What is clinical research?
Research is a methodical investigation into a subject. Clinical research is carried out to obtain new knowledge of a disease or condition and to add to already known facts. New or improved medical treatments are often the result of clinical research.
What are clinical trials?
A single clinical research study is called a clinical trial. Clinical trials are done with human volunteers. Clinical trials attempt to answer specific health questions.
There are different types of clinical trials. A clinical trial may study::
- Disease prevention
Disease prevention trials look for better ways to prevent diseases in people. They also study ways to prevent a disease from returning. Topics that may be studied include the roles that medicines, vitamins, minerals, vaccines, or lifestyle changes play in keeping individuals healthier.
- Disease Treatment
Treatment trials study new treatments or alternative ways to use existing treatments for a specific disease or condition. Treatments including newly developed drugs or medical devices, different combinations of existing drugs, and new advances in surgery or radiation therapy are different types of disease treatment trials.
- New Screening and Diagnostic Techniques
Diagnostic and screening technique trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
- Quality of Life Trials
Quality of Life trials explore ways to improve the comfort and quality of life for individuals with acute or chronic illnesses and conditions.
Why are clinical trials done?
It is not really known whether a new treatment or intervention offers any benefits to humans until the clinical research is complete. Federal law states that clinical trials must be conducted before a new drug or treatment may be marketed in the United States.
What is the difference between a clinical trial and standard medical care?
Clinical trials are not the same as standard medical care. Standard medical care is the customary approach currently used in caring for the health of individuals. Clinical trials are research studies looking at potential new or improved treatments and interventions before they are marketed.
What are the phases of clinical trials that use investigational drugs?
Each phase of a clinical trial has a different purpose.
Phase 1: Phase I trials initiate the introduction of a new drug in humans. Phase I trials usually involve 20-80 individuals and are closely monitored by the investigator and sponsor. These trials are designed to determine the safety of the investigational drug. Side effects associated with increasing doses and the safe dosage range are studied. How the drug is absorbed, metabolized, and its mechanism of action in humans is also determined during a Phase I trial.
Phase II: Phase II trials begin the evaluation of a drug’s effectiveness for a particular condition or disease. Common short-term side effects and risks associated with the new drug are determined. Approximately 100-300 patients with the specific disease condition are enrolled into Phase II trials.
Phase III: Phase III trials are carried out after preliminary evidence from Phase I and Phase II trials suggests that the new drug is safe and effective. Phase III trials usually include several thousand individuals. Most Phase III trials are randomized and blinded. This means that the standard medical care for a disease (or placebo if appropriate) is compared to the study drug. Often neither the subject nor the study investigator knows what the subject has received until the trial is completed. Phase III trials allow a more complete understanding of the drug’s effectiveness, benefits, and associated adverse reactions.
Post-Marketing – Phase IV: These trials may be carried out to monitor a drug’s long-term effectiveness and long-term side effects. The cost-effectiveness of the new drug therapy compared to other traditional therapies may also be studied.
Why volunteer for a clinical trial?
By taking part in a clinical trial, an individual can try a new treatment that may be better than those that already exist. Individuals can also help researchers better understand how the treatment works in diverse populations.
Are clinical trials safe?
Treatments used in a clinical trial have first been studied in laboratories and animal experiments. Treatments that have acceptable safety reports and show the most promise are then moved into clinical trials.
An individual must qualify for a clinical trial. Inclusion and exclusion criteria (requirements such as age, previous medical history and type of disease) are used to determine whether a person may or may not be allowed to enter a clinical trial. An individual will be allowed to participate in the clinical trial if all inclusion criteria are satisfied. If an individual meets any of the exclusion criteria participation will not be allowed. Inclusion and exclusion criteria help identify appropriate participants and help to exclude those who may be put at risk by participating in a particular clinical trial. If an individual does not meet eligibility requirements for one trial that does not mean she or he cannot possibly participate in a different trial.
Efforts are made to control risks to clinical trial participants. Some risk may be unavoidable because of the uncertainty fundamental to clinical research that uses new medical treatments or products. It is important that people make their decision to participate only after they have a full understanding of the entire process and the risks that may be involved.
Are clinical trials overseen by a regulatory board?
Clinical trial procedures are reviewed by Institutional Review Boards (IRBs). These boards are composed of at least five individuals that include scientists, doctors, nurses, pharmacists, and lay people. Every clinical trial that takes place must be approved by an IRB. The IRB reviews the clinical trial to ensure that appropriate steps are taken to protect the rights and welfare of the participants. If the risks to the participants are found to be too great the IRB will not approve the research or it will specify changes that must be made before the research can be done.
