A clinical trial (commonly referred to as a clinical study, research study, or research urgent treatment study) is one of the final steps in the extensive research process for confirming the effectiveness of new medical advances. These trials are crucial not only for finding ways to better treat conditions or diseases, but also finding improved ways to detect problems early as well as prevention in the first place. Trials allow doctors and researchers to gain information on the benefits, side effects, and possible applications to the new treatment modality. They may gain information on different combinations of treatments, doses of drugs, devices, and possible applications of therapies. As a result, the medical community may be able to determine new ways to detect, diagnose, avoid, and control clinical factors responsible for disease.
To determine the effectiveness of a particular strategy for fighting or preventing a disease, a clinical trial will compare its effect to that of the best existing alternative. To make the comparison as accurate as possible, researchers often randomly select which clinical trial participants receive the new agent or treatment and which participants receive the current standard. What the “standard” is depends on the type of trial. For a treatment trial, it might be the most commonly used drug, while for a prevention trial it might be no intervention at all.
Types of Clinical Trials
There are several different types of clinical trials:
- Community Trials – an entire community receives a treatment or preventative measure to determine if it works in the “real world”; or
- Diagnostic Trials – scrutinizes avenues to find better tests or procedures for diagnosing a particular disease or condition; or
- Interventional or Treatment Trials – evaluates the effectiveness of new drugs, new types or approaches of surgery or radiation/ancillary therapies, different combinations of therapies, and new methods of stopping or reversing the disease course; or
- Observational Trials – addresses health issues in large groups of people or populations in natural settings; or
- Prevention Trials – tests strategies for reducing the risk of acquiring the disease, which include strategies in taking medicines, vitamins supplements, vaccines, mineral supplements, learning new dietary or exercise strategies, or making lifestyle changes; or
- Quality of Life/Supportive Care Trials – examines ways to make the lives of people with terminal diseases as comfortable as possible and to help them maintain a high quality of living despite their condition; or
- Randomized Controlled Trials – a true experiment, in which the researcher randomly assigns some patients to at least one treatment and other patients to a placebo, or usual treatment; or
- Screening Trials – explores the best ways to find or detect the disease at the earliest and most treatable stage, such as comparing the effectiveness of a blood test in detecting a disease with that of a physical examination as a screening procedure.
Phases of a Clinical Trial
Clinical Trials are conducted in phases or stages. The trials at each phase have a different purpose and help scientists answer different questions:
- Phase I – researchers test a new drug or treatment in a small group of people (20 to 80, even as few as 10) at first to evaluate its safety, determine a safe dosage or modality range, and identify side effects.
- Phase II – the study drug or treatment is given to a larger group of people (100 to 300) to see if it is effective and to further evaluate its safety.
- Phase III – the study drug or treatment is given to large groups of people (1,000 to 3,000) to confirm its effectiveness, monitor side effects, compare to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
- Phase IV – post marketing studies delineate additional information including the risks, benefits, and optimal uses of the drug or treatment.
Pros and Cons of participating in a Clinical Trial
In addition to receiving high-quality care, there are many benefits to enrolling in a clinical trial. A patient’s participation could potentially benefit others by increasing knowledge about a disease and its potential treatments. Also consider that, as a clinical trial participant, a patient may be one of the first patients to experience positive results from a new therapy. On the other hand, it is possible that new treatments may not be better than the standard care, and unknown side effects may be worse than those experienced with standard treatments. Also, the patient may be placed in a control group that receives the standard care instead of the experimental study treatment. These are the factors a patient needs to be comfortable with before deciding to join a clinical trial. The patient, whether in the clinical trial or control group, will receive the same close monitoring and evaluations. So no matter which group a patient is in, he or she will receive excellent care for the condition being treated by a team of researchers.
Rights and Measures that Protect Clinical Trial Participants
Clinical trials are regulated by a number of governing groups and processes to help protect patient safety and the ethicality of scientific research. The rights of clinical trial patients are notably protected by:
- Institutional Review Board (IRB) – All research organizations have an IRB which oversees the actions and documents related to the clinical trial process. This group is made up of medical specialists, nurses, social workers, medical ethicists, and sometimes patient advocates and clergy, who have no special interest in any specific trial. Each member is responsible for ensuring that all trials are conducted safely, fairly, ethically, and with full patient consent and involvement.
- Peer Review (PR) – Clinical trials are often reviewed by experts such as the National Cancer Institute, or a pharmaceutical company chosen by the study sponsor. Prior to IRB submission, these groups review the trials for scientific merit, patient safety, and ethical considerations. Many research organizations have peer review groups in addition to their IRBs.
- Informed Consent (IC) – IC is a process designed to inform the clinical trial patient and to help foster good communication between the patient and the research team. This process includes providing the patient with information about significant safety issues that might surface during the course of the study. The IC document will relate facts about the trial, such as its purpose, duration, required or alternative procedures, benefits and risks. Once the IC document and written material are given to the clinical trial patient, he or she will have time to think over the decision. If he or she elects to participate in the study, they will be asked to sign the IC document, verifying the clinical trial patient understands and agrees to its conditions. Once the clinical trial begins, the IC patient rights continue. Throughout the trial, the patient may receive new information which may affect their decision to remain in the trial study. The healthcare team will routinely evaluate how the treatment is affecting the patient and will inquire if the patient would like to continue participation. The clinical trial patient is free to leave a trial at any time.
Where do the ideas for trials come and who sponsors them?
Ideas for clinical trials usually come from researchers. After the researchers test new therapies and procedures in the laboratory and in animal studies, the treatment with the most promising laboratory results are moved into clinical trials. The clinical trials are then sponsored or funded by a variety of organizations or individuals, such as physicians, medical institutions, voluntary groups, and pharmaceutical companies. The studies will then be conducted in a variety of places, such as hospitals, universities, physician offices, or community clinics.
Approved Clinical Trials
The term approved clinical trials means a phase I, phase II, phase III, or phase IV clinical trial that is conducted in relation to the prevention, detection, or treatment of cancer or other life-threatening disease or condition, as determined by the treating physician. The following is a listing of federally sponsored, approved or funded (which may include funding through in-kind contributions), clinical trials:
Federally Funded Trials:
- The National Institutes of Health (NIH);
- The Centers for Disease Control (CDC) and Prevention;
- The Agency for Health Care Research and Quality (AHCRQ);
- The Centers for Medicare & Medicaid Services (CMS);
- The Department of Veterans Affairs (VA);
- The Department of Defense (DOD);
- The Department of Energy (DOE);
- Cooperative group or center of the entities listed above; or
- A qualified non-governmental research entity identified in the guidelines issued by the NIH for center support grants.
Review by Governmental Agency:
- The study or investigation is conducted under an investigational new drug application reviewed by the Food and Drug Administration (FDA).
- The study or investigation is a drug trial that is exempt from having such an investigational new drug application.