The Canadian Centre for Clinical Trials (CCCT) would like to take this opportunity to address some of the most common and relevant questions regarding trial participation because we believe in the importance of making an informed decision towards your health.
What is a clinical trial?
A clinical trial is a type of research that investigates whether a particular drug can be used to treat human conditions and diseases. Clinical trials follow a protocol which clearly defines what the trial is investigating (e.g. how effective 5 mg of drug A is in reducing blood pressure) and how the study should be conducted (e.g. drug A is administered orally every morning at 8 am and blood pressure is measured every evening at 4 pm). Clinical trials are designed specifically to determine whether the drug under investigation would improve outcomes (in our example, this would mean lower blood pressure) while ensuring patient safety.
How, as a participant, is my safety ensured while involved in a clinical trial?
As previously mentioned, clinical trials are carried out following a strict protocol. Patient safety is the number one priority in a study and every clinical study is executed under the supervision of a medical doctor (also known as a principal or qualified investigator) and qualified medical staff who have received training in research ethics and safety (i.e. a certification in Good Clinical Practice). All studies are also monitored by ethical and legal regulatory bodies such as the Institutional Review Board and Health Canada that ensure patient well-being remains a priority. All information collected is kept confidential and never disclosed to individuals outside of the research.
Why should I consider participating in a clinical trial?
Clinical trials are meant to offer innovative treatment options to patients who suffer from various conditions. By electing to participate in a clinical trial, you are electing to further investigate the treatment of a given medical condition or disease, and to help identify a potential cure. Individuals who participate in clinical trials are often those who have unsuccessfully tried available treatment options. As a participant in a clinical trial, you are essentially afforded the opportunity to be more attentive in your health care while aiding the progression of medical research.
Is it possible that I will experience side effects from the medication under study?
Side effects are a possibility for drugs like Tylenol that have been on the market for years, and are not just a reality of clinical trials. It is important to note that clinical trials minimize the chance of side effects through the rigorous screening process and if side effects do occur, they occur in an environment of regular medical monitoring and priority access to health care professionals.
What is informed consent?
Participants are asked to provide informed consent for participation which means that they fully understand the treatment, risks and benefits of the trial. Informed consent provides an excellent opportunity for the participant to ask questions about the treatment and/or the nature of their condition. It is worth recognizing that informed consent is an ethical consideration and is NOT a contract to participate. Patients are free to leave the trial at any point.
Will I be compensated for trial participation?
In addition to receiving potentially beneficial treatment in a trial, participants usually receive monetary compensation for their time and any expenses associated with participation. The amount of compensation is based on factors such as the length of stay in the clinic, travel, and the frequency of follow up visits. As a result, phase I trials are often associated with greater compensation because they can involve overnight stays at the clinic.
What is a Placebo?
A placebo is a mock medication (often in the form of a sugar pill) which actually has no therapeutic effect in the body. Placebos are important because of the tendency for patients to feel better, and even have physical improvements in parameters such as blood pressure, after taking what they think is a medication. This is referred to as the placebo effect and is thought to be due to the strong connection between our mind and bodies (e.g. if you think you are going to feel better, you tend to feel better and show physical signs of improvement). The placebo effect makes it difficult to know whether the medication under study is actually working or not, so some participants are given placebos in a clinical trial. Since both placebo and non-placebo groups are treated the same way in a trial (e.g. they both take a medication and undergo the same assessments) we can measure the effect of the medication by cancelling out the placebo effect.
Will my Participation Really Make a Difference? A Note on Sample Size
Without a good number of participants in clinical research (i.e. a low sample size), researchers cannot say for certain whether treatments will benefit patients. The reason for this is because there is always the possibility that any observed benefit is due to chance.
For example, if you tested a treatment on one patient you may observe a benefit, but it is possible that the patient’s improvement had nothing to do with the treatment itself (for example if the body suddenly decided to heal itself). When you test the same treatment on 100 patients and 95 of them show improvements, there is a lower probability that all 95 patients are improving for a reason unrelated to the treatment.
Therefore, a higher study sample size increases researchers’ ability to make conclusions about the effectiveness of treatments. This same concept applies when there are many studies examining the effectiveness of a treatment.