The IRB requires any new information about the clinical trial to be sent to it for review. If new information changes the risks to the participants the IRB may stop the clinical trial or require changes by the investigator to decrease the increased risk. In addition, at a minimum of every year an IRB requires the investigator to submit a summary of how the clinical trial is proceeding. The IRB examines the summary and then decides if the clinical trial may continue to be conducted.
What is a protocol?
A protocol outlines exactly what is going to take place during a clinical trial. The protocol describes what is being studied and why it is an important question, how the research team will protect the health and safety of the participants, what procedures are planned and when they will be performed, possible risks and benefits to the participant, and how the data will be analyzed.
What is informed consent?
Potential participants in a clinical trial must be given complete information about the clinical trial before study procedures begin. The informed consent process provides an opportunity for the researcher and potential participant to exchange information and ask questions. Information that must be talked about with a potential participant is:
- That the clinical trial involves research
- The purpose of the clinical trial
- The expected duration of the clinical trial
- A description of planned procedures with the experimental procedures identified as being experimental
- Reasonably foreseeable risks or discomforts
- Benefits to the participant or to others
- An explanation of how confidentiality will be maintained
- If compensation or treatment is available if a clinical trial related an injury occurs
- A name and phone number of an impartial individual to contact for questions about the clinical trial
- A name and phone number of an impartial individual or party to contact for concerns about a participant’s rights during the clinical trial
- A statement that participation is voluntary
- That refusal to participate will not involve penalty or loss of benefits the subject is otherwise entitled to
- That the participant may discontinue participation without loss of any benefits
- Any financial conflicts of interest on the part of the investigator(s)
Individuals invited to enter a clinical trial are not obligated to participate. If an individual does enter a clinical trial the consent form must be signed by the participant before enrollment into the trial and before any study procedures can be performed. The signed informed consent means that the participant has had the opportunity to talk with the clinical research staff and has agreed to participate in the trial.
Trial participants have the right to leave a trial at any time. Participants also need to know that circumstances may arise under which their participation in a trial may be ended by the researcher without their consent.
Who may participate in a clinical trial?
Some clinical trials involve people with a particular illness or condition to be studied while other clinical trials ask for healthy volunteers. Each clinical trial has specific criteria that must be met before an individual may enter a clinical trial. Requirements as to age, gender, previous medical history, and other medical conditions are always outlined. In addition other inclusion and exclusion criteria are used to further refine who may enter into a clinical trial.
Volunteering for a clinical trial is no guarantee of acceptance into the trial. There is also no guarantee that a participant in a clinical trial will receive the treatment being studied unless specifically told that it actually is the treatment being studied.
What should you ask before entering a clinical trial?
- Who is sponsoring the trial?
- Who has reviewed and approved the trial?
- Who is in charge of the research at this site?
- What is the purpose of the clinical trial?
- Are there any benefits to me if I participate in the trial?
- Are there any risks to me if I participating in the trial?
- What will happen to me during the trial?
- What do I have to do for the trial?
- It is possible that I will receive a placebo (a look-alike substance containing no active drug ingredients).
- How long is the trial?
- Do I have other options for my condition besides participating in the trial?
- What if I want to leave the trial before it is completed?
- Will it cost me anything personally?
- Who should I contact with questions?
- Will I be able to know the results of the trial when it is ended?
What factors should you consider before entering a clinical trial?
- A research trial may or may not help you personally
- Are you comfortable with the idea of participating in a clinical trial?
- How do you feel that you may possibly receive a placebo?
- There might be unknown side effects from the research treatment
- What happens if your condition worsens?
- How will your study responsibilities affect your daily life?
- Are people close to you (spouse, relatives, religious leaders, etc.) in agreement with trial participation, if this is important to you
- Are there any other questions or concerns you need answered?
For a summary on volunteering for clinical research, you can read our brochure here: Becoming a Research Volunteer (PDF)
If you have questions that are not addressed in the FAQs, or to learn more about studies that are open for enrollment, please contact:
Carla Erickson, RN, MS, CCRP
Director, Education & Quality in Clinical Research
For current, prospective, or past research participants to discuss problems, concerns, or questions; obtain information; or offer input with an individual who was unaffiliated with the specific research protocol, please contact:
Craig Peine, MD
Karen Heim-Duthoy, PharmD
Vice Chair, IRB
Research Secretary, Sr.
Carla Erickson, RN, MS, CCRP
Director, Education & Quality in Clinical Research