Clinical trials are an integral part of advancing scientific research and delivering promising new treatments. Research gathered from a clinical trial can help to advance our knowledge in preventing, detecting or treating a disease. This research could lead to safer and more effective treatments, an improved quality of life for those affected by a disease and could ultimately lead to a cure.
Why participate in a clinical trial?
There are several reasons why one might want to participate in a clinical trial. A healthy volunteer may choose to be involved to advance medical research. A person living with an illness may choose to get involved in a clinical trial because existing treatments have failed and their participation may give them access to new, possibly cutting-edge treatments that may improve their health condition. Getting involved in a clinical trial may also give a participant access to expert care and detailed health monitoring. As well, participating in a clinical trial is a step toward bringing improved treatments to the market and increasing treatment options for those suffering from the same disease or condition.
What is a clinical trial?
A clinical trial is a rigorous study involving human volunteers to test the safety and/or effectiveness of an experimental treatment, a medical device or procedure. It may also involve an alternative use of an existing treatment or a combination of treatments.
Typically, prior to trials in humans, safety and effectiveness studies are conducted in animals providing ‘preclinical’ safety and efficacy data. If there is sufficient data to support advancing an investigational treatment to human trials, the pharmaceutical company developing the treatment then submits an application to one or more regulatory agencies. This application contains data from laboratory studies, including research in animals, to support research of the investigational product in humans. It also contains a clinical trial protocol which details how long the clinical trial will take, how many participants will be involved, the tests and medical procedures involved and the goals of the study. In parallel to this regulatory review, an ethics board also reviews these materials and any information that will be provided to a potential trial participant in order to protect the rights of participants and to ensure transparent, fair and ethical communication.
It typically takes 5 – 10 years of laboratory, animal and human clinical research before an investigational treatment, medical device or procedure can be considered for market approval. There are a number of clinical trial stages before an experimental drug or device becomes commercially available.
- Phase 1 – This clinical stage usually involves a small number of healthy volunteers (those with no significant health concerns) and assesses safety, side effects and/or dosage range of the investigational treatment.
- Phase 2 – This clinical stage involves a larger number of participants with active disease and involves testing safety and effectiveness, side effects and optimal dose.
- Phase 3 – This clinical stage involves hundreds or thousands of participants with active disease and further evaluates safety, effectiveness and side effects and may involve comparing the investigational treatment with commonly-used treatments. At this stage, pharmaceutical companies, experts, investigators and regulators are ensuring the drug is effective and that the investigational treatment benefits outweigh the risks associated with the treatment. After completing this stage, the company developing the treatment compiles the data and assesses whether the investigational product shows a benefit to patients and should be considered for market approval. An application containing all the data gathered to date is then presented to the regulatory agency or agencies for consideration of market approval.
- Phase 4 –This clinical stage occurs after the product is commercially available. The pharmaceuticalcompany continues to monitor and evaluate benefits, risks, side effects and best use. They may also explore the use of an approved drug in a new disease area.
At each stage, a pharmaceutical company may determine there is not sufficient evidence to support advancing the product to the next successive stage and may choose to discontinue development.
Preparing to participate in a clinical trial
Anyone considering involvement in a clinical trial should conduct due diligence to ensure the clinical trial is a fit for their needs. If you are considering participating in a clinical trial, here are some important tips to think about:
- Read about the company developing the investigational treatment or device. Learn about the investigational treatment and the results of research conducted prior to the clinical trial. Learn about any safety studies that have been done on the investigational treatment to date and the side effect profile of the drug.
- Understand the process and time involved to ensure that the clinical trial fits into the demands of your day-to-day activities.
- Speak with those close to you about your plans to participate. Have them understand what will be involved and how they may be able to help you.
- Learn if there are any costs associated with participating in the trial such as travel, lodging, drug costs and tests and/or procedures.
- Weigh the benefits and risks of being involved in the clinical trial. If existing treatments have failed you, an investigational treatment with a new mechanism/method may benefit you.
- Talk to your doctor about the trial. It’s possible your doctor(s) may not have heard of the investigational treatment. It’s best to bring detailed information to your doctor about the investigational treatment or device.
- Prepare a list of questions for the clinical trial nurse and/or investigator. The National Institutes of Health provides a list of suggested questions to ask if you are considering a clinical trial: http://www.nih.gov/health/clinicaltrials/basics.htm#5
- You can decline participation in a trial at any time, so speaking with a clinical trial nurse or investigator about a trial does not mean that you have to participate. Ask lots of questions and join the trial only if it is right for you.
Clinical trials ensure that any investigational treatment, medical device or procedure is thoroughly investigated to assess risks and benefits. Without clinical trials, it would not be possible to bring ground-breaking new therapies to those who would benefit from them. Each clinical trial participant contributes to improving our understanding of a disease and advancing a potential new treatment, medical device or procedure. Clinical trial participants have contributed and will continue to contribute to scientific research that will lead to improving diagnosis, prevention and the treatment of disease and improving the quality of life of those living with illnesses and advancing towards a cure.
Dr. Hal Gunn is the CEO of Qu Biologics, a biotechnology company developing Site Specific Immunomodulators (SSIs), new investigational immunotherapies designed to activate our body’s innate immune response and restore normal immune function in the targeted organ or tissue to reverse the chronic inflammation underlying many diseases, such as inflammatory bowel diseases. Qu Biologics is recruiting participants for three clinical trials in Crohn’s disease, ulcerative colitis and lung cancer.