Clinical trials (sometimes called clinical studies) are done to answer a specific research or medical question. Clinical trials are where participants receive specific interventions according to a research plan or protocol. These interventions may be medical products, such as drugs or devices; procedures; natural history, etc. These trials are essential to test whether a potential treatment is safe and effective before approval can be granted for use in the wider population.
Clinical trials are one of the later steps in a long process of drug development which involve extensive pre-clinical research often including animal testing. Only interventions that are evaluated as reasonably safe and show the most promise, progress to clinical trials in patients. Clinical trials are required by regulatory agencies such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) before a new drug can be brought to the market. The FDA and EMA are committed to protecting participants in clinical trials and to providing reliable information to the public.
Participating in a clinical trial
Participating in a clinical trial is a voluntary choice and there are many factors that need to be weighed before making a decision that is right for you and your family. Issues such as time away from school and work, travel requirements to appointments, and the medical procedures required during the trial, need to be considered. Ultimately it is a personal choice that you and your healthcare team have to make together. Deciding not to participate in a clinical trial will not affect the care you and your family will receive.
To volunteer for a clinical trial you must meet the eligibility criteria. Eligibility criteria are the rules for who can be recruited for a clinical trial, and include inclusion and exclusion criteria which are specific for each trial. These may include: age, sex, specific genetic mutation, stage of disease, previous treatment history, and presence of other medical conditions. The eligibility criteria aim to give the trial the best possible chance of success and help protect volunteers and ensure that it is safe for them to participate.
Before enrolling in a clinical trial each participant will undertake a complex and rigorous screening process. There is no guarantee that a participant will be enrolled until the screening data has been evaluated. Similarly, many trials have a placebo control arm, which is a group of individuals who will not receive the treatment and will be the control group. There is therefore no guarantee that an individual in a clinical trial will actually receive the active treatment /intervention.
Prior to enrolling the study doctor will discuss elements of the trial such as: the length of the study, the number and interval of visits required, the lab work and other tests or questionnaires required during the study. Any study related physical exam or outcome measures will be described. The study consent (for a parent, if participant will be under age 18) and assent (for participants age 7 -17) will be described, mailed and later discussed prior to enrolment.
Once an interest in participating in the clinical trial is expressed, a screening visit will be scheduled to discuss and answer any questions prior to signing of study consent and/or assent and to complete all questionnaires, exams and lab work associated with the screening process. It is important to note that you and/or your child’s participation is totally voluntary and that you may, at any time, resign from the study without penalty and without compromising the right to receive the standard treatment. The cost of all the tests and examinations associated with clinical studies are covered and there are no trial-related fees.