Clinical Trials Explained
Volunteering to participate in a clinical trial is a very personal choice that only you can make. To gather the necessary information to help you make a decision that is right for you, first seek advice from your hematologist or oncologist, and talk to the team coordinating the study you are considering. Before deciding whether or not to participate in a clinical trial, it is essential to discuss with your doctor the purposes of the study and the risks and benefits as they relate to you, making certain that you understand all available options for treatment.
What are clinical research studies?
What happens in a typical clinical trial?
How will my safety be protected if I decide to participate in a clinical trial?
What are the phases of clinical trials?
How do I decide whether or not to participate?
Questions to Ask About a Clinical Trial
Bone Marrow Failure Disease Consortium
Additional Resources
Contact an AA&MDSIF staff member with questions about clinical trials
What are clinical research studies?
Clinical research studies are a way for doctors to find new and better methods to understand, prevent, control, and treat diseases. Carefully conducted research studies are the fastest and safest way to find treatments that work for patients and new approaches to improve health. Patients do not have to receive a new treatment to participate in a research study. There are different types of studies designed for specific purposes and each is important. Some types of research studies are:
Observational and epidemiological studies – Observational and epidemiological studies are research studies where patients do not receive a new treatment. In these studies patients are observed over time and answer questions about their disease.
In observational studies researchers watch patients over time to learn more about the causes of their disease and if and how it progresses. For example, in a natural history study (a type of observational study) researchers follow and observe the medical conditions of groups of patients and collect information about their illnesses over a period of time.
Epidemiological studies try to figure out how many people have a disease, which geographic locations have the most patients, and how the disease affects patients. During epidemiological studies, researchers usually talk with patients about their past history to try to explain their current medical condition.
Observational and epidemiological studies are beneficial to patients because they help researchers learn more about why patients develop the disease and how it progresses. This information may be key in developing new treatments or ways to prevent the disease.
Clinical trials – Clinical trials, also called interventional trials, are research studies that examine the effects and safety of a new treatment in patients. Clinical trials are designed to evaluate a new treatment, a new combination of established treatments, or to learn more about a current successful treatment. Clinical trials are valuable because the research data they provide leads to the development of safer and more effective procedures and treatment options. Clinical trials are beneficial to patients because they may receive access to new treatments before they are available to the public and may be among the first to benefit.
What happens in a typical clinical trial?
The researcher leading the study carefully designs a plan called a protocol to describe exactly what will happen during the study and why and how the results of the study are expected to answer specific research questions. A protocol describes the schedule of tests, procedures, medications and dosages, and the length of the study. It also explains the way that you and other participants would receive treatment while participating in the study.
A protocol is created to protect the health and wellbeing of participants, and it is explained in detail to each patient before they agree to participate in the study. A protocol must be approved by an independent committee called an Institutional Review Board, which is responsible for making sure that research studies are run safely and ethically.
Depending on the phase of the study and the scientific questions it is designed to answer, treatments could be given to participants in different ways. Sometimes every participant in a clinical trial receives the new treatment being studied so researchers can learn more about its safety and effectiveness in treating a disease.
In other clinical trials, participants are randomly assigned to different groups and receive different treatments so researchers can compare the results to gather more information about the new treatment. One of these groups of participants, called the control group, receives the standard treatment, which is a current treatment considered to be effective in treating a disease. The other group, called the treatment group, receives the new treatment being studied in the clinical trial. In some cases, the new treatment being studied may be a combination of treatments, possibly including a standard treatment for the disease.
All research studies have a list of guidelines, called inclusion and exclusion criteria, specifying who is appropriate to participate. Inclusion criteria are conditions that patients must meet in order to participate in a study. For example, patients may have to be transfusion dependent in order to participate. Exclusion criteria are factors that exclude patients from participating in a study. For example, patients may not be able to participate in a study if they also have diabetes or heart disease.
How will my safety be protected if I decide to participate in a clinical trial?
Protection for people participating in clinical trials is of primary importance to the United States government. The Common Rule, a Department of Health and Human Services regulation effective 12/13/01, mandates that research studies must be conducted under strict guidelines to protect participants' rights and welfare and to ensure that risks to participants are minimal compared to likely outcomes. This policy also enforces oversight by Institutional Review Boards (IRBs) and requires informed consent by all study participants. These rules apply to all medical research involving human participants subject to federal regulation. To ensure that medical progress is made safely and ethically, the protections described below make certain that participants in research studies are protected from harm and unnecessary risk.
Institutional Review Boards are independent committees established at each medical center conducting clinical trials to ensure that the risks to study participants are minimal and are offset by the potential benefits of their participation. IRB members include individuals who do not have any personal interest in the results of the study, such as doctors, other health care professionals, and community advocates. It is the IRB's responsibility to ensure that the highest ethical standards are met and that participants' rights are protected.
Informed Consent is the process by which patients learn about a clinical trial to help them decide whether or not to participate. The doctors and nurses explain the protocol (details of the study) to you and answer all of your questions. The research team then provides an informed consent document that includes information about the study, including its: purpose, duration, procedures, risks and potential benefits, and key contacts. While the informed consent document is not a legally binding contract, participants must sign it to both acknowledge their understanding of the protocol and to agree to participate in the study. Informed consent continues throughout the study because new information gained by researchers is constantly communicated to participants. Participants are always free to leave a study at any time without compromising their future medical care.
What are the phases of clinical trials?
Clinical trials are conducted in phases. Each phase has a different purpose and helps researchers answer different questions.
Phase I: Researchers evaluate a new medication or treatment for the first time in a small group of people with a specific disease to evaluate its safety, determine a safe dosage range, and identify side effects. (Small number of participants)
Phase II: The identified safe dosage range of the treatment being studied is given to people with a specific disease to see how effective it is in treating the disease. These trials also continue to evaluate the safety of the treatment. (Larger number of participants)
Phase III: The treatment is given to larger groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the treatment to be used safely. These trials are larger than the previous two phases; they often involve a "randomization" in which participants are assigned to different treatment groups by chance and either receive the new treatment being studied or a standard treatment for the disease. (Largest number of participants)
Phase IV: These studies usually take place after a drug has been approved by the Food and Drug Administration (FDA) and has been made available to the public. They are designed to collect additional information about a treatment, particularly about its long-term side effects in a larger population.
How do I decide whether or not to participate?
It is important to consider the pros and cons of participating in a research study before making the personal choice about whether or not to participate. Discuss the risks and benefits with your hematologist or oncologist and family members or advocates to help you to make an informed decision that is right for you. Refer to AA&MDSIF's list of Questions to Ask About a Clinical Trial.
For more information about the risks and benefits of participation in a clinical trial, call 800.747.2820 to request AA&MDSIF's brochure Clinical Trials for Patients With Bone Marrow Diseases: Basic Explanations.
Additional Resources
Contact an AA&MDSIF staff member with questions about clinical trials
To learn more about clinical trials, contact the AA&MDSIF at 410.867.0242 or 800.747.2820 via email at clinicaltrials@aamds.org.
Last Modified: December 20, 2007
