Why Participate In A Clinical Trial
Clinical trials are studies that evaluate the effectiveness of new drugs or treatment strategies. The development of more effective disease treatments requires that new and innovative therapies be evaluated for patients. Each clinical trial is designed to find new or better ways to treat our patients’ disease. Clinical trials are especially important because, in the absence of high cure rates, nearly all therapeutic approaches are developmental in nature. Our goal is to provide patients with all forms of investigational and approved therapies without the need to travel to other parts of the country.
What are Clinical Trials?
A clinical trial is a research study designed to evaluate potential new treatment options. These studies are the result of a long and deliberate research process that often takes years. Clinical trials test the safety and effectiveness of new or modified drugs, new drug doses, unique approaches to surgery or therapy, 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. In the United States all new treatment products must proceed through an orderly clinical trials evaluation process to ensure that they have an acceptable level of safety and demonstrate benefit to helping patients with a specific disease before they become commercially available to other patients.
Clinical trials essentially fall into two general categories:
The first general category of clinical trials is designed to evaluate new drugs, compounds, or biologic agents that have not yet been approved by the Food and Drug Administration (FDA) for administration to patients. All substances must go through several phases of clinical trials to document their safety and effectiveness before the FDA approves them for routine use to treat patients. Prior to FDA approval, these substances are only available through clinical trials; however, after FDA approval, they are commercially available.
Clinical trials may also evaluate drugs, compounds, or biologic agents already approved by the FDA for the treatment of one type of disease. These substances have already been determined to be safe by the FDA and they are now being evaluated in different doses, schedules, and combinations to determine how to optimally use them for the treatment of a variety of diseases.
Phases of Clinical Trials
Development of treatment strategies occur in four phases. Each phase is designed to determine specific information about the potential new treatment such as its risks, safety and effectiveness compared to standard therapy. The hope is that the new therapy will be an improvement over the previous standard therapy.
Phase I Trials:
This phase is probably the most important step in the development of a new drug or therapy. These trials usually involve a small number of patients for whom other standard therapies have failed or no known alternative therapy is available. Phase I therapy may produce anti-disease effects and a small number of patients may benefit. However, the primary goals of this phase are to determine anti-disease activity in humans, the maximum tolerated dose of the treatment, the manner in which the drug works in the body, the toxic side effects related to different doses and whether toxic side effects are reversible. Upon completion of phase I trials, the information that has been gathered is used to begin phase II trials.
Phase II Trials:
Once the information is gathered and analyzed from phase I trials, phase II trials are designed to determine the effectiveness of the treatment in a specific patient population at the dose and schedules determined in phase I. These trials usually require a slightly higher number of patients than phase I trials. This number may increase depending on the number of responses as the phase II trial progresses. Drugs or therapies that are shown to be active in phase II trials may become standard treatment or be further evaluated for effectiveness in phase III trials.
Phase III Trials:
Phase III trials compare a new drug or therapy with a standard therapy in a randomized and controlled manner in order to determine proof of effectiveness. Phase III trials require a large number of patients to measure the statistical validity of the results because patient age, sex, race, and other unknown factors could affect the results. To obtain an adequate number of patients, several physicians (investigators) from different institutions typically participate in phase III clinical trials.
Phase IV Trials:
Once the drug or treatment becomes part of standard therapy, the manufacturer of the drug may elect to initiate phase IV trials. This phase includes continued evaluation of the treatment effectiveness and monitoring of side effects as well as implementing studies to evaluate usefulness in different types of diseases.
How Are Clinical Trials Conducted?
Clinical trials are designed to test treatments under very specific scientific and ethical guidelines. Clinical trials use written protocols to define the purpose, design and conduct of a specific clinical trial. All of the research centers participating in a particular study use the same research protocol. The protocol is written by the sponsor of the study and explains what the trial will do, how it will be conducted, where it will be conducted, who may participate and how and when the participants will be evaluated. In order to protect patients participating in clinical trials, the sponsor reviews the protocol for safety and appropriateness and then the protocol must undergo a second neutral review by an Institutional Review Board (IRB). The Institutional Review Board is responsible for overseeing any clinical trials that are performed in the specific healthcare institution where the clinical trial is offered / conducted. An Institutional Review Board includes physicians, healthcare providers and individuals not involved in healthcare, including the clergy or ordinary citizens/consumers. Institutional Review Board members do not have any personal interest in the results of the trial and, therefore, can ensure that the study is conducted fairly and safely. Committee members serving on Institutional Review Boards address the following questions in reviewing protocols:
- Does this protocol have scientific value?
- Does the protocol have scientific validity?
- Does the study have a valid scientific design and yet pose an inappropriate risk for subjects?
- Are risks to subjects minimized?
- Are the risks to subjects reasonable in relation to anticipated benefits, if any, to subjects and the importance of the knowledge that may reasonably be expected to result?
- Is the selection of subjects equitable? Are additional safeguards in place for subjects likely to be vulnerable to coercion or undue influence?
- Will informed consent be obtained from research subjects or their legally authorized representatives?
- Is there adequate provision for monitoring the data collected to ensure the safety of subjects?
- Are there adequate provisions to protect the privacy of subjects and to maintain the confidentiality of data? The Institutional Review Board also reviews all informed consent documents to make sure that they provide clear and complete information for those evaluating the merits of enrolling in a specific clinical trial.
Clinical trials evaluating disease treatments or strategies may be offered in large university hospitals, local community hospitals and/or physician practices. The location where a clinical trial is conducted depends on the specific resources required to conduct the trial, the number of patients needed for enrollment in the trial to answer the question and the individual or institution's interest in performing clinical trials in general.
Clinical Trials Safeguards
Both standard care and clinical trials have risks, side effects and benefits that vary depending on the individual. However, there are rigorous guidelines in place to protect the well being and safety of clinical trial participants. The physician and research nurse conducting the study will explain any known or anticipated risks ahead of time. Once a patient's eligibility for a specific trial is established, the research nurse will explain the informed consent process. Informed consent is one of the patient's most important rights in the research process, as it outlines the purpose of the study, the exact treatments that will be administered, all possible side effects, and the patient's right to withdraw from the study at any point. Signing the consent form acknowledges that the trial was explained and is understood. Also, clinical trial participants are constantly monitored to identify any changes in their condition.
Why Participate in Clinical Trials?
Clinical trials are designed to evaluate the effectiveness of new treatment interventions. The objective of these clinical trials is to test new therapies in patients with a particular disease. Patients participate in clinical trials for several reasons, including: The potential to benefit from a new drug or treatment procedure, improved management of symptoms resulting from treatment of disease, the opportunity to directly contribute to improving the understanding of how to treat a disease and ultimately benefit other patients. Patients who participate in clinical trials receive either a promising new treatment or the best available conventional treatment. If a new treatment option is proven to work, patients who are participating in the clinical trail will be among the first to benefit. While there is no guarantee that any treatment will be successful, clinical trials have been proven to offer some of the most effective disease treatments currently available today.