PATIENT INFORMATION BRIEF
QUESTIONS AND ANSWERS FOR PATIENTS CONSIDERING PARTICIPATION IN A CLINICAL TRIAL
Clinical Trials are at the heart of all medical advances. They provide an avenue to explore new and effective ways to prevent, detect, or treat disease. The focus of clinical trials may include:
• New drugs or new combinations of drugs to treat complex diseases and conditions
• New ways of doing surgery
• New medical devices
• New ways to use existing treatments to treat complex diseases and conditions
• New ways to improve the quality of life for people with acute or chronic illnesses.
What do I gain from participating in a clinical trial?
Clinical trials assist researchers and doctors in uncovering new and effective ways to prevent, diagnose and treat diseases and conditions. For many patients the reward is not simply improved treatment for their particular disease or condition, but as well, a feeling of strength and satisfaction in knowing they have contributed to an effort to combat a disease or condition that affects the human population.
Who participates in clinical trials?
Participation in a clinical trial is often determined by the nature and scope of the trial. In some trials you may participate as a healthy volunteer (i.e., a person with no known significant health problems who participates in clinical research to test a new drug, device, or intervention), or you can participate as a patient volunteer (i.e., a person who has a known health problem and participates in research to better understand, diagnose, treat, or cure that disease or condition).
What questions should I ask before deciding if I want to take part in a clinical trial?
Before indicating a willingness to participate in a clinical trial you may want to ask your doctor or the individual in charge of the trial some important questions. The following represent some suggested questions:
Questions Regarding the Clinical Trial
• What is the purpose of the trial?
• Why do researchers think the approach may be effective?
• Who has reviewed and approved the clinical trial?
• How are study results and safety of participants being checked?
• How long will the study last?
• What will my responsibilities be if I participate?
Questions Regarding Possible Risks and Benefits
• What are the possible short-term benefits of the trial for me?
• What are the possible long-term benefits of the trial for me?
• What are my short-term risks and potential side effects?
• What are my possible long-term risks?
• What other options besides the trial do people with my disease have?
• How do the possible risks and benefits of this trial compare with those options?
Questions Regarding Participation and Care
• Will I be able to take my regular medications while participating in the clinical trial?
• Where will I get my medical care?
• During the trial, who will be in charge of my care?
• How will participation in the trail affect my daily life?
Questions Regarding Cost
• Will I have to pay for any part of the trial, such as tests or the study drug? If so, what will the charges likely be?
• What is my health insurance likely to cover?
• Who can help answer any questions from my insurance company or health plan?
• Will there be any travel costs that I need to consider while I am in the trial?
What is informed consent?
Informed consent is the process of learning about a clinical trial before you decide to take part in it. The research team running the trial will provide a complete explanation of the:
• Purpose of the trial
• How long the trial will take
• What will happen during the trial
• All potential risks and potential benefits
• Information in the privacy and confidentiality of your personal medical records.
If you decide to participate in a trial, you will be asked to sign a consent form that details all of the information that has been discussed with the research team. The consent form is yours to keep and you will be able to take the form home and refer to it at any time.
It is important to note that even though you have signed the consent form, you still have the right to leave the study at any time and for any reason. Informed consent continues throughout the trial.
Clinical trials follow a specific plan known as a protocol. The protocol is carefully designed to balance the potential benefits and risks to participants, and answer specific research questions. In general, protocol describes the following:
• The goal or intent of the study
• Who is eligible to take part in the trial
• What protections are established against risks to participants
• Details regarding tests, procedures, and treatments
• How long the trial is expected to last
• What information will be gathered
Most, but not all, clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are reduced and are outweighed by potential benefits. IRBs are committees that are responsible for reviewing research in order to protect the rights and safety of people who take part in research, both before the research starts and as it proceeds. You should ask the sponsor or research coordinator whether the research you are thinking about joining was reviewed by an IRB.
What is meant by Inclusion and Exclusion Criteria?
All clinical trials have specific inclusion and exclusion criteria for participation. Inclusion criteria are factors that allow you to take part in a clinical trial (for example type of disease or condition you are experiencing and your current treatment). Factors that exclude or prevent participation are “exclusion criteria.” These criteria are based on factors such as age, gender, the type and stage of a disease, treatment history, and other medical conditions you may have. In general, inclusion and exclusion criteria for a given clinical trial are strict. Strict adherence to inclusion and exclusion criteria allows researchers to gain confidence that whatever findings are derived from a given trial are not the result of random uncontrolled factors.
Clinical trials are conducted in a series of steps called “phases.” Each phase has a different purpose and helps researchers answer different questions.
• Phase I trials: This phase is designed to test a drug or treatment in a small group of people (20–80) for the first time. The purpose is to study the drug or treatment to learn about safety and identify side effects.
• Phase II trials: In this phase, a new drug or treatment is given to a larger group of people (100–300) to determine its effectiveness and to further study its safety.
• Phase III trials: In Phase III Trials, a new drug or treatment is given to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information that will allow the new drug or treatment to be used safely.
Phase IV trials: Phase IV Trials occur after a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits, and optimal use.
After a clinical trial is completed, the researchers carefully examine information collected during the study before making decisions about the meaning of the findings and about the need for further testing. After a phase I or II trial, the researchers decide whether to move on to the next phase or to stop testing the treatment or procedure because it was unsafe or not effective. When a phase III trial is completed, the researchers examine the information and decide whether the results have medical importance.
Results from clinical trials are often published in peer-reviewed scientific journals. Peer review is a process by which experts review the report before it is published to ensure that the analysis and conclusions are sound. If the results are particularly important, they may be featured in the news, and discussed at scientific meetings and by patient advocacy groups before or after they are published in a scientific journal. Once a new approach has been proven safe and effective in a clinical trial, it may become a new standard of medical practice.
Clinical trials and clinical research represents a rigorous, highly controlled, and effective avenue for gaining important insights and answers about the safety and effectiveness of new treatments and procedures to address the complex diseases and conditions that affect human populations. Groundbreaking scientific advances in the past, the present, and indeed in the future are only possible because of participation of volunteers, both healthy and those with an illness, in clinical research. If you are asked to participate in the clinical trial, make sure you ask several questions and reach a comfort zone before committing.