What is a Clinical Trial?
There are many definitions of clinical trials but generally they are health-related research studies in human beings that follow pre-defined protocol. Usually the research is done to determine the safety and effectiveness of a drug or treatment and to discover any side effects.
If someone volunteers to be part of a clinical study there are various kinds of studies to be part of. Drug studies may be a new medication and not tested on people before or it might be an existing medication being used in a new way. Sometimes a new medication or treatment is being compared to the best-known standard of care to see whether it is more effective or causes fewer side effects.
The information obtained during clinical trials helps regulatory authorities around the world determine if they will approve a drug or treatment to use in their country, The information can also guide health professions in the decisions about prescribing medications or other treatments for patients. It also helps determine the proper use of medications both prescribed and over-the-counter.
Are there other Clinical Trial Phases?
Yes, there are more phases than just Phase I.
- Phase I – Screening for safety of drug or therapy in a small number of people.
- Phase II – Test the efficacy and safety of the drug or therapy in a larger number of people.
- Phase III – Confirm effectiveness, monitor side effects, compare to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely, on large groups of people usually 1000+ individuals are enrolled in Phase III studies.
- Phase IV – Study drugs or therapies after they are approved and gather more information on drug’s risks, benefits and optimum use.
What happens during a clinical trial?
The process during a clinical trial depends greatly on the kind of study being conducted. The study trial team includes, doctors, nurse practitioners, nurses, study coordinators, and other health care professionals. The study team will check the health of the potential volunteer at the beginning of the trial, give specific instructions for being part of the study, monitor the volunteer carefully for the duration of the trial and stay in touch once it has been completed.
Who funds clinical trials?
Clinical trials are funded, or sponsored by a variety of organizations, foundations, voluntary groups, and pharmaceutical companies, additionally clinical trials can also be sponsored by federal agencies such as the National Institutes of Health, the Department of Defense and the Department of Veteran’s Affairs. Trials can take place in many different settings, such as hospitals, universities, doctors’ offices, or community clinics.
What is a protocol?
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. It describes what types of people may volunteer for the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study.
What is a placebo?
A placebo is an inactive, pill, liquid or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the experimental effectiveness.
How does someone volunteer for a clinical trial?
There are thousands of clinical research studies conducted around the world every year. Your doctor may be able to refer you to clinical studies in your area. Sometimes you can find information about clinical studies in ads run on TV, radio and in newspapers. Universities with medical facilities, patient groups, and disease associations often are places where you can find information about clinical studies.
Who can participate in a clinical trial?
All clinical trials have set of guidelines about who can participate. The factors that allow someone to be in a clinical trial are called "inclusion criteria" and those that don’t allow someone to participate are called "exclusion criteria". These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
Before joining a clinical trial, a potential volunteer must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical trial, and others need healthy volunteers. The inclusion and exclusion criteria are not used to reject people personally, the criteria are used to identify appropriate participants and keep them safe.
What should someone consider before volunteering in a clinical trial?
Potential volunteers should know as much as possible about the clinical trial and feel comfortable asking the members of the study team questions about it, the care expected in a trial, and the cost of the trial. The following questions could be useful for the potential volunteer to discuss with the study staff some of the answers are also found in the informed consent document. (See what is informed consent)
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers think 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 potential risks, side effects, and the benefits in the study compare with my current treatment?
- How might this study affect my daily life?
- How long will the study last?
- Who will pay for the experimental treatment?
- Will I be reimbursed for expenses?
- What type of long-term follow up is part of this trial?
- How will I know that the experimental treatment is working? Will results of the study be provided to me?
- Who will be in charge of my care?
Although each study population is different each potential volunteer receives the same high quality care. This will include a medical history, list of all current medications, screening exams, and study specific diagnostic tests.
Sometimes the study staff will require a copy of a volunteer’s medical record for review, and sometimes approval from their current personal physician before you can be enrolled into a study.
All of this is to make sure that each possible volunteer is suitable for the study they are being screened for.
Before a potential volunteer meets with a research coordinator or doctor they should plan ahead and write down possible questions to ask. It is ok to have a friend or relative come along for support and to hear the answers to the questions. They can take notes or ask to record the discussion on a tape recorder to replay later.
Who protects a volunteer’s safety if they participate in a clinical trial?
Every clinical trial in the U.S must be approved and monitored by an Institutional Review Board (IRB). The IRB is there to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support medical research involving people must, by federal regulation have an IRB that initially approves and periodically reviews the research.
Every research participant must go through the informed consent process; this is so that the volunteer understands the risks and freely consents to participate in the study.
Does a volunteer continue to work with a primary health care provider while in a trial?
Absolutely. Most clinical trials provide short-term therapy or treatments related to a specific illness or condition, but do not provide extended or complete primary health care. When the health care provider works with the study team, it helps ensure that other medications or treatments will not conflict with the study protocol.
What is informed consent?
Informed consent is the process by which a fully informed volunteer can participate in the decision to be part of a research study.
The major elements of informed consent are:
- the purpose of the study/research being conducted
- duration of the study
- The relevant risks and benefits of being part of the study
- the required procedures
- key contacts
- and the acceptance to participate in study by volunteer
The informed consent is not a contract and a volunteer can withdraw from a clinical trial at any time.