their study participation. Physical examinations are often performed as part of clinical trials, as are laboratory tests. Sometimes important information about participants’ health can be learned from the examinations and studies performed as part of clinical trials. Some clinical trials offer payment to compensate participants for their time and travel. All clinical trials offer the opportunity to be involved in improving the knowledge available about the care of health problems for future patients.
How are new medications tested?
The clinical testing of experimental drugs is normally done in phases. There are progressively larger numbers of participants enrolled in each study phase. Once these phases have been completed, New Drug Approval may be granted by the Food and Drug Administration allowing medications to be marketed. Pharmaceutical companies often continue to conduct post-marketing studies to learn more about medications.
Phase I clinical trials are the first studies investigating potential new treatments in humans. These studies are conducted on treatments which have been shown to have potential value based on the results of laboratory testing or other research. Initial Phase I clinical trials are generally performed using small numbers of healthy volunteers. These clinical trials attempt to determine the appropriate doses of medication, and to learn more about how medication travels through the body. Phase I clinical trials also study, under very close supervision, the potential side effects of medications. Phase I clinical trials generally start with low doses of medication and patients are observed closely for any side effects. If the doses appear safe, higher dosages may be tested. Phase I clinical trials primarily assess safety of medications.
Once the safety of a medication has been established in Phase I clinical trials, the medication must be studied for effectiveness. This is done initially in Phase II clinical trials. Phase II clinical trials involve a relatively small number of participants who generally have the health problem being studied. Phase II studies may involve up to a few hundred participants. Often Phase II clinical trials are randomized. Randomization is done in order to scientifically test medications for their safety and effectiveness. Participants are assigned to one of two or more potential treatment groups. Possible assigned treatment groups may include an experimental treatment, a control treatment in which participants receive a standardly used treatment, or a placebo. A placebo is an inactive treatment that looks like the study treatment. A placebo is used when the study treatment is being compared to no active treatment so that it is not possible for the evaluator or the patient to tell whether or not the study medication is active.
Randomization of treatments is done in a manner similar to flipping a coin, or like drawing a name from a hat. This is done to avoid the doctors and/or patients biasing the study results based on which treatment they think might be better. Usually randomization is done by a computer, and most of the time the study personnel do not know which treatment a participant is receiving. The participant and doctor are both “blinded”, or unaware of which treatment has been assigned. Blinding is done to help insure that the conclusions drawn from clinical trials are as accurate as possible. Patients involved in Phase II clinical trials are very closely monitored for any potential problems that might develop.
If the results of Phase II clinical trials show that a medication has the potential to be effective with an acceptable safety profile, the medication may proceed to Phase III clinical trials. Phase III clinical trials often involve more patients, perhaps hundreds or even thousands, and often take place at multiple investigational centers across the country or in multiple countries. There is continued evaluation of the effectiveness of the medication under study, and more information is obtained on how well the medication is tolerated, and how it affects the quality of life of the trial participants. Most Phase III clinical trials are randomized and blinded. Once a Phase III clinical trial is successfully completed, a pharmaceutical company can ask the FDA for approval to market the medication.
Phase IV, or post-marketing, clinical trials may be performed for a number of reasons. A pharmaceutical company may wish to compare a new drug to other drugs which are already on the market. A Phase IV clinical trial may give information on the safety and effectiveness of a medication on a long term basis. The cost-effectiveness of using a new treatment may be compared to that of using a routinely used treatment.
What is a protocol?
A protocol is a document that gives background information on a treatment being tested, including any available safety information. A protocol must contain information required by the Food and Drug Administration. A protocol outlines the exact steps and procedures that will be taken in a clinical trial. It contains details such as the number of patients that may be enrolled in the clinical trial, the number of visits that will take place, and treatment dosing. It also describes known potential risks and methods for monitoring these risks.
What is an informed consent?
A person must give informed consent prior to participating in a clinical trial. In order to explain what is involved in a particular clinical trial, an informed consent document is used. This document contains information on how and why a study is being done, the known potential risks and benefits, the rights of participants, and how confidentiality is maintained. Once a consent form has been thoroughly reviewed with a potential participant, it must be signed in order for a person to become enrolled in a clinical trial. Taking part in a clinical trial is voluntary, and participation may be declined prior to enrollment or at any time after enrollment into the clinical trial.
Who approves a clinical trial before it can begin?
All clinical trials are also reviewed by an Institutional Review Board. Members of the Institutional Review Board, which consists of persons in both the scientific and lay communities, thoroughly review the protocol and communicate to the investigator any questions they may have relating to the protocol. The protocol and informed consent must be approved by the Institutional Review Board prior to the initiation of a clinical trial. Depending on the phase and type of testing, a clinical trial may also be reviewed and approved by the Food and Drug Administration.
How do I know if I am eligible to participate?
Each study protocol contains a list of criteria that define who is eligible to participate in a clinical trial and who is not eligible to participate. These eligibility criteria are developed to help insure that the medication being studied can be accurately assessed for effectiveness. The eligibility criteria also help to protect the safety of potential participants. Eligible participants generally need to be in reasonably good health, so that the risk of significant side effects occurring as a result of a treatment are minimized.
If I volunteer, will I be treated like a guinea pig?
Participants in clinical trials are followed very closely. In fact, participants often get more attention than the average patient because they are monitored so closely.
What safety measures are taken?
There is generally some degree of risk involved with any medical treatment. There is generally less known about the risks associated with investigational new drugs because there has been less experience using these drugs than using standard treatments. Clinical trial participants are informed of the known potential risks associated with an investigational therapy prior to participating in the trial. If there is new information discovered about the therapy that would potentially affect the participants, that information is also communicated to them. Clinical trial participants are followed very closely, usually with more office visits and sometimes with more tests performed, than would be done with standard therapies, so that any side effects which may occur can be monitored closely. Because there may not be much information on the effect of an experimental drug on pregnant women and developing fetuses, pregnant women or those unwilling to avoid pregnancy may not be allowed to enter some clinical trials. Women of child-bearing potential may be monitored closely with pregnancy tests to insure that a pregnancy has not occurred.
What do clinical trials cost?
Funding for clinical trials comes from both private industry (pharmaceutical companies and biotechnology companies), and the government (National Institutes of Health). Some clinical trials are not funded at all. The medical care that is part of the clinical trial is often provided free to the patient. The study treatment and study-related tests are also frequently provided without expense to the patient. Care which is not directly related to the clinical trial, or treatment needed following completion of the clinical trial, is not covered by clinical trial funds. In some instances, participants may be reimbursed for transportation expenses, and may also be paid a small fee for the trouble they take to participate in a study.
What are a participant’s rights and how are they protected?
Taking part in a clinical trial is voluntary, and a participant may choose to leave the trial at any time. A person does not sign away any legal rights by volunteering to participate in a study. An Institutional Review Board examines a proposed study and ascertains that patients’ rights are protected.
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