What is a Clinical Trial
A clinical trial is a carefully planned study with human volunteers to find out if a treatment is safe and effective. This kind of scientific study is key to all medical advances, because it helps answer these questions:
Does the Treatment work?
Does it work better than other treatments for the same condition?
Is it safe for different groups of patients?
Each clinical trial focuses on finding a better way to understand, prevent, diagnose, or treat a disease. All clinical trials are carefully scrutinized by independent experts to make sure the health of the participants is safeguarded. In addition, different phases of trials must be carried out to track safety and efficacy before a new treatment is approved and made widely available.
What are the different kinds of clinical trials?
There are two different kinds of clinical trials, interventional and observational, and both types are important for increasing our understanding of diseases and their treatment.
They assign a specific therapy to each participant, so they are an important part of figuring out what health effects are specifically caused by the treatment.
They track health in more natural settings by studying what people already do. These trials give scientists a more realistic idea of how a certain factor influences health (normally a drug or other medical intervention), but they cannot help determine causal relationships.
Phases of clinical trials
Researchers are primarily concerned with safety, so they test a new treatment in a small group of people usually in a healthy population
A larger group of patients receive the treatment to test both safety and efficacy
Even more patients receive the treatment to confirm its efficacy and safety and to compare it with other possible treatments
These trials occur after the treatment is approved and in public use, in order to track effects of long-term use and commonly reported side effects
How to find a clinical trial
Companies that develop new treatments commit to sharing the results of their studies so doctors and patients around the world can benefit from the information. When sharing these details, they must protect participants’ privacy while following the regulatory guidelines of their country or jurisdiction. The ClinicalTrials.gov website was developed as a place to post details about clinical studies taking place all around the world. Trials that investigate Ipsen therapies are listed in the ClinicalTrials.gov database, showing that Ipsen is committed to transparency and the sharing of information necessary for advancing medicine.
Patients who want to volunteer for a clinical trial can search ClinicalTrials.gov to find suitable studies in their geographic area. After finding a study on the website that looks like a good fit, the patient can contact the study investigators directly for more information on how to take part.
Patient clinical trials journey
The clinical trial
During a clinic visit, Jane asks about clinical trials happening locally. The doctor tells her about a clinical trial that might be a good fit for her.
The doctor explains the study and the specific tests, including how much time and how many visits are required.
The informed consent form
A doctor of another member of the research team gives Jane the informed consent form. *
After taking home the informed consent form and reading it through, Jane decides whether or not to go ahead as a participant in the clinical trial.
The signed informed consent form
Jane signs the form and returns it to the researchers. The researchers also sign it and give her a copy to keep.
After getting the signed form, researchers go through a checklist to make sure Jane is eligible to participate. The researchers then confirm that Jane can join the trial.
The baseline testing
In the clinic, researchers carry out the first set of tests according to the study plan. Jane, for example, might be asked to fill out a questionnaire, give a blood sample or do a set of tasks.
The data manager of the clinical trial assigns Jane to one of the groups in the trial. In a « blinded » study, Jane will not know whether she has been assigned to the experimental treatment group or to the placebo (non treatment) group.
Jane receives the treatment (or placebo depending on her group) according to the study design. What if Jane wants to withdraw the study?
At any time in the process, she is free to withdraw by speaking with the researchers.
The clinic visits
Jane returns to the clinic at specific times for testing, carrying out the study plan that was outlined at the beginning.
The final tests
Jane returns to the clinic for the final set of tests that the researchers need to carry out.
Jane might receive a summary of the study from the sponsor, but she can also look for the overall results on the clinicaltrials.gov website 12 month after the study is completed.
*What is the informed consent form (ICF)? The ICF is a document that outlines key information about a study – especially the possible risks and benefits – to help a person decide whether or not to take part. Jane needs to read the ICF carefully, since it includes all the « fine print » about the clinical trial and exactly how the researchers’ plan will be carried out.