Physicians explain what randomization, standard of care arm and experimental arm mean in clinical trial study design. Randomization is where participants are assigned by chance to separate groups that compare different treatments, and neither the participant, nor the doctor, nor the medical team can choose which group the participant is assigned to. Typically this is assigned by computer allocation. They also explain that to help support the development of new drugs there needs to be a reference, and that reference is called a standard approach or ‘standard of care.’ Participants may be assigned to the standard of care arm of a study where they receive the standard of care treatment plus placebo, or to the experimental arm where they receive the standard of care plus the new treatment of interest. If a study design is ‘blinded’ it means that one or more parties involved in the trial (such as the participant, or researchers, or sometimes both) do not know which study arm the participant is assigned to.
This animation explains what clinical trials are, how they are conducted, and why they are important for patients with diseases like pancreatic cancer. The animation also provides an overview of study design, eligibility criteria, informed consent, safeguards, different phases of clinical trials, and the potential benefits and potential risks of participation.
Physicians describe what a clinical trial is. Clinical trials are research studies that evaluate new ways to improve treatments and quality of life for people with diseases, like pancreatic cancer. A clinical trial can be part of a number of scenarios and patients can participate in many ways. A clinical trial is how we make progress in cancer research. All the drugs and tools that we have today came about because patients volunteered for clinical trials.
Physicians discuss why clinical trials are important. When it comes to a disease like pancreatic cancer, even though we have made progress we still have a long way to go to improve outcomes for pancreatic cancer patients - and the best way to do that is to test new treatments and new diagnostic tools through clinical trials. By being in a clinical trial patients may benefit from a new treatment, as well as contribute to science and moving the field of medicine forward.
Physicians explain how clinical trials work and the different types of clinical trials and stages – from early stage to late stage. In a clinical trial, there is an individual called the principle investigator, who has primary responsibility for the conduct of the trial, and he or she will work with a team, usually a research nurse and several study coordinators and data managers. The trial is overseen by an institutional review board (IRB) and other committees that govern the scientific merits of the study, and patient welfare and safety. A clinical trial has to be done under rigorous oversight and controls, so it usually means that patients receive treatment in a clinical trial center and not always with their local community oncologist.
Physicians discuss the series of steps (called phases) of clinical trials. Each phase is designed to answer important questions that lead to the next phase. If a patient is thinking about a clinical trial, it’s important to understand which phase the trial is in and what the unique protocol (or plan) is for the study and the eligibility criteria.
Physicians discuss who can participate in a clinical trial. Whether a patient can be enrolled in a clinical trial depends on what’s referred to as “eligibility criteria”. Eligibility criteria differ from one clinical trial to the next and there is a great deal of variation. The highest priority for a clinical trial is to minimize the potential for serious risk and harm. It is a lot of work to have a patient enroll in a clinical trial and it requires careful explanation and discussion on the pros and cons to make sure it is the right fit for that person and that they are truly comfortable with the decision to participate.
Physicians explain the advantages of participating in a clinical trial. By participating in a clinical trial, patients can often expect a higher standard of care, and there is the hope that the clinical trial will benefit the individual patient. We need clinical trials to advance new medical discoveries and to make progress against cancer, so there is also an altruistic aspect to participating in a clinical trial in terms of helping others and advancing medical science.
Physicians discuss the risks and benefits of volunteering for a clinical trial. In terms of benefits of the clinical trial, basic standard of care is usually covered by a patient’s insurance (for example, physician visits and routine blood tests will be covered by insurance) but patients should check to be sure. Also, a research biopsy (taking a piece of the tissue to look at the genetic makeup of the tumor) would typically be covered, and the experimental drug that’s being studied is provided free of cost as part of the trial. An important part of the risks for patients are side effects. Patients need to have a detailed understanding of what the side effects are, and this is part of the consent process which they need to discuss with the researchers. Participating in a clinical trial is not for the faint of heart. It has to be something that patients are really committed to participating in because it will be a bit more intensive than just regular treatments. Of course there is the potential for higher reward. Patients need to understand both the benefits and risks very clearly before they agree to volunteer.
Physicians explain what “informed consent” is in a clinical trial. Informed consent is the process of giving clinical trial participants all of the facts about a trial. Informed consent is greatly important because patients cannot participate in a study without their clear understanding of everything involved. Informed consent means that the patient is making an informed decision on their own, and understands all the risks and benefits for participating in a study. There is a verbal conversation that goes along with this and also a written document. The informed consent document is vetted by an institutional review board (IRB) to make sure it’s not missing anything. Both the physician and the patient will sign the consent form, and then the next step is screening for eligibility and, if the patient qualifies, moving forward with the clinical trial.
