I was doing some research on clinical trials and it is much more complex than I dreamed! First of all, any new treatment must go through numerous stages of testing before its benefits and risks can completely be known. New treatments are discovered in the laboratory and it can take many years of research before they are given to patients. Why? Because it is essential to identify that the new treatment is actually better than what is already available. These research studies are also called clinical trials. If a treatment has definite potential in the final stages of development, then research is carried out in patients with the particular type of illness that the treatment aims to help. Furthermore, I always believed that clinical trials were only regarding drugs, and that is not true. The most commonly performed clinical trials, in addition to drugs, evaluate new medical devices (like a new catheter), biologics, psychological therapies or other interventions. A new treatment does not always turn out to be better and clinical trials are extremely important in helping to decide if one treatment is safer and more effective than another.
I found my best resource for this topic was ClinicalTrials.gov. ClinicalTrials.gov currently reports there are 110,353 active trials with locations in 174 countries. That is unbelievable! Their website broke down the different types of clinical trials, which are:
Treatment trials test experimental 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, vaccines, vitamins, 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.
In addition to the different types of trials, there are four specific phases that have a different purpose to help scientists answer different questions. In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely. Finally, in Phase IV trials, post marketing studies delineate additional information including the drug’s risks, benefits and optimal use.
Before starting a clinical trial on a patient, it is imperative to have an informed consent. This process involves learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. It is important to note that an informed consent is not a contract, and the participant may withdraw from the trial at any time. All clinical trials are based on a study plan, referred to as a protocol. The plan is carefully designed to 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.
In this article, we are going to cover some of the most important questions to ask if you are contemplating participating in a clinical trial and what the benefits might be as well as the risks. The questions each participant needs to ask are as follows:
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
- What kinds of tests and experimental treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the experimental treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the experimental treatment is working? Will results of the trials be provided to me?
- Who will be in charge of my care?
The benefits of participating in a clinical trial include gaining access to new research treatments before they are widely available, obtaining expert medical care at leading health care facilities during the trial, helping others by contributing to medical research and, most important, playing an active role in one’s own health care. The risks, on the other hand, include unpleasant, serious or even life-threatening side effects to experimental treatments, the experimental treatment may not be effective for the participant and the protocol may require more of a person’s time and attention than would a non-protocol treatment. For instance, more trips to the study site, more treatments, hospital stays or complex dosage requirements.
Clinical trials really are important, but they are a very complex part of research as well. Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work. Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veterans Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices or community clinics.