What are clinical trials?
Have you ever been scrolling down your social media account and come upon advertisements asking you if you want to participate in a clinical trial? Well, many of the medication in the market today that you take for the flu, diabetes, an infection, even cancer, all started with a clinical trial. These medications would probably not be available had people not participated in clinical trials.
What are they/what they are for: The World Health Organization (WHO) defines a clinical trial as a “type of research that studies new tests and treatments and evaluates their effects on human health outcomes[1].” These trials study things ranging from vaccines, medication, blood tests, risk factors, etc. Participating in these trials is completely voluntary and you may even receive some sort of compensation for your participation. You may ask yourself “what is the point of a clinical trial?” or “why should I participate in a clinical trial?” Well, to answer that, let’s look a little more into the specifics of clinical trials.
History: First, a little bit of history. Clinical trials have been around since 562 BC. The first clinical trial is recorded in the “Book of Daniel” in the bible! Of course, this study was done inadvertently by King Nebuchadnezzar, ruler of Babylon. The King’s trial consisted of ordering his people to adhere to a diet of only meat and wine, as he thought this diet would keep them in better physical conditions. However, some men of royal blood objected and asked to eat vegetables as well. The King allowed these men to eat vegetables and water for 10 days only and at the end of this “trial” the King noticed that the men who had eaten vegetables were better nourished that the men who exclusively ate meat. Due to these results, the King allowed his subjects to continue eating vegetables[2].
Another important clinical trial was conducted by Dr. Lind in 1747. This was the first physician led trial and the results are highly relevant to this day. While he was working as a surgeon on a ship, Dr. Lind observed a high mortality in sailors due to scurvy. All the sick sailors slept in the same room for disease containment and received the same diet daily. So, Lind devised a study in which he would give different diets to these sailors to ascertain whether this would help or cure the disease. Two sailors would receive a quart of cider a day, another two would take 25 drops of elixir vitriol three times a day, another two would take two spoonfuls of vinegar three times a day. The sailors that had more severe illness were given the following diet: two were on a course of seawater, two would take two oranges and one lemon a day and the final two received an electuary recommended by a hospital physician. From this, Dr Lind observed that the two sailors who consumed oranges and lemons were physically better and after only six days, they were fit for duty[2]! Thanks to this trial, today we know that scurvy is a disease caused by a Vitamin C deficiency and is easily treatable.
Different types: There are two main types of clinical trials: observational and interventional.
Observational clinical trials do not administer any drugs or vaccines and only monitor the participants in order to gather information[1].
Interventional clinical trials test safety and efficacy of medications, tests, procedures or devices in patients[3].
Interventional clinical trials are further divided into phases 1-4.
Phase 1 clinical trials usually only takes a small number of people (less than 100) and test a new medication’s safety, side effects and dose.
Phase 2 clinical trials include a larger number of participants, and the focus is whether the new drug works in a given disease.
Phase 3 clinical trials will gather information from hundreds to thousands of participants. These trials are comparing the efficacy of the drug being studies versus another drug that is already in the market. After a phase 3 clinical trial, the FDA may approve the intervention being studied.
Phase 4 clinical trials study a drug after the FDA has approved it, but it a much larger group of people and it happens over a longer period[2].
What happens during a trial: If you decide you want to participate in a clinical trial, there a few steps you go through before receiving the medication or procedure. First, you will be given an informed consent form (ICF). This form will have all the information you need regarding the trial, such as information about the medication/device, blood tests you will need, possible side effects, etc[4]. The research staff will answer any questions you have regarding the trial and once they have been answered to your satisfaction, you may sign the form if you wish to participate. Then, you will be screened by the research staff to assess if you qualify for the trial. This stage may include questionnaires, blood tests, or imaging studies. If you are accepted into the trial, you will be schedule for your first visit. Depending on the trial, during this visit you may be assigned to receive the intervention being tested or that may come at a later visit. Once you start receiving the intervention, you will continue to have follow up visits with a physician to ensure everything is going okay. Once the study is finished (and this may take months to years depending on the trial), all the information will be analyzed to determine the drug or procedure’s efficacy and safety.
Reasons to participate: As you can see there is a lot that goes into clinical trials. It can take years to get a trial off the ground. But without these clinical trials, we would not have any of the advancements that we have today for conditions such as cancer, HIV, diabetes, heart disease, etc. Without the people who participate in these trials, these drugs would not exist, and nobody would benefit from them. You may also benefit from a trial. For example, if a drug regimen that you are currently on is no longer working, this is a way to receive a new and promising medication at no charge and you may also be compensated for your time and travel. Also, remember that participating in a clinical trial is completely voluntary and you may decide to withdraw whenever you want. They are also safe, and you are always monitored by a physician. The safety and health of the participants always comes first.
References:
1. [2023Apr9]. Available from: https://www.who.int/health-topics/clinical-trials#tab=tab_1
2. Bhatt A. Evolution of clinical research: A history before and beyond James Lind [Internet]. Perspectives in clinical research. U.S. National Library of Medicine; 2010 [cited 2023Apr9]. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3149409/#:~:text=James%20Lind%20is%20considered%20the,trial%20of%20the%20modern%20era.&text=Dr%20Lind%20(1716%2D94),most%20promising%20cure%20for%20scurvy.
3. Day JA. Clinical research: What is it?: Johns Hopkins Division of Gastroenterology and Hepatology [Internet]. Clinical Research: What Is It? | Johns Hopkins Division of Gastroenterology and Hepatology. 2022 [cited 2023Apr9]. Available from: https://www.hopkinsmedicine.org/research/understanding-clinical-trials/clinical-research-what-is-it.html
4. Commissioner Of the Informed consent for clinical trials [Internet]. U.S. Food and Drug Administration. FDA; [cited 2023Apr9]. Available from: https://www.fda.gov/patients/clinical-trials-what-patients-need-know/informed-consent-clinical-trials