Physicians describe the safeguards that are put in place to protect patients in clinical trials. At the beginning, the initial idea for the study undergoes a series of reviews to make sure the study idea is appropriate, that patients will be safe, that the right drugs are being used, and the right end points are going to be answered. A big part of the safeguard process is institutional review board (IRB) oversight. An IRB is a committee that reviews the trial to ensure the science and safety aspects for patient care. Furthermore, the principal investigator has an ethical and professional obligation to both the patient and to the study. In addition, on a national level, there are federal safeguards in place that oversee clinical trials. For example, periodically, audits can be done at institutions of clinical trials to make sure there isn't an important safety signal being missed to make sure that the data is reported accurately and in a timely fashion.
Physicians discuss what happens if a patient changes their mind and wants to leave a clinical trial. A patient has every right to choose to stop participating in a clinical trial at whatever time point they want, even if they don't meet formal criteria for having to be taken off the trial. A patient will be removed from a study if the treatment is not working, if their cancer is continuing to grow, if researchers observe an unacceptable level of potentially serious side effects that may be life-threatening, or if the clinical trial is affecting their quality of life too much. There are also occasions where a patient finds that a clinical trial is just too time-intensive. It’s always important for a patient to talk with the researchers about their reasons for leaving the trial and to make sure that everything is done in the safest manner possible.
Physicians discuss whether patients will be able to pick what medications they will be receiving in a clinical trial. Every clinical trial is different, and sometimes this is possible and sometimes it is not. Sometimes one group of patients will want to choose one option and another group will want the opposite choice. Sometimes a “randomized” trial will be in place, where neither the patient nor the physician can choose which medication the patient will receive. The process of randomization is typically coordinated by computer allocation so that there is no bias in the study design. Randomization can make the results more accurate and more reliable.
Physicians discuss what a “placebo” is in a clinical trial, and if is it always used. Placebos can have some real impacts on a clinical trial. Researchers know that there is an effect from the trial, but they need to isolate that effect to make sure it is really the medicine or device that is causing that effect. In some clinical trials, doctors want to learn if adding a new drug to the standard therapy makes it work better. In these studies, some patients get the standard drug(s) and the new one being tested, while other patients get the standard drug(s) and a placebo. But none of the patients would get only a placebo. The placebo may look like medicine but it has no active ingredient in it. This clinical trial context is typically “blinded”, meaning neither the doctors nor the patients know which group they are in. This ensures there are no doctor or patient biases that impact the outcomes of the clinical trial.
Physicians discuss how patients can find out the results of a clinical trial that they participated in, how results for the larger group of patients are announced, and that individual patient information is always kept confidential. Clinical trial results can be made available to the general public and patients in a number of ways, such as at big scientific meetings, or in journal publications. The goal is to let as many people know as possible and to enhance knowledge sharing amongst the oncology research community to stimulate further ideas and clinical trials. Announcing results often happens when clinical trials are completed and an update will be put on the clinicaltrials.gov website. Occasionally a study will reach an end point and researchers may have to close the study early. As for patient privacy, patients will sign a disclosure that acknowledges that the study team looking after them will have access to their information, however individual information is never shared outside the trial without patient consent. Often the institutional review board (IRB) will have oversight, as well as the company or agency that's funding the trial. The Federal Drug Authority (FDA) can also have access to the information, as well as certain collaborating sites if they are involved in the clinical trial. All of this information should be provided up front to patients and is part of the informed consent process. There are many safety measures in place to protect confidential (also called “Privileged”) information as much as possible.
This slide show explains what clinical trials are, how they are conducted, and why they play an important role in moving science forward and improving treatments and quality of life for people with diseases like pancreatic cancer. The animation also provides an overview of study design, eligibility criteria, informed consent, safeguards, different phases of clinical trials, and the potential benefits and potential risks of participation.
This educational activity has been developed by:
The National Pancreas Foundation and Mechanisms in Medicine Inc.
This educational activity is supported by:
This website is part of the Animated Patient™ series that provides highly visual formats of learning for patients to improve their understanding, make informed decisions, and partner with their health care professionals for optimal outcomes.